补体抑制剂在罕见病的临床应用  

Complement Inhibitors in Rare Diseases

作　　者：乐偲[1] 杜亚丽[2] 黄杨钰 陈丽萌[1] 管宇宙[3] 韩冰[2] YUE Cai;DU Yali;HUANG Yangyu;CHEN Limeng;GUAN Yuzhou;HAN Bing(Department of Nephrology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;Department of Hematology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;Department of Neurology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医院肾内科,北京100730 [2]中国医学科学院北京协和医院血液科,北京100730 [3]中国医学科学院北京协和医院神经科,北京100730

出　　处：《罕见病研究》2022年第4期391-399,共9页Journal of Rare Diseases

基　　金：中国医学科学院医学与健康科技创新工程项目(2021-I2M-1-003)。

摘　　要：补体系统是人体的一种自我保护机制,补体系统的异常活化参与多种疾病的发生、发展。补体抑制剂的使用为很多罕见疾病,如阵发性睡眠性血红蛋白尿症(PNH)、非典型溶血尿毒综合征(aHUS)等,带来了里程碑式的进步。近年来,补体抑制剂的应用也逐渐向其他补体相关疾病领域拓展。本文结合近年文献,就补体抑制剂在较为成熟的罕见病领域的应用进行总结,也对其今后在相关罕见病领域的应用前景予以展望。The complement system is a self-protection mechanism of the human body. The abnormal activation of the complement system is involved in the occurrence and development of various diseases. The application of complement inhibitors in many rare diseases was a milestone in leading to the progress of such disease as paroxysmal nocturnal hemoglobinuria(PNH), atypical hemolytic uremic syndrome(aHUS), and others. Recently, the application of complement inhibitors has gradually expanded to other complement-related diseases. This review summarizes the literature on the current application of complement inhibitors in rare diseases and looks into the prospects of the application in the rare diseases.

关 键 词：补体抑制剂 阵发性睡眠性血红蛋白尿症 非典型溶血尿毒综合征 重症肌无力 

分 类 号：R453.9[医药卫生—治疗学]