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ASH 2025 | First promising results of exa-cel in pediatric patients with TDT or SCD and recurrent severe VOCs
Haydar Frangoul, MD, MS, Sarah Cannon Research Institute at TriStar Centennial Children’s Hospital, Nashville, TN, discusses the first results with exagamglogene autotemcel (exa-cel) in pediatric patients aged 5-11 years with transfusion-dependent β-thalassemia (TDT) or sickle cell disease (SCD) and recurrent severe vaso-occlusive crises (VOCs). Dr Frangoul notes the encouraging findings thus far and highlights the potential benefits of treating patients at a younger age, before irreversible damage from their disease occurs. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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