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

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

May 7, 2026 · 13 min · Season 11

About this episode

Dr. Laura Michaelis interviews Dr. Alexis Thompson about the long-term efficacy and safety of betibeglogene autotemcel for treating transfusion-dependent 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.

People in this episode

Host: Dr. Laura Michaelis

Guest: Dr. Alexis Thompson

Mentioned in this episode

Organizations: American Society of Hematology

Products: betibeglogene autotemcel

Books & works: Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia

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