Treating cytopenia in myelofibrosis: real-world data

In this video, Dr Adriano Salaroli (Institut Jules Bordet) discusses two poster presentations on myelofibrosis, with a particular focus on the use of momelotinib and the management of cytopenic disease.

A retrospective analysis from a German cohort evaluated 77 patients with myelofibrosis treated with momelotinib in a real-world setting. Approximately half of the patients were treatment-naïve, while the remainder had prior exposure to JAK inhibitors. Treatment with momelotinib was associated with a median hemoglobin improvement of 1.3 g/dL in both groups, with a more pronounced benefit observed among treatment-naïve patients. This improvement was accompanied by a reduction in transfusion burden, consistent with findings from previous clinical trials that supported the drug’s approval. An unexpected observation was an improvement in platelet counts, potentially related to optimal dosing and reductions in splenomegaly¹. These data provide real-world evidence supporting the use of momelotinib in cytopenic patients and suggest potential advantages when used earlier in the treatment pathway.

A second retrospective analysis from the ERNEST trial examined disease evolution in 418 patients with myelofibrosis, of whom 55% had a myeloproliferative phenotype and 45% a cytopenic phenotype. Patients with cytopenic disease demonstrated larger spleen volumes, greater molecular complexity and more advanced bone marrow fibrosis, indicating a more challenging disease course. Interestingly, the analysis showed that the disease phenotype is dynamic, with transitions occurring between myeloproliferative and cytopenic states over time. Patients with cytopenic disease had fewer available therapeutic options, underscoring the need for regular reassessment throughout the disease course to ensure that treatment strategies remain aligned with evolving clinical characteristics.²