2012年美国血液病学会会议热点：阵发性睡眠性血红蛋白尿症  

2012 American Society of Hematology report:advances in paroxysmal nocturnal hemoglobinuria

作　　者：何广胜[1] Peter Hillmen 

机构地区：[1]苏州大学附属第一医院江苏省血液研究所,江苏苏州215006 [2]St James's University Hospital,University of Leeds,LS9 7TF

出　　处：《中国实用内科杂志》2013年第8期623-626,共4页Chinese Journal of Practical Internal Medicine

摘　　要：阵发性睡眠性血红蛋白尿症(PNH)糖化磷脂酰肌醇-缺陷中性粒细胞(GPI-DG)>50%者乳酸脱氢酶(LDH)高,易发生血栓。儿童PNH患者血栓事件和肾脏疾病发生率较低。GPI基因缺陷后引发T细胞和DC活化,并攻击GPI+髓系细胞,PNH克隆通过免疫逃逸而扩张。抗C5单抗Eculizumab能有效控制血管内溶血,改善临床症状。新的针对补体C3的抗体能够解决eculizumab引起的红细胞膜C3沉积所致血管外溶血。People suffering from paroxysmal nocturnal hemoglobinuria（PNH）with glycosylphosphatidy-linositol-anchored deficient granulocyte（GPI-DG）〉50% are associated with a higher level of LDH and a higher incidence of thrombosis.Pediatric PNH patients have lower rates of thrombosis and renal diseases.GPI gene defects induce activation of T cells and DC,and attack GPI＋myeloid cells and PNH colony expands by escaping the immune injury.The anti-C5 antibody eculizumab has been proven effective in controlling intravascular hemolysis,leading to remarkable clinical benefits in almost all PNH patients.The new antibodies are effective for C3 deposition-induced extravascular hemolysis caused by administration of eculizumab.

关 键 词：阵发性睡眠性血红蛋白尿症 免疫逃逸 ECULIZUMAB 补体C3 

分 类 号：R551[医药卫生—血液循环系统疾病]