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Management of luspatercept therapy in patients with transfusion‐dependent β‐thalassaemia

Year: 2023
Type: review
Abstract: 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.
DOI: 10.1111/bjh.18801
Institutions: Weill Cornell Medicine, American University of Beirut Medical Center, University of Southern California, Children's Hospital Los Angeles, National and Kapodistrian University of Athens, University of Milan, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Language: English
Subfield: Genetics
Field: Medicine
Open Access status: hybrid
Estimated APC paid: $4,940
OpenAlex ID: W4363679472
FWCI: 0.984
Cites: 27
Cited by: 21
Related to: 10