首页|罗特西普对中国 β地中海贫血患者的临床疗效观察

罗特西普对中国 β地中海贫血患者的临床疗效观察

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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.
Clinical efficacy of luspatercept in patients with β-thalassemia in China
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.

thalassemialuspatercepttransfusion

黄秋莹、周亚丽、周天红、杨蓓蓓、韦长青、尹晓林、廖桂萍

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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)

地中海贫血 罗特西普 输血

广州市生命绿洲公益服务中心血液病领域科研基金广西卫生健康委自筹经费科研课题

GZLZ-HEMA-008Z-A20231086

2024

临床血液学杂志
华中科技大学同济医学院血液病研究所 北京医科大学血液病研究所

临床血液学杂志

CSTPCD
影响因子:1.063
ISSN:1004-2806
年,卷(期):2024.37(3)
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