阵发性睡眠性血红蛋白尿症的临床进展  

Clinical Progres of Paroxysmal Nocturnal Hemoglobinuria——Review

作　　者：龚亚文[1] 何广胜[1] 

机构地区：[1]苏州大学附属第一医院,江苏省血液研究所,卫生部血栓与止血重点实验室,江苏苏州215006

出　　处：《中国实验血液学杂志》2013年第6期1627-1630,共4页Journal of Experimental Hematology

基　　金：国家科技支撑计划(编号2008BAI61B02;2008ZX09312-026);江苏省医学重点人才资助项目(编号H201126);高校自然科学研究项目(编号09KJB320015);江苏省临床医学中心(编号ZX201102)

摘　　要：随着FLAER高灵敏度流式细胞仪诊断技术和补体C5单克隆抗体的应用,许多学者对阵发性睡眠性血红蛋白尿症(PNH)的认识发生了改变。这些新的研究成果表明,人源型抗C5单克隆抗体的治疗能改变PNH的自然病史,能从根本上减少血栓栓塞、控制血管内溶血、减少或消除输血的需求,减少了疾病相关的死亡率。本文对PNH和骨髓衰竭症之间的关系、抗C5单克隆抗体治疗PNH的长期疗效、控制PNH血管内和血管外溶血的新治疗策略进行综述。Through the applications of high-sensitivity flow cytometry of FLAER and the treatment of eculizumab, it is necessary to understand of paroxysmal nocturnal hemoglobinuria （PNH） from a new point of view. The results of studies demonstrate that treatment with eculizumab alters the natural history of PNH by virtually eradicating thromboem- bolic complications, inhibiting of intravascular hemolysis and reducing or eliminating transfusion requirements. Eculi- zumab treatment may also reduce disease-related mortality. This review focuses on the studies to define the relationship between PNH and bone marrow failure syndromes and to characterize the long-term outcome of patients with PNH treated with eculizumab. New therapeutic strategies aimed at controlling extravascular and intravascular hemolysis are discussed.

关 键 词：抗C5单克隆抗体 溶血 阵发性睡眠性血红蛋白尿症 再生障碍性贫血 

分 类 号：R556.64[医药卫生—血液循环系统疾病]