Schrezenmeier Hubert, Höchsmann Britta
German Red Cross Blood Transfusion Service Baden-Württemberg-Hessia, Institute of Clinical Transfusion Medicine and Immunogenetics Ulm, and University of Ulm, Helmholtzstraße 10, 89081 Ulm, Germany.
Transfus Apher Sci. 2012 Feb;46(1):87-92. doi: 10.1016/j.transci.2011.11.012. Epub 2011 Dec 13.
The complement system is an important part of the innate immune system. Complement plays a crucial role in the pathophysiology of many disorders. Despite the pivotal role of the complement system, an approved targeted inhibitor of a complement factor became available only recently. Eculizumab is a humanized monoclonal antibody that inhibits complement factor C5. It is a targeted, disease modifying, treatment of paroxysmal nocturnal hemoglobinuria (PNH). It was approved be the US FDA and the European Commission in 2007. In this review we will update the experience with eculizumab in PNH and discuss potential use of eculizumab in other disorders (e.g. cold agglutinin disease; atypical HUS) and new approaches to complement inhibition with drugs other than eculizumab.
补体系统是固有免疫系统的重要组成部分。补体在许多疾病的病理生理学中起着关键作用。尽管补体系统具有关键作用，但直到最近才出现一种经批准的补体因子靶向抑制剂。依库珠单抗是一种抑制补体因子C5的人源化单克隆抗体。它是阵发性夜间血红蛋白尿（PNH）的一种靶向性、疾病修饰性治疗药物。2007年它获得了美国食品药品监督管理局（FDA）和欧盟委员会的批准。在本综述中，我们将更新依库珠单抗治疗PNH的经验，并讨论依库珠单抗在其他疾病（如冷凝集素病；非典型溶血尿毒综合征）中的潜在用途以及除依库珠单抗外其他药物抑制补体的新方法。
