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EBMT 2026 | Exa-cel for sickle cell disease and transfusion-dependent beta thalassemia: a real-world experience
In this interview, Mohammed Essa, MD, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia, briefly outlines the initial findings from a real-world single center trial evaluating the efficacy and safety of exagamglogene autotemcel (exa-cel) in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), highlighting that the primary endpoint of the trial has been met but that follow-up remains relatively short. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.
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Transcript
As you know, exa-cel has been approved late 2023, early 2024 in different countries. And in the last meeting in EBMT, we have tried to share our initial outcome that is mimicking the clinical trial outcome in terms of efficacy and safety as well. We have included in our abstract in EBMT, the first nine patients, three of them were with transfusion-dependent thalassemia and another six with sickle cell disease...
As you know, exa-cel has been approved late 2023, early 2024 in different countries. And in the last meeting in EBMT, we have tried to share our initial outcome that is mimicking the clinical trial outcome in terms of efficacy and safety as well. We have included in our abstract in EBMT, the first nine patients, three of them were with transfusion-dependent thalassemia and another six with sickle cell disease. They all reached the first primary objective or endpoint for our short follow-up, which included transfusion dependency in TDT and being pain crisis-free in patients with sickle cell disease.
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Disclosures
Advisory board: Vertex.
