Targeted Therapies, Key Subgroup Insights to Watch at ASH 2025

The upcoming American Society of Hematology (ASH) 2025 meeting is poised to spotlight several pivotal developments across myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and rare bleeding disorders.

Ivo Carre, PhD, a senior business analyst at Lifescience Dynamics, discussed updates within the MDS space; one of the most closely watched updates will be the new subgroup analyses from the high-risk MDS trial evaluating venetoclax. Although the study failed to meet its primary end point earlier this year, emerging data suggest that certain patient subgroups may still derive meaningful benefit. These insights could help refine future treatment strategies and guide more personalized approaches for patients with high-risk disease. These innovations extend into AML, particularly for patients who progress from inadequately treated MDS. This continues to be an area of rapid innovation, with targeted therapies reshaping the treatment landscape.

Shayla Bergmann, MD, a clinical professor in pediatric hematology at the Medical University of South Carolina, discussed a study on the perioperative management of bleeding in pediatric patients with severe von Willebrand (VWF) disease using recombinant VWF. Key priorities include improving prophylactic treatment protocols and expanding access to therapies that can be administered more easily, such as subcutaneous formulations that reduce dependence on intravenous delivery. A particularly important focus is addressing the unique challenges experienced by women and adolescent females with bleeding disorders, including issues related to menstruation, fertility, pregnancy, and childbirth. Strengthening care models for these underserved populations remains a critical area of progress.

Collectively, these developments set the stage for impactful discussions and potential practice-changing insights at ASH 2025.