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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 maturation 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 according to drug instruction. The efficacy and safety data of patients were collected.Results A total of 6 patients with β-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 dependent β-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 with β-thalassemia.
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Key words:
- thalassemia /
- luspatercept /
- transfusion
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表 1 接受罗特西普治疗的6例β地贫患者的临床特征
患者编号 性别 年龄/岁 基因型 开始输血年龄/岁 是否切脾 用药前6个月输血量/U 用药前6个月最大输血间隔/d 诊断 1 女 31 CD41-42/βE 13 是 6 不适用 NTDT 2 女 20 CD-28/CD-28 3 是 6 不适用 NTDT 3 男 18 CD41-42/CD17 1 是 20 30 TDT 4 女 20 CD41-42/IVS-2-654 1 无 36 20 TDT 5 男 21 CD17/17-SEA/αα 4 无 12 30 TDT 6 女 19 CD17/CD17 1 是 24 30 TDT 
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表 2 6例患者使用罗特西普治疗后平均Hb和输血量变化
患者编号 用药次数 疗程/d 平均Hb/(g/dL) 平均输血量/(U/月) 疗效 用药前 用药后 提高值 用药前 用药后 下降/% 1 7 134 5.8 7.6 1.8 1.0 0.45 55 有效 2 4 81 5.9 8.2 2.3 1.1 0 无法判断 有效 3 5 86 6.0 7.0 1.0 3.3 4.9 -48.5 无效 4 6 106 8.8 10.3 1.5 6.0 1.1 81.7 有效 5 4 75 7.4 10.1 2.7 2.0 1.2 40.0 有效 6 2 21 5.8 7.8 2.0 3.0 5.0 -66.7 无法判断
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