罗特西普治疗β-地中海贫血的研究进展
Research Progress of Luspatercept in the Treatment of β-Thalassemia
DOI: 10.12677/acm.2025.1592656, PDF,   
作者: 唐彩练, 李昱佳：右江民族医学院研究生学院，广西 百色；王小超*：右江民族医学院附属医院血液科，广西 百色
关键词: β-地中海贫血；罗特西普；靶向药物；研究进展；β-Thalassemia； Luspatercept； Targeted Drugs； Research Progress
摘要: 地中海贫血(thalassemia，简称地贫)是一种以珠蛋白肽链合成障碍为特征的遗传性疾病，其发病机制为基因变异导致血红蛋白α链及非α链合成比例失衡，根据受累基因不同，主要分为α地贫和β地贫，传统上，改善其贫血最常用的方式为输血治疗，鉴于我国血源紧张和经济条件等因素，我国的大多数患者难以做到规范输血，能有效改善贫血的药物治疗一直在探索中。罗特西普(luspatercept)是一种促红细胞成熟剂，已被国内批准用于治疗β-地中海贫血，本文旨在对罗特西普治疗β-地中海贫血的相关研究进展进行综述。
Abstract: Thalassemia is a genetic disorder characterized by the disorder of globin peptide chain synthesis. Its pathogenesis is that genetic variations lead to an imbalance in the synthesis ratio of hemoglobin α chains and non-α chains. According to the different involved genes, it is mainly divided into α-thalassemia and β-thalassemia. Traditionally, the most commonly used way to improve anemia is blood transfusion therapy. Given factors such as the tight blood supply and economic conditions in our country, most patients in China find it difficult to receive standardized blood transfusions. Drug treatments that can effectively improve anemia have been under exploration. luspatercept is a erythropoietic maturation agent and has been approved in China for the treatment of β-thalassemia. This article aims to review the relevant research progress of luspatercept in the treatment of β-thalassemia.
文章引用：唐彩练, 王小超, 李昱佳. 罗特西普治疗β-地中海贫血的研究进展[J]. 临床医学进展, 2025, 15(9): 1563-1569. https://doi.org/10.12677/acm.2025.1592656

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