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 with β-thalassemia in China
黄秋莹 1周亚丽 1周天红 1杨蓓蓓 1韦长青 1尹晓林 1廖桂萍1
作者信息
- 1. 中国人民解放军联勤保障部队第九二三医院血液科(南宁,530021)
- 折叠
摘要
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.
Abstract
Objective:To summarize the clinical efficacy and safety of luspatercept,the first erythroid matura-tion agent,in Chinese β-thalassemia patients in real-world study.Methods:A retrospective analysis was carried out in patients diagnosed with β-thalassemia from May 2022 to January 2023.Luspatercept was prescribed accord-ing 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 pa-tients 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 trans-fusion were increased to 10.1 g/dL and 10.3 g/dL.Conclusion:Luspatercept can improve the hemoglobin level in non-transfusion dependent β-thalassaemia patients,and reduce the need for blood transfusion in transfusion de-pendent patients,and has a good efficacy in Chinese population.More accumulated data is needed to improve the prognosis of Chinese patients with β-thalassemia.
关键词
地中海贫血/罗特西普/输血Key words
thalassemia/luspatercept/transfusion引用本文复制引用
基金项目
广州市生命绿洲公益服务中心血液病领域科研基金(GZLZ-HEMA-008)
广西卫生健康委自筹经费科研课题(Z-A20231086)
出版年
2024
NETL
NSTL
万方数据