依库珠单抗治疗阵发性睡眠性血红蛋白尿症1例并文献复习  

A case of paroxysmal nocturnal hemoglobinuria treated with eculizumab and review of literature

作　　者：周茉 邵钰[1,2,3] 周燕 孙乃同[1,2,3] ZHOU Mo;SHAO Yu;ZHOU Yan;SUN Naitong(Department of Hematology,the Sixth Affiliated Hospital of Nantong University,Yancheng,Jiangsu,224000,China;Department of Hematology,the Yancheng School of Clinical Medicineof Nanjing Medical University;Department of Hematology,Yancheng Third People's Hospital)

机构地区：[1]南通大学第六附属医院血液科,江苏盐城224000 [2]南京医科大学盐城临床医学院血液科 [3]盐城市第三人民医院血液科

出　　处：《临床血液学杂志》2025年第3期240-242,共3页Journal of Clinical Hematology

摘　　要：阵发性睡眠性血红蛋白尿症(PNH)是一种罕见的获得性造血干细胞克隆性疾病,其特征是由于PIG-A基因的获得性突变导致的血管内溶血和血红蛋白尿。依库珠单抗是一种C5补体抑制剂,可以抑制补体系统,从而减少红细胞的破坏,但由于经济、可及性等原因,目前中国PNH患者使用依库珠单抗的病例还罕有报道。我院最近采用依库珠单抗治疗1例病程长达11年的年轻男性PNH患者,取得了较为满意的疗效,现将患者的诊疗经过和疗效情况进行总结。Paroxysmal nocturnal hemoglobinuria(PNH)is a rare,acquired clonal disorder of hematopoietic stem cells.It is primarily characterized by intravascular hemolysis and hemoglobinuria,resulting from acquired mutations in the PIG-A gene.Eculizumab,a C5complement inhibitor,effectively impedes the complement system,thereby mitigating red blood cell destruction.Nonetheless,due to economic constraints and limited accessibility,there are scant reports on the use of Eculizumab in Chinese patients with PNH.Recently,our medical team treated a young male patient who had an 11-year history of PNH using Eculizumab,which resulted in remarkable clinical improvement.We are now summarizing the diagnostic and therapeutic process,as well as the effectiveness of the treatment.

关 键 词：阵发性睡眠性血红蛋白尿症 难治性 依库珠单抗 

分 类 号：R55[医药卫生—血液循环系统疾病]