杨卫 张国香

    【摘要】 阵发性睡眠性血红蛋白尿(PNH)源于磷脂酰肌醇多糖A类(PIG-A)基因的体细胞突变引起，该突变导致糖基磷脂酰肌醇锚连蛋白缺乏。而缺乏这种锚连蛋白(CD55、CD59)之一會导致溶血。临床表现包括慢性溶血，血栓栓塞性疾病，感染性并发症，慢性肾脏损伤，肺动脉高压和平滑肌功能障碍。依库珠单抗(Eculizumab)是一种针对末端补体蛋白C5的人源化单克隆抗体，可以使PNH患者的存活率显著提高，并减少并发症。但带来强大疗效的同时，也面临着许多的难题。例如：骨髓衰竭、突破性溶血、感染等。本文回顾了依库珠单抗在PNH治疗中取得的研究进展，并介绍目前尚未解决的难题。

    【关键词】 阵发性睡眠性血红蛋白尿 依库珠单抗

    Research Progress of Eculizumab in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Review/YANG Wei， ZHANG Guoxiang. //Medical Innovation of China， 2021， 18(10)： -181

    [Abstract] Paroxysmal nocturnal hemoglobinuria (PNH) is caused by somatic mutations in the phosphatidylinositol polysaccharide class A (PIG-A) gene， which results in glycosylphosphatidylinositol anchoring protein deficiency. The deficiency of one of these ancholectin proteins (CD55， CD59) can lead to hemolysis. Clinical manifestations include chronic hemolysis， thromboembolic disease， infectious complications， chronic kidney injury， pulmonary hypertension， and smooth muscle dysfunction. Eculizumab is a humanized monoclonal antibody against terminal complement protein C5， which can significantly improve the survival rate of PNH patients and reduce complications. But at the same time ......