ABSTRACT

Introduction

Red blood cell transfusions and iron chelation therapy are the cornerstone of treatment for β-thalassemia, with allogeneic hematopoietic stem cell transplantation and gene therapy offering further disease-management options for eligible patients. With up to 90% of severe cases of β-thalassemia occurring in resource-constrained countries, and estimates indicating that 22,500 deaths occur annually as a direct consequence of undertransfusion, provision of adequate treatment remains a major issue.

Areas covered

In this review, we provide an overview of luspatercept, a first-in-class erythroid maturation agent, and present the available clinical data related to the treatment of β-thalassemia.

Expert opinion

The recent approval of luspatercept offers a new, long-term therapeutic option for adult patients with transfusion-dependent β-thalassemia to reduce red blood cell transfusion burden, anemia, and iron overload.

中文翻译：

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Luspatercept 治疗β-地中海贫血：超越红细胞输血

摘要

介绍

红细胞输血和铁螯合疗法是治疗β-地中海贫血的基石，异基因造血干细胞移植和基因疗法为符合条件的患者提供了进一步的疾病管理选择。高达 90% 的严重 β-地中海贫血病例发生在资源有限的国家，估计每年有 22,500 人死于输血不足的直接后果，因此提供充分的治疗仍然是一个主要问题。

涵盖的领域

在这篇综述中，我们概述了 luspatercept，一种一流的红细胞成熟剂，并介绍了与治疗 β-地中海贫血相关的可用临床数据。

专家意见

最近批准的 luspatercept 为患有输血依赖性 β-地中海贫血的成年患者提供了一种新的长期治疗选择，以减少红细胞输血负担、贫血和铁过载。