依库珠单抗治疗阵发性睡眠性血红蛋白尿的研究进展  

Research advances of Eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria

作　　者：邱婧妍 秘红岭 QIU Jingyan;MI Hongling(Department of Haematology,Nanjing Pukou People's Hospital,Nanjing,Jiangsu 210029,China)

机构地区：[1]南京市浦口人民医院血液科,江苏南京210029

出　　处：《医药前沿》2024年第3期37-39,43,共4页Journal of Frontiers of Medicine

摘　　要：依库珠单抗是最早被美国食品药品监督管理局(FDA)批准用于治疗阵发性睡眠性血红蛋白尿(PNH)的一种新型的补体单抗。PNH是造血干细胞X染色体上PIG-A基因突变,补体活化异常所致的一种罕见的良性造血干细胞克隆性疾病。依库珠单抗治疗PNH的安全性及疗效良好,最主要的潜在严重不良反应是脑膜炎奈瑟氏菌感染。本文对依库珠单抗治疗PNH的有效性、安全性及局限性进行综述,以期为临床提供参考。Eculizumab,a new type of complement monoclonal antibody,was first approved by the US Food and Drug Administration(FDA)for the treatment of paroxysmal nocturnal hemoglobinuria(PNH).PNH is a rare,benign hematopoietic stem cell clonal disease caused by mutation of the PIG-A gene on the X chromosome of hematopoietic stem cells and abnormal complement activation.The safety and efficacy of escuzumab in the treatment of PNH are good,and the main potential serious adverse reaction is Neisseria meningitidis infection.This article reviews the efficacy,safety and limitations of eculizumab in the treatment of PNH,in order to provide reference for clinical practice.

关 键 词：阵发性睡眠性血红蛋白尿 依库珠单抗 血管内溶血 补体抑制剂 

分 类 号：R556.7[医药卫生—血液循环系统疾病]