【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。更多还原

【Abstract】 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.更多还原