How I manage luspatercept in transfusion-dependent beta-thalassemia.

ABSTRACT: Beta-thalassemia is an inherited anemia characterized by a broad spectrum of clinical manifestations. The most severe form is transfusion-dependent β-thalassemia, in which patients need regular blood transfusions to survive, since no adequate amount of hemoglobin is produced by the bone marrow. The therapeutic landscape for this disease is constantly evolving, and new therapies have been recently approved. Luspatercept is the first and only approved drug for treating anemia in transfusion-dependent β-thalassemia. Most available information regarding its safety and efficacy is derived from clinical trials, with limited data on real-world experiences. Thus, a significant gap remains in the literature concerning patient management in everyday clinical settings, particularly in terms of assess

SUBMITTER: Panzieri DL 

PROVIDER: S-EPMC12919478 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature