Division of Pediatric Hematology/Oncology, Weill Cornell Medicine, New York, New York, USA.
Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
Br J Haematol. 2023 Jun;201(5):824-831. doi: 10.1111/bjh.18801. Epub 2023 Apr 10.
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.
依赖输血的β-地中海贫血患者为了生存需要终生定期输注红细胞；然而，频繁输血会增加铁过载、输血传播疾病和同种免疫以及降低生活质量的风险。促红细胞生成素成熟剂 luspatercept 可独立于促红细胞生成素促进晚期红细胞成熟，已证明其在降低依赖输血的β-地中海贫血患者的输血负担方面具有疗效。在这篇综述中，我们讨论了接受 luspatercept 治疗的依赖输血的β-地中海贫血患者的治疗起始、持续评估、剂量调整和不良反应管理，并就如何确定患者是否获得临床获益提供了指导。
