Long-term efficacy and safety of betibeglogene autotemcel for β-thalassemia

In this week's episode, Blood editor Dr. Laura Michaelis interviews Dr. Alexis Thompson, former ASH president, on her latest article published in Blood. Dr. Thompson discusses "Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia." She explains transfusion-dependent β-thalassemia (TDT) requires rigorous, lifelong transfusion therapy and iron chelation to manage iron overload. Dr. Kwiatkowski and colleagues discuss the long-term efficacy and safety of this gene therapy in 63 patients with TDT, documenting sustained transfusion independence for up to 10 years and a safety profile consistent with that of myeloablative autologous transplantation.