治疗造血干细胞移植相关血栓性微血管病的新药NarsoplimabOA
A novel drug for treating thrombotic microangiopathy associated with hematopoietic stem cell transplantation-Narsoplimab
Narsoplimab于2025年12月24日由美国食品药品监督管理局批准,成为首个用于治疗2岁及以上儿童与成人造血干细胞移植相关血栓性微血管病(TA-TMA)的药物.TA-TMA是造血干细胞移植(HSCT)后较为常见且病情凶险的一类并发症,具有较高死亡率.TA-TMA与内皮损伤有关,可激活补体凝集素途径,甘露聚糖集合凝集素相关丝氨酸蛋白酶-2(MASP-2)是该途径中的关键酶.Narsoplimab是一种人源单克隆抗体,能够特异性结合并抑制MASP-2,可降低受损组织中的补体激活,同时不破坏补体在抗感染中的有益作用,在多项真实世界研究中显示出了较高的缓解率.现对Narsoplimab的作用机制、药效学特征、药代动力学特点、临床研究进展及安全性研究现状等做一分析与介绍.
Narsoplimab was first approved by the U.S.Food and Drug Administration(FDA)on December 24,2025,for the management of thrombotic microangiopathy following hematopoietic stem cell transplantation(TA-TMA)in children aged 2 years and older and adults.As a serious complication of hematopoietic stem cell transplantation(HSCT),TA-TMA is characterized by high mortality.TA-TMA is associated with endothelial injury and can activate the complement agglutinin pathway,where mannose-binding agglutinin-related serine protease-2(MASP-2)is a key enzyme.Narsoplimab is a humanized monoclonal antibody that specifically binds to and inhibits MASP-2,thereby reducing complement activation in damaged tissues without compromising the beneficial role of complement in anti-infection.Multiple real-world studies have demonstrated a high response rate.This article provides an overview of the mechanism of action,pharmacodynamics,pharmacokinetics,clinical studies,and safety profile of Narsoplimab.
阮婷婷;贾岩岩;杜华情;卞婷婷
南京医科大学附属妇产医院/南京市妇幼保健院药学部,江苏南京 210004南京医科大学附属妇产医院/南京市妇幼保健院药学部,江苏南京 210004南京医科大学附属妇产医院/南京市妇幼保健院药学部,江苏南京 210004扬州大学医学院附属扬州妇幼保健院药学部,江苏扬州 225006
医药卫生
Narsoplimab造血干细胞移植相关血栓性微血管病甘露聚糖集合凝集素相关丝氨酸蛋白酶-2
narsoplimabhematopoietic stem cell transplant-associated thrombotic microangiopathymannan-binding lectin-associated serine protease 2
《中国临床药理学杂志》 2026 (4)
548-551,4
江苏省药学会-奥赛康医院药学科研基金资助项目(A201926)
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