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Overcoming Delayed Engraftment: UM171 Expansion Technology and ATG-free Regimens Reshape the CBT Landscape
As the landscape of hematopoietic stem cell transplantation (HSCT) continues to evolve, umbilical cord blood transplantation (CBT) maintains a critical position in the treatment of hematologic malignancies due to its unique immunological advantages. However, delayed engraftment and high non-relapse mortality (NRM) were once significant bottlenecks limiting its clinical utility. At a recent academic symposium, Professor…
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Breaking the Bottleneck in Severe Aplastic Anemia: Insights from EBMT Registry Big Data, Long-term IST Follow-up, and Novel Biological Predictors
Severe Aplastic Anemia (SAA), as a highly heterogeneous bone marrow failure (BMF) disorder, continues to present a clinical challenge regarding the optimal choice between immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT). During the recent European Society for Blood and Marrow Transplantation (EBMT) meeting, Professor Antonio Risitano provided a comprehensive overview of the Severe…
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Safety Profiles in the Real World: An EBMT Registry Study on the Evolution of Non-Relapse Mortality in 6,928 CAR-T Recipients
The advent of Chimeric Antigen Receptor T-cell (CAR-T) therapy has fundamentally transformed the treatment landscape for relapsed/refractory (R/R) hematologic malignancies. However, as clinical adoption expands and follow-up durations extend, the management of treatment-related toxicities—specifically Non-Relapse Mortality (NRM)—has emerged as a critical bottleneck for long-term patient survival. At the 2026 Annual Meeting of the European Society…
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Filling the Void in Myeloid Neoplasm Evaluation: Prof. Donal McLornan on Defining Remission, Relapse, and Outcome Optimization in MDS/MPN Overlap Syndromes
llogeneic hematopoietic cell transplantation (allo-HCT) remains the only potentially curative intervention for Myelodysplastic Syndromes/Myeloproliferative Neoplasm (MDS/MPN) overlap syndromes. However, due to the high heterogeneity and low incidence of these disorders, the clinical field has long lacked unified criteria for evaluating post-transplant remission and relapse. At the recent European Society for Blood and Marrow Transplantation (EBMT)…
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Deciphering VEXAS Syndrome: From UBA1 Somatic Mutations to Precision Management and Allo-HSCT
During the recent workshop “Focus on Allogeneic HSCT in Myelodysplastic Syndromes,” Professor Fabio Ciceri (IRCCS San Raffaele Scientific Institute / Vita-Salute San Raffaele University), Chair of the EBMT Scientific Council and the Acute Leukemia Working Party (ALWP), provided a comprehensive overview of “Contemporary Approaches to VEXAS.” He systematically detailed the genetic foundations, clinical progression, and…
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12-Month OS Reaches 54%: Microbiome-Based Therapy MAAT013 Addresses the Therapeutic Crisis in Refractory GI-aGVHD
At the recent Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Professor Florent Malard from Sorbonne Université, lead of the Microbiome Research Project within the EBMT Cellular Therapy & Immunobiology Working Party, presented the final results of the ARES Phase III clinical trial. The presentation focused on the efficacy and safety…
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Up to 13 Years of Follow-up: Integrated Analysis of Etuvetidigene Autotemcel Shows Sustained Clinical Benefit in WAS Patients
During the EBMT Annual Meeting, Dr. Francesca Ferrua from the IRCCS San Raffaele Scientific Institute presented an integrated analysis of the lentiviral hematopoietic stem and progenitor cell (HSPC) gene therapy, etuvetidigene autotemcel (ototemcel), for the treatment of Wiskott-Aldrich Syndrome (WAS). The report highlighted remarkable efficacy and safety outcomes with a follow-up period extending up to…
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A Milestone in Gene Editing: Prof. Locatelli Presents Durable 6-Year Data for Exa-cel in Thalassemia and Sickle Cell Disease
At the EBMT 2026 Annual Meeting, Professor Franco Locatelli from Bambino Gesù Children’s Hospital presented long-term follow-up data for exagamglogene autotemcel (exa-cel), a CRISPR/Cas9-based gene-editing therapy. The study included patients with transfusion-dependent thalassemia (TDT) and sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). With a median follow-up of over three years and some patients…