中国临床药理学杂志2026，Vol.42Issue(4)：548-551,4.DOI:10.13699/j.cnki.1001-6821.2026.04.015
治疗造血干细胞移植相关血栓性微血管病的新药Narsoplimab
A novel drug for treating thrombotic microangiopathy associated with hematopoietic stem cell transplantation-Narsoplimab
摘要
Abstract
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.关键词
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-2Key words
narsoplimab/hematopoietic stem cell transplant-associated thrombotic microangiopathy/mannan-binding lectin-associated serine protease 2分类
医药卫生引用本文复制引用
阮婷婷,贾岩岩,杜华情,卞婷婷..治疗造血干细胞移植相关血栓性微血管病的新药Narsoplimab[J].中国临床药理学杂志,2026,42(4):548-551,4.基金项目
江苏省药学会-奥赛康医院药学科研基金资助项目(A201926) （A201926）
