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

Page: https://stenobird.com/podcast/blood-podcast-664776/long-term-efficacy-and-safety-of-betibeglogene-autotemcel-for-thalassemia
Text version: https://stenobird.com/podcast/blood-podcast-664776/long-term-efficacy-and-safety-of-betibeglogene-autotemcel-for-thalassemia.md
Podcast: [Blood Podcast](https://stenobird.com/podcast/blood-podcast-664776)
Published: 2026-05-07T16:00:00+00:00
Episode link: https://share.transistor.fm/s/b9b05532
Audio file: https://prfx.byspotify.com/e/media.transistor.fm/b9b05532/4244f1e4.mp3
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JSON: https://stenobird.com/v1/public/podcasts/blood-podcast-664776/episodes/long-term-efficacy-and-safety-of-betibeglogene-autotemcel-for-thalassemia
Duration seconds: 784

## Resource

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.

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## Transcript

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