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ASH 2024 | The impact of luspatercept and mitapivat on transfusion dependence in patients with thalassemia
Sophia Delicou, MD, Hippokration General Hospital, Athens, Greece, comments on the impact of luspatercept and mitapivat on transfusion dependence in thalassemia. Both agents have shown promising results in reducing transfusion needs, with luspatercept helping some patients become entirely transfusion-independent. In the ENERGIZE-T trial (NCT04770779), mitapivat showed a significant reduction in transfusion needs of patients with alpha- and beta-thalassemia, with up to 10% of participants not requiring transfusions for at least eight weeks. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript
Luspatercept has had a big impact in reducing transfusion needs in beta-thalassemia patients. We know it, we use it. For those who are transfusion-dependent, the study showed that about one in five patients needed fewer transfusions and some even became completely independent. For the non-transfusion and from my experience in my department, I can say that patients saw almost 90% stayed transfusion-free during the treatment...
Luspatercept has had a big impact in reducing transfusion needs in beta-thalassemia patients. We know it, we use it. For those who are transfusion-dependent, the study showed that about one in five patients needed fewer transfusions and some even became completely independent. For the non-transfusion and from my experience in my department, I can say that patients saw almost 90% stayed transfusion-free during the treatment. So it’s helping, it’s a good player and it’s helping patients to produce more red cells and improving their quality of life. Now the new study that will be announced by Professor Capellini has showed that mitapivat, which is a new oral agent that reduces the need for blood transfusion in people with alpha and beta thalassemia, shows that about 30% of the patients taking mitapivat managed to cut their blood transfusion by at least half, which is very important. And even better, almost 10% of the mitapivat group didn’t need any transfusion for at least eight weeks. These are very good results and we are waiting to hear it from Professor Capellini. It was a well-tolerated drug, so it has some common side effects, but I think that we are walking in a safe path.
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Disclosures
Honoraria: ELPEN, DEMO, Novo Nordisk, Pfizer, Bristol Myers Squibb, Novartis; Research Funding: ISIS, Vifor, Novo Nordisk, Bristol Myers Squibb, Novartis, Afimmune, Agios, Novartis; Consultancy: Pfizer, Bristol Myers Squibb, Novartis; Membership on an Entity’s Board of Directors or Advisory Committees: Afimmune, Novartis; Current Employment: Hippokrateio General Hospital.
