Maria Domenica Cappellini

Summary Patients with transfusion‐dependent β‐thalassaemia require lifelong, regular red blood cell transfusions for survival; however, frequent blood transfusions are associated with an increased risk of iron overload, transfusion‐transmitted disease and alloimmunization, as well as reduced quality of life. Luspatercept, an erythroid maturation agent that promotes late‐stage erythroid maturation independently of erythropoietin, has demonstrated efficacy in reducing transfusion burden in patients with transfusion‐dependent β‐thalassaemia. In this review, we discuss treatment initiation, ongoing evaluation, dose adjustment and management of adverse events in transfusion‐dependent patients with β‐thalassaemia receiving luspatercept, and we provide guidance on how to determine whether patients are deriving clinical benefit.