β-Thalassemia is an autosomal recessive hemoglobinopathy characterized by the absence or reduction of β-globin chains synthesis [1]. Advancements in β-thalassemia therapy [2, 3] include emerging treatments like gene therapy [4, 5] and erythroid maturation agents [6, 7]. Luspatercept, a first-in-class erythroid maturation agent, is FDA and EMA-approved for treating anemia in adults with transfusion-dependent (TD) β-thalassemia [8]. This paper discusses a real-life study on luspatercept’s use in β-thalassemic patients.
Real-life experience with luspatercept in transfusion-dependent β-thalassemia
Roccotelli D.;Grande D.;Cicco G.;Albano F.;Musto P.
2023-01-01
Abstract
β-Thalassemia is an autosomal recessive hemoglobinopathy characterized by the absence or reduction of β-globin chains synthesis [1]. Advancements in β-thalassemia therapy [2, 3] include emerging treatments like gene therapy [4, 5] and erythroid maturation agents [6, 7]. Luspatercept, a first-in-class erythroid maturation agent, is FDA and EMA-approved for treating anemia in adults with transfusion-dependent (TD) β-thalassemia [8]. This paper discusses a real-life study on luspatercept’s use in β-thalassemic patients.File in questo prodotto:
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