阵发性睡眠性血红蛋白尿症抗补体治疗现状  

Current situation of anti-complement treatment in paroxysmal nocturnal hemoglobinuria

作　　者：付蓉[1] FU Rong(Department of Hematology,Tianjin Medical University General Hospital,Tianjin 300052,China)

机构地区：[1]天津医科大学总医院血液内科,天津300052

出　　处：《中国实用内科杂志》2020年第9期722-727,共6页Chinese Journal of Practical Internal Medicine

基　　金：国家自然科学基金(81770110)

摘　　要：阵发性睡眠性血红蛋白尿症(PNH)是一种获得性造血干细胞克隆性疾病,其病变细胞X染色体PIG-A基因突变与PNH细胞克隆增殖及疾病进展密切相关。伴随着PNH发病机制研究的不断深入,其诊断和治疗模式也取得了重要进展,尤其是靶向模式的出现为PNH的治疗提供了新的策略,依库珠单抗的问世使PNH的治疗进入补体通路抑制时代,由于其存在原发耐药、血管外溶血、停药后病情易反复等不足,使针对补体通路活化的新药研发成为热点。文章主要围绕PNH的抗补体治疗现状作一阐述。Paroxysmal nocturnal hemoglobinuria(PNH)is an acquired clonal disorder of hematopoietic stem cells due to the mutation of PIG-A gene which is closely related to the proliferation of PNH cells and the progress of disease.Knowledge of the molecular mechanisms leading to PNH has substantially increased in the past decades,which remarkably advance the diagnostic modalities and treatment approaches of PNH patients.Targeted mode provides a new strategy for the treatment of PNH.The emergence of eculizumab enables the treatment of PNH to enter the era of complement pathway inhibition.Due to its shortcomings such as primary drug resistance,extravascular hemolysis,and easy relapse after drug withdrawal,the research and development of new drugs for complement pathway activation has become a hot spot.This article mainly reviews the status quo of anti-complement therapy for PNH.

关 键 词：阵发性睡眠性血红蛋白尿 补体 靶向治疗 

分 类 号：R556.64[医药卫生—血液循环系统疾病]