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# Medimix Hematology

## Sitemaps

- [XML Sitemap](https://medimix.be/hematology/sitemap.xml): Contains all public & indexable URLs for this website.

## Posts

- [Long-term safety of liso-cel in large B-cell lymphoma](https://medimix.be/hematology/eha-stockholm-2026-poster-13/) - In this video, Prof Dr Stephan Mielke presents a pooled longitudinal safety analysis of lisocabtagene maraleucel (liso-cel) in 420 patients with relapsed or refractory large B-cell lymphoma.
- [Bela-Vd vs. Tec-Dara for RRMM: an indirect comparison](https://medimix.be/hematology/eha-stockholm-2026-poster-11/) - In this video, Prof Dr Marie-Christiane Vekemans discusses an indirect comparison of two BCMA-targeted regimens in relapsed or refractory multiple myeloma.
- [Healthcare resource utilization with axi-cel vs. liso-cel in LBCL](https://medimix.be/hematology/eha-stockholm-2026-poster-10/) - In this video, Prof Dr Catherine Thieblemont discusses real-world data on healthcare resource utilization after CAR T-cell therapy in relapsed or refractory large B-cell lymphoma.
- [The REMNANT study](https://medimix.be/hematology/eha-stockholm-2026-poster-9/) - In this video, Dr Frida Bugge Askeland discusses primary results from the phase II portion of the REMNANT study in newly diagnosed, transplant-eligible multiple myeloma.
- [The impact of obesity on the risk of progression to MM in MGUS patients](https://medimix.be/hematology/eha-stockholm-2026-poster-8/) - Data presented as a poster at EHA 2026 by Dr Saemundur Rögnvaldsson and researcher at the University of Iceland, examined the relationship between obesity and the development of multiple myeloma (MM) and its precursor conditions.
- [Interactions between AML cells and mature osteoblasts](https://medimix.be/hematology/eha-stockholm-2026-poster-3/) - During EHA 2026, Lotfi Laaouimir shared his research findings on the interaction between acute myeloid leukemia (AML) cells and osteoblasts within the bone marrow microenvironment.
- [Genomic & transcriptomic profiling of post-transplant lymphoma](https://medimix.be/hematology/eha-stockholm-2026-poster-4/) - As part of her PhD research Ana-Lucía Rocha is investigating the molecular landscape of post-transplant lymphomas, a group of lymphoid malignancies that develop following solid organ or stem cell transplantation.
- [The IRAKLIA trial: impact of prior anti-CD38 exposure](https://medimix.be/hematology/eha-stockholm-2026-poster-2/) - In this video, Dr Albert Oriol discusses a subgroup analysis of the IRAKLIA trial in relapsed or refractory multiple myeloma.
- [The non-canonical function of EZH2 in T-ALL](https://medimix.be/hematology/eha-stockholm-2026-poster-1/) - In this video, Stef Van Den Bergh discusses his poster on the non-canonical function of EZH2 in T-cell acute lymphoblastic leukaemia.
- [ctDNA for risk stratification of SMM progression](https://medimix.be/hematology/eha-stockholm-2026-poster-6/) - In this video, Dr Sigrún Thorsteinsdóttir discusses the use of circulating tumour plasma cells for risk stratification and monitoring in smoldering multiple myeloma.
- [Blinatumomab for infants with KMT2A-rearranged ALL](https://medimix.be/hematology/eha-stockholm-2026-poster-5/) - Long-term follow-up data of a phase II, single arm interventional study presented by Dr Miguel Vieira at EHA 2026 provide evidence supporting the incorporation of blinatumomab into the frontline treatment for these patients.
- [MF symptoms following an immediate switch from ruxolitinib to momelotinib](https://medimix.be/hematology/eha-stockholm-2026-poster-12/) - In this video, Dr Jan Brijs discusses a post hoc analysis of the phase 3 SIMPLIFY-1 and SIMPLIFY-2 trials presented at the 2026 European Hematology Association (EHA) Congress.
- [Treating cytopenia in myelofibrosis: real-world data](https://medimix.be/hematology/eha-stockholm-2026-in-depth-23/) - In this video, Dr Adriano Salaroli discusses two poster presentations on myelofibrosis, with a particular focus on the use of momelotinib and the management of cytopenic disease.
- [Subcutaneous blinatumomab](https://medimix.be/hematology/eha-stockholm-2026-in-depth-22/) - New data presented at the 2026 annual meeting of the European Hematology Association (EHA)by Prof Dr Elias Jabbour highlight the potential of a new subcutaneous formulation of blinatumomab in patients with relapsed or refractory, Philadelphia chromosome (Ph)-positive acute lymphoblastic leukemia (ALL).
- [Updates from DREAMM-7 and DREAMM-8](https://medimix.be/hematology/eha-stockholm-2026-in-depth-21/) - In this video, Dr Marie Vercruyssen summarizes updated results from the pivotal phase III DREAMM-7 and DREAMM-8 trials presented during the 2026 annual meeting of the European Hematology Association. Both these studies are evaluating belantamab mafodotin-based regimens in patients with relapsed/refractory multiple myeloma (RRMM).
- [Risk factors for infection in multiple myeloma](https://medimix.be/hematology/eha-stockholm-2026-poster-7/) - At EHA 2026, Prof Cecilia Hveding Blimark presented findings from a population-based study investigating risk factors for infections in patients with multiple myeloma (MM).
- [The SENTRY trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-17/) - In this video, Prof Claire Harrison summarizes the key findings of this study and discusses their potential implications for the management of patients with MF.
- [The SEQUOIA trial: long-term follow-up](https://medimix.be/hematology/eha-stockholm-2026-in-depth-20/) - Long-term data from the SEQUOIA trial, presented at EHA 2026, provided a seven-year follow-up analysis of this pivotal randomized study evaluating zanubrutinib versus bendamustine plus rituximab in patients with previously untreated chronic lymphocytic leukemia (CLL).
- [The EPCORE FL-1 study: outcomes in clinically relevant subgroups](https://medimix.be/hematology/eha-stockholm-2026-in-depth-19/) - At the 2026 annual European Hematology Association (EHA) Congress, Prof Benoit Tessoulin presented an oral abstract discussing an updated subgroup analysis from EPCORE FL-1, a pivotal phase III study evaluating epcoritamab plus rituximab and lenalidomide (R²) versus R² alone in patients with relapsed or refractory follicular lymphoma (R/R FL).
- [CLL-associated RPS15 mutations rewire transcription through codon-specific tRNA accommodation defects](https://medimix.be/hematology/eha-stockholm-2026-in-depth-18/) - As a postdoctoral researcher at KU Leuven, Dr Marino Caruso is investigating the functional consequences of cancer-associated ribosomal protein mutations, with a particular focus on mutations in RPS15 that occur in chronic lymphocytic leukemia (CLL).
- [The MonumenTAL-3 study](https://medimix.be/hematology/eha-stockholm-2026-in-depth-15/) - In this video, Prof Dr Claudio Cerchione discusses the plenary presentation of the phase III MonumenTAL-3 trial at EHA 2026. The study evaluated talquetamab-based combinations in earlier lines of therapy for patients with relapsed/refractory multiple myeloma (RRMM), demonstrating significant improvements in efficacy while maintaining a manageable safety profile.
- [The SUCCESSOR-2 trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-13/) - In this video, Prof Meletios Dimopoulos and Dr Marie Vercruyssen discuss the groundbreaking results of the phase III SUCCESSOR-2 trial, which were presented as a late breaking abstract during the 2026 annual EHA meeting.
- [The ImmunoPRISM study](https://medimix.be/hematology/eha-stockholm-2026-in-depth-16/) - During the European Hematology Association (EHA) 2026 Congress, Prof Omar Nadeem presented the results of the ImmunoPRISM trial as a late-breaking abstract. This phase II study is comparing the efficacy and safety of the BCMA-directed bispecific antibody teclistamab to lenalidomide plus dexamethasone in patients with high-risk smoldering multiple myeloma (SMM).
- [EPCORE NHL-1: long-term follow-up data](https://medimix.be/hematology/eha-stockholm-2026-in-depth-14/) - At the 2026 annual meeting of the European Hematology Association (EHA), Prof Catherine Thieblemont presented long-term follow-up results from the phase II EPCORE NHL-1 study evaluating epcoritamab monotherapy in patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) after at least two prior lines of systemic therapy.
- [The BRUIN CLL-322 trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-12/) - In this video, Prof Matthew Davids, hematologist summarizes the key findings and discusses their potential implications for the treatment landscape of relapsed or refractory CLL.
- [The BlinPredict Risk model for pediatric B-ALL patients](https://medimix.be/hematology/eha-stockholm-2026-in-depth-11/) - During the 2026 European Hematology Association (EHA) congress, the BlinPredict model was presented as a novel risk stratification tool developed from a real-world UK cohort of patients with acute lymphoblastic leukemia (ALL) treated with frontline blinatumomab in combination with reduced-intensity chemotherapy.
- [The LATE-R trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-10/) - During EHA 2026 in Stockholm, Dr Mariana Bastos-Oreiro presented preliminary results from the phase 2 LATE-R trial. This trial is evaluating CAR T-cell therapy in patients with aggressive B-cell lymphoma experiencing a late relapse following frontline treatment.
- [Updated results of the COMMANDS-trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-9/) - Updated results from the phase 3 COMMANDS trial, presented by Prof Dr Valeria Santini demonstrated sustained clinical benefit with luspatercept compared with epoetin alfa in erythropoiesis-stimulating agent (ESA)-naive patients with transfusion-dependent lower-risk myelodysplastic syndromes (LR-MDS).
- [The COG AALL1731 study](https://medimix.be/hematology/eha-stockholm-2026-in-depth-8/) - During the 2026 European Hematology Association (EHA) meeting in Stockholm, Dr Sumit Gupta presented findings from the Children's Oncology Group (COG) AALL1731 trial, focusing on the prognostic significance of high-throughput sequencing (HTS)-based minimal residual disease (MRD) assessment in pediatric standard-risk B-cell acute lymphoblastic leukemia (ALL).
- [The AGMT MM04 trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-6/) - During the 2026 annual meeting of the European Hematology Association (EHA), Prof Heinz Ludwig presented results from a prospective, randomized international study (AGMT MM04) evaluating the addition of isatuximab to lenalidomide and dexamethasone (Rd) in elderly patients with newly diagnosed multiple myeloma (NDMM).
- [The ASC4FIRST study](https://medimix.be/hematology/eha-stockholm-2026-in-depth-7/) - In this video, Dr Adriano Salaroli discusses these important findings with Prof Dr Timothy Hughes ASC4FIRST represents the first direct comparison of asciminib with established frontline tyrosine kinase inh
- [Sequencing therapy in DLBCL: the issue of preserving CD19 expression](https://medimix.be/hematology/eha-stockholm-2026-in-depth-2/) - In this video recorded during the 2026 annual meeting of the European Hematology Association, Dr Johannes Düll discusses the evolving treatment landscape of relapsed or refractory diffuse large B-cell lymphoma (DLBCL), with a particular focus on the role of tafasitamab and its sequencing with other CD19-directed therapies.
- [KITE-753 for R/R LBCL](https://medimix.be/hematology/eha-stockholm-2026-in-depth-5/) - As explained in this video by Dr Sairah Ahmed, KITE-753 incorporates a unique bicistronic construct with 4-1BB and CD28 costimulatory domains designed to enhance target binding, promote T-cell proliferation, and support the generation of naïve and memory T-cell populations through a rapid manufacturing process.
- [The ALIDHE trial](https://medimix.be/hematology/eha-stockholm-2026-in-depth-4/) - In this in depth video interview, Dr Remco Molenaar shares the updated results of the ALIDHE trial presented during the 2026 annual EHA meeting.
- [The evolving role of immunotherapy in adult ALL](https://medimix.be/hematology/eha-stockholm-2026-in-depth-3/) - During an interview at the European Hematology Association (EHA) 2026 Congress, Prof Sabina Chiaretti discussed the evolving role of immunotherapy in the treatment of adult acute lymphoblastic leukemia (ALL), highlighting its growing integration into first-line therapy and its potential to substantially reduce the reliance on conventional chemotherapy.
- [The FrontMIND study](https://medimix.be/hematology/eha-stockholm-2026-in-depth-1/) - In this video, Prof Lenz discusses these findings with Dr Adriano Salaroli. FrontMIND evaluated the addition of tafasitamab and lenalidomide to R-CHOP in previously untreated patients with high-risk diffuse large B-cell lymphoma (DLBCL), aiming to improve outcomes beyond the long-standing standard of care.
- [Follicular lymphoma](https://medimix.be/hematology/eha-stockholm-2026-highlight-6/) - During the 2026 annual meeting of the European Hematology Association (EHA), Dr Umut Yilmaz co-chaired the oral abstract session on follicular lymphoma. In this highlights video, Dr Yilmaz shares his key takeaways from this session.
- [Diffuse large B-cell lymphoma](https://medimix.be/hematology/eha-stockholm-2026-highlight-5/) - In this video summarizing key findings in diffuse large B-cell lymphoma (DLBCL) presented at EHA 2026, Dr Gilles Crochet highlighted four studies addressing frontline treatment optimization, innovative treatment strategies for patients with relapsed or refractory disease, the management of elderly patients, and the use of bispecific antibodies as a bridge to CAR T-cell therapy.
- [Myeloproliferative neoplasms](https://medimix.be/hematology/eha-stockholm-2026-highlight-4/) - In this highlights video from the 2026 annual meeting of the European Hematology Association (EHA) Dr Adriano Salaroli discussed four studies that illustrate ongoing therapeutic advances across myeloproliferative neoplasms.
- [Multiple myeloma & other plasma cell disorders](https://medimix.be/hematology/eha-stockholm-2026-highlight-1/) - In this highlights video from EHA 2026, Dr Marie Vercruyssen Haematologist discusses six studies illustrating the expanding role of bispecific antibodies across the spectrum of plasma cell disorders.
- [Myelodysplatic syndromes](https://medimix.be/hematology/eha-stockholm-2026-highlight-3/) - For this highlights video from EHA 2026, Prof Dr Marie-Christiane Vekemans reviews five studies that highlight emerging therapeutic strategies for patients with low-risk and high-risk myelodysplastic syndromes (MDS).
- [Chronic lymphocytic leukemia](https://medimix.be/hematology/eha-stockholm-2026-highlight-2/) - In this highlights video from EHA 2026, Prof Dr Virginie De Wilde reviews the evolving treatment landscape of chronic lymphocytic leukemia (CLL), highlighting recent developments that further consolidate fixed-duration therapies as a preferred first-line approach for most patients and discussing emerging strategies that may redefine future treatment standards
- [SEQUOIA](https://medimix.be/hematology/sequoia/) - Zanubrutinib as an effective 1st line treatment for CLL, irrespective of del(17p) status
- [SEQUOIA & ALPINE](https://medimix.be/hematology/sequoia-alpine/) - Zanubrutinib in the treatment of chronic lymphocytic leukemia Six-year results from SEQUOIA & ALPINE with Long Term Extension
- [Clinical benefit of luspatercept in ESA-naive patients with very low-, low-, or intermediate-risk MDS: post-hoc data from COMMANDS](https://medimix.be/hematology/ash-orlando-2025-in-depth-21/) - At ASH 2025, Prof Dr Valeria Santini presented the results of an ad hoc analysis of the COMMANDS trial focusing on clinically meaningful predictors of response to first-line therapy in transfusion-dependent patients with lower-risk myelodysplastic syndromes (MDS).
- [Highlights in Multiple Myeloma](https://medimix.be/hematology/ash-orlando-2025-highlight-7/) - In this video, Dr Marie Vercruyssen, hematologist at the Institut Jules Bordet in Brussels shares the key takeaways from 4 presentations related to multiple myeloma (MM) presented at ASH 2025.
- [Real-world data with ivosidenib-azacitidine as 1st line treatment for unfit AML patients](https://medimix.be/hematology/ash-orlando-2025-poster-11/) - At ASH 2025, Prof Dr Joshua Zeidner presented a poster reporting results from a real-world analysis of frontline treatment with ivosidenib plus azacitidine (IVO-AZA) in older, unfit patients with IDH1-mutated acute myeloid leukemia (AML). This analysis addresses an important clinical question, as the optimal initial treatment strategy for this patient population remains uncertain.
- [inMMyCAR: in vivo CAR-T therapy for multiple myeloma](https://medimix.be/hematology/ash-orlando-2025-in-depth-20/) - At ASH 2025, Prof Dr Phoebe Joy Ho presented a late-breaking abstract at ASH 2025 describing a first-in-human clinical trial of in vivo CAR T-cell therapy for multiple myeloma.
- [Overall survival in mCRC patients with persistent chemotherapy-induced thrombocytopenia](https://medimix.be/hematology/ash-orlando-2025-poster-15/) - In this video, Prof Hanny Al-Samkari, hematologist and clinical investigator, summarized findings from a poster presented at ASH 2025 examining overall survival (OS) in patients with metastatic colorectal cancer (mCRC) who develop persistent chemotherapy-induced thrombocytopenia (CIT).
- [BVd or BPd for functional high-risk MM: subgroup data from DREAMM-7 & DREAMM-8](https://medimix.be/hematology/ash-orlando-2025-poster-14/) - In this video, Prof Vania Hungria, hematologist, summarizes the results from a subgroup analysis of the phase III DREAMM-7 and DREAMM-8 studies, evaluating belantamab mafodotin–based combinations in patients with relapsed or refractory multiple myeloma (RRMM).
- [Isatuximab-Rd vs. Rd induction, followed by isa-R vs. R maintenance in very elderly patients with NDMM: results of AGMT-MM04](https://medimix.be/hematology/ash-orlando-2025-poster-13/) - At ASH 2025, Prof Heinz Ludwig, haematologist presented a poster describing the results of a randomized phase II AGMT-MM04 study. This trial evaluated the efficacy and safety of isatuximab plus lenalidomide–dexamethasone (Isa-Rd) versus lenalidomide–dexamethasone (Rd) alone in very elderly patients with newly diagnosed multiple myeloma (NDMM).
- [Subcutaneous blinatumomab for relapsed/refractory ALL](https://medimix.be/hematology/ash-orlando-2025-poster-12/) - At the 2025 American Society of Hematology (ASH) Annual Meeting, Prof Elias Jabbour, hematologist, presented updated results from a phase 1b study evaluating subcutaneous blinatumomab in patients with relapsed or refractory acute lymphoblastic leukemia (R/R ALL).
- [Highlights in Acute Myeloid Leukemia](https://medimix.be/hematology/ash-orlando-2025-highlight-6/) - In this video, Dr Fabio Andreozzi, hematologist at the Institut Jules Bordet in Brussels and doctoral researcher at the laboratory of molecular biology of leukemia at the VIB-KU Leuven, provides an overview of selected highlights in acute myeloid leukemia from ASH 2025.
- [The VAYHIT2 study: ianalumab + eltrombopag for ITP after corticosteroid failure](https://medimix.be/hematology/ash-orlando-2025-in-depth-19/) - In this video, Dr Hanny Al-Samkari summarized results from the VAYHIT2 study, a pivotal randomized, placebo-controlled trial presented as a Late-Breaking Abstract at ASH 2025.
- [iMMagine-1: anitocabtagene autoleucel for relapsed/refractory multiple myeloma](https://medimix.be/hematology/ash-orlando-2025-in-depth-18/) - ASH 2025 featured a key update from the phase II iMMagine-1 study, evaluating the BCMA-targeting CAR T construct anitocabtagene autoleucel (anito-cel) in patients with relapsed/refractory (R/R) multiple myeloma (MM). In this video, Dr Krina Patel, Associate Professor and Multiple Myeloma Section Chief, shares the key takeaways from this trial.
- [MajesTEC-3: teclistamab + daratumumab for relapsed/refractory multiple myeloma](https://medimix.be/hematology/ash-orlando-2025-in-depth-16/) - In this video, Dr Alain Kentos, hematologist at Jolimont Hospital in La Louvière (HELORA Hospital Group), shares the key takeaways from this presentation in addition to a critical interpretation of the data in a Belgian clinical context.
- [The CLL17 study](https://medimix.be/hematology/ash-orlando-2025-in-depth-15/) - The CLL17 trial is a randomized, phase III study evaluating two major therapeutic paradigms in previously untreated chronic lymphocytic leukemia. These results were presented in the Plenary Session of ASH 2025 by Dr Othman Al-Sawaf.
- [The GRAALL/B-QUEST study](https://medimix.be/hematology/ash-orlando-2025-in-depth-17/) - In this video, Prof Carlos Graux, hematologist at CHU UCL Namur, discusses the key results of the GRAALL/B-QUEST study presented during ASH 2025.
- [Highlights in myeloproliferative neoplasms](https://medimix.be/hematology/ash-orlando-2025-highlight-5/) - In this daily highlights video, Dr Adriano Salaroli discusses several practice-changing advances in myeloproliferative neoplasms (MPNs) and chronic myeloid leukemia (CML) presented at ASH 2025.
- [Liso-cel in the treatment of R/R DLBCL: long-term follow-up of the TRANSFORM trial](https://medimix.be/hematology/ash-orlando-2025-poster-4/) - At ASH 2025, Prof Dr Manali Kamdar presented the four-year follow-up results from the TRANSFORM study, a global randomized phase III trial evaluating lisocabtagene maraleucel (liso-cel) in patients with primary refractory or early relapsing diffuse large B-cell lymphoma following frontline chemoimmunotherapy.
- [MARVIN](https://medimix.be/hematology/ash-orlando-2025-poster-10/) - During ASH 2025, Dr Adrien de Voeght, hematologist, presented results of MARVIN, a deep generative artificial intelligence model specifically designed to analyze high-dimensional flow cytometry data. 
- [Thiotepa dosing following haplo-HSCT in AML patients](https://medimix.be/hematology/ash-orlando-2025-poster-9/) - In a poster presented at ASH 2025, Dr Fabio Andreozzi, hematologist, reported the results of a retrospective, registry-based study conducted within the Acute Leukemia Working Party of the EBMT, evaluating the impact of busulfan and thiotepa dosing on outcomes after matched identical allogeneic stem cell transplantation in patients with acute myeloid leukemia (AML) in first or second complete remission.
- [Outcomes in t(6;9)-positive AML following an allo-SCT](https://medimix.be/hematology/ash-orlando-2025-poster-8/) - In a poster at ASH 2025, Dr Fabio Andreozzi, hematologist, presented the results of a retrospective registry-based study within the EBMT, evaluating outcomes of allogeneic stem cell transplantation in patients with acute myeloid leukemia harboring the t(6;9) translocation.
- [GMMG-HD7: health related QoL](https://medimix.be/hematology/ash-orlando-2025-poster-7/) - At ASH 2025, quality-of-life (QoL) data of this trial were presented, addressing an important clinical question in the treatment of newly diagnosed, transplant-eligible multiple myeloma: does intensification from a triplet to a quadruplet regimen compromises patient-reported outcomes? The main message from this poster are summarized by Prof Tobias Slørdahl, hematologist at the Norwegian University of Science and Technology in Trondheim (Norway)
- [JAK inhibition in the treatment of myelofibrosis](https://medimix.be/hematology/ash-orlando-2025-in-depth-14/) - In this video, Dr Michael Loschi from Nice University Hospital in France provides a brief overview of how JAK inhibitors have changed the treatment landscape for myelofibrosis (MF) over the last decade.
- [FRONTIER2: patient reported outcomes with Mim8](https://medimix.be/hematology/ash-orlando-2025-in-depth-13/) - The FRONTIER2 study evaluates a novel bispecific antibody designed to mimic factor VIII activity for the treatment of patients with severe hemophilia. In this video, Prof Cedric Hermans, hematologist, shares his key takeaways from these presentations.
- [Altered metabolisms of platelet disorders](https://medimix.be/hematology/ash-orlando-2025-in-depth-12/) - At ASH 2025, Prof Kathleen Freson presented new insights into the biology of Glanzmann thrombasthenia, the most common inherited platelet function disorder
- [ALIDHE: ivosidenib + azacitidine for adult patients with IDH1-mutant AML](https://medimix.be/hematology/ash-orlando-2025-in-depth-11/) - During ASH 2025, results were presented of the phase 3b ALIDHE trial, a real-world follow-up study of AGILE. In this video, Dr Koen Theunissen (Jessa Hospital, Hasselt) shares his key takeaways from this poster.
- [BCMA targeting for patients with multiple myeloma in 1st relapse](https://medimix.be/hematology/ash-orlando-2025-in-depth-10/) - In this video, Dr Koen Theunissen shares his perspective on the evolving role of belantamab mafodotin within this rapidly changing treatment landscape and discusses how BCMA-targeted approaches are reshaping clinical decision-making in relapsed multiple myeloma.
- [The IRAKLIA trial: new insights on the use of subcutaneous isatuximab in multiple myeloma](https://medimix.be/hematology/ash-orlando-2025-in-depth-9/) - In this video, Prof Sikandar Ailawadhi shares updated results from the IRAKLIA trial presented at ASH 2025.
- [KITE-363 and KITE-753: dual CD19/CD20 targeting CAR-T constructs for the treatment of LBCL](https://medimix.be/hematology/ash-orlando-2025-in-depth-7/) - During ASH 2025, Dr Saurabh Dahiya presented updated efficacy data with KITE-363 in addition to initial safety and preliminary efficacy results with KITE-753.
- [CLL & indolent lymphoma](https://medimix.be/hematology/ash-orlando-2025-highlight-4/) - In this daily highlights video, Dr Fulvio Massaro (Institut Jules Bordet, Brussels) shares the key takeaways from a selection of impactful ASH presentations related to chronic lymphocytic leukemia (CLL) and indolent non-Hodgkin lymphoma (NHL).
- [ZUMA-25: brexucabtagene autoleucel for patients with R/R Burkitt lymphoma](https://medimix.be/hematology/ash-orlando-2025-in-depth-8/) - At ASH 2025, results from the ZUMA-25 trial, sub-study C, evaluating brexucabtagene autoleucel (brexu-cel), an autologous anti-CD19 CAR T-cell therapy, in patients with relapsed or refractory Burkitt lymphoma were presented. In the video, Dr Suzanne Van Dorp, hematologist at the Radboud UMC in Nijmegen, discusses the key takeaways from this presentation.
- [GIMEMA ALL2820: ponatinib + blinatumomab for adults with Ph+ ALL](https://medimix.be/hematology/ash-orlando-2025-in-depth-6/) - At ASH 2025, Prof Sabina Chiaretti presented the first results of the phase III GIMEMA ALL2820 trial, a landmark study evaluating a chemotherapy-free regimen in adult patients with newly diagnosed Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL). The objective of the study was to formally demonstrate that a chemo-free strategy combining ponatinib and blinatumomab could improve the event-free (EFS) and overall survival (OS) in this setting compared to the historical standard of care consisting of imatinib plus chemotherapy.
- [Blinatumomab partly mitigates the impact of traditional adverse prognosticators among children standard risk B-ALL: updated results from AALL 1731](https://medimix.be/hematology/ash-orlando-2025-in-depth-5/) - At ASH 2025, Dr Sumit Gupta, pediatric oncologist, presented updated results of the Children’s Oncology Group study ALL 1731 evaluating the long-term impact of incorporating blinatumomab into the frontline treatment for children with standard-risk, B-cell acute lymphoblastic leukemia (B-ALL).
- [Toxicity-free progression-fee survival and complete response at day 100: new endpoints for the evaluation of CAR-T therapy in DLBCL](https://medimix.be/hematology/ash-orlando-2025-in-depth-4/) - To this end, Prof Veronika Bachanova, hematologist, and colleagues developed 2 new composite endpoints combining efficacy and toxicity in the first 100 days post CAR T: toxicity-free complete response at day 100 (tfCR100) and toxicity-free, progression free survival at day 100 (tfPFS100).
- [Highlights in acute lymphoblastic leukemia](https://medimix.be/hematology/ash-orlando-2025-highlight-3/) - While ASH 2025 did not feature any transformative breakthroughs in the field of acute lymphoblastic leukemia (ALL), Dr Koen Theunissen did find three interesting studies that may provide meaningful progress in the management of this disease
- [Brexu-cel in R/R MCL: 2-year update of ZUMA-2 cohort 3](https://medimix.be/hematology/ash-orlando-2025-poster-6/) - Cohort 3 of the phase II ZUMA-2 trial has been designed to evaluate the safety and efficacy of brexucabtagene autoleucel (brexu-cel), an autologous anti-CD19 CAR T-cell therapy, in adults with BTK inhibitor–naive relapsed or refractory (R/R) mantle cell lymphoma (MCL).
- [IgG2a-formatted 4-1BB agonism combined with S100A9 inhibition enhances T cell activation and tumor control in a preclinical model of multiple myeloma](https://medimix.be/hematology/ash-orlando-2025-poster-5/) - In a poster at ASH 2025, Hatice Satilmis (Vrije Universiteit Brussel) presented the results of her PhD project investigating strategies to enhance durable T-cell responses in multiple myeloma, a disease in which relapse remains common despite the availability of CD38-targeting antibodies, bispecific T-cell engagers, and CAR T-cell therapies.
- [Steroid toxicity in newly diagnosed patients with multiple myeloma treated with a limited dexamethasone regimen](https://medimix.be/hematology/ash-orlando-2025-poster-3/) - An analysis presented as a poster at ASH 2025 evaluated the impact of omitting dexamethasone on patient reported steroid toxicity in this trial.
- [Efficacy of Isa-VRd in older and/or frail patients with multiple myeloma](https://medimix.be/hematology/ash-orlando-2025-poster-2/) - In this light, Dr Enrique Ocio, presented the results of an analysis looking into the efficacy and safety of the quadruplet combination of isatuximab, bortezomib, lenalidomide and dexamethasone (Isa-VRd) in older multiple myeloma patients with, or without frailty.
- [Liso-cel as 2nd line treatment for LBCL: real-world data from the French DESCAR-T registry](https://medimix.be/hematology/ash-orlando-2025-poster-1/) - During ASH 2025, Dr Gabriel Brisou presented updated, real-world data on the outcomes of French patients with large B-cell lymphoma treated with second line lisocabtagene maraleucel (Liso-cel) in the context of an Early Access Program.
- [The role of SOX-11 in the development of MCL and γδ-like T-ALL](https://medimix.be/hematology/ash-orlando-2025-in-depth-2/) - At the ASH 2025 meeting, Dr Tim Pieters, had two presentations centered on the pioneering transcription factor SOX11 and its role in the pathogenesis of different hematologic malignancies.
- [Zanubrutinib in the treatment of CLL: long-term results from ALPINE & SEQUOIA](https://medimix.be/hematology/ash-orlando-2025-in-depth-3/) - The SEQUOIA and ALPINE trials established zanubrutinib as a standard of care treatment for patients with chronic lymphocytic leukemia (CLL), both in the treatment-naïve and relapsed setting. During ASH 2025, Prof Constantine Tam, presented 6-year follow-up data of both studies.
- [Axi-cel vs. liso-cel as 2 nd line treatment for LBCL: real world evidence from theDESCAR-T registry](https://medimix.be/hematology/ash-orlando-2025-in-depth-1/) - In the absence of a prospective head-to-head comparison of both therapies, Gabriel Brisou and colleagues performed a comparative analysis using real-world data from the DESCAR-T registry.
- [Highlights in Haemophilia and Haemostasis](https://medimix.be/hematology/ash-orlando-2025-highlight-2/) - In this video, Prof Cédric Hermans, haematologist at the Cliniques Universitaires Saint-Luc in Brussels summarizes his key take aways form the meeting related to haemophile and haemostasis.
- [CAR-T therapy for aggressive lymphoma](https://medimix.be/hematology/ash-orlando-2025-highlight-1/) - In this daily highlights video from ASH 2025, Prof Koen Debackere offers an integrated scientific overview of the most relevant advances in CAR-T therapy for aggressive lymphoma. In recent years, CD19-directed CAR-T therapies such as axicabtagene ciloleucel and lisocabtagene maraleucel have reshaped the treatment landscape for relapsed or refractory (R/R) large B-cell lymphoma (LBCL), yet approximately half of treated patients still relapse. The developments showcased at ASH 2025 illustrate how the next generation of CAR-T constructs is being engineered to address this unmet need through enhancements in antigen targeting, manufacturing platforms, and graft composition.
- [Belgian real-world date on the use of teclistamab in RRMM](https://medimix.be/hematology/ims-2025-poster-4/) - During the 2025 IMS meeting, Prof Dr Marie-Christiane Vekemans shared the real-world experience with teclistamab in the treatment of relapsed/refractory multiple myeloma (RRMM) patients across three centres in Belgium
- [Transitioning from SMM to MM](https://medimix.be/hematology/ims-2025-in-depth-8/) - During the 2025 IMS meeting in Toronto, Prof Dr Sigurdur Kristinsson gave an educational talk on the factors determining the risk of progression, apart from genetics and the microenvironment.
- [Immune remodeling after Isa-KRd in newly diagnosed multiple myeloma](https://medimix.be/hematology/ims-2025-in-depth-7/) - In the plenary session of IMS 2025, Dr Ludovic Martinet presented the result of his research into the response of the immune system following quadruplet therapy in multiple myeloma (MM) patients. In these quadruplets, an immunomodulatory drug is combined with a proteasome inhibitor, an anti-CD38 antibody, and dexamethasone. While several studies have convincingly demonstrated that these regimens are highly effective, their precise impact on the immune system remains unclear. Gaining more insights into this effect is of particular relevance given the role of the immune environment as a foundation for future T-cell-based immunotherapies such as CAR-T therapy and bispecific antibodies.
- [Impact of MRD negativity on QoL](https://medimix.be/hematology/ims-2025-in-depth-5/) - At the 2025 IMS meeting, Prof Dr Jesús San-Miguel presented a new analysis evaluating the impact of MRD negativity on patients treated in the pivotal PERSEUS and CEPHEUS studies. In these trials, patients were randomized to receive either daratumumab, bortezomib, lenalidomide, and dexamethasone (DVRd) induction/consolidation followed by daratumumab and lenalidomide (DR) maintenance, or the standard regimen of bortezomib, lenalidomide, and dexamethasone (VRd) with lenalidomide (R) maintenance (PERSEUS), or DVRd vs. VRd alone (CEPHEUS).
- [Patient preferences and treatment decisions at relapse](https://medimix.be/hematology/ims-2025-in-depth-6/) - During the 2025 IMS meeting, Dr Lisa Leypoldt, haematologist at the University Medical Center in Hamburg addressed the critical topic of patient preferences at relapse in multiple myeloma care.
- [IZALCO & IRAKLIA](https://medimix.be/hematology/ims-2025-poster-2/) - At the 2025 annual IMS meeting, several presentations highlighted the use of the new on-body injector (OBI) designed to facilitate the administration of the subcutaneous (SC) formulation of isatuximab. In this video, Prof Vania Hungria shares the key takeaways.
- [IRAKLIA study](https://medimix.be/hematology/ims-2025-poster-3/) - According to Prof Claudio Cerchione these findings are highly relevant—particularly given the growing role of isatuximab in the evolving therapeutic landscape of multiple myeloma.
- [DREAMM-7 study – subgroup analysis in lenalidomide-refractory patients](https://medimix.be/hematology/ims-2025-poster-1/) - During IMS 2025, Prof Dr Vania Hungria presented the results of a subgroup analysis of this trial, focussing on lenalidomide-refractory patients. The results in this subgroup were in line with the overall DREAMM-7 results, with a median PFS of 35.7 months for BVd-treated patients as compared to 13.5 months with DVd (HR[95%CI]: 0.39[0.17-0.88]). In addition, BVd was associated a higher rate of deep responses and a significantly longer duration of response compared to DVd.
- [When to initiate treatment in a relapsing MM patient?](https://medimix.be/hematology/ims-2025-in-depth-4/) - During the 2025 IMS meeting in Toronto, Prof Fredrik Schjesvold addressed the increasingly relevant question when to start a treatment for a multiple myeloma (MM) patient with a disease relapse. In fact, as clinicians become more focused on achieving and sustaining measurable residual disease (MRD) negativity in frontline therapy, the question has become what to if MRD pops up in a patient who was initially MRD negative. Should this patient immediately start treatment or is it ok to wait until a true biochemical relapse is detected
- [The future of multiple myeloma care](https://medimix.be/hematology/ims-2025-in-depth-3/) - Inspired by the wealth of new data and insights presented at the 2025 IMS Meeting, Prof Dr Claudio Cerchione shares his outlook on the future of myeloma care.
- [The evolving role of BCMA targeted therapies in MM](https://medimix.be/hematology/ims-2025-in-depth-2/) - In this video, Dr Marie Vercruyssen shares her insights on the shifting role of BCMA targeted therapy in the management of multiple myeloma (MM).
- [Revised diagnostic criteria for ligh-chain MGUS](https://medimix.be/hematology/ims-2025-in-depth-1/) - During IMS 2025, Dr Laerke Sloth Andersen presented the results of a study that validate these revised diagnostic criteria for LC-MGUS. Using the Danish nationwide "Danish Lymphoid Cancer Research" (DaLyCaRe) dataset, the study included patients diagnosed with MGUS without heavy chain involvement, excluding those with a lymphoproliferative disorder diagnosis prior to or within 90 days of the MGUS diagnosis. Only individuals with free light chain measurements from the same assay used to define the new reference intervals, and with recent kidney function data, were included.
- [Precursor disease](https://medimix.be/hematology/ims-2025-highlight-4/) - In this video, Dr Marie Vercruyssen summarizes the key takeaways from these discussions. While IMS 2025 did not feature any true practice changing presentations related to precursor stages of multiple myeloma (MM), several educational sessions featured animated discussions on the optimal management of patients with (high-risk) smoldering multiple myeloma (SMM).
- [Relapsed refractory multiple myeloma](https://medimix.be/hematology/ims-2025-highlight-3/) - In this video, Prof Dr Marie-Christiane Vekemans shares her key takeaways from the 2025 IMS meeting, focussing on the management of relapsed refractory multiple myeloma (RRMM).
- [Plenary & Late breaking session](https://medimix.be/hematology/ims-2025-highlight-1/) - In this video, Dr Nicolas Kint shares his key takeaways from these two sessions. At the IMS 2025 meeting in Toronto, the focus of the scientific community was clearly shifting from managing relapsed/refractory disease to treating earlier stages of multiple myeloma (MM) and improving frontline therapy. This transition was also apparent in the plenary and late breaking abstract session of the meeting.
- [Newly diagnosed multiple myeloma](https://medimix.be/hematology/ims-2025-highlight-2/) - In this video, Dr Isabelle Vande Broek, shares key takeaways from four important presentations from IMS 2025 focused on the management of NDMM.
- [New EHA/EMN treatment guidelines for MM](https://medimix.be/hematology/eha-milan-2025-in-depth-17/) - In this video, Prof Michel Delforge talks us through the most important changes in these updated guidelines relative to the previous edition released in 2021.
- [Updated efficacy results of DREAMM-8](https://medimix.be/hematology/eha-milan-2025-in-depth-5/) - In this video, Dr Rutger Callens summarizes the key take-aways from this update and reflects on the impact of these findings on our Belgian clinical practice.
- [FYN: TRAF3IP2 fusion in PTCL-NOS](https://medimix.be/hematology/eha-milan-2025-poster-4/) - In a poster presented at EHA 2025 by Yilke Schoenmaekers, PhD student at the laboratory of experimental hematology of the KU Leuven, a mouse model was used to demonstrate how the presence of this FYN::TRAF3IP2 fusion led to changes in the tumor microenvironment (TME) of PTCL-NOS.
- [Cognitive and psychometric predictors of return to work after allo-HSCT](https://medimix.be/hematology/eha-milan-2025-poster-8/) - Quality of life plays a crucial role in the ability of long-term survivors of allogeneic stem cell transplantation (allo-HSCT) to return to work (RTW). The SOPRALLO project investigates long-term outcomes in patients who underwent allo-HSCT at the Jules Bordet Institute and remained in remission for at least one year posttransplant.
- [ISKIA](https://medimix.be/hematology/eha-milan-2025-in-depth-16/) - The ISKIA trial is a phase 3 study that investigated the sustained MRD negativity in patients with newly diagnosed multiple myeloma who were treated with a combination of carfilzomib, lenalidomide, and dexamethasone (KRd) with or without isatuximab (Isa).
- [SEQUOIA 5-year follow-up in Arm C](https://medimix.be/hematology/icml-lugano-2025-in-depth-1/) - At ICML Lugano, Prof Dr Shadman presented updated results from SEQUOIA ARM C cohort, in patients with del(17p) after approximately 5 years of follow-up.
- [Lisocabtagene maraleucel for 2nd line DLBCL: real-world data from the French DESCAR-T registry](https://medimix.be/hematology/icml-lugano-2025-poster-4/) - During the 2025 ICML meeting, Dr Gabriel Brisou presented French real-world data of DLBCL patients treated with 2nd line liso-cel in the French DESCAR-T registry.
- [Bendamustine as Lymphodepleting Chemotherapy Prior to Axicabtagene Ciloleucel for the Treatment of Relapsed / Refractory Large B-Cell Lymphoma](https://medimix.be/hematology/icml-lugano-2025-in-depth-2/) - Dr Alaa Ali presents an analysis from the CIBMTR registry, comparing the real-world safety and effectiveness of single-agent bendamustine versus standard fludarabine/cyclophosphamide (Flu/Cy) as lymphodepleting chemotherapy (LDC) before axicabtagene ciloleucel (axi-cel) for the treatment of relapsed/refractory large B-cell lymphoma.
- [Tafasitamab + lenalidomide in R/R DLBCL](https://medimix.be/hematology/icml-lugano-2025-in-depth-3/) - During this ICML, Dr Bailly and Dr Johannes Düll had an interesting discussion around the combination of tafasitamab and lenalidomide, an FDA and EMA-approved treatment for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in patients who are not candidates for autologous stem cell transplantation (ASCT).
- [SWOG S1826 TRIAL](https://medimix.be/hematology/icml-lugano-2025-in-depth-4/) - At the ICML meeting, the results from the S1826 study were discussed, which focused on patients with advanced-stage Hodgkin Lymphoma (HL). This randomised phase 3 study compared brentuximab vedotin plus AVD (control) with nivolumab plus AVD for six cycles. The study previously showed that nivolumab-AVD improved PFS compared to brentuximab vedotin plus AVD and was better tolerated.
- [Phase 2 TRANSCEND FL study](https://medimix.be/hematology/icml-lugano-2025-in-depth-5/) - At ICML 2025 in Lugano, new data from the phase 2 TRANSCEND study for lisocabtagene maraleucel (liso-cel) CAR-T cell therapy for indolent lymphoma were communicated.
- [Real-world outcomes of axi-cel](https://medimix.be/hematology/icml-lugano-2025-in-depth-6/) - Dr Allison Winter presented at ICML 2025 data that highlight a collaboration between KITE and the CIBMTR data registry, examining the outcomes of axicabtagene ciloleucel (axi-cel) for the treatment of patients with Richter's transformation to large B-cell lymphoma (Richter). Richter is an aggressive form of lymphoma that arises from chronic lymphocytic leukaemia (CLL), and historically, patients with this condition have had poor outcomes, with median overall survival often reported as low as five months, especially for clonally related Richter.
- [Axicabtagene Ciloleucel (axi-cel) in Relapsed/Refractory Central Nervous System Lymphoma (CNSL)](https://medimix.be/hematology/icml-lugano-2025-poster-3/) - At ICML 2025, Prof Caron Jacobson presented a poster on a pilot study evaluating axicabtagene ciloleucel (axi-cel) in patients with primary and secondary CNS lymphoma, primarily in the second line and beyond. The study involved 18 patients, most of whom had refractory disease, with a mix of primary (two-thirds) and secondary (one-third) CNS lymphoma.
- [Real-world outcomes with brexucabtagene-autoleucel in R/R mantle cell lymphoma](https://medimix.be/hematology/icml-lugano-2025-poster-2/) - During the 2025 ICML meeting, Prof Olalekan Oluwole shared the results of a systematic literature review and meta-analysis evaluating real-world outcomes for patients with relapsed/refractory mantle cell lymphoma (R/R MCL) treated with brexucabtagene-autoleucel (brexu-cel).
- [SEQUOIA – Results in Arm D](https://medimix.be/hematology/icml-lugano-2025-poster-1/) - The SEQUOIA Arm D study explored the combination of zanubrutinib and venetoclax for untreated CLL/SLL patients, including those with abnormal TP53. Both normal and abnormal TP53 cohorts showed high response rates, with a 60% undetectable minimal residual disease (uMRD) rate.
- [Hodgkin Lymphoma](https://medimix.be/hematology/icml-lugano-2025-highlights-7/) - At the 18th ICML in Lugano, Dr Fulvio Massaro highlighted notable presentations on the management of Hodgkin lymphoma, with the primary focus of interest being the integration of novel immunotherapies in the earlier phases of the disease.
- [NK/T cell lymphoma](https://medimix.be/hematology/icml-lugano-2025-highlights-6/) - At the 18th ICML held in Lugano, Dr Alice Wolfromm highlighted some interesting presentations regarding the management of NK/T-cell lymphoma.
- [Mantle Cell Lymphoma](https://medimix.be/hematology/icml-lugano-2025-highlights-5/) - At the 18th ICML in Lugano, Prof Dr Virginie De Wilde from the Institut Jules Bordet, HUBruxelles, highlighted notable presentations on the management of mantle cell lymphoma. A wide range of information was presented, covering four key areas: biomarkers, MRD results, first-line treatment options for both young and elderly patients, and some second-line treatment approaches.
- [Diffuse large B-cell lymphoma](https://medimix.be/hematology/icml-lugano-2025-highlights-4/) - At ICML, several important updates were presented regarding the treatment of diffuse large B-cell lymphoma (DLBCL), with a particular focus on novel therapeutic strategies for frail and elderly patients, as well as advances in the relapsed/refractory setting. Dr Gilles Crochet outlined key developments reflecting an evolving treatment landscape increasingly oriented toward chemo-free regimens and targeted antibody-based combinations.
- [Indolent lymphomas](https://medimix.be/hematology/icml-lugano-2025-highlights-3/) - At the 18th ICML held in Lugano, several notable updates were presented regarding the management of indolent lymphomas. Dr Fulvio Massaro highlighted two key areas of progress: the renewed interest in low-dose radiotherapy and the increasing adoption of chemo-free regimens in frontline treatment. Both developments reflect a broader trend toward treatment strategies that aim to reduce toxicity while preserving efficacy in indolent lymphomas.
- [Highlights Plenary session](https://medimix.be/hematology/icml-lugano-2025-highlights-2/) - At this year’s ICML meeting in Lugano, Prof Dr Koen Debackere reviewed highlights from the plenary session, which this year focused atypically on diffuse large B-cell lymphoma (DLBCL) biology and treatment innovation.
- [CLL](https://medimix.be/hematology/icml-lugano-2025-highlights-1/) - At ICML, several important updates were presented on CLL, focusing on longer-term follow-up data from key clinical trials. Prof Ann Janssens summarised these findings, which largely confirmed and extended earlier results.
- [Highlights in acute lymphoblastic leukemia](https://medimix.be/hematology/eha-milan-2025-highlights-6/) - In the selection of his key highlights related to acute lymphoblastic leukemia, Dr Jan Brijs focussed on the integration of immunotherapeutic agents such as blinatumomab or inotuzumab ozogamicin (Ino) in first line treatment regimens for patients with B-ALL.
- [Momelotinib in the treatment of myelofibrosis: new insights from SIMPLIFY-1 & MOMENTUM](https://medimix.be/hematology/eha-milan-2025-in-depth-6/) - In this video, Prof Dr Francesca Palandri, myeloproliferative neoplasm specialist at the IRCCS Azienda Ospedaliero in Bologna summarizes the results of two interesting posters discussing new insights from the SIMPLIFY-1 and MOMENTUM studies, evaluating momelotinib in patients with myelofibrosis.
- [The contemporary Belgian treatment landscape for patients with myelofibrosis](https://medimix.be/hematology/eha-milan-2025-in-depth-2/) - In this video, Prof Dr Timothy Devos provides a helicopter view of the contemporary treatment landscape for patients with myelofibrosis in Belgium.
- [The MIDAS trial: MRD guided therapy for newly-diagnosed multiple myeloma](https://medimix.be/hematology/eha-milan-2025-in-depth-15/) - At EHA 2025, early results of the MRD-driven consolidation phase of the trial were presented. In this video, Prof Claudio Cerchione explains why MRD-driven treatment strategies like the one evaluated in MIDAS is the way to go in the future management of MM.
- [Continuous zanubrutinib vs. fixed-duration VenO for newly diagnosed CLL](https://medimix.be/hematology/eha-milan-2025-poster-7/) - Prof Dr Talha Munir and colleagues performed an unanchored, matching-adjusted indirect comparison (MAIC) between continuous zanubrutinib and fixed duration VenO using data from SEQUOIA and CLL14.
- [Long term follow-up of clinical trials in lymphoid malignancies (CLL/LGL/MM)](https://medimix.be/hematology/eha-milan-2025-highlights-4/) - During EHA 2025, Prof Dr Krzysztof Giannopoulos chaired a much-anticipated oral abstract session featuring long-term updates of 5 randomized-controlled, clinical trials evaluating new treatment options across different lymphoid malignancies.
- [Highlights in indolent non-Hodgkin lymphoma](https://medimix.be/hematology/eha-milan-2025-highlights-5/) - At EHA 2025, Dr Dima El-Sharkawi chaired the oral abstract session on indolent non-Hodgkin lymphoma.
- [The RESTORE trial: elritercept alone or in combination with ruxolitinib for the treatment of myelofibrosis](https://medimix.be/hematology/eha-milan-2025-in-depth-14/) - During EHA 2025, Prof Timothy Devos presented results of the ongoing, phase II RESTORE trial in which the activin receptor type IIA ligand trap elritercept is being evaluated in the treatment of myelofibrosis. Elritercept is specifically designed to inhibit activin A and select TGF-β superfamily ligands (activin B, GDFs 8&11) to improve hematopoiesis.
- [A new subcutaneous formulation of isatuximab for the treatment of multiple myeloma](https://medimix.be/hematology/eha-milan-2025-in-depth-13/) - In this video, Dr Fredrik Schjesvold discusses two studies evaluating a subcutaneous (SC) formulation of the anti-CD38 monoclonal antibody isatuximab in patients with multiple myeloma.(MM)
- [Highlights in sickle cell disease](https://medimix.be/hematology/eha-milan-2025-highlights-3/) - In this video, Prof Catherine Lambert talks us through the key take home messages from EHA 2025 related to sickle cell disease (SCD).
- [Adding blinatumomab to consolidation therapy in the treatment of younger adults with B-ALL: subgroup data from ECOG-ACRIN E1910](https://medimix.be/hematology/eha-milan-2025-in-depth-12/) - During EHA 2025, Dr Shira Dinner presented the results of a subgroup analysis of this trial specifically looking at patients below 55 years of age.
- [Polatuzumab vedotin plus R-GemOx for the treatment of relapsed/refractory DLBCL](https://medimix.be/hematology/eha-milan-2025-in-depth-11/) - During the plenary session at EHA 2025, Prof Matthew Matasar presented the results of the randomized, phase III POLARGO trial in which the combination of polatuzumab vedotin, rituximab, gemcitabine and oxaliplatin (pola-R-GemOx was compared to R-GemOx alone in DLBCL patients who received at least 1 prior line of therapy and were ineligible for an allogeneic stem cell transplantation.
- [Real-world data with axicabtagene ciloleucel in second line LBCL patients](https://medimix.be/hematology/eha-milan-2025-in-depth-10/) - During EHA 2025, Dr Dasom Caroline Lee presented the results of a large real-world study evaluating the safety and efficacy of axicabtagene ciloleucel (axi-cel) as second line treatment for patients with large-B-cell lymphoma (LBCL). This analysis included a total of 461 LBCL patients from the American CIBMTR® registry who received commercial axi-cel between April 2022 and July 2023.
- [Subcutaneous blinatumomab for the treatment of adult relapsed/refractory ALL patients](https://medimix.be/hematology/eha-milan-2025-in-depth-9/) - Previously,Prof Jabbour and colleagues demonstrated that a subcutaneous (SC) formulation of blinatumomab comes with a high efficacy and a tolerable safety profile. During EHA 2025, results were presented for 88 adult B-ALL patients treated with SC blinatumomab monotherapy in a phase 1 dose expansion study.
- [iMMagine-1: Anitocabtagene autoleucel for the treatment of relapsed/refractory multiple myeloma](https://medimix.be/hematology/eha-milan-2025-in-depth-8/) - During EHA 2025, Dr Gurbakhash Kaur presented updated results of the phase II registrational iMMagine-1 study.
- [Highlights in acute myeloid leukemia](https://medimix.be/hematology/eha-milan-2025-highlights-2/) - In this video, Dr Adrien De Voeght discusses some of the key take-aways from the 2025 annual EHA meeting in the field of acute myeloid leukemia (AML).
- [Highlights in multiple myeloma](https://medimix.be/hematology/eha-milan-2025-highlights-1/) - In this video, Prof Dr Michel Delforge shares his key take-aways form EHA 2025 in the field of multiple myeloma (MM). A first of the presentations selected by Prof Dr Delforge consists of the final 5-year follow-up data of the CARTITUDE-1 trial, evaluating ciltacabtagene autoleucel (cilta-cel) in patients with heavily pretreated relapsed/refractory MM.
- [Axicabtagene ciloleucel is cost-effective as 2nd line treatment for DLBCL](https://medimix.be/hematology/eha-milan-2025-poster-6/) - The results of the pivotal ZUMA-7 trial have convincingly established axicabtagene ciloleucel (axi-cel) as a preferred second line treatment for patients with large B-cell lymphoma (LBCL). More recently, data from the US have confirmed these findings in a large, real-world patient cohort.
- [Cyclosporin-cyclophosphamide: a new standard for GVHD prevention after an allogeneic stem cell transplantation?](https://medimix.be/hematology/eha-milan-2025-in-depth-7/) - In this video, Prof Dr David Curtis clinical hematologist. Australia summarizes the key take home messages from this presentation.
- [5-year ZUMA-3 - brexu-cel ALL](https://medimix.be/hematology/eha-milan-2025-poster-5/) - At EHA 2025, Prof Dr Olalekan Oluwole presented the 5-year follow-up data of this study. Patients in ZUMA-3 continued to experience a survival benefit with a 5-year overall survival (OS) rate of 40%. As could be expected, responders had the greatest treatment benefit with a median OS of more than 5 years.
- [Risk stratification for double- and triple-exposed relaped/refractory multiple myeloma](https://medimix.be/hematology/eha-milan-2025-poster-3/) - The results of this effort were presented as a poster at EHA 2025, with Prof Dr Michel Delforge as first author. A risk stratification model was developed based on age, ECOG performance status, time since diagnosis, cytogenetic risk, refractoriness status, number of prior treatment lines, prior use of a stem cell transplantation, platelet count and the levels of LDH, β2 microglobulin, albumin, hemoglobin, platelet count and calcium.
- [Optical genome mapping in NHL](https://medimix.be/hematology/eha-milan-2025-poster-2/) - In this video, Amber Verhasselt, PhD student at the department of human genetics at KU Leuven discusses the key take-aways of a poster presented at EHA 2025 evaluating the use of optical genome mapping (OGM) as a diagnostic tool in non Hodgkin lymphoma (NHL).
- [Blina ALL - real world](https://medimix.be/hematology/eha-milan-2025-poster-1/) - In this video, Prof Sabina Chiaretti from the Sapienza University of Rome discusses real-world data with blinatumomab in adult patients with B-cell acute lymphoblastic leukemia (B-ALL) across 13 European countries.
- [Integrating blinatumomab in the treatment of adult B-ALL patients](https://medimix.be/hematology/eha-milan-2025-in-depth-4/) - In this video, Dr Fabian Lang summarizes the key take aways from this interesting session.
- [MMOVE](https://medimix.be/hematology/eha-milan-2025-in-depth-3/) - During EHA 2025, Marleen Bakkus and Eleni Linskes shared the results that MMOVE generated so far.
- [New treatment options in hemophilia](https://medimix.be/hematology/eha-milan-2025-in-depth-1/) - During an educational session at EHA 2025, Prof Dr Cédric Hermans provided an overview of the most important innovations in the treatment armamentarium for patients with hemophilia A and B.
- [Multiple Myeloma](https://medimix.be/hematology/asco-chicago-2025-in-depth-2/) - At ASCO 2025 in Chicago, Prof Claudio Cerchione, a haematologist at the Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy, presented significant data on multiple myeloma. The revolution in diagnosis and treatment is ongoing, with a particular focus on the frontline setting, encompassing both transplant-eligible and ineligible patients. There is a growing ambition for deeper responses and minimal residual disease (MRD) negativity, key endpoints linked to long-term outcomes.
- [Plenary: The VERIFY trial](https://medimix.be/hematology/asco-chicago-2025-in-depth-1/) - In this ASCO 2025 plenary highlight, Dr Andrew Kuykendall presents the primary results of the pivotal VERIFY trial, introducing rusfertide as a potential new standard in the treatment of polycythaemia vera.
- [The ALLELE trial](https://medimix.be/hematology/ebmt-florence-2025-in-depth-6/) - In this video, Prof Daan Dierickx, hematologist at the University Hospitals Leuven, discusses the updated results of this trial presented during EBMT 2025.
- [Quality standards](https://medimix.be/hematology/ebmt-florence-2025-in-depth-5/) - In this video, Prof Dr Ivan Van Riet discusses the recent update of the JACIE quality standards for the accreditation of cell therapy centers in Europe. These quality standards are revised every 4 years and cover the different aspects of cell therapy, ranging from processing, and collection to the clinical use of cell products. The goal of these standards is to guarantee the best quality of care and ultimately improve outcomes for patients.
- [AML transplant or not](https://medimix.be/hematology/ebmt-florence-2025-in-depth-4/) - In this video, Prof Charles Craddock discusses how improvements in the pre- and post-transplant management of these patients can lead to better outcomes.
- [healing bonds](https://medimix.be/hematology/ebmt-florence-2025-poster-2/) - Dr Victoria Bordon discussed the results of a qualitative study that explored the lived experiences and relational significance of minor sibling donors to enhance psychosocial care and address their unique needs effectively.
- [Fungal & bacterial infections](https://medimix.be/hematology/ebmt-florence-2025-highlight-4/) - In this daily highlights video, Dr Doris Ponce shares the results of 3 key abstracts presented in the oral abstract session on bacterial and fungal infections during the 2025 annual EBMT meeting in Florence.
- [Conception after transplant](https://medimix.be/hematology/ebmt-florence-2025-in-depth-3/) - During EBMT 20225, Ms Iebe De Quick, nurse and oncofertility coordinator at Brussels IVF, delivered a presentation on the available options for fertility presentation in oncological patients.
- [OS21-07 - MDS very poor risk](https://medimix.be/hematology/ebmt-florence-2025-in-depth-2/) - During EBMT 2025, Prof Dr Xavier Poiré presented the results of an analysis performed by the EBMT chronic malignancies working party looking into the outcome of MDS patients with very poor risk cytogenetics after an allogenic stem cell transplantation (alloSCT).
- [Nurse symposia](https://medimix.be/hematology/ebmt-florence-2025-in-depth-1/) - During EBMT 2025, Ms. Marijke Quaghebeur chaired an interesting series of sessions dedicated to nurses working in stem cell transplantation and cell therapy.
- [Pediatric](https://medimix.be/hematology/ebmt-florence-2025-highlight-3/) - Dr Victoria Bordon Cueto De Braem, pediatrician responsible for bone marrow transplantations in the pediatric department of the Ghent University Hospital, shares some of the highlihghts related to SCD and β-Thalassemia presented during EBMT 2025.
- [Hemorrhagic cystitis](https://medimix.be/hematology/ebmt-florence-2025-poster-1/) - Dr Pauline Mazilier conducted a retrospective study to analyze the characteristics and treatments received by pediatric patients who developed HC following a HSCT.
- [Acute leukemia](https://medimix.be/hematology/ebmt-florence-2025-highlight-2/) - In this daily highlights video, Prof Dr Xavier Poiré, hematologist at the Cliniques Universitaires Saint-Luc in Brussels discusses some of the more resonating studies in the field of acute leukemia presented during the 2025 EBMT meeting in Florence.
- [Lymphoma](https://medimix.be/hematology/ebmt-florence-2025-highlight-1/) - In this daily highlights video, Dr Guillaume Dachy, clinical hematologist at the Cliniques Universitaires Saint-Luc in Brussels shares his key take-aways from EBMT 2025 related to lymphoma.
- [Decluttering responses and dynamic risk in multiple myeloma: how can we improve prognostication?](https://medimix.be/hematology/ash-sandiego-2024-in-depth-9/) - In this video, Prof Meera Mohan, haematologist at the Medical College of Wisconsin in Milwaukee (WI, USA) discusses the highlights of an oral abstract session at ASH 2024 dedicated to response assessment and prognostication in patients with multiple myeloma (MM).
- [Highlights in MPN](https://medimix.be/hematology/ash-sandiego-2024-highlight-10/) - In this video, Dr Nikki Granacher, haematologist at the Ziekenhuis aan de Stroom in Antwerp talks us through her highlights from ASH 2024 related to myeloproliferative neoplasms (MPN).
- [Momelotinib for the treatment of myelofibrosis: real-world data](https://medimix.be/hematology/ash-sandiego-2024-poster-8/) - Dr Alexandros Rampotas presented a poster with real-world data on the use of momelotinib in patients with myelofibrosis. The analysis included a total of 119 myelofibrosis patients who received momelotinib across 20 centers in the UK. The study confirmed that momelotinib effectively improves anemia, reduces the spleen volume and alleviates symptoms in a significant proportion of patients, both as a first- and second-line JAK inhibitor. Overall, the safety profile of momelotinib was in line with previous reports, although this analysis did reveal a higher-than-expected rate of thrombocytosis.
- [Longer survival with momelotinib than with best available therapy in ruxolitinib-pretreated myelofibrosis: results of a matching-adjusted indirect comparison](https://medimix.be/hematology/ash-sandiego-2024-poster-5/) - In a poster presented at the 2024 annual ASH meeting, Prof Francesca Palandri, haematologist in the IRCCS Azienda Ospedaliero at the University of Bologna (Italy), shared the results of a matching-adjusted, indirect comparison of momelotinib and best available therapy (BAT) in ruxolitinib-pretreated myelofibrosis patients. According to this analysis, momelotinib was associated with a longer overall survival (OS) than BAT, both in the overall study population and in the subgroup of patients with anemia at baseline. Together with the results of the SIMPLIFY-2 and MOMENTUM trials, these findings provide further support for the use of momelotinib as standard of care for myelofibrosis patients who discontinue their first line treatment with ruxolitinib.
- [Tafasitamab-lenalidomide for relapsed/refractory DLBCL: real-world data from the GELTAMO study](https://medimix.be/hematology/ash-sandiego-2024-poster-12/) - During ASH 2024, Prof Dr Antonio Gutierrez (University Hospital Son Espases, Palma de Mallorca, Spain) presented updated results of the GELTAMO study evaluating the real-life efficacy and safety of tafa-len in R/R DLBCL patients treated with this regimen across 39 Spanish centers. In the efficacy cohort (N=83) tafa-len proved to be associated with an overall response rate (ORR) of 61% (42% complete responses) and a median progression-free (PFS) and overall survival (OS) of 10.9 and 21.8 months, respectively. The best results with tafa-len were obtained in patients with an ECOG performance status of 0-1, in patients with relapsing rather than refractory disease, non-double hit patients and patients who received tafa-len in 2nd or 3rd line. Furthermore, the analysis revealed that the relative dose intensity of tafa-len was associated with a better PFS and a longer duration of response.
- [Promising efficacy and a low rate of cytokine release syndrome with ABBV-383 in patients with released/refractory multiple myeloma](https://medimix.be/hematology/ash-sandiego-2024-poster-17/) - Dr Muhamed Baljevic is a haematologist at the Vanderbilt University Medical Center, in Nashville, Tennessee (USA). In this vide, he discusses the results of a phase 1b study evaluating different doses of the anti-BCMA, anti-CD3 bispecific antibody ABBV-383 in patients with relapsed/refractory multiple myeloma (RRMM). This study revealed that a single step-up dose (SUD) of 2 mg ABBV-383 followed by a full dose of 60 mg in combination with a modified dexamethasone regimen reduced the incidence of cytokine release syndrome (CRS) to 30% (26% grade 1, 4% grade 2). Furthermore, preliminary efficacy results with this dosing schedule indicated a high rate of early treatment responses. Based on these results, future studies with ABBV-383 will adopt a dosing schedule using a 2 mg SUD in combination with modified dexamethasone premedication.
- [Patient-reported outcomes for Hemophilia A patients treated with Mim8](https://medimix.be/hematology/ash-sandiego-2024-poster-16/) - Prof Cédric Hermans is the head of the haematology department at the Cliniques Universitaires St.-Luc in Brussels (Belgium). During ASH 2024 he presented the patient-reported outcomes for patients treated with Mim8 in the phase III FRONTIER2 study. In this study, Mim8 proved to be associated with a lower annualized bleeding rate (ABR) for treated bleeds compared to on-demand treatment and/or previous clotting factor concentrate (CFC) prophylaxis. In addition to this haemostatic efficacy, Mim8 prophylaxis also improved the physical functioning and reduced the treatment burden compared with on-demand treatment. Among patients who previously received CFC prophylaxis, physical functioning improved to a lesser extent. Joint pain intensity was not severe at baseline in all arms and did not change notably with Mim8 PPX. These findings demonstrate the holistic benefits of Mim8 beyond bleed protection and provide insights into opportunities for individualized care.
- [CD19 expression is maintained after treatment with tafasitamab in patients with DLBCL](https://medimix.be/hematology/ash-sandiego-2024-poster-15/) - During ASH 2024, Dr Johannes Duell from the University of Würzburg (Germany) presented a poster evaluating the CD19 expression before and after treatment with tafasitamab-lenalidomide in patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL). This analysis demonstrated that the CD19 antigen was maintained on tumor cells after tafasitamab treatment. Furthermore, no somatic mutations in the CD19 gene were detected on end-of-treatment tumor and blood samples collected from patients treated with tafasitamab. As such, these data confirm that CD19 expression is retained after treatment with tafasitamab and underline the potential to treat tafasitamab-exposed R/R DLBCL patients with an alternative anti-CD19 targeting therapy (e.g., CAR-T therapy) in a subsequent treatment line.
- [Tafasitamab-lenalidomide for relapsed/refractory DLBCL: real-world data from the United States](https://medimix.be/hematology/ash-sandiego-2024-poster-14/) - During ASH 2024, Dr Kim Saverno from the Incyte Corporation (Wilmington, DE, USA) presented real-life data with tafa-len in a cohort of 181 R/R DLBCL patients treated with this regimen across 23 centers in the US. The median age of patients in the study was 71 years, 52.5% had an ECOG performance status of 0-1 and 80% had an R-IPI score of 3-5 at the start of the tafa-len therapy. Overall, 71.8% of patients received tafa-len in 2nd line. The real-world progression-free (PFS) and overall survival (OS) with tafa-len in this study were reported at 11.3 and 24.8 months, respectively. Across the entire study cohort, a real-world objective response rate of 73.5% was reported, including 23.2% of complete responses. Interestingly, a multivariate analysis of the real-world PFS and OS suggested a greater benefit for tafa-len in patients who received this therapy in 2nd line compared to later treatment lines.
- [Efficacy and safety of selinexor-pomalidomide-dexamethasone in patients with released/refractory multiple myeloma](https://medimix.be/hematology/ash-sandiego-2024-poster-13/) - Dr Muhamed Baljevic is a haematologist at the Vanderbilt University Medical Center, in Nashville, Tennessee (USA). During ASH 2024 he presented data from a phase 1b/2 study evaluating the combination of pomalidomide-dexamethasone (Pd) with different doses of selinexor (S) in patients with relapsed/refractory multiple myeloma (RRMM). In this trial, the all-oral SPd regimen showed promising signs of efficacy and was generally well-tolerated in RRMM patients who previously received multiple treatment lines, including an anti-CD38 antibody. While the 60mg qw dose of selinexor was associated with a higher response rate than the 40mg qw dose, the median progression-free survival (PFS) proved to be longer among patients who received the 40mg qw dose. The latter can be explained by the fact that the 40mg dose came with a lower rate of adverse events, resulting in a longer treatment exposure and a higher relative dose intensity. As such, this study provides valuable information for future studies with selinexor-based regimens in RRMM.
- [Adding blinatumomab to consolidation therapy in older newly-diagnosed patients with Philadelphia-negative B-ALL](https://medimix.be/hematology/ash-sandiego-2024-poster-11/) - During ASH 2024, Dr Nikolai Podoltsev from the Yale School of Medicine in New Haven, CT (USA) presented the results of a subgroup analysis of this trial looking into the effect of blinatumomab in patients aged 55 years or older. Surprisingly, in this cohort of older patients, adding blinatumomab to standard consolidation therapy did not lead to a longer relapse-free (RFS) or OS. According to Dr Podoltsev, this lack of a treatment effect may be linked to the small sample size (N=211) but may also point towards a different disease biology in older B-ALL patients. Further studies are needed to better determine the tolerance and benefit of blinatumomab added to consolidation therapy in older ALL patients.
- [Isa-VRd in transplant-ineligible patients with newly diagnosed multiple myeloma and high-risk features](https://medimix.be/hematology/ash-sandiego-2024-poster-10/) - During ASH 2024, Prof Xavier Leleu (Hôpital La Mileterie, CHU Poitiers, Poitiers, France) presented updated results of this trial looking at the efficacy results in function of the risk profile of patients. Interestingly, the clinical benefit obtained with Isa-VRd over Isa-Rd proved to be more pronounced in the subgroup of patients with high-risk disease according to the new IMS HR definition than in patients with non-high risk disease.
- [Isatuximab-lenalidomide-bortezomib and low-dose dexamethasone: an effective steroid sparing regimen for transplant-ineligible MM](https://medimix.be/hematology/ash-sandiego-2024-poster-9/) - The phase II REST study is evaluating a steroid-sparing regimen consisting of isatuximab, lenalidomide, bortezomib and a limited dose of dexamethasone (Isa-VRd) as a first line treatment for transplant ineligible patients with newly diagnosed multiple myeloma (NDMM).
- [Lower response rates to cevostamab in triple-class refractory multiple myeloma patients who previously received a bispecific antibody](https://medimix.be/hematology/ash-sandiego-2024-poster-7/) - During ASH 2024, Prof Michel Delforge, haematologist at the University Hospitals Leuven (Belgium) presented updated results of this trial with a specific focus on the impact of the type of prior BCMA-directed therapies. These updated results confirmed the manageable safety profile of cevostamab in this setting. Furthermore, a lower response rate was observed among patients who previously received a BCMA-targeted bispecific antibody compared to patients in whom the prior anti-BCMA treatment consisted of an antibody-drug conjugate (ADC) or CAR-T cell therapy. A possible explanation for this lower response rate can be found in the fact that patients who previously received an anti-BCMA bispecific antibody had lower levels of soluble BCMA at baseline than patients in the prior ADC or prior CAR-T group.
- [Predictive value of D-dimer concentrations in patients on long-term direct oral anticoagulation after a venous thrombo-embolism](https://medimix.be/hematology/ash-sandiego-2024-poster-6/) - During the 2024 annual ASH meeting, Prof Cédric Hermans, shared the results of an analysis looking into the levels and predictive potential of D-Dimers in patients on long-term anticoagulation with a direct oral anticoagulant for a venous thrombo-embolism (VTE). During secondary VTE prevention, the levels of D-Dimer were generally low and did not predict for a VTE recurrence. The age-adjusted D-dimer levels were somewhat higher in men with gonosomal aneuploidy and in patients with a deep vein thrombosis complicated by post-thrombotic syndrome.
- [Blood-derived cell-free DNA for disease monitoring in patients with multiple myeloma](https://medimix.be/hematology/ash-sandiego-2024-poster-4/) - In his poster presented at ASH 2024, Dr Robbe Heestermans, clinical biologist in training at the University Hospital Brussels (Brussel, Belgium), shares the results of his research into peripheral blood-based monitoring in the follow-up of multiple myeloma (MM) patients.
- [Tagraxofusp induces a rapid restoration of normal hematopoiesis in patients with newly-diagnosed BPDCN](https://medimix.be/hematology/ash-sandiego-2024-poster-3/) - During ASH 2024, Prof Dr Marina Konopleva from the Albert Einstein College of Medicine (Bronx, NY, USA) presented the results of a sub analysis of a phase II study evaluating the anti-CD123 agent tagraxofusp as first line treatment for patients with BPDCN.
- [Impact of emicizumab on mental liberation in haemophilia A patients](https://medimix.be/hematology/ash-sandiego-2024-poster-2/) - Prof Dr Cédric Hermans, a haematologist at Cliniques Universitaires Saint-Luc in Brussels presented data on emicizumab, a new treatment for haemophilia A.
- [Highlights in Multiple Myeloma](https://medimix.be/hematology/ash-sandiego-2024-highlight-9/) - In this video, Dr Claudio Cerchione, Haematologist at the Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy shares his key take aways from ASH 2024 related to the management of multiple myeloma (MM).
- [Updates from the EAE115 and IONA-MM studies](https://medimix.be/hematology/ash-sandiego-2024-in-depth-5/) - In this video, Prof Fredrik Schjesvold, head of the Oslo myeloma center in Oslo (Norway), discusses the results of the EAE 115 study evaluating this regimen as a second line treatment for patients with relapsed/refractory MM.1
- [Updates from GMMG-HD7](https://medimix.be/hematology/ash-sandiego-2024-in-depth-8/) - In this video, Dr Elias Mai, haematologist at the Heidelberg University Hospital in Germany and one of the lead investigator of GMMG-HD7, shares the key take-aways of these abstracts.
- [Five-year follow-up of the phase II OPTIC study](https://medimix.be/hematology/ash-sandiego-2024-in-depth-7/) - In this video, Prof Jorge Cortes, haematologist at the Georgia Cancer Centre in Augusta, Georgia (USA), discusses the 5-year follow up data of this trial presented at ASH 2024.
- [Momelotinib for myelofibrosis with anaemia: Spanish real-world data](https://medimix.be/hematology/ash-sandiego-2024-poster-1/) - Dr Lucia Pérez-Lamas, a haematologist at Hospital Universitario Puerta de Hierro, Madrid, Spain, presented real-world data on momelotinib for myelofibrosis patients with anaemia, collected from 54 patients across 74 centres.
- [Highlights in ALL](https://medimix.be/hematology/ash-sandiego-2024-highlight-8/) - During ASH 2024, Dr Guru Subramanian Guru Murthy from the Medical College of Wisconsin in Milwaukee chaired an oral abstract session discussing innovations in the treatment of Philadelphia chromosome-positive acute lymphoblastic leukaemia (Ph+ ALL) and early T-precursor ALL (ETP-ALL).
- [Frontline ponatinib plus blinatumomab for Ph+ ALL](https://medimix.be/hematology/ash-sandiego-2024-in-depth-6/) - In this video, Prof Robin Foà, emeritus professor of haematology at the Sapienza University of Rome discusses two abstracts presented at ASH zooming in on the use of ponatinib plus blinatumomab as frontline therapy for patients with Philadelphia chromosome-positive acute lymphoblastic leukaemia (Ph+ ALL).
- [Highlights in aggressive lymphoma](https://medimix.be/hematology/ash-sandiego-2024-highlight-7/) - In this video, Dr William Basem, haematologist at the OhioHealth Physician Group in Columbus, Ohio, shares some of the key take aways of an oral abstract session at ASH 2024 focussing on innovative, chemotherapy-free treatment options for patients with aggressive B-cell lymphomas.
- [Highlights in chronic lymphocytic leukemia (CLL)](https://medimix.be/hematology/ash-sandiego-2024-highlight-6/) - In this daily highlights video from ASH 2024, Dr Lucrecia Yanez San Segundo, a haematologist at Hospital Universitario Marqués de Valdecilla (IDIVAL), Santander, Spain, summarised key findings from the oral session on CLL, which she co-chaired at ASH.
- [MRD negativity dynamics in the IMROZ study](https://medimix.be/hematology/ash-sandiego-2024-in-depth-4/) - Dr Robert Orlowski, a haematologist at the University of Texas MD Anderson Cancer Center in Houston, Texas, provided a comprehensive summary of his presentation on MRD testing in the context of the IMROZ trial.
- [Highlights in follicular lymphoma](https://medimix.be/hematology/ash-sandiego-2024-highlight-5/) - In this daily highlights video from ASH 2024, Dr Erika Haydu, heamatologist at the Massachusetts General Hospital in Boston, shares the key take home messages from an oral abstract session discussing new treatment options for patients with follicular lymphoma (FL).
- [Highlights in chronic myeloid leukaemia (CML)](https://medimix.be/hematology/ash-sandiego-2024-highlight-4/) - Dr Adriano Salaroli, a haematologist at the Institut Jules Bordet in Brussels, discussed new data on CML. First of all, he focused on the ASC4FIRST study's 96-week findings. This trial explored asciminib as a first-line treatment following its established efficacy and safety in third-line settings.
- [inMIND study in R/R follicular lymphoma](https://medimix.be/hematology/ash-sandiego-2024-in-depth-3/) - Prof Marek Trneny, haematologist at Charles University General Hospital in Prague, Czech Republic, presented findings from the inMIND study, featured as a late-breaking abstract at the ASH meeting.
- [DREAMM-7 study in R/R MM](https://medimix.be/hematology/ash-sandiego-2024-in-depth-2/) - Prof Dr Claudio Cerchione, a haematologist at the Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy, co-authored the DREAMM-7 study and shared updated efficacy data presented at ASH 2024.
- [Highlights in thrombosis and bleeding disorders](https://medimix.be/hematology/ash-sandiego-2024-highlight-3/) - Prof Dr Cédric Hermans, a haematologist at the Cliniques Universitaires Saint-Luc (Brussels, Belgium) presented notable updates regarding thrombosis and bleeding disorders.
- [AALL1731 trial](https://medimix.be/hematology/ash-sandiego-2024-in-depth-1/) - Dr Sumit Gupta, a paediatric oncologist and assistant professor at the Hospital for Sick Children and the University of Toronto, Canada, presented the main findings of the AALL1731 study, of which he is the co-author.
- [Highlights in Acute Myeloid leukaemia (AML)](https://medimix.be/hematology/ash-sandiego-2024-highlight-2/) - While ASH 2024 did not feature any major, practice changing studies in the field of acute myeloid leukaemia (AML), Dr Koen Theunissen, heamatologist at the Jessa Hospital in Hasselt (Belgium), did find some interesting presentations that are building further on the advancements of the last decade.
- [Highlights in multiple myeloma](https://medimix.be/hematology/ash-sandiego-2024-highlight-1/) - In this video, Prof Dr Michel Delforge, Haematologist at the University Hospitals Leuven (Leuven, Belgium) shares his key take aways from ASH 2024 related to the management of patients with multiple myeloma (MM).
- [Management of cytopenic myelofibrosis](https://medimix.be/hematology/eha-madrid-2024-indepth-14/) - Dr Adriano Salaroli, a haematologist at the Institut Jules Bordet in Brussels, provides an update on cytopenic myelofibrosis, emphasising the need for increased awareness regarding this specific subtype of myelofibrosis.
- [Real-world outcomes of momelotinib in myelofibrosis patients with anaemia](https://medimix.be/hematology/eha-madrid-2024-indepth-15/) - Dr Lucia Perez-Lamas, a haematologist at the Puerta de Hierro University Hospital in Madrid, Spain, presented real-world evidence on the use of momelotinib in myelofibrosis (MF) patients with anaemia.
- [Transfusion practice for sickle cell disease in a Belgian reference centre](https://medimix.be/hematology/eha-madrid-2024-poster-5/) - Dr Yvan Momo, a haematologist at the Hopital Universitaire de Bruxelles in Brussels, presents an analysis of transfusion practices in a Belgian sickle cell reference centre, assessing their adherence to European guidelines. This retrospective study examined all transfusions administered to sickle cell patients between January 2013 and October 2022. For each transfusion, the indication and its conformity to the guidelines were documented. A similar study conducted at the same institution a decade ago, before the introduction of specific guidelines for transfusion in sickle cell disease, provides a basis for comparison. The results indicate a higher adherence to European transfusion guidelines over the past ten years. Nevertheless, some transfusions still do not comply with the guidelines. Despite the inherent limitations of a retrospective study, these findings underscore the importance of raising awareness about the specific management of sickle cell disease and the need for local initiatives to enhance transfusion practices.
- [Return to work after allogeneic stem cell transplantation (SOPRALLO project)](https://medimix.be/hematology/eha-madrid-2024-poster-9/) - Dr Yana Stepanishyna, a haematologist at the Institut Jules Bordet in Brussels, presents a preliminary analysis from the SOPRALLO project. Allogeneic hematopoietic stem cell transplantation (HSCT) has significantly extended the survival of recipients. However, this progress is accompanied by the potential for long-term side effects, which can adversely impact the quality of life and reduce the capacity for return to work (RTW) among these patients. The current analysis focuses on socio-demographic predictive factors that may impact social maladjustment and RTW.
- [Matching-adjusted indirect comparisons of bispecific antibodies in R/R FL](https://medimix.be/hematology/eha-madrid-2024-poster-8/) - Epcoritamab (EPCOR) is a novel, subcutaneous CD3xCD20 bispecific antibody (bsAb) that has demonstrated deep and durable responses with manageable safety in patients with R/R FL following at least two systemic therapies, as evidenced by the EPCORE NHL-1 trial. Intravenous mosunetuzumab (MOSUN) and intravenous odronextamab (ODRO) are also CD20xCD3 bsAbs that have shown efficacy and safety in the same patient population, based on findings from the GO29781 and ELM-2 trials, respectively. To date, no direct comparisons of the efficacy and safety of these bsAbs have been conducted. Professor Alexey Danilov, a haematologist at the City of Hope National Medical Center in Duarte, CA, USA, presented the outcomes of matching-adjusted indirect comparisons of the efficacy and safety of EPCOR versus MOSUN or ODRO in patients with R/R FL after at least two systemic therapies.
- [Targeting the IL-4 pathway in T-ALL patients](https://medimix.be/hematology/eha-madrid-2024-poster-7/) - Wouter Sleeckx, a doctoral researcher at the University of Ghent, presented his research on the therapeutic potential of targeting the IL-4 signalling pathway in a subset of T-ALL patients. His study focuses on patients harbouring genomic aberrations that drive abnormal IL-4 receptor expression that can be activated by paracrine IL-4 present in the microenvironment or acquire the ability to produce and excrete autocrine IL-4.
- [Functional characterisation of  kdm6b in immature leukemia](https://medimix.be/hematology/eha-madrid-2024-poster-6/) - Cristina Borin, a doctoral researcher at the University of Ghent and the Cancer Research Institute Ghent (CRIG) presented her work on the functional characterization of Kdm6b in immature leukemia. Her research aims to evaluate the potential of Kdm6b as an oncogenic driver of leukemia in vivo. Additionally, she seeks to elucidate the role and molecular partners of Kdm6b in the initiation of immature leukemia, with the ultimate goal of proposing novel therapeutic combinations for the treatment of this malignancy.
- [Brentuximab vedotin + pembrolizumab in R/R cHL](https://medimix.be/hematology/eha-madrid-2024-poster-4/) - The current standard treatment for R/R cHL involves salvage chemotherapy followed by ASCT. However, the optimal salvage therapy regimen remains undefined. Recent evidence suggests that the combination of brentuximab vedotin (BV) with PD-1 inhibitors may yield promising outcomes in this context. Dr. Hanne Massa, a haematologist in training at the Jules Bordet Institute in Brussels, presented a single centre retrospective analysis evaluating the efficacy and safety of BV and pembrolizumab as salvage therapy prior to ASCT, followed by BV maintenance, in patients with R/R cHL.
- [Dual targeting of EZH2 and HDAC in haematological malignancies](https://medimix.be/hematology/eha-madrid-2024-poster-3/) - Thomas Slegers, a doctoral researcher, and Dr Marlies Vanden Bempt, a postdoctoral researcher, both from KU Leuven, presented their research on the dual targeting of EZH2 and HDAC in haematological malignancies. Both EZH2 and HDAC inhibitors are currently utilized in clinical practice for treating various haematological cancers. Previous studies have shown strong synergistic effects between EZH2 degradation and HDAC inhibition in peripheral T cell lymphoma. Their ongoing research aims to demonstrate the broad applicability of combined EZH2 and HDAC targeting for haematological cancers and to elucidate the mechanism of action underlying the synergism of this combination treatment.
- [The IMROZ trial](https://medimix.be/hematology/eha-madrid-2024-indepth-13/) - Dr Marie Vercruyssen, a haematologist at the Institut Jules Bordet in Brussels, summarised the outcomes of the IMROZ trial, which evaluated the addition of the anti-CD38 monoclonal antibody isatuximab to the VRd regimen in transplant-ineligible patients with newly diagnosed MM. The trial included over 400 patients from various global regions. The primary endpoint of the study, PFS, was 54 months for the VRd arm, while the PFS was not reached for the Isa-VRd arm. Notably, the projected median PFS for the Isa-VRd arm is approximately 90 months, indicating a significant improvement. Secondary key endpoints included CR and MRD negativity at a sensitivity level of 10-5, as assessed by NGS. A CR was achieved by three out of four patients, with nearly 60% attaining MRD negativity. Importantly, nearly 50% of these patients achieved sustained MRD negativity for 12 months. Given that MRD negativity, particularly sustained MRD negativity, is widely accepted as a surrogate marker for patient outcomes in MM, these results are considered a substantial advancement in the field. Regarding safety, the Isa-VRd regimen was well-tolerated, with slightly increased incidences of myelosuppression and infection as expected, but no new safety concerns emerged from the trial. In conclusion, the IMROZ trial demonstrates that the addition of an anti-CD38 monoclonal antibody to the VRd regimen significantly improves outcomes for transplant-ineligible patients with newly diagnosed MM, establishing Isa-VRd as a new standard of care in this patient population.
- [The PREAMBLE registry](https://medimix.be/hematology/eha-madrid-2024-poster-2/) - Prof Dr Claudio Cerchione, a haematologist at the Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy, shares insights from the PREAMBLE registry. This registry documents treatment patterns and clinical outcomes in MM patients after lenalidomide exposure. Understanding current real-world treatment patterns and clinical outcomes in patients receiving lenalidomide-containing regimens may help identify unmet needs and optimise future management strategies for these patients.
- [Results from the screened iSTOPMM study](https://medimix.be/hematology/eha-madrid-2024-indepth-12/) - Dr Sigrún Thorsteinsdóttir, a haematologist at Rigshospitalet in Copenhagen, Denmark, also holds a position as a postdoctoral researcher in the iStopMM study at the University of Iceland. The iSTOPMM study (Iceland Screens, Treats, or Prevents Multiple Myeloma) aims to investigate smoldering multiple myeloma (SMM) and monoclonal gammopathy of undetermined significance (MGUS). All Icelandic residents over the age of 40 were offered screening through serum protein electrophoresis (SPEP) and free light chain (FLC) assay. Epidemiological data consistently demonstrate that MM and its precursor MGUS are more prevalent in men compared to women. In the iSTOPMM study, individuals with abnormal screening results were classified as MGUS cases, while those with normal screening results served as controls. The study identified 3,554 individuals with MGUS and 71,049 controls. Among those with MGUS, 54% were male, compared to 45% of the controls. Males exhibited a higher risk of MGUS and were more likely to present with clinical features indicative of more aggressive disease, such as elevated M-protein concentration, abnormal FLC ratios, and non-IgG isotypes. Furthermore, it was observed that exogenous oestrogen exposure for a duration of at least five years potentially offered a protective effect against MGUS in women. SMM is recognised as a precursor to MM. Cases of MM that exclusively secrete FLC proteins are classified as light chain multiple myeloma (LC-MM). The entity LC-SMM was identified based on the presence of urinary monoclonal light chain excretion. However, with the availability of more sensitive FLC assays for serum, there is a need to redefine this precursor condition. Based on our population-based screening of 75,422 individuals, we propose a new definition for LC-SMM: the presence of pathological FLC values as determined by FLC assay, absence of detectable intact immunoglobulins, and bone marrow plasma cells constituting over 10%, in the absence of myeloma-defining events. Our findings underscore the limitations of traditional urine protein electrophoresis-based diagnostics, which failed to identify 28% of individuals diagnosed with LC-SMM in our cohort. Consequently, we conclude that LC-SMM is a distinct precursor of LC-MM, which can be effectively diagnosed using serum FLC testing and bone marrow sampling. This warrants further investigation into its clinical outcomes and the formulation of evidence-based management guidelines.
- [Vaccination against mutated RAS in MM: the phase I/II TG01 study](https://medimix.be/hematology/eha-madrid-2024-indepth-11/) - Dr Hanne Norseth, a haematologist at the Oslo Myeloma Center in Norway, presented the results of the phase I/II TG01 study. This study evaluates the safety and efficacy of the TG01/QS-21 vaccine as a single agent in patients harbouring KRAS or NRAS codon 12/13 mutations. Eligible patients include those with measurable MM following at least one prior line of therapy or those with high-risk smoldering MM. The primary endpoint of the study is safety and tolerability of TG01/QS-21. Secondary endpoints include evaluating response according to IMWG criteria, measuring the immunological response to the vaccine through TG01-specific cytokine production and assessing the size of the RAS-mutated clone before and after treatment or at disease progression. Out of the 37 patients screened, nine have been enrolled since the initiation of the study. Of these, three patients have high-risk smoldering MM, while six patients have MM. No patients experienced adverse events of grade 3 or higher. However, there was no observed clinical response to the vaccine, with six out of nine patients showing disease progression, with a median time to progression of two months. Currently, three patients remain in the study with stable disease, having initiated a median of five treatment cycles (range 4-9). These preliminary findings suggest that while the TG01/QS-21 vaccine is well-tolerated, its clinical efficacy in this cohort of patients remains to be further evaluated.
- [The BENEFIT trial: Isa-VRd in non-frail patients with NDMM TI](https://medimix.be/hematology/eha-madrid-2024-indepth-10/) - At the EHA 2024 conference, Prof Dr Xavier Leleu, Head of the Myeloma Clinic and Department of Haematology at the University Hospital of Poitiers (France), presented the results of the BENEFIT trial. BENEFIT is a prospective, multicenter, randomised study designed to evaluate the efficacy and safety of the IsaRd regimen versus the Isa-VRd regimen in non-frail, NDMM TI patients aged 65-79 years. The primary endpoint, MRD at a sensitivity of 10-5 at 18 months from the start of treatment, was significantly higher in the Isa-VRd arm compared to the IsaRd arm. Additionally, MRD at a sensitivity of 10-6 at 18 months was superior in the Isa-VRd cohort relative to the IsaRd arm, with benefits observed as early as 12 months into treatment. The safety profile was consistent with the addition of bortezomib. These findings suggest that the Isa-VRd regimen should be considered a new standard of care for non-frail patients with NDMM TI.
- [New treatment combinations in RRMM patients](https://medimix.be/hematology/eha-madrid-2024-indepth-9/) - Prof Dr Claudio Cerchione, a haematologist at the Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy, highlights the necessity for novel treatment combinations in lenalidomide-refractory MM patients. Belantamab mafodotin (belamaf), a B-cell maturation antigen targeted ADC has demonstrated clinical efficacy and a manageable safety profile, supporting its use in combination with standard of care therapies for RRMM patients. The DREAMM-7 trial is a global, randomised, open-label, phase III study comparing the efficacy and safety of the belamaf plus bortezomib and dexamethasone (BVd) triplet against daratumumab, bortezomib and dexamethasone (DVd) in patients with RRMM who have received at least one prior line of therapy. The trial demonstrated a statistically significant PFS benefit for BVd as well as a strong and clinically meaningful OS benefit. Additionally, BVd resulted in a greater depth of response and a doubling of median DOR compared to DVd, while maintaining a manageable safety profile. These findings support BVd as a potential new standard of care for this patient population. In the DREAMM-8 study, another belamaf combination, belamaf plus pomalidomide and dexamethasone (BPd) was evaluated against pomalidomide plus bortezomib and dexamethasone (PVd) in RRMM patients previously treated with lenalidomide. This study also demonstrated a statistically significant and clinically meaningful PFS benefit for BPd compared to PVd in RRMM patients. BPd led to deeper and more durable responses, showed a favourable trend in OS and had a manageable safety profile. These results indicate that BPd could serve as an effective treatment option for RRMM patients previously treated with lenalidomide, further advancing the therapeutic landscape for this challenging condition.
- [How to treat frail patients with multiple myeloma?](https://medimix.be/hematology/eha-madrid-2024-indepth-8/) - During EHA 2024, an entire session was dedicated to the management of difficult to treat multiple myeloma (MM) populations. In this session, Dr Claudia Stege, haematologist at the Erasmus Medical Center in Rotterdam (The Netherlands), specifically addressed the treatment of elderly, frail MM patients. Over the last decade, the introduction of many new treatment modalities dramatically improved the outcome for patients with MM. However, a higher age and a poor performance status continue to be associated with a worse outome. In this respect, a complete geriatric assessment of elderly MM patients can help to determine the prognosis of patients. Over the years, several frailty scores have been developed for MM patients, of which the IMWG frailty score and the simplified frailty score are the ones that are used most frequently.1,2 Unfortunately, elderly, and frail patients are usually excluded from clinical studies, resulting in a data gap on the performance of the currently available treatment regimens for MM patients in patients with a poorer performance status. Based on the results of the phase III MAIA study, the combination of daratumumab-lenalidomide and dexamethasone (DRd) has become the standard of care first line treatment of transplant-ineligible MM patients. Interestingly, this study demonstrated that DRd significantly outperformed Rd irrespective of the frailty status of patients.3 Nevertheless, also with this regimen, frail patients continue to have a worse prognosis than their fitter counterparts. The main reason for this can be found in a higher rate of early treatment discontinuation due to tolerability issues. To generate more data on the efficacy and safety of novel MM treatment regimens, there is a need to include frail patients in future randomized controlled trials and to generate prospective real-world data with these regimens in elderly and/or frail patients. In addition, treatment strategies should be evaluated in which the treatment intensity is adapted to the frailty of the individual patient.
- [Blinatumomab added to prephase and consolidation therapy for adult B-ALL patients](https://medimix.be/hematology/eha-madrid-2024-indepth-7/) - During EHA 2024, Dr Anita Rijneveld, haematologist at the Erasmus Medical Center Cancer Institute in Rotterdam, presented the final results of the phase II HOVON-146 study. This study evaluated the addition of blinatumomab to the prephase and consolidation treatment of adult patients with B-cell acute lymphoblastic leukemia (B-ALL). Despite a continuous optimization of chemotherapy regimens, the long-term prognosis for adult patients with B-ALL remains to be poor. To improve on this, several studies are evaluating the integration of novel therapeutic agents in the first line treatment of adult B-ALL patients. In HOVON-146, 71 adult B-ALL patients were treated with a paediatric-inspired prephase and consolidation treatment protocol. After 2 cycles of blinatumomab in the prephase, already 63% of patients had a complete response (CR), with an MRD-negativity rate of 53%. Importantly, this high rate of CR after the prephase was obtained without the use of any chemotherapy. After the consolidation phase, during which patients received an additional 4 weeks of blinatumomab in conjunction with consolidation chemotherapy, the CR rate had increased to 97%, with MRD-negativity in 91% of patients. The 4-year overall survival (OS) rate obtained with the blinatumomab-containing regimen in this study was reported at 86% in the cohorts of patients ≤40 years of age and in patients aged 40-60 years. In patients above the age of 60, the OS rate at 4 years was somewhat lower at 50%. In the subgroup of patients with Philadelphia chromosome positive (Ph+) ALL, the 4-year OS rate reached 85%. The survival rates obtained in HOVON-146 compare favourably to the results obtained in HOVON-100, a clinical trial evaluating a similar chemotherapy regimen without blinatumomab (4-year OS in patients ≤40 years: 76% [vs. 86%], >40 years: 51% [vs. 71%]). For Dr Rijneveld, these data underscore the potential of blinatumomab in the first line treatment of adult B-ALL, especially for elderly patients and for those with Ph+ disease. To evaluate this in more detail, dedicated phase III randomized controlled trials are warranted in which blinatumomab is added to first line chemotherapy, or in which (parts of) the chemotherapy backbone are substituted for blinatumomab.
- [CAR-T cell therapy as second line treatment for high-risk multiple myeloma (MM)](https://medimix.be/hematology/eha-madrid-2024-indepth-6/) - During EHA 2024, Prof Dr Xavier Leleu, Head of the Myeloma Clinic and Head of the Department of Haematology at the University Hospital of Poitiers (France), presented the results of cohort 2B of the KARMMA-2 study. In this study cohort, transplant ineligible multiple myeloma (MM) patients with an early relapse following first line therapy were treated with the anti-CD-19 CAR-T product idecabtagene vicleucel (ide-cel). The study cohort at hand included 35 MM patients who did not receive an autologous stem cell transplantation in first line and relapsed within 18 months following their diagnosis on a first line treatment regimen including a proteasome inhibitor, an immunomodulatory agent and dexamethasone. The median age of patients in the study was 60 years, about 40% carried high-risk cytogenetic abnormalities and 67.7% had double-class refractory disease. Overall, 87.1% of patients received bridging therapy prior to CAR-T infusion. Of the 31 patients who received the CAR-T therapy, 71.0% obtained a complete response following CAR-T therapy (overall response rate: 93.5%). This is a remarkable finding given the fact that the complete response rate in first line was only 3.2%. At data cut-off, 51.6% of patients had an ongoing complete response, with a 24-month duration of response rate among patients with at least a complete response of 75.7% (median not reached). Importantly, this high complete response rate was accompanied by a steady improvement in overall quality of life, pain, and fatigue. In conclusion, ide-cel demonstrated a favourable benefit-risk profile in transplant-ineligible patients with clinical high-risk MM who experienced an early relapse following first line therapy.
- [IMPACT-AML project](https://medimix.be/hematology/eha-madrid-2024-indepth-5/) - Prof Dr Giovanni Martinelli, a haematologist at the "L. e A. Seragnoli" Institute in Bologna, Italy, presented Impact-AML, a European master framework designed for cohort studies and pragmatic clinical trials in R/R AML. The primary goals of this initiative are to establish an inclusive framework for R/R AML patients, design and conduct a randomized pragmatic clinical trial comparing low-intensity and standard chemotherapy rescue, and increase patient engagement in clinical trials. Additionally, standard operating procedures for local biobanking will be developed, including sample collection for MRD quantification, to enhance biological understanding of R/R AML
- [Multidisciplinary approach to myeloproliferative neoplasms (MPN)](https://medimix.be/hematology/eha-madrid-2024-indepth-1/) - Prof Timothy Devos, a haematologist at UZ Leuven, chaired a session on myeloproliferative neoplasms (MPN) organised by the specialised working group of the EHA. He provided a comprehensive summary of the presentations delivered by four distinguished speakers. The first speaker, Prof Passamonti from Milan, Italy, introduced the topic. He elucidated the objectives and key elements of multidisciplinary meetings in the context of MPNs, emphasizing the necessity of involving experts from various disciplines. Such collaboration is essential for accurate diagnosis, informed treatment decision-making, and coordinated follow-up strategies. Dr Lyon, a cardio-oncologist from London, UK, presented on the critical vascular risk factors affecting patients with MPNs and offered guidance on reducing cardiovascular events in these patients. He concluded his presentation by referencing the 2022 ESC guidelines on cardio-oncology, which were developed in collaboration with the EHA. Prof Kiladjian from Paris, France, concentrated on the management of splanchnic vein thrombosis in JAK2-positive MPN patients. He emphasized the necessity of collaboration with hepatologists for effective management of these patients. At his hospital, a risk score has been developed for MPN patients to assess their risk for splanchnic vein thrombosis, as well as secondary myelofibrosis, acute leukaemia and mortality. Prof Sobas from Wroclaw, Poland, presented her research on young MPN patients, a group often underrepresented in clinical trials. She highlighted the importance of collaborating with gynaecologists and obstetricians to manage pregnancies in these patients. Prof Devos concluded his summary by emphasizing that young MPN patients are a key focus of the scientific working group. He underscored the importance of enrolling these patients in registries to collect data on an international scale, given their rarity. This approach aims to generate more conclusive and comprehensive data.
- [GMMG-HD7: Isatuximab plus lenalidomide, bortezomib, dexamethasone as induction therapy for transplant-eligible NDMM](https://medimix.be/hematology/eha-madrid-2024-indepth-2/) - Dr Fredrik Schjesvold, director of the Oslo Myeloma Center at the Oslo University Hospital in Norway, previously discussed the randomized phase III GMMG-HD7 study which showed that the addition of isatuximab (isa) to induction therapy with lenalidomide, bortezomib, and dexamethasone (RVd) significantly increased the rate of MRD negativity after three cycles of therapy in patients with transplant-eligible, newly diagnosed multiple myeloma (NDMM).1
- [The BENEFIT trial](https://medimix.be/hematology/eha-madrid-2024-indepth-3/) - Prof Dr Nathalie Meuleman, haematologist at the Institut Jules Bordet in Brussels summarized the outcomes of the BENEFIT trial presented at EHA 2024 CD38-targeting immunotherapy, in combination with lenalidomide and dexamethasone, is currently approved for the treatment of newly diagnosed, transplant-ineligible multiple myeloma (NDMM TI) and is considered the standard of care (SOC). To enhance the existing SOC, the potential benefit of extending the use of bortezomib over 18 months with a reduced intensity weekly schedule added to IsaRd was evaluated. The objective was to assess the impact of incorporating a proteasome inhibitor into a quadruplet regimen on improving the depth of response. The BENEFIT trial is a prospective, multicenter, randomized, parallel study involving patients aged 65-79 years who are non-frail and diagnosed with NDMM TI. This trial compares the efficacy and safety of the IsaRd regimen versus the Isa-VRd regimen. The primary endpoint, MRD 10-5 at 18 months from the start of treatment was significantly higher in the Isa-VRd arm compared to the IsaRd arm. The safety profile is consistent with the addition of bortezomib. The inclusion of a weekly, "light" bortezomib schedule did not significantly impact the relative dose intensity of the IsaRd regimen. These findings suggest that the Isa-VRd regimen should be considered a new standard of care for non-frail patients with NDMM TI.
- [The changing frontline treatment landscape for adult patients with acute lymphoblastic leukemia](https://medimix.be/hematology/eha-madrid-2024-indepth-4/) - In this video, Prof Dr Selina Luger, haemato-oncologist at the hospital of the University of Pennsylvania discusses the recent changes in the treatment landscape for adult patients with acute lymphoblastic leukemia (ALL). The standard first line treatment for patients with ALL consists of multi-agent chemotherapy. While the continuous optimization of these chemotherapy regimens has led to significant improvements in the survival of adult ALL patients, still about 30-50% of patients will eventually suffer a disease relapse. In recent years, several new treatment modalities have proven their worth in the treatment of relapsed/refractory (R/R) ALL patients. The most prominent of these therapies consist of inotuzumab ozogamicin, blinatumomab and CAR-T cell therapy. The success of these agents in the R/R setting inspired clinicians to also evaluate their potential in earlier disease stages. In this respect, several studies have demonstrated a significant clinical benefit with the incorporation of blinatumomab in first line treatment regimens for adult ALL patients.1-3 For example, the randomised, phase III ECOG-ACRIN E1910 study recently showed that the addition of blinatumomab to consolidation chemotherapy significantly improved the overall survival of patients with BCR-ABL-negative ALL who achieved MRD negativity after consolidation chemotherapy (median OS not reached vs. 71.4 months).4 Importantly, the benefit obtained with blinatumomab in ECOG-ACRIN E1910 and other studies was also seen in older patients, a subgroup of patients who historically have a markedly worse outcome then their younger counterparts. The results of these studies are reshaping the treatment paradigm of adult ALL patients, offering them less toxic treatment regimens with a higher likelihood for a durable disease remission.
- [EHA 2024 – Highlights in multiple myeloma](https://medimix.be/hematology/eha-madrid-2024-highlight-5/) - In this video, Prof Dr Claudio Cerchione, haematologist at the Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy shares his congress highlights from EHA 2024 in the field of multiple myeloma (MM). EHA 2024 featured many interesting presentations across different treatment lines in MM. In the frontline setting, especially the results of the BENEFIT trial caught the attention of Prof Cerchione. In this trial, transplant ineligible, newly diagnosed MM patients were treated with isatuximab-lenalidome and dexamethasone (Isa-Rd) with or without bortezomib (V). At the 18-month landmark, the rate of MRD negativity (10 -5) with Isa-VRd was reported at 47%, which was significantly better than the 24% seen with Isa-Rd (OR[95%CI]: 2.96[1.73-5.07; p< 0.001). The regimen was well-tolerated, with only 10% of patients who discontinued V due to adverse events.1 Also the position of CAR-T cell therapy in the treatment of MM continues to evolve. In this respect, interesting data were presented from cohort D of the CARTITUDE-2 study, evaluating ciltacabtagene autoleucel (cilta-cel) with or without lenalidomide maintenance in patients with newly diagnosed multiple myeloma who achieved less than a complete response (
- [ASH Highlights on Multiple Myeloma](https://medimix.be/hematology/ash-highlights-on-multiple-myeloma/) - Prof Claudio Cerchione, haematologist at the Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, Meldola, Italy, was so kind to share his highlights on multiple myeloma from ASH 2023. The EMN 17 Perseus study, focusing on frontline intervention in transplant-eligible patients with MM, has unveiled promising outcomes. The incorporation of daratumumab into the VRd regimen, in comparison to VRd, with daratumumab and lenalidomide-based maintenance, has exhibited notable advancements. Particularly, the study has demonstrated remarkable achievements in terms of both conventional efficacy parameters and more significantly, the depth of response. In the same setting, the IsKIa study, focusing on the comparison between isatuximab + KRd and KRd yielded compelling outcomes. Beyond demonstrating notable improvements in the overall response rate, Isa-KRd has exhibited noteworthy efficacy in achieving MRD negativity. The emphasis on MRD negativity is not only confined to the induction phase but extends across various temporal points, including post-transplantation and consolidation phases. The attainment of MRD negativity has emerged as a pivotal endpoint in the evaluation of treatment efficacy. The proposition to consider MRD negativity as a primary endpoint for patients underscores a paradigm shift in the treatment approach for young, transplant-eligible patients, with an overarching objective of striving towards a potential cure for MM. The assessment of MRD in the context of MM warrants consideration beyond the confines of clinical trials, extending into routine clinical practice. The updated data from the MonumenTAL-1 study with talquetamab presents noteworthy advancements in the realm of MM treatment. The observed overall response rate of approximately 70% is indeed remarkable, indicative of talquetamab's pronounced efficacy in this patient cohort. Of particular significance is the substantial 50% response rate observed in individuals who had previously been exposed to other bispecific antibodies. This suggests a promising trajectory toward establishing a new standard of care. Alnuctamab, a BCMA x CD3 TCE exhibits notable attributes, particularly in the context of subcutaneous administration and favourable tolerability. An additional key feature is the noteworthy MRD negativity rate, with 100% of the evaluated population achieving MRD negativity at the specified dosage, slated for use in upcoming trials. The imminent initiation of the phase 3 iLLUMINATE study, comparing alnuctamab to the standard of care within the same clinical context holds significant promise for elucidating its comparative efficacy. The Asian Myeloma Network (AMN) conducted a phase 3 study that scrutinized the efficacy of pomalidomide, cyclophosphamide, dexamethasone (PCD) in comparison to pomalidomide and dexamethasone (PD) in the relapsed/refractory setting. While the observed PFS of 11 months may not be considered extensive, the study's focus on the sequencing of treatments and the combination of PD with cyclophosphamide offers intriguing possibilities for future therapeutic approaches. Ongoing initiatives are poised to make substantial contributions to the earlier diagnosis of MM. An illustrative example is the "Iceland Screens, Treats, or Prevents Multiple Myeloma" study (iStopMM), which constitutes a population-based screening initiative specifically targeting MGUS. The iStopMM study represents a significant step towards the early identification and intervention for individuals at risk of developing MM. Moreover, advancements in the molecular characterization of MM, such as gene expression profiling, particularly utilizing signatures like the SKY92 gene signature, coupled with state-of-the-art imaging tools like PET-CT in conjunction with full-body MRI, hold the potential to formulate a new prognostic score. The integration of advanced genomic profiling with cutting-edge imaging techniques aims to enhance the precision and accuracy of prognostic assessments in MM.
- [QuANTUM-First: Efficacy by age in newly diagnosed FLT3-ITD–positive AML](https://medimix.be/hematology/eha-madrid-2024-poster-1/) - Dr Pau Montesinos, a haematologist at the Hospital Universitario y Politècnico La Fe in Valencia, Spain, presented a post hoc analysis of the phase 3 QuANTUM-First study. This analysis evaluated the impact of age (
- [Complementary role of WB-MRI & FDG PET/CT on management of MM](https://medimix.be/hematology/asco-chicago-2024-in-depth1-cerchione/) - Prof Claudio Cerchione, a haematologist at the Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori in Meldola, Italy, presented pioneering research at the ASCO24 rapid oral abstract session. The study focused on the combined use of FDG PET/CT and whole-body MRI (WB-MRI) for staging newly diagnosed and relapsed/refractory multiple myeloma (MM). In this prospective trial, 73 MM patients were enrolled, with 71 patients included in the final analysis. WB-MRI was performed alongside FDG PET/CT. Results showed a 73% concordance rate between WB-MRI and FDG PET/CT. In the 25% of discordant cases, 83% (15 out of 18) were negative on FDG PET/CT but positive on WB-MRI, while 17% (3 out of 18) were positive on FDG PET/CT for focal lesions but negative on WB-MRI. The accuracy of WB-MRI stood at 97% (69 out of 71), significantly higher than the 77% accuracy of FDG PET/CT (55 out of 71). The study suggests that WB-MRI and FDG PET/CT can complement each other in managing MM patients at diagnosis and relapse stages. The next phase of this prospective research will combine imaging and molecular analyses to develop a risk-adapted prognostic index in MM.
- [Multiple myeloma - DREAMM-7 update](https://medimix.be/hematology/asco-chicago-2024-in-depth2-cerchione/) - Prof Claudio Cerchione, haematologist at the Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, Meldola, Italy, one of the authors of the DREAMM7 study, gave a summary of the main outcomes of the study presented at ASCO24. In the DREAMM-7 clinical trial, the combination of belantamab mafodotin with bortezomib and dexamethasone (BVd) exhibited a statistically significant and clinically meaningful PFS advantage compared to the standard-of-care regimen of daratumumab, bortezomib and dexamethasone (DVd) in patients with RRMM who had received at least one prior line of treatment. The PFS was 36.6 months in the experimental BVd arm versus 13 months in the DVd control group. At ASCO, subgroup analyses were presented, confirming a consistent benefit across all prespecified subgroups, including patients refractory to lenalidomide and patients with one or more high-risk cytogenetic abnormalities. These findings, demonstrating significant efficacy in subgroups with high unmet medical needs, support the potential of BVd as a new standard of care in this clinical setting.
- [Highlights in acute myeloid leukaemia (AML)](https://medimix.be/hematology/eha-madrid-2024-highlight-4/) - In this video, Dr Fabio Andreozzi, haematologist at the Institut Jules Bordet in Brussels, discusses the key highlights from EHA 2024 in the field of acute myeloid leukaemia (AML) Within the context of the BEAT AML Master Trial, investigators assessed the effect of adding the menin inhibitor revumenib to azacitidine + venetoclax in patients with KMTA2 rearranged or NPM1 mutated newly diagnosed AML who were ineligible for intensive chemotherapy. In the efficacy evaluable population (N=24) of this trial, the composite complete remission (CRc) rate was reported at 96%, with 92% of patients attaining minimal residual disease (MRD) negativity. Three patients proceeded to hematopoietic stem cell transplant (HSCT). Extended follow-up data were available for patients in the first cohort of this trial indicating a 12-month overall survival (OS) rate of 100%.1 In a phase I/II study reported by Bataller et al., the safety and efficacy of decitabine/cedazuridine in combination with venetoclax and gilteritinib was evaluated in patients with FLT3-mutated, newly diagnosed, or relapsed/refractory (R/R) AML or myelodysplastic syndrome (MDS). Among newly diagnosed patients, an overall response rate (ORR) of 83% was reported while this was 44% in the R/R cohort. No dose-limiting toxicities were observed, although myelosuppression was frequent.2 Olutasidenib is a potent, selective, oral inhibitor of mutant IDH1. During EHA 2024, 5-year results were presented of a phase II study evaluating this agent in 153 patients with R/R IDH1-mutated AML. In this trial, an ORR of 48% was reported with a median duration of response of 15.5 months and a median OS of 11.6 months. Of note, this high rate of response was obtained in a patient population with a high percentage of intermediate or poor cytogenetic risk (90%) and 1-7 prior lines of therapy (median 2), including venetoclax in 8% of patients.3 by Dr Andreozzi also selected two abstracts evaluating the integration of venetoclax in a high-intensity chemotherapy regimen. In the phase I/II SAL-RELAX trial, the combination of venetoclax with high-dose cytarabine and mitoxantrone resulted in a composite complete remission rate (CR and CR with incomplete blood count recovery) of 75% in a cohort of R/R AML patients. The safety profile of this regimen was acceptable.4 In a second study, the combination of intensive chemotherapy (FLAG-IDA) with venetoclax was evaluaeted in patients with newly diagnosed or R/R AML. This combination led to a complete response with complete blood count recovery (cCR) in 96% of newly diagnosed patients, with MRD-negativity in 88% of patients. After 30 months of follow, the median OS and event-free survival (EFS) were not reached (2 year rates 75% and 58%, respectively). In the R/R cohort, a cCR rate of 66% was observed, with MRD negativity in 76% of patients. The median OS and EFS in this patient group were 12 and 7 months, respectively. 5 During the plenary session of EHA 2024, Platzbecker and colleagues presented the first results of the APOLLO trial, a randomized phase III study evaluating the combination of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) in patients with newly diagnosed, high-risk acute promyelocytic leukaemia (APL). Patients in the ATRA-ATO arm received two doses of idarubicin (12 mg/m2 on days 1 and 3) and daily ATO (0.15 mg/kg) and ATRA (45 mg/m2) until a complete response was obtained. Subsequent consolidation therapy consisted of ATO (5 days/week, 4 weeks on 4 weeks off for 4 courses), in parallel with ATRA (2 weeks on 2 weeks off for 7 courses). Patients in the control arm received the AIDA regimen (i.e., ATRA + Idarubicin induction followed by chemotherapy-based consolidation [3 cycles] and maintenance therapy). After a median follow-up of 31 months, the ATRA-ATO regimen proved to be associated with a significantly better event-free survival (EFS) than the AIDA regimen, with 2-year EFS rates of 89% and 70%, respectively (p= 0.02). In addition, a trend for a better OS was seen with ATRA-ATO, with a 2-year OS rate of 93% as compared to 87% with AIDA (p= 0.33). 6 References 1. Zeidner, J. EHA2024. #S134. 2. Bataller, A. EHA2024. #139. 3. Cortes, J. EHA2024. #S144. 4. Ruhnke, L. EHA2024. #S135 5. Jen, W-Y. EHA2024. #S136. 6. Platzbecker, U. EHA2024. #S102.
- [EHA highlights](https://medimix.be/hematology/eha-madrid-2024-highlight-3/) - In this video, Dr Adriano Salaroli, haematologist at the Institut Jules Bordet in Brussels, shares his congress highlights from EHA 2024. For patients with chronic myeloid leukaemia (CML), the most interesting data at EHA 2024 came from the ASC4FIRST study, a randomized phase III trial comparing asciminib to all the current standard of care frontline BCR:ABL tyrosine kinase inhibitors (TKIs). At week 48, asciminib proved to be associated with a significantly higher rate of major molecular response (MMR) compared to the standard first line TKIs (67.7% vs. 49.0%; p< 0.001). Compared to the established TKIs, asciminib also came with a more favourable safety profile.1 In the context of myelofibrosis (MF), EHA 2024 featured two interesting studies evaluating novel combination regimens with ruxolitinib. In the phase III MANIFEST-2 trial, combining ruxolitinib with pelabresib, an oral inhibitor of bromodomain and extraterminal proteins, resulted in a significantly higher percentage of JAK inhibitor naïve MF patients obtaining a spleen volume reduction of ≥35% (SVR35) compared to ruxolitinib alone (65.9% vs. 35.2% at week 24; p< 0.001). Furthermore, the addition of pelabresib to ruxolitinib also came with a trend for a lower symptom burden and improved anemia.2 In a second phase III trial (TRANSFORM-1), adding the BCL2 inhibitor navitoclax to ruxolitinib resulted in a twofold increase in the percentage of JAK inhibitor naïve MF patients obtaining a SVR35 at week 24 (63% vs. 32%; p< 0.0001). Also in this trial, the combination therapy improved haemoglobin levels. Interestingly, the benefit obtained with navitoclax in this trial was particularly pronounced in patients with poor prognostic features.3 In the field of acute lymphoblastic leukaemia (ALL), Dr Salaroli discussed the results of a study evaluating the addition of blinatumomab to a paediatric-inspired prephase and consolidation treatment protocol (HOVON-146). The 4-year overall survival (OS) rate with the blinatumomab-containing regimen was reported at 86% in the cohorts of patients ≤40 years of age and in patients aged 40-60 years, while it was 50% in the subgroup of patients >60 years. This survival rate compares favourably to the OS rates observed in HOVON-100 evaluating a similar chemotherapy regimen without blinatumomab (4-year OS in patients ≤40 years: 76% [vs. 86%], >40 years: 51% [vs. 71%]). During the plenary session of EHA 2024, Platzbecker and colleagues presented the first results of the APOLLO trial, a randomized phase III study evaluating the combination of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) in patients with newly diagnosed, high-risk acute promyelocytic leukaemia (APL). Patients in the ATRA-ATO arm received two doses of idarubicin (12 mg/m2 on days 1 and 3) and daily ATO (0.15 mg/kg) and ATRA (45 mg/m2) until a complete response was obtained. Subsequent consolidation therapy consisted of ATO (5 days/week, 4 weeks on 4 weeks off for 4 courses), in parallel with ATRA (2 weeks on 2 weeks off for 7 courses). Patients in the control arm received the AIDA regimen (i.e., ATRA + Idarubicin induction followed by chemotherapy-based consolidation [3 cycles] and maintenance therapy). After a median follow-up of 31 months, the ATRA-ATO regimen proved to be associated with a significantly better event-free survival (EFS) than the AIDA regimen, with 2-year EFS rates of 89% and 72%, respectively (p= 0.02). In addition, a trend for a better OS was seen with ATRA-ATO, with a 2-year OS rate of 93% as compared to 87% with AIDA (p= 0.33). Based on these results, Dr Salaroli beliefs that this almost chemotherapy-free ATRA-ATO regimen will become the new standard of care for patients with newly diagnosed, high-risk APL.
- [Frontline therapy for multiple myeloma](https://medimix.be/hematology/eha-madrid-2024-highlight-2/) - In this video, Dr Rutger Callens, haematologist at the AZ Delta hospital in Roeselare, talks us through the highlights of the oral abstract session at EHA 2024 discussing innovations in the frontline treatment of patients with multiple myeloma (MM). CASSIOPEIA is randomized phase III trial including 1,085 transplant eligible, newly diagnosed MM patients (NDMM). Patients were initially randomized to 4 cycles of pre-ASCT induction (ind) and 2 cycles of post-ASCT consolidation (consol) therapy with daratumumab (D) + bortezomib-thalidomide-dexamethasone (VTd) or VTd alone. In a second phase, patients with a partial response or better were randomized to D maintenance for up to 2 years or observation. After >6.5 years of median follow-up, D-VTd ind/consol, prolonged the median progression-free survival (PFS) with ~2.5 years vs. VTd alone, corresponding to a 39% lower risk of disease progression or death (median: 83.7 vs. 52.8 months; HR[95%CI]: 0.61[0.52-0.72]; p< 0.0001). Also the overall survival (OS) was significantly improved with D-VTd, with a hazard ratio of 0.55 (95%CI: 0.442-0.73). In part II of the study, D maintenance reduced the risk of progression or death by 51%, with a 20% higher 72-month PFS rate compared to observation (57.1% vs. 36.5%). The incorporation of D in the different treatment phases also led to high rates of durable MRD-negativity.1 In the phase III PERSEUS study, adding D to bortezomib, lenalidomide, and dexamethasone (VRd) ind/consol followed by maintenance therapy with D + lenalidomide (R) significantly delayed disease progression and increased the depth of response compared to VRd alone followed by R maintenance in patients with transplant eligible NDMM. EHA 2024 featured the presentation of a detailed analysis of MRD in this trial. The rate of MRD-negativity proved to be consistently higher with the D-based therapy and this rate gradually increased over time. With D-VRd, the percentage of patients with sustained MRD-negativity (i.e., ≥12 months) at a level of 10-6 was 47.3% as compared to 18.6% in the control arm (p< 0.0001). Among patients who were MRD positive at the end of consol therapy, D-VRd was associated with a significantly higher proportion of patients achieving MRD negativity at10–6 during maintenance therapy (62.3% vs. 31.0%; p
- [Chronic Lymphocytic Leukemia (CLL)](https://medimix.be/hematology/eha-madrid-2024-highlight-1/) - In this video, Prof EM Dominique Bron, haematologist at the Jules Bordet Institute in Brussels provides us with a helicopter view of the contemporary treatment landscape for patients with chronic lymphocytic leukemia (CLL) and summarizes some of the key results in the field of CLL presented during the 2024 annual EHA meeting. Anno 2024, the treatment for patients with CLL is no longer steered by the fitness of the patients, but rather by the presence or absence of a del(17p), or TP53 mutation. In patients without this dismal prognostic feature, the current standard of care consists of a fixed duration treatment combination of either venetoclax + ibrutinib, or venetoclax + obinutuzumab. With these regimens, a median progression-free survival (PFS) of ≥80 months can be achieved, with MRD negativity in about 50% of patients. In patients with a TP53 mutation, there is consensus on the fact that a BTK inhibitor should be part of the first line treatment, either as a fixed duration combination of ibrutinib with venetoclax or as continuous therapy with a next-generation BTK inhibitor, such as acalabrutinib, or zanubrutinib. While significant progress has been made in the treatment of these patients, their prognosis continues to be inferior to that of patients without a TP53 mutation, indicating that there is still room for improvement. In this respect, data from arm D of the SEQUOIA trial, presented at EHA 2024 demonstrated impressive results with a combination of zanubrutinib and venetoclax.1 In a cohort of 66 treatment-naïve CLL patients with a TP53 mutation, this combination led to an objective response rate (ORR) of 100%, including 45% complete responses (CR). Furthermore, MRD negativity was obtained in 50% of patients, with a 2-year PFS rate of 94%.1 A second interesting study presented at EHA 2024 evaluated the safety and efficacy of the bispecific antibody epcoritamab in CLL patients with Richter’s transformation (RT), a notoriously difficult to treat patient population. Of the 35 patients enrolled in this study, 91% received at least 1 prior line of therapy for CLL or RT, including a BTK inhibitor in 57%. Of the patients who were pre-treated for RT, 76% received R-CHOP. The primary results of this study showed an ORR of 62%, with a CR in 35% of patients.2 A final presentation that was discussed by Prof Bron consists of a phase II study evaluating a triplet combination of the non-covalent BTK inhibitor pirtobrutinib, with venetoclax and obinutuzumab.3 The study enrolled a total of 40 treatment-naïve CLL patients, of whom 10% had a del(17p)/TP53 mutation. The first results with this triplet regimen are impressive, with an MRD negativity rate of 76%. More mature data are eagerly awaited.
- [ELEVATE-TN: Impact of first-year dose modifications in treatment-naive CLL patients treated with acalabrutinib](https://medimix.be/hematology/elevate-tn-impact-of-first-year-dose-modifications-in-treatment-naive-cll-patients-treated-with-acalabrutinib/) - Prof George Follows, a consulting haematologist from Nuffield Health Cambridge Hospital, presents a post-hoc analysis of the ELEVATE-TN trial, a prospective, randomized study involving treatment-naïve elderly CLL patients. Patients were assigned to receive either acalabrutinib monotherapy or a combination of acalabrutinib with obinutuzumab, compared to the standard arm of obinutuzumab plus chlorambucil. This poster specifically delves into the performance of patients based on dose reductions during the first year of treatment. Patients were categorized into four groups, ranging from those without dose reductions and treatment breaks to those who, for various reasons, were unable to continue acalabrutinib. An encouraging finding regarding OS emerged, indicating that over a 4-5-6 year follow-up period, OS remains largely consistent when patients can resume acalabrutinib treatment. The implication is that if a patient is medically fit to resume the drug, there is minimal compromise in terms of OS, notwithstanding dose reductions or treatment interruptions. Notably, 80% of patients in this trial did not experience dose reductions or treatment breaks. Among them, half received additional obinutuzumab during the first year, and this subgroup exhibited a distinct, statistically significant survival advantage. Although the overall 6 year survival outcome for all patients did not reach statistical significance, clinical observations suggest that considering the addition of obinutuzumab to acalabrutinib for patients who are deemed medically fit is a worthwhile consideration in practice.
- [cfDNA as alternative to bone marrow cells for molecular diagnostics and MM monitoring](https://medimix.be/hematology/cfdna-as-alternative-to-bone-marrow-cells-for-molecular-diagnostics-and-mm-monitoring/) - In this presentation, Dory Abelman, a PhD candidate at the Princess Margaret Cancer Centre, University of Toronto, assesses the concordance of molecular profiles between cancer cells derived from bone marrow (BM) and those obtained using cell-free DNA (cfDNA) from peripheral blood and BM plasma. Samples from 27 patients underwent sequencing using targeted panels encompassing exons from 37 genes, all immunoglobulin loci, translocations and copy number variations, employing NGS and sWGS. A noteworthy observation emerged when tumour fraction estimates exceeded 5%, indicating that more than 5% of the genome was altered by copy number aberrations. Under these conditions, a high concordance in copy number variations and factors such as del(1p,13q) and amp(1q) was identified in both peripheral blood and BM plasma cfDNA compared to BM cells. Given the cost-effectiveness of sWGS, this assay could potentially conserve BM cells, which are conventionally utilized in FISH assays. Across the targeted panel, immunoglobulin rearrangements demonstrated a concordance of over 90% between BM cells and cfDNA in peripheral blood and BM plasma. Over time, monitoring immunoglobulin rearrangements may prove valuable for MM surveillance.
- [The SKylaRK trial](https://medimix.be/hematology/the-skylark-trial/) - Dr Betsy O’Donnell, a haematologist at the Dana-Farber Cancer Institute (US), presents the findings from the SKylaRK trial, a phase 2 study in NDMM patients eligible for transplantation, who received a four-drug combination comprising isotuximab, carfilzomib, lenalidomide, and dexamethasone. The trial enrolled patients with all-risk cytogenetics, and participants underwent four cycles of the quadruplet regimen. Subsequently, after stem cell collection, patients had the option to defer transplant or proceed with transplantation. A majority of patients opted to defer transplant and underwent an additional four cycles of therapy. Results indicate a noteworthy OR of 100%, with a PFS of 92% and an OS of 95% at the two-year mark. Post-consolidation, an excellent MRD negativity rate was achieved. Noteworthy, maintenance therapy was stratified by risk, posing a critical question in NDMM patients, especially those achieving deep responses. Additional insights into the optimal duration of maintenance therapy will be gained as the study matures. High-risk patients, defined by del(17p), 1q gains, t(4:14), t(14:20), t(14:16), received a triplet maintenance combination comprising both the proteasome inhibitor and the monoclonal antibody, in contrast to the standard lenalidomide alone for standard-risk patients. Both arms exhibited outstanding results, underscoring the importance of addressing this question. An essential endpoint in this study is the QoL, which demonstrated significant improvement across most metrics for MM after a temporary decline post-transplant. This study underscores the overall efficacy and tolerability of the quadruplet regimen in NDMM patients eligible for transplantation.
- [Jmjd3 Drives Immature T-Cell Lymphoblastic Leukemia](https://medimix.be/hematology/jmjd3-drives-immature-t-cell-lymphoblastic-leukemia/) - T-ALL represents a highly aggressive haematological malignancy, characterized by an unmet clinical need, particularly in refractory patients or those resistant to standard treatments. Dr Tim Pieters, PhD at Ghent University describes his investigative approach, utilising mouse models that overexpress specific oncogenes to delve into the intricate biology of immature T-ALL. His focus centres on unravelling the role of Jmjd3, a demethylase with a D-metallase domain, in tumour initiation. This exploration involves the overexpression of Jmjd3 within the hematopoietic system, driving the development of a distinctly immature subtype of T-ALL. To dissect the molecular mechanisms at play, single-cell RNA sequencing on control spleens and splenic tumours within the mouse model was conducted. This analysis revealed the presence of genes associated with very immature T-ALL, alongside more mature T-cell markers such as CD3, CD4, and CD8. Further scrutiny identified elevated expression of the SPI-1 gene in the tumour fraction of the single-cell RNA sequencing dataset. This discovery was corroborated through proteomics analysis. Ongoing experiments are underway to elucidate the precise molecular mechanisms underlying these findings.
- [AML real world evidence initiative: venetoclax versus control in patients unfit for intensive chemotherapy](https://medimix.be/hematology/aml-real-world-evidence-initiative-venetoclax-versus-control-in-patients-unfit-for-intensive-chemotherapy/) - In this study, Dr Aaron Goldberg from the Memorial Sloan Kettering Cancer Center presents a comparative analysis of outcomes and healthcare research utilization in patients with AML treated with venetoclax (VEN) versus conventional therapeutic approaches. As part of the AML Real World Evidence initiative, a substantial retrospective study encompassing over 500 AML patients was conducted. Among them, 267 received VEN-based treatments, while another 267 underwent conventional regimens. The subgroup under investigation comprised patients deemed unsuitable for intensive chemotherapy, specifically those aged over 75 or with comorbidities. Ineligible chemotherapy recipients treated with VEN were matched with counterparts on non-VEN-based regimens, based on age categories (
- [Results from the Phase 1/2 Atalanta-1 trial](https://medimix.be/hematology/results-from-the-phase-1-2-atalanta-1-trial/) - An innovative decentralized and automated PoC manufacturing paradigm was devised to facilitate the expeditious administration of freshly prepared autologous CAR-T therapies within a 7-day timeframe following apheresis in patients diagnosed with relapsed/refractory non-Hodgkin lymphoma (NHL). Prof Marie José Kersten, a haematologist from UMC Amsterdam presented an update of the Atalanta-1 trial. As of the current phase, 14 participants have undergone treatment in the initial phase 1 segment of the investigation, while an additional 9 subjects have been enrolled in the subsequent phase 2 dose expansion segment. The outcomes reveal noteworthy OR and CR rates in both the phase 1 and phase 2 components, coupled with a commendably low incidence of adverse effects. Instances of Cytokine Release Syndrome and severe immune-related events are infrequently observed. Notably, there is substantial proliferation of CAR-T cells in the bloodstream, with marginal distinctions between the 50 million and 110 million dose cohorts. The persistence of CAR-T cells is robust, and patient recruitment for the study remains ongoing.
- [The Impact of Isatuximab HGG in R/R MM patients](https://medimix.be/hematology/the-impact-of-isatuximab-hgg-in-r-r-mm-patients/) - Dr Cesar Rodriguez, affiliated with Mount Sinai in New York, presents a research study examining the impact of isatuximab (Isa) on the occurrence of hypogammaglobulinemia (HGG) and the concomitant risk of infection in patients with R/R MM, whether administered as a standalone agent or in combination. The study compiled data from all investigations involving Isa as a monotherapy, including results from the ICARIA-MM and IKEMA studies. These datasets were systematically analyzed to assess the incidence of HGG in the control group as compared to patients treated with Isa. Upon incorporating Isa into therapeutic regimens, there was observed HGG, yet this did not necessarily result in a heightened susceptibility to infections. Notably, the correlation between infection rates in these therapeutic interventions was more closely associated with neutropenia rather than HGG. This observation is substantiated by the discovery that the occurrence of grade 3 and grade 4 infections did not significantly escalate in the Isa treatment groups. Remarkably, this finding persists despite the absence of prophylactic IVIG use, a preventive measure currently employed with anti-CD38 agents and bispecific T-cell engagers to mitigate infection risks. In conclusion, our study underscores the importance of directing attention toward neutropenia in combination therapies rather than exclusively focusing on HGG, which emerges as the primary driver of infections in anti-CD38 and combination therapy contexts. As part of the AML Real World Evidence initiative, a substantial retrospective study encompassing over 500 AML patients was conducted. Among them, 267 received VEN-based treatments, while another 267 underwent conventional regimens. The subgroup under investigation comprised patients deemed unsuitable for intensive chemotherapy, specifically those aged over 75 or with comorbidities. Ineligible chemotherapy recipients treated with VEN were matched with counterparts on non-VEN-based regimens, based on age categories (
- [Clonal Heterogeneity and Evolution during Treatment in High Hyperdiploid  B-Cell ALL](https://medimix.be/hematology/clonal-heterogeneity-and-evolution-during-treatment-in-high-hyperdiploid-b-cell-all/) - Dr Margo Aertgeerts from UZ Leuven presented her PhD research in ALL, the most prevalent cancer in the pediatric population. The aetiology of ALL involves the incremental acquisition of mutations by lymphoid progenitor cells, resulting in the formation of a heterogeneous population of leukaemia cells. Each of these cells may harbour distinct mutational profiles, potentially contributing to varying treatment sensitivities. Our hypothesis posits that greater heterogeneity within the leukemic cell population correlates with an increased susceptibility to relapse. To scrutinize this heterogeneity, we employed single-cell DNA sequencing, which enabled the identification of single nucleotide variants within individual cells. This approach not only facilitated the exploration of diverse mutations but also provided insights into the chronological order in which they were acquired. These findings were subsequently correlated with bone marrow samples obtained from the same patients after the induction phase, where residual leukaemia cells persist. Notably, KRAS-positive cells exhibited reduced sensitivity to induction treatment compared to their NRAS-positive counterparts; however, a favourable response was observed after the initial consolidation phase. Analysis of a relapsed patient revealed that the clones identified at diagnosis exhibited greater complexity compared to samples from non-relapsed individuals. Tracking these clones from diagnosis through the end of induction to relapse unveiled the presence of a dominant clone at relapse, albeit at a minimal percentage during the diagnostic phase. Furthermore, a comprehensive examination of proteins in various cell populations revealed distinctions between normal cells and B-ALL cells. Systematic investigation of individual B-ALL samples at different treatment stages, including identification of CD19-negative cells following anti-CD19 CAR-T cell therapy, highlighted the dynamic nature of the leukemic cell population. In conclusion, our study successfully characterized distinct clones at different disease stages—diagnosis, end of induction, and relapse. Further exploration, particularly of relapse samples, holds promise for refining risk stratification strategies, ultimately enhancing the management of pediatric ALL patients.
- [A dual Role of SOX11 expression in the formation of T-cell ALL in mouse models](https://medimix.be/hematology/a-dual-role-of-sox11-expression-in-the-formation-of-t-cell-all-in-mouse-models/) - André De Almeida, PhD student at the University Hospital Ghent presented his research on investigating the role of SOX11 in T-ALL, which is motivated by its expression within a distinct subset of T-ALLs. To delineate the impact of aberrant SOX11 expression, diverse mice models were generated. These encompassed a model with T-cell-specific overexpression of SOX11, another featuring concurrent overexpression of SOX11 and Lmo2, and a third with Pten loss. The latter models are recognized for spontaneously developing TLL. The overexpression of SOX11 induced perturbations in T-cell differentiation in both mice and human cells. Specifically, an augmentation in immature T-cell populations was observed, concomitant with a reduction in the mature T-cell subset. Importantly, this phenomenon was determined not to be attributable to enhanced self-renewal properties of thymocytes induced by SOX11. The co-expression of Lmo2 was found to accelerate the onset of leukaemia formation, contrasting with the delayed leukemogenesis observed in the Pten-deficient mice. In summary, the successful development of murine models demonstrated a substantial impact of SOX11, though the underlying mechanistic intricacies necessitate further elucidation.
- [A 46 long non-coding RNAs expression signature as independent prognostic factor in pediatric AML](https://medimix.be/hematology/a-46-long-non-coding-rnas-expression-signature-as-independent-prognostic-factor-in-pediatric-aml/) - Approximately 30% of pediatric AML patients encounter relapse, emphasizing the imperative need for refining risk stratification in this cohort. Consequently, Zhiyao Ren, PhD student at University Hospital Ghent, constructed a signature comprising 46 lncRNAs associated with relapse and assessed its prognostic efficacy for RFS in pediatric AML. Internal validation substantiated the robust performance of the signature in predicting 1, 2, and 3-year RFS. Moreover, external validation across independent cohorts corroborated its prognostic utility, establishing the signature as an independent prognostic determinant. Intriguingly, our signature not only served as an independent predictor of RFS but also stratified patients into distinct risk groups, thereby diverging from conventional risk classification factors.
- [Pragmatic recommendations for treating patients with myelofibrosis, particularly those experiencing cytopenias](https://medimix.be/hematology/pragmatic-recommendations-for-treating-patients-with-myelofibrosis-particularly-those-experiencing-cytopenias/) - Prof Claire Harrison, a haematologist at Guy's and St. Thomas' Hospital in London (UK), presents collaborative efforts involving colleagues and patients to offer pragmatic recommendations for treating patients with myelofibrosis, particularly those experiencing cytopenias. Utilizing Modified Delphi technology driven by consensus, the process involved defining topics of unmet need and formulating agreed-upon recommendations. A steering committee, comprising global experts, and an extended faculty of over 30 individuals and representatives from patient advocacy organizations, were involved. The inclusion of patients in shaping these recommendations introduced a unique and essential perspective. The study addressed various unmet needs in myelofibrosis, focusing on prognostic criteria, their selection, and timing. A comprehensive table is presented to summarize the applicable prognostic tools, accommodating scenarios where next-generation sequencing and an extended genetic profile might not be feasible. Recommendations were crafted for managing critical areas of unmet need in patients, such as anaemia and thrombocytopenia. A structured approach for investigating and managing these patients was outlined. Additionally, the study identified key factors signalling the need to reconsider therapy, highlighting indicators of treatment ineffectiveness. Given the array of available therapy options, deciding when to change therapy can be challenging, and this work aims to assist colleagues internationally in navigating these decisions. The next step involves preparing a publication and exploring additional communication methods to disseminate this valuable information.
- [First results of a Lysa study from the French Descar-T registry](https://medimix.be/hematology/first-results-of-a-lysa-study-from-the-french-descar-t-registry/) - Dr Gabriel Brisou presents preliminary findings on the utilization of axi-cel in the second-line treatment of patients with early R/R LBCL. In France, real-world data encompassing all patients subjected to approved CAR T-cell therapy are systematically recorded in the DESCAR-T database. The administration of axi-cel infusion has received approval from French regulatory authorities for patients experiencing LBCL recurrence within one year of initial treatment or exhibiting refractoriness after the first-line therapy. As of July 2022, approximately 200 patients have undergone axi-cel infusion. Notably, 12% of cases were excluded from infusion primarily due to disease progression. Among those who did receive the infusion, over 85% underwent bridging therapy, with less than half of them exhibiting a positive response. Regarding safety assessments, observed data include CRS and neurotoxicity, aligning with findings from other trials such as ZUMA-7 and ALYCANTE. Available efficacy data extend only up to a three-month follow-up period. Once again, OR and CR rates mirror those observed in ZUMA-7 and ALYCANTE, with approximately 60% of patients achieving CR. However, the immature nature of the data due to the brief follow-up period is acknowledged. In contrast to the ZUMA-7 trial, where bridging therapy was not administered, real-world data presented here indicate that the majority of patients undergo bridging therapy. This discrepancy highlights the need for further investigation into optimizing CAR-T outcomes through the strategic use of bridging therapy. An additional noteworthy distinction lies in the performance of intermediate PET-CT scans, and the selection of some patients based on a positive PET after four cycles of R-CHOP. A dedicated subgroup analysis of these patients is planned, emphasizing the ongoing exploration of refined strategies in CAR-T therapy.
- [Risk of infections in multiple myeloma in the era of novel agents](https://medimix.be/hematology/risk-of-infections-in-multiple-myeloma-in-the-era-of-novel-agents/) - Dr Cecilie Blimark, haematologist at the Sahlgrenska University Hospital in Sweden summarized her extensive population-based investigation encompassing symptomatic MM patients from the Swedish Myeloma Registry, ensuring a robust 98% coverage within the cancer registry. A cohort of 8,600 MM patients was meticulously tracked from 2008 to 2022 or until the time of death, and was systematically paired with 4 controls matching in terms of age, gender, and geographical region. The findings reveal a 5-fold increase in the risk of bacterial infections, a 7-fold increase in the risk of viral infections, and an 11-fold increase in the risk of fungal infections among MM patients. These augmented risks manifested in susceptibility to severe conditions such as septicemia, pneumonia, meningitis, pyelonephritis, endocarditis, as well as infections like herpes zoster, influenza, CMV, and COVID. Furthermore, all documented infections were of clinical significance, with a likelihood of being underreported, indicating a potentially higher prevalence. Approximately 30% of deaths occurring within one year post-diagnosis were attributed to infections. Thus, the concern extends beyond the mere incidence of infections in these patients; rather, the life-threatening ramifications pose a substantial challenge. In light of these findings, forthcoming clinical guidelines ought to extend beyond the prevention of bacterial infections and also encompass strategies for mitigating the more severe viral and fungal infections, given their pronounced impact and heightened risk among MM patients.
- [The REMNANT study: will treatment at MRD relapse improve survival in MM?](https://medimix.be/hematology/the-remnant-study-will-treatment-at-mrd-relapse-improve-survival-in-mm/) - Dr Frida Bugge Askeland, associated research doctor and PhD student at the Oslo Myeloma Center in Norway outlined the REMNANT study. The REMNANT study aims to assess whether addressing MRD relapses following initial treatment enhances PFS and OS in myeloma patients. The clinical design of this phase 2/3 study involves 400 transplant-eligible MM patients undergoing standard of care treatment in part 1. This treatment comprises four pre-transplant induction and four post-transplant consolidation cycles of bortezomib, lenalidomide, and dexamethasone (VRd). After induction, patients undergo either tandem or single transplant. All patients receive lenalidomide maintenance, the sole approved and reimbursed therapy in Norway. The inclusion criteria are extensive, encompassing patients with kidney failure, amyloidosis, plasma cell leukaemia and other comorbidities. MRD is evaluated using flow cytometry with a sensitivity of 10-5 at diagnosis and post-consolidation in the first line. Patients achieving CR MRD status post-consolidation proceed to part 2 of the study, while others are monitored for 24 months on lenalidomide maintenance and join part 2 upon attaining MRD negativity. Part 2 constitutes the randomized segment, wherein 176 patients receive second-line treatment at either loss of CR or MRD negativity or PD. In the MRD-guided experimental arm, MRD negativity is assessed every four months. Data currently available pertains only to part 1, focusing on standard treatment. In first-line treatment, an ORR of 98% was observed, with 55% of patients achieving an MRD-negative complete response. Bortezomib was initially administered twice weekly during first-line treatment, but nearly half of the patients had to discontinue prematurely due to neuropathy. Consequently, the protocol was modified after 200 patients to administer bortezomib once a week, facilitating a later comparison of side effects and efficacy. Given the significance of MRD for prognosis and the incurable nature of MM, ongoing trials exploring MRD status as a guide for treatment intensification or de-escalation are crucial. Definitive conclusions are pending the completion of these trials. Currently, there is no conclusive outcome of the REMNANT study, as this will be derived from the second part.
- [The REST study](https://medimix.be/hematology/the-rest-study/) - Dr Frida Bugge Askeland, associated research doctor and PhD student at the Oslo Myeloma Center in Norway summarised the REST study. In Norway, the use of VRd (bortezomib, lenalidomide and dexamethasone) or Rd alone represents the standard of care for patients with newly diagnosed multiple myeloma (NDMM) who are ineligible for transplant. The MAIA trial demonstrated that adding a CD38 monoclonal antibody to Rd results in better and deeper responses and prolonged PFS. The REST study evaluated isatuximab (Isa) combined with VRd as first-line treatment in transplant-ineligible NDMM patients with a median age of 77 years. Isa was introduced as a replacement for corticosteroids in the Isa-VRd regimen. The rationale behind limiting or substituting corticosteroids in MM treatment stems from their potential to adversely impact both short-term and long-term quality of life, while also increasing susceptibility to infections. Infections emerge as a predominant cause of complications and mortality, with an escalating incidence correlated to age. In the REST study, bortezomib was administered on a once-weekly basis for the initial eight cycles. Notably, only six cases of peripheral neuropathy attributable to bortezomib were observed, comprising three cases of grade 2 severity and three cases of grade 3 severity. Adverse events primarily manifested as infections and neutropenia. The majority of infections were categorized as grade 2. The predominant haematological toxicity observed was neutropenia, with a significant proportion graded as level 3. Many patients received growth-stimulating factors to manage this haematological toxicity. After a median follow-up of 12 months, the ORR was reported at 100%, with a very good partial response or better achieved in 80.3% of cases. In conclusion, the Isa-VRd regimen demonstrates a well-tolerated safety profile and promising preliminary efficacy in transplant-ineligible NDMM patients with a median age of 77 years. Follow-up is ongoing.
- [Proactive and systematic hemophilia carrier screening in females with hemophilia](https://medimix.be/hematology/proactive-and-systematic-hemophilia-carrier-screening-in-females-with-hemophilia/) - Hemophilia recognized as a disorder characterized by excessive bleeding, has conventionally been considered exclusive to male individuals. However, emerging research highlights the potential manifestation of haemophilia in females who carry the affected X-chromosome, thereby transmitting the condition to their male offspring. It is noteworthy that a substantial number of affected women remain undiagnosed. This research endeavour focuses on the proactive and systematic screening of all female family members associated with haemophilia patients to identify carriers of the disease. Retrospective data from previously screened individuals were compiled, and efforts were made to extend the screening to previously unscreened individuals. Within the cohort of 236 families afflicted by haemophilia, a total of 900 female individuals were identified, comprising 454 carriers and 118 non-carriers. The carrier status of 320 patients was unknown, and their coagulation factor levels had not been previously assessed. Significantly lower coagulation factor levels were observed in carriers compared to non-carriers, and it was determined that those with affected deficiency typically exhibited moderate haemophilia, with severe cases being rare. In the context of women with undiagnosed haemophilia, heavy menstrual bleeding emerged as a potential indicator of the disease. Distinctions between HA and HB were noted in terms of bleeding patterns and hemostatic treatment requirements. No discernible differences were observed in mean age at diagnosis or mean coagulation factor levels. This study contributes a comprehensive inventory of the patient and carrier populations within the haemophilia cohort, albeit acknowledging that not all females have undergone screening. Challenges such as loss of contact with family members, study fatigue, or inadequate awareness about the significance of carrier status may impede comprehensive screening efforts. Consequently, this study underscores the necessity for ongoing haemophilia carrier screening as an integral component of every comprehensive haemophilia clinic, regardless of gender. In conclusion, it is imperative to ensure that individuals, irrespective of gender, who are potentially affected by haemophilia, have access to timely diagnosis and appropriate care. This necessitates a sustained commitment to haemophilia carrier screening within the clinical setting to enhance our understanding of the disease and facilitate optimal management strategies.
- [Health related QoL data of KarMMa-3](https://medimix.be/hematology/health-related-qol-data-of-karmma-3/) - KarMMa-3 constitutes an open-label, phase 3 randomized clinical trial aimed at investigating the efficacy of a single infusion of the BCMA-targeting CAR-T cell product ide-cel in comparison to conventional standard-of-care drug-based regimens in patients with RRMM. The enrolled patient cohort was characterized by extensive prior treatments, with all participants having been heavily pretreated, and a notable proportion (2 out of 3 patients) demonstrating refractoriness to triple-class therapies. The primary endpoint of this study was PFS, and the results indicated a statistically and clinically significant prolongation of PFS in patients treated with ide-cel. The main objective of the study is to establish durable responses but without compromising QoL for the patients. Consequently, an important sub-study within the KarMMa-3 trial focused on HRQoL. This investigation employed various standardized scales, including the EORTC QLQ-C30, EORTC QLQ-MY20, and the EuroQol utility index, assessing patients at different intervals over an observation period exceeding two years. An initial decrease in global health status was observed, attributable to the fact that patients were precluded from receiving any new treatments during the CAR-T manufacturing process. Following the infusion of ide-cel, a prompt and statistically significant improvement in QoL parameters was observed, manifesting not only as statistical significance but also as clinical relevance. Overall health status enhanced, encompassing physical well-being, mental functionality, and mitigation of core side effects such as fatigue and pain. This improvement stood in marked contrast to the control group, wherein QoL either stabilized or exhibited deterioration. Notably, the reported HRQoL enhancement manifested earlier in treated patients than in those undergoing the standard regimen. In conclusion, the singular administration of ide-cel not only yielded a markedly superior haematological response but also significantly enhanced the overall quality of life for the patients.
- [Primary results of the Perseus trial investigating daratumumab in NDMM patients](https://medimix.be/hematology/primary-results-of-the-perseus-trial-investigating-daratumumab-in-ndmm-patients/) - The current standard of care for patients with NDMM involves four cycles of VRd induction pre-ASCT, followed by two VRd cycles of consolidation post-ASCT, and subsequently, a maintenance phase with lenalidomide. Under this regimen, the mean PFS is approximately 5 years, with approximately half of the patients achieving complete remission. The Perseus study seeks to evaluate the impact of incorporating daratumumab (D) into the established standard of care. In this treatment paradigm, patients undergo a minimum of 2 years of maintenance with sustained MRD negativity for at least 12 months, after which D can be discontinued, and patients continue with lenalidomide monotherapy. This study involves the randomization of 700 patients in a 1:1 ratio between the standard and experimental arms. Baseline patient characteristics were comparable and representative of typical NDMM patients. The primary endpoint of the study is PFS, and with a median follow-up of 4 years, PFS has not yet been reached in the experimental arm. Notably, the 4-year PFS in the control arm was 68%, while in the experimental arm, 84% of patients remained alive and without disease progression. This translates into a remarkable 58% risk reduction in progression with the addition of D. Furthermore, the incorporation of D into the standard regimen led to an increased depth of response, with an 88% CR compared to 70% in the control arm. In two-thirds of patients, MRD negativity in the bone marrow was achieved at a sensitivity level of 10-6, sustained at the level of 10-5, allowing for the discontinuation of D during maintenance. Importantly, the addition of D did not compromise the collection of stem cells for ASCT, and no new side effects were observed. In conclusion, the findings from this study indicate a shift in the standard of care for transplant-eligible NDMM patients, with the establishment of D-VRd as a new and highly effective treatment approach.
- [Pirtobrutinib in relapsed/refractory MCL patients: update from the BRUIN trial](https://medimix.be/hematology/pirtobrutinib-in-relapsed-refractory-mcl-patients-update-from-the-bruin-trial/) - BTKi has demonstrated efficacy in MCL; however, a substantial number of patients experience relapse during treatment. Pirtobrutinib, a non-covalent third-generation BTKi, exhibits enhanced effectiveness in the post-covalent BTKi setting and greater selectivity compared to earlier agents. The OR for pirtobrutinib is 58%, with a CR observed in 20% of patients. The favourable pharmacokinetics of pirtobrutinib, maintaining concentrations above the IC90 24/7, facilitate once-daily oral administration. Its limited occurrence of grade 3 adverse effects enhances its usability and potential for combination with future therapeutic agents. The Bruin study, a phase 1/2 trial, established the optimal daily dose of pirtobrutinib at 200 mg. In this study 152 MCL patients previously treated with BTKi and 14 treatment-naive MCL patients were enrolled. Approximately half of these patients with known TP53 mutation status exhibited the mutation, while two-thirds with known Ki-67 status had a proliferative index of 30% or greater. A remarkable improvement in the overall burden of disease was evident, with an overall median time to the first response of 1.8 months, corresponding to the initial disease assessments in the trial. Regardless of the subgroup, the OR remained consistently around 45-50%. Patients discontinuing previous BTKi treatment due to toxicity exhibited a notably high OR, aligning with expectations. Updated survival curves revealed a median DoR of 21.6 months, a median PFS of 5.6 months, and a median OS of 23.5 months. Analysis of high-risk subgroups indicated that TP53-mutated patients and those with a Ki-67 status over 30% exhibited a lower response rate and shorter PFS to pirtobrutinib. However, when a response occurred, its duration was prolonged, suggesting potential benefits for this patient subset. In a smaller cohort of treatment-naive patients, the OR was an impressive 86%, with half achieving CR and the other half partial response. After almost 2 years of follow-up, median DoR, OS, and PFS were not reached. Despite the limited patient pool, these findings are encouraging. Throughout the study, no unexpected safety issues emerged, and few grade 3 adverse events were noted, affirming the safety and ease of use of pirtobrutinib in relapsed/refractory MCL patients.
- [Patient-Reported Outcomes in the QuANTUM-First Trial: Quizartinib Versus Standard Chemotherapy in FLT3-ITD AML patients](https://medimix.be/hematology/patient-reported-outcomes-in-the-quantum-first-trial-quizartinib-versus-standard-chemotherapy-in-flt3-itd-aml-patients/) - At the ASH meeting, Professor Oliva presented the patient-reported outcomes from the QuANTUM-First trial, a phase 3 clinical investigation assessing the efficacy and safety of quizartinib. Quizartinib, is approved in the US, Europe, and Japan for concurrent use with chemotherapy in the induction, consolidation phase and maintenance monotherapy for adults with newly diagnosed FLT3-ITD positive AML. Quizartinib demonstrated a clinical benefit over placebo, yielding a 17-month increase in OS as the primary endpoint. Patient-reported outcomes on QoL were evaluated using the EORTC QLQ-C30 and EuroQol EQ-5D-5L instruments. Baseline measurements were collected on day 8 of the first induction cycle and compared to measurements at specific intervals throughout induction, consolidation, and continuation cycles. For patients receiving quizartinib, a clinically significant improvement in QoL was observed post-induction and consolidation, persisting during the continuation phase. No statistical differences were noted between treatment arms for QoL and TUDD measurements. In summary, the findings suggest that quizartinib treatment does not induce short- or long-term detrimental effects on QoL compared to placebo while demonstrating a clear benefit in OS, making it one of the best therapeutic choices for FLT3-ITD AML patients receiving induction chemotherapy.
- [IsKia trial: results of Isa-KRd in transplant-eligible NDMM patients](https://medimix.be/hematology/iskia-trial-results-of-isa-krd-in-transplant-eligible-ndmm-patients/) - Dr Annemiek Broijl, haematologist at the Erasmus MC hospital in Rotterdam, the Netherlands and co-principal investigator of the IsKia study was so kind to present the results. The study aims to assess the effectiveness of incorporating isatuximab into the standard treatment regimen of carfilzomib, lenalidomide and dexamethasone (KRd) during the pre-ASCT induction and post-ASCT consolidation phases in patients with NDMM. The induction and consolidation phases each comprised four cycles, followed by a 12-week maintenance phase with a reduced dose of Isa-KRd administered less frequently. The data about the primary endpoints, specifically MRD negativity with NGS sensitivity set at 10-5 and 10-6, are presented. The incorporation of isatuximab into the KRd regimen significantly enhanced MRD negativity at the 10-5 sensitivity level, with a rate of 77% compared to 67% in the control arm. This difference was more pronounced at a higher sensitivity of 10-6, where the Isa-KRd group demonstrated a rate of 67% compared to 48% in the control group. Interestingly, this improvement was observed across all risk groups, including normal-risk patients, those with a single high-risk abnormality such as del(17p), t(14;16) or t(4;14), and higher-risk patients with two or more of these cytogenetic abnormalities or with gain(1q21) or amp(1q21). In all risk groups, MRD at the 10-5 level was comparable in patients receiving Isa-KRd, while a decline was noted in those on the KRd regimen. At the 10-6 sensitivity level, this trend was even more pronounced, with risk groups exhibiting a high MRD negativity ranging between 77-79% in the Isa-KRd arm. At each measured time point, including post-induction, post-high dose and post-consolidation, there was a 20% increase in MRD negativity. The safety data from the combined treatment revealed minimal cardiotoxicity, with less than 5% incidence in both arms, addressing concerns related to carfilzomib. Peripheral neuropathy was notably low, and while neutropenia was observed, it was within the expected range. While the results are encouraging for the Isa-KRd arm, the focus has been primarily on MRD negativity. Evaluation of the impact on PFS awaits further investigation. Initial PFS data after one year did not show any significant differences. Future research aims to establish sustained MRD results that translate into improved PFS outcomes for patients.
- [Safety and PK/PD data on the use of ALLO-647 for lymphodepletion in R/R LBCL and FL patients](https://medimix.be/hematology/safety-and-pk-pd-data-on-the-use-of-allo-647-for-lymphodepletion-in-r-r-lbcl-and-fl-patients/) - Allogeneic CAR T-cell therapies represent a potentially readily accessible therapeutic alternative derived from healthy donors. However, ensuring a secure and efficient method to regulate host lymphocyte rejection (allo-rejection) is imperative. This presentation elucidates the safety and PK/PD outcomes of ALLO-647, an anti-CD52 monoclonal antibody, in patients with R/R LBCL and FL. A total of 87 patients participated in the ALPHA and ALPHA2 studies, encompassing those with R/R LBCL (n=61), including 34 who were naive to CAR T-cell therapy, and FL (n=26). The patients were treated with ALLO-647, before undergoing CAR T-cell infusion, with the doses administered 39, 60, and 90 mg in 11 (13%), 39 (45%), and 37 (43%) patients, respectively. The median follow-up period was 29.5 months (range, 6.8-47.5). There were no unforeseen safety issues, GvHD or ICANS of ≥ grade 3 observed. Approximately 24% of the patients experienced low-grade CRS, with only one patient experiencing ≥ grade 3 CRS. The predominant infection event was low-grade and manageable CMV reactivation. Patients who exhibited a positive lymphoma response generally received higher doses of ALLO-647, indicating a dose-dependent relationship. In conclusion, this data suggests that ALLO-647 provides an immediately available, safe, and single-dosing option for patients eligible for CAR T-cell therapy.
- [Efficacy of Brexu-cel in the treatment of high risk R/R MCL patients](https://medimix.be/hematology/efficacy-of-brexu-cel-in-the-treatment-of-high-risk-r-r-mcl-patients/) - In this presentation, data sourced from the CIBM-TR registry were utilized to assess the differential outcomes of Brexu-cel therapy in patients with R/R MCL exhibiting high-risk features, such as delTP53/17P, high Ki-67 (≥50%), and eligibility for ZUMA-2 clinical trials. The data analysis encompassed 456 adult patients who underwent Brexu-cel therapy, with a median follow-up period of 12.3 months. Notably, 42% of these patients presented with delTP53/17P and/or high Ki-67 and 57% of the cohort would have been ineligible for ZUMA-2, due to the presence of comorbidities before infusion. The examination of efficacy and safety revealed consistent ORR and CR rates across all treated high-risk subgroups. The median DOR had not yet been reached. No statistically significant differences were observed in OS and PFS at any designated time point. Following multivariate adjustment, Bruxa-cel exhibited comparable efficacy and safety profiles in patients both with and without high-risk features. While numerically longer OS and PFS were noted in patients without high-risk features, statistical significance was not demonstrated. This real-world study lends support to the proposition that Bruxa-cel should be considered a standard of care in the treatment of R/R MCL patients, including those presenting with high-risk features.
- [The impact of bridging therapy before Axi-cel in R/R LBCL patients](https://medimix.be/hematology/the-impact-of-bridging-therapy-before-axi-cel-in-r-r-lbcl-patients/) - Dr Fred Locke from Moffitt Cancer Center in Florida shares the data that were presented at the oral session on real world data of cellular therapies for B cell lymphomas at ASH2023. The results presented explored the impact of pre-treatment bridging therapy before administering Axi-cel, a CD19-directed CAR T-cell therapy, on outcomes for patients with R/R LBCL. Data from 1200 patients were collected through the CIBM-TR registry, using propensity score weighting to assess the effects of bridging therapy. Analysis revealed that mild hepatic impairment, over three prior lines of therapy, or recent Axi-cel infusion increased the likelihood of receiving bridging therapy. Conversely, patients with histologic transformation or a longer time from diagnosis to leukapheresis (>12 months) were less likely to undergo bridging therapy. Bridging therapy selection showed considerable heterogeneity, with 58% receiving CT with an anti-CD20 mAB, predominantly rituximab. Other approaches included CT alone, non-CT bridging therapies and in 10% of cases radiotherapy, either with or without systemic therapy. After 36 months, patients with bridging therapy had an ORR of 70% and a CR of 51% with a mean PFS of 3.6 months. In patients without bridging therapy ORR and CR were 79% and 64%, respectively, with a mean PFS of 14.7 months. However, multivariate analysis did not find significant differences in axi-cell treatment outcomes between patients receiving and not receiving bridging therapy. Toxicity profiles were generally similar, except for prolonged neutropenia and thrombocytopenia in the bridging therapy group. Axi-cel therapy consistently demonstrated effectiveness regardless of the bridging therapy type, and overall, bridging therapy did not enhance outcomes, except for responders. In conclusion, bridging therapy is recommended only when disease progression is expected within the 3-week turnaround time required for Axi-cel manufacturing.
- [Pros and cons of emerging therapeutic targets in multiple myeloma](https://medimix.be/hematology/pros-and-cons-of-emerging-therapeutic-targets-in-multiple-myeloma/) - Dr Rutger Callens, a haematologist at AZ Delta in Roeselare, delved into the intricacies of emerging therapeutic targets in multiple myeloma (MM), weighing the pros and cons associated with these advancements. Within the BCMA-directed therapeutic domain, notable modalities include CAR T cells, T-cell engagers (TCE) and antibody-drug conjugates (ADC). A pivotal advantage of CAR T therapy lies in its singular administration, leading to a protracted treatment-free remission, a distinctive feature within the relapsed/refractory paradigm of MM. However, the non-off-the-shelf nature of CAR T cells poses a drawback, necessitating bespoke fabrication and potentially mandating a bridging treatment. Despite encountering CRS, ICANS and infections post-therapy, the observed degrees of CRS and ICANS are generally milder and more manageable than those witnessed in CD19-directed CAR T therapies. BCMA-directed TCE offer the notable advantage of being readily available off the shelf and are amenable to combination with other therapeutic molecules. Reflecting on historical developments with proteasome inhibitors, IMIDs, and anti-CD38 antibodies, monotherapy yielded an overall response rate of approximately 30%. However, combining these agents demonstrated a remarkable extension. In the case of BCMA TCE, the overall response rate stands at around 60-70%, providing a promising foundation for future combinatorial approaches that may further enhance patient survival. A distinctive characteristic of TCE is their potential to induce CRS. To mitigate these associated risks, a common strategy involves a step-up dosing regimen. Additionally, infections represent a prevalent side effect of CAR-T and BCMA-directed therapy. A recent study from a French group presented at ASH indicated that approximately half of patients undergoing TCE treatment experienced a grade 3 or higher infection. Notably, these infections were predominantly pulmonary or systemic, encompassing bacterial and viral origins. Consequently, it remains imperative to administer appropriate anti-infectious prophylaxis following TCE and BCMA-directed CAR T therapy to optimize patient outcomes and safety. The third modality within the BCMA-directed therapeutic landscape involves ADC, exemplified by belantamab mafodotin. Belantamab is notably linked to ocular toxicity, a factor that has impeded its routine utilization. However, recent findings in a second-line therapy context demonstrated that the combination of belantamab with bortezomib and dexamethasone exhibited superior PFS compared to the VRd regimen. This development raises intriguing prospects for a potential resurgence in the use of belantamab. A secondary therapeutic target in focus is GPRC5D, with talquetamab serving as a representative TCE extensively examined in the MonumenTAL study. This intervention yielded an overall response rate of approximately 70%. Distinctive to GPRC5D-directed therapies is the occurrence of less common side effects, including taste aberrations, as well as nail and skin alterations. While these effects are generally non-life-threatening, they significantly impact patients' quality of life and frequently evoke concerns. In the phase 1b MonumenTAL study, patients who achieved a partial response or better were afforded the option of dose de-escalation. Notably, this approach revealed that a significant majority of these patients demonstrated a deepening of responses. Moreover, some individuals experienced improvements in side effects. Furthermore, in the realm of GPRC5D-directed therapies, a recently presented CAR T therapy demonstrated promise, boasting an impressive overall response rate of 87% within a heavily pretreated patient cohort, all of whom had undergone prior BCMA-directed therapies. In a distinct category of molecules, iberdomide and mezigdomide represent two Cereblon E3 ligase modulators (CeLMoDs). CeLMoDs are recognized for their cerebral modulation properties, and both iberdomide and mezigdomide have demonstrated promising results, particularly in heavily pretreated patient populations. These agents primarily manifest haematological toxicities, which, thus far, appear to be manageable. Lastly, in the context of translocation involving chromosome 14, BCL2 inhibitors have emerged as promising therapeutic candidates. While the BELLINI trial did not achieve its intended endpoint of overall survival benefits, noteworthy outcomes were observed in the combination of venetoclax with daratumumab and dexamethasone. This regimen exhibited robust responses, a high rate of MRD negativity and an extended PFS. These findings underscore the potential efficacy of BCL2 inhibition in the treatment of MM. A novel agent, sonrotoclax, appears to boast a more favourable toxicity profile compared to venetoclax. The anticipation is that this improved safety profile could pave the way for broader utilization of BCL2 inhibitors in the MM treatment landscape.
- [The role of NETs and thrombosis in different disease states](https://medimix.be/hematology/the-role-of-nets-and-thrombosis-in-different-disease-states/) - The therapeutic potential of inhibiting NETs in various pathological conditions is elucidated in a scientific discourse by Dr Martinod. Dr Martinod's research primarily delves into the PAD4 molecule's involvement in the early stages of NET formation, offering a promising target for drug intervention. These drugs, currently undergoing preclinical development through in vitro and animal studies, aim to modulate the activity of the PAD4 enzyme. Citrullinated histones, a byproduct of PAD4 enzymatic activity, emerge as a potential biomarker, exhibiting elevated levels in several thrombotic diseases. The anticipation surrounding the imminent future stems from the prospective impact of inhibiting NETs. Dr Martinod's investigation encompasses a comprehensive examination of NETs' binding targets in thrombosis, including activated platelets, various coagulation factors, red blood cells, and the scaffold of thrombus growth. Notably, the study utilizing genetically modified mice lacking the PAD4 enzyme reveals a diminished thrombus formation, elucidating the enzyme's pivotal role. While NETs play a crucial role in neutralizing bacteria, their potential impact on infections warrants investigation. Notably, in a mouse model of endocarditis, where infected blood clots at the heart valve are mimicked, the role of NETs in either promoting thrombosis or combating infection is explored. Surprisingly, targeting NETs showed no discernible effect. Further scrutiny revealed that coagulation of Staphylococcus aureus bacteria formed a fibrin barrier, hindering neutrophils' entry for releasing NETs. Genetic mutation preventing this staphylocoagulation underscored the importance of NETs in fighting bacterial infection, implicating the bacteria in thrombosis. Future endeavors involve evaluating the efficacy of NET formation inhibitors in human diseases, and transitioning candidate drugs from preclinical stages to clinical studies. A deeper comprehension of the conditions and contributing factors that elevate NET levels to a prothrombotic extent may provide valuable insights for identifying patients suitable for targeted NET inhibition.
- [3 year follow up of ZUMA-12: CAR T-cell as a first line treatment in patients with high-risk LBCL](https://medimix.be/hematology/3-year-follow-up-of-zuma-12-car-t-cell-as-a-first-line-treatment-in-patients-with-high-risk-lbcl/) - The ZUMA-12 study represents a phase 2 clinical trial aimed at investigating the efficacy of Axi-Cel as a primary therapeutic intervention for individuals diagnosed with high-risk LBCL. High-risk categorization involved the assessment of MYC and BCL2 and/or BCL6 rearrangements or an IPI score of ≥3, coupled with a positive PET scan following two cycles of chemotherapy. This presentation highlights outcomes observed three years after the initial findings, revealing sustained advantages associated with the early implementation of Axi-Cel. Following a treatment duration exceeding 40 months, the evaluation of 37 patients demonstrated a notable overall response rate (OR) exceeding 90%. The PFS and OS rates among these patients, as determined at the three-year mark, stood at 75% and 81%, respectively. AE was universally reported among all patients, exhibiting a profile consistent with that observed during the primary analysis. In light of these observations, we deduce a heightened efficacy of CAR T-cell therapy in high-risk LBCL patients. This substantiates the rationale for initiating frontline investigations comparing this innovative approach against the current standard of care involving first-line chemoimmunotherapy.
- [Acalabrutinib treatment in CLL, insights from the ELEVATE-TN study](https://medimix.be/hematology/cd20-antibody-treatment-in-cll-insights-from-the-elevate-tn-study/) - Dr Sharman presents the 6-year follow-up outcomes of the ELEVATE-TN study, a prospective, randomized phase 3 investigation involving previously untreated patients with CLL. Three cohorts were subjected to distinct treatments: standard chemotherapy with obinutuzumab (O) and chlorambucil, monotherapy with acalabrutinib (A), and the combined regimen of A and O. The data explore subtle nuances within the study findings. Key findings reveal that patients receiving the combined A+O treatment exhibited a 6-year PFS rate of 78%, whereas those receiving A alone demonstrated a PFS of 62%. Notably, the PFS was substantially lower in the chemo-immunotherapy group, leading to the approval of A monotherapy or its combination in previously untreated patients. This presentation addresses the question of the added value of including O with A, given the lack of consensus in the field regarding whether the use of an anti-CD20 monoclonal antibody confers additional benefits to A monotherapy. The study establishes an absolute 16% improvement in PFS with the addition of O. However, as the incorporation of O is not yet widely adopted in the treatment landscape, this study aimed to elucidate this aspect. Pooling data from both A treatment arms, it was observed that patients achieving CR experienced significantly better PFS, a novel finding that has not been previously demonstrated. Furthermore, individuals receiving O exhibited higher rates of CR, indicating that doublet therapy enhances CR and, consequently, PFS. Exploring the impact of O addition on various molecular subgroups, it was evident that the presence of uIGHV mutation had no discernible effect on A or A+O treatment. Interestingly, patients with del(17P) did not derive added benefit from the inclusion of O. Therefore, considering these factors could assist physicians in identifying optimal candidates for anti-CD20 antibody therapy. This prompts the question of the optimal frontline therapy for CLL TN patients. Various treatment options, including BTKi monotherapy, BTKi and anti-CD20 therapy, BCL-2 and CD20, or BTKi and BCL-2 combinations, can be considered. However, a definitive answer remains elusive and awaits further investigations such as the MAGIC study, which aims to shed light on optimal doublet therapy. In summary, this presentation underscores that the addition of O results in both clinically meaningful and statistically significant improvements in outcome. The IsKia EMN24 trial, presented in the plenary session, evaluated the efficacy and safety of isatuximab-carfilzomib-lenalidomide-dexamethasone (IsaKRd) as a pre-autologous stem-cell transplant (ASCT) induction and post-ASCT consolidation compared to KRd in newly diagnosed multiple myeloma patients. The addition of isatuxamab to KRd exhibited significantly higher MRD negativity rates, both post-induction and post-consolidation. Particularly noteworthy was the comparable MRD rates in both the standard risk and very high-risk groups within the ISA-KRd arm, in contrast with the KRd arm. Given the distinctive mechanisms of action of CAR T cells and T-cell engagers (TCE) in multiple myeloma treatment, a reassessment of conventional prognostic factors, such as MRD, is imperative. A study conducted by a Spanish group revealed that BCMA-directed CAR T yielded approximately twice the MRD negativity rates compared to BCMA-directed TCE. However, once MRD negativity was achieved, the PFS was approximately 20 months in both groups, with MRD-positive patients exhibiting a markedly shorter median PFS of only 3 months. Several BCMA-directed T-cell engagers are currently under investigation, with two, teclistamab (recently reimbursed in Belgium) and elranatamab, advancing furthest in clinical development. Although no direct comparisons exist, an unanchored matching-adjusted indirect comparison showcased elranatamab's superior overall response rate and PFS compared to teclistamab in patients with relapsed/refractory multiple myeloma (RRMM). T-cell engagers, such as talquetamab, present the potential for combination therapies. Talquetamab, a T-cell redirecting bispecific antibody targeting GPRC5D and CD3, combined with the established immunomodulatory drug (IMiD) pomalidomide, demonstrated synergistic antimyeloma effects in the phase 1b MonumenTAL-2 study. Encouragingly, responses were achieved rapidly, with deep and enduring effects. At 6 months, 100% of responders maintained their response, and the median duration of response and PFS were not reached, with 6-month PFS rates hovering around 90%. Additionally, a phase 1, multicenter, open-label, dose-finding study explored a GPRC5D-targeted autologous CAR T-cell therapy in heavily pretreated and BCMA-exposed RRMM patients. The outcomes revealed profound and persistent responses, including MRD negativity. In conclusion, the field of multiple myeloma is witnessing ongoing development with the introduction of novel drugs and combinations. MRD negativity rates are on an upward trajectory, and the understanding of when and how to measure MRD negativity is deepening.
- [ASH 2023 Highlight 4](https://medimix.be/hematology/ash-2023-highlight-4/) - Two investigations provide novel insights into optimizing the therapeutic approach for patients with newly diagnosed multiple myeloma (NDMM) presenting high-risk features. High-risk categorization is contingent upon specific FISH abnormalities, such as del(17p), t(4;14), and t(14;16). Ultra-high-risk or double-hit myeloma patients exhibit two or more of these unfavourable cytogenetic features, warranting distinct management due to the lack of benefit from standard treatments. The IFM 2018-04 trial, a pivotal phase 2 study, selectively enrolled high-risk patients, with over half classified as ultra-high risk. The regimen comprised induction therapy with 6 cycles of Dara-KRd before ASCT, followed by a consolidation phase involving 4 cycles of Dara-KRd, a second ASCT, and Dara-lenalidomide as a 2-year maintenance therapy. Notably, challenges were encountered in collecting sufficient stem cells for transplantation after 6 cycles, prompting a protocol modification to initiate stem cell collection after 3 cycles. Clinically, the outcomes were unprecedented, with approximately 80% of patients alive and without progression at a median follow-up of 30 months. A profound depth of response was evident, exemplified by 81% of patients achieving complete remission after the second consolidation, and over 90% exhibiting MRD negativity—an unprecedented result. This proof-of-concept study advocates for a tailored, intensive treatment approach for this subgroup of high-risk patients. The Iskia study, a phase 3 prospective investigation, randomized transplant-eligible patients into two arms, receiving 4 cycles of KRd with or without isatuximab. Post-stem cell transplantation, patients underwent 4 consolidation cycles of KRd with or without isatuximab. Rather than the typical maintenance treatment, patients received a light consolidation with IsaKRd every 2 weeks after the first consolidation. The primary endpoint centred on MRD negativity, assessed at various intervals. Throughout the study, MRD depth improved after each treatment phase, with a conspicuous advantage observed in the quadruplet treatment arm, particularly in achieving more MRD negativity compared to those on KRd alone. Notably, high-risk patients, constituting 20% of the cohort, demonstrated substantial benefits from the quadruplet treatment, particularly in achieving MRD negativity at the 10-6 cut off. In summary, both studies offer a novel perspective on managing high-risk NDMM patients who are candidates for ASCT, suggesting the merit of more intensive and tailored therapeutic approaches for this subset.
- [ASH 2023 Highlight 3](https://medimix.be/hematology/ash-2023-highlight-3/) - We take pleasure in presenting the third daily highlight from the ASH 2023 event, featuring Evelien Krumb, a PhD candidate and medical trainee affiliated with Cliniques Universitaires Saint-Luc in Brussels. She delivered an exhaustive synopsis of three presentations concentrating on the management of bleeding disorders across the entirety of an individual's lifespan. The initial presentation, conducted by Dr Steven Pipe, delved into the primary analysis of the Haven 7 study, scrutinising the utilisation of emicizumab in infants aged ≤12 months and diagnosed with severe haemophilia A (HA) and devoid of FVIII inhibitors. Challenges associated with the administration of FVIII in paediatric patients with HA have impeded the global consensus advocating early bleeding prophylaxis. The availability of emicizumab presents a potential alleviation of the treatment-related burdens. The Haven 7 study enlisted 55 children, with over half categorised as minimally treated patients. Emicizumab demonstrated remarkably low annualised bleed rates (ABRs), with traumatic bleeding occurrences noted in those who experienced bleeding episodes and an absence of spontaneous treated bleeds. Treatment-related adverse events, confined to injection-site reactions, were reported by nine participants. Thirty serious adverse events were documented, none of which were attributed to the treatment. The deduction drawn from these findings is that emicizumab is both safe and efficacious for application in children aged ≤12 months, with a slated seven-year follow-up expected to yield valuable long-term data. Subsequently, Dr Flora Peivandi presented pharmacokinetic (PK) data emanating from the XTEND-Kids study, evaluating efanesoctocog alfa in children below 12 years afflicted with severe HA. Efanesoctocog alfa represents a novel category of FVIII replacement therapy meticulously engineered to surmount the FVIII half-life ceiling imposed by von Willebrand factor. The study enrolled a total of 74 participants, with 37 included in the pharmacokinetic (PK) subgroup (n=19,
- [ASH 2023 Highlight 2](https://medimix.be/hematology/ash-2023-highlight-2/) - Dr Rutger Callens, a haematologist at AZ Delta in Roeselare, graciously provided key insights into multiple myeloma. The IsKia EMN24 trial, presented in the plenary session, evaluated the efficacy and safety of isatuximab-carfilzomib-lenalidomide-dexamethasone (IsaKRd) as a pre-autologous stem-cell transplant (ASCT) induction and post-ASCT consolidation compared to KRd in newly diagnosed multiple myeloma patients. The addition of isatuxamab to KRd exhibited significantly higher MRD negativity rates, both post-induction and post-consolidation. Particularly noteworthy was the comparable MRD rates in both the standard risk and very high-risk groups within the ISA-KRd arm, in contrast with the KRd arm. Given the distinctive mechanisms of action of CAR T cells and T-cell engagers (TCE) in multiple myeloma treatment, a reassessment of conventional prognostic factors, such as MRD, is imperative. A study conducted by a Spanish group revealed that BCMA-directed CAR T yielded approximately twice the MRD negativity rates compared to BCMA-directed TCE. However, once MRD negativity was achieved, the PFS was approximately 20 months in both groups, with MRD-positive patients exhibiting a markedly shorter median PFS of only 3 months. Several BCMA-directed T-cell engagers are currently under investigation, with two, teclistamab (recently reimbursed in Belgium) and elranatamab, advancing furthest in clinical development. Although no direct comparisons exist, an unanchored matching-adjusted indirect comparison showcased elranatamab's superior overall response rate and PFS compared to teclistamab in patients with relapsed/refractory multiple myeloma (RRMM). T-cell engagers, such as talquetamab, present the potential for combination therapies. Talquetamab, a T-cell redirecting bispecific antibody targeting GPRC5D and CD3, combined with the established immunomodulatory drug (IMiD) pomalidomide, demonstrated synergistic antimyeloma effects in the phase 1b MonumenTAL-2 study. Encouragingly, responses were achieved rapidly, with deep and enduring effects. At 6 months, 100% of responders maintained their response, and the median duration of response and PFS were not reached, with 6-month PFS rates hovering around 90%. Additionally, a phase 1, multicenter, open-label, dose-finding study explored a GPRC5D-targeted autologous CAR T-cell therapy in heavily pretreated and BCMA-exposed RRMM patients. The outcomes revealed profound and persistent responses, including MRD negativity. In conclusion, the field of multiple myeloma is witnessing ongoing development with the introduction of novel drugs and combinations. MRD negativity rates are on an upward trajectory, and the understanding of when and how to measure MRD negativity is deepening.
- [ASH 2023 Highlight 1](https://medimix.be/hematology/ash2023-highlight-1/) - The first daily highlight on ASH 2023 is done by Dr Bert Heyrman, haematologist at the ZNA Hospitals, Antwerp. He selected 4 presentations from the session on Myeloid Malignancies: Real-World Treatment Patterns and Outcomes. Hypomethylating agents (HMAs) represent the established therapeutic standard for individuals afflicted with higher-risk myelodysplastic syndromes (HR-MDS). Dr. Zeidan presented real-life data on the use of oral decitabine, in conjunction with a decitabine inhibitor, relative to intravenous (IV) and subcutaneous (SC) formulations, predominantly encompassing decitabine and azacitidine. Despite lacking approval in Belgium, the oral combination of decitabine and cedazuridine (DEC-C) obtained FDA endorsement in July 2020 for MDS treatment. Real-world data trends underscore an increasing utilization of oral DEC-C, indicative of a shifting treatment landscape. During the early phases of therapy there’s an initial equivalence in treatment persistence between DEC-C and IV/SC HMAs. An improved persistence is evident with oral DEC-C, particularly extending beyond the 6-month threshold. Oral DEC-C is correlated with a significantly reduced therapeutic burden for the patient. Dr. Herrera delved into the intricate interplay between MDS, cardiovascular disease (CVD) and the potential mitigating effects of HMA therapy. He had previously demonstrated that MDS is independently associated with an increased risk of CVD in older patients. Approximately 25% of MDS patients reportedly die from CVD, with a plausible mechanism involving CH-mediated accelerated atherosclerosis attributed to inflammatory vascular responses. The central hypothesis of his presentation postulated that HMA therapy could potentially diminish the risk of CVD by addressing clonal hematopoiesis and mitigating inflammation. An extensive analysis of the SEER database comprising more than 14,000 patients revealed no significant association between HMAs and a reduction in the risk of CVD among MDS patients. As such, follow-up assessments specifically focused on CVD remain necessary, even when MDS patients exhibit favourable haematological responses. In a retrospective analysis conducted by Dr. Alice Mims, an Electronic Medical Record (EMR) database was scrutinized to compare the outcomes of two treatment regimens for patients with newly diagnosed acute myeloid leukemia (AML). The study juxtaposed the efficacy of intensive chemotherapy followed by oral azacitidine (Oral-AZA) maintenance treatment against the regimen of venetoclax plus injectable azacitidine (VEN-AZA). Patients treated with frontline intensive chemotherapy followed by Oral-AZA maintenance had significantly better survival outcomes than patients who were treated and achieved remission with VEN-AZA. These results suggest that patients with newly diagnosed AML eligible for frontline intensive chemotherapy should receive this regimen, followed by Oral-AZA maintenance, if they are transplant-ineligible at remission. Unfortunately, the Oral-AZA maintenance treatment is not yet available in Belgium. The last presentation of this session was on real-life data in primary myelofibrosis (PMF). Notably, the introduction of ruxolitinib (Rux) has demonstrated enhanced survival rates; however, concerns have arisen regarding potential cardiotoxic effects associated with the medication. Given the prolonged exposure of patients to Rux, there is an increasing need for diligent CVD monitoring. A retrospective study of patients diagnosed with PMF between 2003 and 2019 was conducted. The period was split into the pre-Rux (2003-2010) and post-approval Rux eras (2012-2019) and compared the standard mortality ratios (SMRs) of CVD in both periods. The risk of CVM in PMF relative to the general population did not reveal an apparent increase in the era after the approval of Rux. On the contrary, the period following the introduction of Rux demonstrated an association with reduced deaths from CVD. Further studies are needed to examine and validate this observation.
- [ASH Highlights on NK cells: a new frontier for cancer immunotherapy](https://medimix.be/hematology/ash-highlights-on-nk-cells-a-new-frontier-for-cancer-immunotherapy/) - Dr Evren Alici, principal researcher and head of the cell and gene therapy group at the Karolinska Institute in Stockholm, Sweden was so kind to summarise data on NK cell therapy covered during the ASH 2023 congress and to share his insightful perspectives on future developments within the realm of NK cell therapies. In recent years, significant strides have been made in the realm of NK cell-based therapies, particularly evident in the notable increase in newly registered clinical trials focusing on haematological indications. Despite the ongoing assessment of clinical outcomes, a growing body of evidence indicates the potential efficacy of NK cell-based therapy when utilized as a standalone treatment. Notably, this effectiveness has been observed across a spectrum of NK cell products, whether they undergo genetic modifications or not. Several promising strategies have emerged in recent discussions, notably checkpoint strategies, which have shown considerable promise in improving the survival rates of patients previously deemed difficult to treat effectively. Additionally, novel approaches, such as retargeted NK cells, CAR modified NK cells and TCR modified NK cells were presented. Of particular interest is the TCR modified approach, which involves engineering NK cells to target neoantigens or neoepitopes, effectively endowing them with dual functionality. Moreover, the potential for abrogating escape mechanisms presents a powerful tool in this context. Furthermore, there has been exploration into new targeting antigens using CAR technology. However, persisting challenges include ensuring the sustained functionality and in vivo expansion of these engineered cells. Consequently, the focus has shifted towards enhancing the retention of function and prolonging the survival of these cells within the patient, with the ultimate goal of achieving more effective treatments and exploring longitudinal potential for combination therapies. The integration of NK cell-based therapies with various treatment modalities is garnering increasing attention within the scientific community. Particularly notable is the exploration of NK cell-based therapies in conjunction with NK cell engagers or combinations of cytokines. This synergistic approach holds significant promise for enhancing therapeutic efficacy. Prof. dr. Katy Rezvani's presentation during the award ceremony underscored the immense potential inherent in NK cells and the possibility of further engineering these cells to achieve complete tumour eradication. While ongoing clinical trials are yet to reach completion, the preliminary results appear promising. The talk served as a source of inspiration, highlighting the pivotal importance of addressing the challenge of cell persistence. Numerous research groups are actively engaged in tackling this hurdle, recognizing that in the NK cell field, persistence correlates with increased anti-tumour activity and retention of function within the tumour microenvironment. Moving forward, the focus will likely be on two key breakthroughs: enhancing the functionality of NK cells within the tumour microenvironment and prolonging their persistence. With a plethora of promising approaches currently under investigation, there is hope that one or more of these strategies will emerge in the near future, heralding a new era in NK cell-based therapies. Our research group is pioneering the exploration of universal grafts, which differs from conventional approaches in the field. Our focus lies in disrupting the immunological synapse from the host while preserving the host's immune function. One notable challenge we encounter is the limitation imposed by the preconditioning of patients. Many individuals excluded from our studies due to frailty are unable to tolerate the lymphodepletion regimens, or preconditioning, necessary for conventional approaches. Addressing this challenge is paramount for our work. We aim to engineer NK cells capable of retaining functionality while ensuring the integrity of the host immune system. This endeavour represents a novel direction in immunotherapy, holding promise for extending treatment options to a broader patient population.
- [The ESMO-Magnitude of Clinical Benefit Scale for Hematological Malignancies](https://medimix.be/hematology/the-esmo-magnitude-of-clinical-benefit-scale-for-hematological-malignancies/) - Prof Dr Michel Delforge, UZ Leuven, was one of the experts contributing to the first version of the ESMO-Magnitude of Clinical Benefit Scale for hematological malignancies (ESMO-MCBS:H). In this video, prof. Delforge gives some background and highlights the importance of this scale. The ESMO-MCBS has been accepted as a robust tool to evaluate the magnitude of clinical benefit of new therapies in oncology trials, but was so far only validated for solid tumors. In a joint project of ESMO and EHA, an ESMO-MCBS version was developed, specifically designed and validated for hematological malignancies (ESMO-MCBS:H).
- [Impact of MYC-R on the outcome of LS DLBCL patients](https://medimix.be/hematology/impact-of-myc-r-on-the-outcome-of-ls-dlbcl-patients/) - Vera De Jonge, PhD student from the VUMC, Amsterdam and Joanna Bult, PhD student from the University Medical Center Groningen assessed the impact of a rearrangement of the MYC oncogene on the outcome of patients with stage I or II diffuse large B-cell lymphoma in the Netherlands.
- [Brain radiotherapy for progressive secondary CNS lymphoma](https://medimix.be/hematology/brain-radiotherapy-for-progressive-secondary-cns-lymphoma/) - Dr. Gustav Cederquist from the Memorial Sloan Kettering Cancer Center, New Yorkevaluated the potential of brain radiotherapy as an effective bridge for chemo-refractory or progressive secondary CNS lymphoma.
- [Impact of the dark zone signature on CNS relapse](https://medimix.be/hematology/impact-of-the-dark-zone-signature-on-cns-relapse/) - Central nervous system (CNS) relapse in diffuse large B-cell lymphoma is associated with poor outcomes necessitating the identification of high-risk patients. To refine CNS risk stratification, a better understanding of the role of molecular risk factors is required. Dr. Waleed Alduaij, hematologist at BC Cancer in Vancouver, CA analysed the impact of the dark zone signature on CNS relapse in a real-world diffuse large B-cell lymphoma population.
- [Enteropathy associated T-cell lymphoma](https://medimix.be/hematology/enteropathy-associated-t-cell-lymphoma/) - Enteropathy associated T-cell lymphoma is a rare peripheral T-cell lymphoma with a poor prognosis. Dr. Frederik Meeuwes, hematologist from the Treant hospital in Emmen presented a population-based cohort study on incidence, treatment and outcome of this type of lymphoma in The Netherlands.
- [Axi-cel in relapsed or refractory large B-cell lymphoma](https://medimix.be/hematology/axi-cel-in-relapsed-or-refractory-large-b-cell-lymphoma/) - Dr Jason Westin, hematologist at Texas MD Anderson Cancer Center, Houston gives insight into the overall survival findings from the ZUMA-7 trial of axicabtagene ciloleucel in patients with relapsed or refractory large B-cell lymphoma. He shares his view on the implications of these findings on the treatment landscape. He also briefly discusses the design of the ZUMA-23 trial, a global, phase 3 study evaluating axicabtagene ciloleucel as first-line therapy in patients with high-risk large b-cell lymphoma that is currently recruiting.
- [Role of BTK-inhibitors in B-cell malignancies](https://medimix.be/hematology/role-of-btk-inhibitors-in-b-cell-malignancies/) - Prof. Stephan Stilgenbauer, hematologist at the Ulm University in Germany shares his view on the role of BTK-inhibitors in B-cell malignancies. He discusses the potential of second-generation BTK-inhibitors and provides insight into selecting the right treatment for the right patient among the various newly available treatments for CLL patients.
- [The SELENE study](https://medimix.be/hematology/the-selene-study/) - Prof. Ann Janssens, hematologist at UZ Leuven, discusses the outcomes of the SELENE study for which she was one of the investigators. This phase 3 study evaluated ibrutinib plus bendamustine-rituximab or R-CHOP in previously treated patients with follicular or marginal zone lymphoma to determine if the addition of ibrutinib would prolong progression-free survival.
- [Highlights on the management of aggressive lymphomas](https://medimix.be/hematology/highlights-on-the-management-of-aggressive-lymphomas/) - Dr. Willem Daneels provides valuable insights on the management of aggressive lymphomas. He presents the outcomes of a non-inferiority trial comparing observation versus radiotherapy in patients with primary mediastinal b-cell lymphoma who had a complete metabolic response after standard immunochemotherapy.
- [Highlights on Hodgkin lymphoma](https://medimix.be/hematology/highlights-on-hodgkin-lymphoma/) - Prof. Marc André discusses the outcomes of the fil-rouge trial comparing frontline intensified ABVD with PET-adapted ABVD in advanced Hodgkin lymphoma. Prof. André also presents results of the SWOG S1826 with nivolumab in advanced stage classic Hodgkin lymphoma (CHL).
- [Highlights on CLL and Richter syndrome](https://medimix.be/hematology/highlights-on-cll-and-richter-syndrome/) - Prof. Ann Janssens from UZ Leuven shares a summary on the informative session on CLL and Richter syndrome.
- [The therapeutic revolution for the management of multiple myeloma](https://medimix.be/hematology/the-therapeutic-revolution-for-the-management-of-multiple-myeloma/) - Dr. Claudio Cerchione, Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, Meldola, Italy, was so kind to share his insights on the therapeutic revolution for the management of multiple myeoloma.
- [The phase 1/2 Bruin study](https://medimix.be/hematology/the-phase-1-2-bruin-study/) - Prof. Wojciech Jurczak discussed the data from the phase 1 / 2 Bruin study evaluating pirtobrutinib in patients with mantle cell lymphoma, pretreated with a covalent BTK-inhibitor.
- [In the spotlight: Jan Cools, editor-in-chief of HemaSphere](https://medimix.be/hematology/in-the-spotlight-jan-cools-editor-in-chief-of-hemasphere/) - MediMix had an interview with Prof. Jan Cools from KU Leuven and editor-in-chief of HemaSphere. He looks back on his career and the start of HemaSphere and closes with his view on clonal hematopoiesis.
- [BTK inhibitors as first line treatment in mantle cell lymphoma: a debate](https://medimix.be/hematology/btk-inhibitors-as-first-line-treatment-in-mantle-cell-lymphoma-a-debate/) - Dr. Jeanette Doorduijn, hematologist at the Erasme hospital in Rotterdam participated in the debate on whether BTK inhibitors should be used as first line treatment in mantle cell lymphoma. She summarised the pro/cons based on the Triangle and the Shine trial and shed her light on current and future management of mantle cell lymphoma in daily practice.
- [The unmet need in patients with multiple myeloma at first relapse](https://medimix.be/hematology/the-unmet-need-in-patients-with-multiple-myeloma-at-first-relapse/) - Prof. Maria Victoria Mateos, hematologist at the Hospital Universitario De Salamanca, Spain discussed the unmet need in patients with multiple myeloma at first relapse and summarised subgroup data from the Boston trial by prior therapies and in lenalidomide-refractory patients.
- [Highlights on infections and SCT complications](https://medimix.be/hematology/highlights-on-infections-and-sct-complications/) - Dr. Alexander Schauwvlieghe chaired the session on infections and SCT complications and gave a nice overview of the most interesting studies relevant for practice.
- [Highlights on platelets and inflammation](https://medimix.be/hematology/highlights-on-platelets-and-inflammation/) - Professor Kimberly Martinod from the Department of Cardiovascular Sciences at KU Leuven had the honor to give a presentation on platelets and inflammation during the closing plenary session at EHA. She focused on the non-classical pathways by which platelets can contribute to inflammation.
- [A Novel Mammalian L-ASNase in the treatment of T-ALL](https://medimix.be/hematology/a-novel-mammalian-l-asnase-in-the-treatment-of-t-all/) - n this study, a research group from Ghent University aims to develop a less toxic an non immunogenic L-ASNase with similar L-ASNase activity as FDA-approved L-ASNase, but without the toxic glutaminase co-activity, for use in the treatment of T-ALL. The result is a novel, mammalian L-ASNase with a high efficacy against T-cell acute lymphoblastic leukemia in vivo. Dr. Maaike Van Trimpont elaborates on the setup of the study.
- [Diagnosis and treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN)](https://medimix.be/hematology/diagnosis-and-treatment-of-patients-with-blastic-plasmacytoid-dendritic-cell-neoplasm-bpdcn/) - Dr. Claudio Cerchione, Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, Meldola, Italy, was so kind to give us an update on the diagnosis and treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare malignant disease with rapid systemic spread. He highlights the importance of the multidisciplinary team in recognising symptoms to make a correct diagnosis and is hopeful with tagraxofusp as new treatment option for this malignancy.
- [José Carreras Award: Ph+ ALL, from the worst malignancy to a chemo/transplant-free management](https://medimix.be/hematology/jose-carreras-award-ph-all-from-the-worst-malignancy-to-a-chemo-transplant-free-management/) - Dr. Tim Pieters discussed his research focusing on modelling of blood cancers in mice. He gave a summary of his presentation at EHA 2023 about the relation between SOX11 overexpression and the formation of mantle cell lymphomas in mice.
- [Advances in the science and care of persons with bleeding disorders](https://medimix.be/hematology/advances-in-the-science-and-care-of-persons-with-bleeding-disorders/) - Prof. Cathérine Lambert gives an overview of the session ‘Advances in the science and care of persons with bleeding disorders’. She discussed a cross-sectional survey evaluating the assessment of joint health in patients receiving prophylaxis for haemophilia.
- [Ageing and hematology: how to cope with the silver wave in hematology](https://medimix.be/hematology/ageing-and-hematology-how-to-cope-with-the-silver-wave-in-hematology/) - Prof. em. Dominique Bron provided a comprehensive summary of the EHA Specialized Working Group on 'Ageing and Hematology'. The group deliberated on the challenges of managing hematological conditions in octogenarians and how to address the "silver wave" in hematology.
- [Extracellular vesicles from chronic lymphocytic leukemia cells induce the differentiation of monocytes into nurse-like cells](https://medimix.be/hematology/extracellular-vesicles-from-chronic-lymphocytic-leukemia-cells-induce-the-differentiation-of-monocytes-into-nurse-like-cells/) - What is the impact of BCR stimulation on miRNA content in CLL cells and EVs? And what is the impact of CLL-EVs on moncytes? That's what this study aims to discover. Researcher and PhD student Nathan Dubois from the Université Libre de Bruxelles tells us the whole story.
- [CARTITUDE-4: the first phase 3 results](https://medimix.be/hematology/eha2023-cartitude-4/) - Prof. Michel Delforge provided a comprehensive summary of the current state of multiple myeloma treatment. He also shared the initial findings from the CARTITUDE-4 study's phase 3 results, which compared cilta-cel to standard of care for patients with lenalidomide-refractory multiple myeloma.
- [SOX11 overexpression and mantle cell lymphomas formation](https://medimix.be/hematology/eha2023-sox11-overexpression/) - Dr. Tim Pieters discussed his research focusing on modelling of blood cancers in mice. He gave a summary of his presentation at EHA 2023 about the relation between SOX11 overexpression and the formation of mantle cell lymphomas in mice.
- [A dual role for PSIP1 in T-ALL](https://medimix.be/hematology/a-dual-role-for-psip1-in-t-all/) - This study identifies the role of PSOP1 in the initiation and maintenance of T-ALL, by analyzing the effects caused by PSIP1 loss, both in mice models and human T-ALL cell lines.
- [Pieter Van Vlierberghe honoured with David Grimwade Award: Exploring novel therapeutic strategies for T-cell acute lymphoblastic leukemia](https://medimix.be/hematology/pieter-van-vlierberghe-honoured-with-david-grimwade-award/) - Prof Pieter Van Vlierberghe has been posthumously honoured with the renowned David Grimwade Award, in recognition of his groundbreaking work in basic and translational research. The award lecture was given by his collegue Prof. Steven Goossens from the Cancer Research Institute of Ghent University who was so kind to highlight Prof Van Vlierberghe's pivotal research and its potential to advance treatment options for T-cell acute lymphoblastic leukemia.
- [Real-world insights on the management of iTTP with Caplacizumab](https://medimix.be/hematology/real-world-insights-on-the-management-of-ittp-with-caplacizumab/) - The aim of this project is to determine the effective concentration (EC) of TXA using the GFC/LT, to facilitate TXA use in surgery or trauma. For this, the research team developed an in vitro urokinase induced hyperfibrinolytic model by spiking plasma with different urokinase concentrations. Anton Evenepoel, a clinical biology trainee from UZ Brussel explains.
- [Effective concentration of TXA using the GFC/LT](https://medimix.be/hematology/effective-concentration-of-txa-using-the-gfc-lt/) - The aim of this project is to determine the effective concentration (EC) of TXA using the GFC/LT, to facilitate TXA use in surgery or trauma. For this, the research team developed an in vitro urokinase induced hyperfibrinolytic model by spiking plasma with different urokinase concentrations. Anton Evenepoel, a clinical biology trainee from UZ Brussel explains.
- [Highlights from ASCO](https://medimix.be/hematology/highlights-from-asco-2023/) - Dr Claudio Cerchione summarised the educational session on the management of multiple myeloma in special patient populations, such as elderly and patients with renal failure.

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- [Contact](https://medimix.be/hematology/contact/) - Get in touch Questions, collaborations or support? We’re here to help. MediMix collaborates with healthcare professionals, medical societies and partners to create high-quality educational content.Feel free to reach out, we’ll get back to you as soon as possible. E-mail LinkedIn Interested in collaborating? Fill in the form Name Email Message “I am a…” Healthcare professional
- [STiP - STudy in the Picture.](https://medimix.be/hematology/stip/) - Where evidence meets expertiseSTudy in the Picture (STiP) brings clinical research into sharp focus through concise, expert-driven summaries. Each STiP distils a single clinical study into its essential elements, from rationale and design to key results and clinical relevance, without compromising scientific depth. Two formats, one mission STiP │ Congress Highlights Tied to the most
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- [EBMT 2025](https://medimix.be/hematology/ebmt-florence-2025/) - This content is accessible exclusively to verified healthcare professionals. If you are a registered user, please log in here. If you do not yet have an account, please register here. Thank you for your understanding.
- [ASH 2024](https://medimix.be/hematology/ash-sandiego-2024/) - This content is accessible exclusively to verified healthcare professionals. If you are a registered user, please log in here. If you do not yet have an account, please register here. Thank you for your understanding.
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- [EHA Madrid 2024](https://medimix.be/hematology/eha-madrid-2024/) - This content is accessible exclusively to verified healthcare professionals. If you are a registered user, please log in here. If you do not yet have an account, please register here. Thank you for your understanding.
- [Post EHA & ICML 2023](https://medimix.be/hematology/post-eha-icml-2023/) - Rewatch the Post EHA & ICLM 2023 meeting Post EHA & ICML 2023 On Thursday 21 September 2023, Esteemed Belgian experts in hematology explored the practical applications of the latest findings in hematology from EHA & ICML 2023. How will these new data change current guidelines? This dynamic and interactive session provides valuable insights for
- [ICML Lugano 2023](https://medimix.be/hematology/icml-2023/) - From 13 till 17 June 2023: HIGHLIGHTS ON MALIGNANT LYMPHOMA Your direct line with Lugano From 13 till 17 June 2023, the International Conference on Malignant Lymphoma (ICML) is – as usually – taking place in Lugano, Switzerland. The MediMix team is onsite, and will – together with your Belgian colleagues – keep you posted
- [EHA Frankfurt 2023](https://medimix.be/hematology/eha-2023/) - From 8 till 11 June 2023: HIGHLIGHTS ON HEMATOLOGY Your direct line with Frankfurt From 8 till 11 Juni 2023, The European Hematology Association (EHA) is taking place in Frankfurt, Germany. The MediMix team is onsite, and will – together with your Belgian colleagues – keep you posted about the hottest topics as they happen.And,
- [ASCO Chicago 2023](https://medimix.be/hematology/asco-chicago-2023/) - From 2 June till 6 June 2023: HIGHLIGHTS FROM ASCO Your direct line with Chicago The American Society of Clinical Oncology (ASCO) 2023 brings together leading experts in the different fields of cancer from around the world. And, as usual, they gather together in Chicago, USA. The MediMix team is also onsite, and will –

## My Templates

- [vb post](https://medimix.be/hematology/?elementor_library=vb-post-2) - Presented by Dr Marie Vercuryssen (Institut Jules Bordet, Brussels, Belgium) In this highlights video from EHA 2026, Dr Marie Vercruyssen Haematologist at the Institut Jules Bordet in Brussels, discusses six studies illustrating the expanding role of bispecific antibodies across the spectrum of plasma cell disorders.The phase III MONUMENTAL-3 study evaluated talquetamab combined with daratumumab, with
- [vb post](https://medimix.be/hematology/?elementor_library=vb-post) - Presented by Prof Dr Toon Van Gorp (University Hospitals Leuven, Belgium) In this in-depth interview, Prof Dr Toon Van Gorp, gynaecologic oncologist at University Hospitals Leuven, discusses results from the NAPISTAR 1-01 study, evaluating TUB-040, a novel antibody-drug conjugate targeting NaPi2b, in platinum-resistant ovarian cancer.1 NaPi2b is a sodium-phosphate transporter that is highly expressed in
- [hema](https://medimix.be/hematology/?elementor_library=hema) - ProvIDHe Presented by Prof Eric Van Cutsem (University Hospitals Leuven, Belgium) & Prof John Bridgewater (University College London Hospitals NHS Foundation Trust – London, UK) Watch video
- [stip](https://medimix.be/hematology/?elementor_library=stip) - Where evidence meets expertiseSTudy in the Picture (STiP) brings clinical research into sharp focus through concise, expert-driven summaries. Each STiP distils a single clinical study into its essential elements, from rationale and design to key results and clinical relevance, without compromising scientific depth. Two formats, one mission STiP │ Congress Highlights Tied to the most
- [thanks](https://medimix.be/hematology/?elementor_library=thanks) - Thanks for getting in touch We received your message Thank you for reaching out through our contact form.We have received your message and will review your request as soon as possible. You can expect a reply from us by email shortly. MediMix Oncology MediMix Hematology MediMix Dermatology MediMix Respirology MediMix Cardiology Home MediMix
- [Contact new](https://medimix.be/hematology/?elementor_library=contact-new) - Get in touch Questions, collaborations or support? We’re here to help. MediMix collaborates with healthcare professionals, medical societies and partners to create high-quality educational content.Feel free to reach out, we’ll get back to you as soon as possible. E-mail LinkedIn Interested in collaborating? Fill in the form Name Email Message “I am a…” Healthcare professional
- [voorbeeld](https://medimix.be/hematology/?elementor_library=voorbeeld) - From 25 till 28 September 2024: HIGHLIGHTS FROM EADV Your direct line with Amsterdam The EADV Congress is Europe’s largest international meeting focused on dermatology and venereology, providing a platform for researchers and clinicians, to exchange knowledge and present their latest research findings. Your Belgian colleagues are onsite and keep you posted about the hottest
- [Home Hermatology](https://medimix.be/hematology/?elementor_library=home-hermatology) - MediMix aims to bring high value medical information to Belgian healthcare professionals via multiple channels. Our team covers congresses and symposia all over the world, particularly in the hematology domain. In collaboration with Belgian and international experts, we bring the newest data to Belgian physicians. Now live Coming up Past congresses Latest publications Long-term safety
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## Categories

- [ASCO 2023](https://medimix.be/hematology/category/asco-2023/)
- [EHA 2023](https://medimix.be/hematology/category/eha-2023/)
- [ICML 2023](https://medimix.be/hematology/category/icml-2023/)
- [NEWS](https://medimix.be/hematology/category/news/)
- [ASH 2023](https://medimix.be/hematology/category/ash-2023/)
- [ASCO 2024](https://medimix.be/hematology/category/asco-2024/)
- [EHA 2024](https://medimix.be/hematology/category/eha-2024/)
- [ASH 2024](https://medimix.be/hematology/category/ash-2024/)
- [EBMT 2025](https://medimix.be/hematology/category/ebmt-2025/)
- [EHA 2025](https://medimix.be/hematology/category/eha-2025/)
- [ICML 2025](https://medimix.be/hematology/category/icml-2025/)
- [ASCO 2025](https://medimix.be/hematology/category/asco-2025/)
- [IMS 2025](https://medimix.be/hematology/category/ims-2025/)
- [ASH 2025](https://medimix.be/hematology/category/ash-2025/)
- [STiP │ Congress Highlights](https://medimix.be/hematology/category/stip-│-congress-highlights/)
- [EHA 2026](https://medimix.be/hematology/category/eha-2026/)

## Tags

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