首个β-地中海贫血治疗药物罗特西普  

The first drug forβ-thalassemia-luspatercept

作　　者：张闪闪 徐文峰[1] 王晶[1] 徐硕[1] 金鹏飞[1] ZHANG Shan-shan;XU Wen-feng;WANG Jing;XU Shuo;JIN Peng-fei(Department of Pharmacy,Beijing Hospital,National Center of Gerontology,Institute of Geriatric Medicine,Chinese Academy of Medical Sciences,Beijing Key Laboratory of Assessment of Clinical Drugs Risk and Individual Application(Beijing Hospital),Beijing 100730,China)

机构地区：[1]北京医院药学部,国家老年医学中心,中国医学科学院老年医学研究院,北京市药物临床风险与个体化应用评价重点实验室(北京医院),北京100730

出　　处：《中国新药杂志》2024年第11期1098-1101,共4页Chinese Journal of New Drugs

摘　　要：罗特西普可通过促进晚期红细胞成熟提高血红蛋白水平,是全球首个且目前唯一一个红细胞成熟剂,也是美国FDA批准的首个用于β-地中海贫血的药物。2022年1月,我国国家药品监督管理局批准其用于治疗需要定期输注红细胞且红细胞输注≤15单位/24周的β-地中海贫血成人患者。临床研究表明,罗特西普可有效降低β-地中海贫血患者的输血负担,不良反应可控且患者耐受性良好。本文对其作用机制、药动学、临床评价、安全性等信息进行综述,旨在为临床用药提供参考。Luspatercept is the world's first and currently only red blood cell maturation agent which increases hemoglobin levels by promoting advanced red blood cell maturation.It's the first FDA-approved drug for beta-thalassemia.In January 2022,China's NMPA approved it for the treatment of adult beta-thalassemia patients who require regular red blood cell infusion and red blood cell infusion≤15 units/24 weeks.Clinical studies have shown that luspatercept can effectively reduce blood transfusion burden in patients with beta-thalassemia with controllable adverse reactions and good tolerance.The mechanism,pharmacokinetics,clinical evaluation and safety are reviewed in this article so as to provide reference for clinical medication.

关 键 词：罗特西普 红细胞成熟剂 Β-地中海贫血 

分 类 号：R973.3[医药卫生—药品]