罗特西普对中国β地中海贫血患者的临床疗效观察

Clinical efficacy of luspatercept in patients withβ-thalassemia in China

目的:总结首个促进红细胞生成药物罗特西普对中国β地中海贫血患者真实世界应用的临床疗效和安全性。方法:回顾性分析2022年5月至2023年1月在我院诊断为β地中海贫血患者的临床资料,罗特西普根据药物说明书给药,收集患者的有效性和安全性数据。结果:共纳入6例接受罗特西普治疗的β地中海贫血患者,2例非输血依赖型患者血红蛋白分别提高了1.8 g/dL和2.3 g/dL;4例输血依赖型患者中2例输血量分别下降了40.0%和80.7%,输血前血红蛋白分别提高到10.1 g/dL和10.3 g/dL。结论:罗特西普能够提高非输血依赖型β地中海贫血的血红蛋白水平,减少输血依赖型患者的输血,在中国人群中有良好的疗效。未来需要积累更多的数据,以期改善中国β地中海贫血患者的预后。

Objective: To summarize the clinical efficacy and safety of luspatercept,the first erythroid maturation agent,in Chineseβ-thalassemia patients in real-world study.Methods: A retrospective analysis was carried out in patients diagnosed withβ-thalassemia from May 2022 to January 2023.Luspatercept was prescribed according to drug instruction.The efficacy and safety data of patients were collected.Results: A total of 6 patients withβ-thalassemia were included.The hemoglobin level increased in two non-transfusion dependent thalassemia patients by 1.8 g/dL and 2.3 g/dL,respectively.Two among the four transfusion dependent thalassemia patients had decreased need for blood transfusion by 40.0%and 80.7%,respectively.The hemoglobin level before transfusion were increased to 10.1 g/dL and 10.3 g/dL.Conclusion: Luspatercept can improve the hemoglobin level in non-transfusion dependentβ-thalassaemia patients,and reduce the need for blood transfusion in transfusion dependent patients,and has a good efficacy in Chinese population.More accumulated data is needed to improve the prognosis of Chinese patients withβ-thalassemia.