Replacing high-dose chemotherapy with blinatumomab in children with newly-diagnosed, high-risk ALL: the phase III AIEOP BFM ALL 2017 trial

As part of the plenary abstract session at EHA 2026 Prof Dr Martin Schrappe (University Medical Center Schleswig-Holstein, Kiel, Germany) presented data from the AIEOP-BFM ALL 2017 study.  In this video, Prof Schrappe and Prof Dr Franco Locatelli (IRCCS Ospedale Bambino Gesù, Rome, Italy) highlight the potentially practice-changing impact of these findings on the treatment of pediatric high-risk acute lymphoblastic leukaemia (ALL). The multinational study, conducted across eight European countries and enrolling several hundred children and adolescents, demonstrated for the first time that selected components of conventional chemotherapy can be safely replaced by blinatumomab.

Blinatumomab is a bispecific T-cell engager that redirects endogenous T cells towards malignant B cells, thereby enhancing anti-leukemic immune responses. Unlike previous studies, which incorporated blinatumomab as an addition to standard chemotherapy backbones, the AIEOP-BFM ALL 2017 study uniquely substituted highly toxic chemotherapy elements with two courses of immunotherapy. This strategy resulted in substantial clinical benefits, including an almost 50% reduction in relapse risk. Importantly, the advantages extended beyond disease control and translated into a marked reduction in treatment-related toxicity, a particularly relevant outcome in pediatric oncology.

The study also demonstrated a lower incidence of both bone marrow and central nervous system relapses, further supporting the efficacy of this chemotherapy-sparing approach. Among patients who subsequently underwent an allogeneic stem cell transplantation, prior treatment with blinatumomab was associated with a significantly lower risk of transplant-related mortality, suggesting that improved disease control combined with reduced cumulative toxicity may positively influence transplant outcomes.

These findings represent a paradigm shift in the management of pediatric high-risk B-cell ALL, showing that effective immunotherapy can replace, rather than merely supplement, intensive chemotherapy. According to both experts, this strategy opens the door to future studies evaluating similar approaches in patients with lower-risk disease. Such an approach could preserve the excellent survival outcomes currently achieved in pediatric ALL while substantially reducing long-term treatment-related sequelae. Given the long life expectancy of this patient population, minimizing late toxicities is becoming an increasingly important therapeutic objective. Collectively, these data suggest that future treatment strategies may focus not only on improving cure rates but also on optimizing the quality of the survival through the progressive replacement of conventional chemotherapy with targeted immunotherapeutic approaches.