超过 25 年的 PNH 经验教训：靶向补体抑制时代。

Learnings from over 25 years of PNH experience: the era of targeted complement inhibition.

机构信息

Bahnhofstrasse 17, 6403 Küssnacht am Rigi, Switzerland.

出版信息

Blood Rev. 2013 Dec;27 Suppl 1:S1-6. doi: 10.1016/S0268-960X(13)00080-5.

Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is a progressive and life-threatening disease that causes thrombosis, end organ damage and impaired quality of life. Chronic uncontrolled complement activation leads to chronic haemolysis, causing progressive morbidities and early mortality. Hence, early diagnosis is essential for improved patient management and prognosis. Eculizumab (SOLIRIS®) specifically inhibits chronic, uncontrolled complement activation and is the first-in-class, humanised, monoclonal antibody targeting C5 within the terminal complement pathway. Eculizumab is the first and only approved treatment for PNH in adults and children.

摘要

阵发性睡眠性血红蛋白尿症（PNH）是一种进行性的、危及生命的疾病，可导致血栓形成、终末器官损伤和生活质量受损。慢性失控的补体激活可导致慢性溶血性贫血，从而导致进行性发病和早逝。因此，早期诊断对于改善患者管理和预后至关重要。依库珠单抗（SOLIRIS®）特异性抑制慢性、失控的补体激活，是首个靶向末端补体途径 C5 的人源化单克隆抗体。依库珠单抗是成人和儿童 PNH 的首个也是唯一获批的治疗药物。