Yıldız Serkan, Demirkan Fatih
Dokuz Eylul University, Division of Nephrology, Department of Internal Medicine, Izmir, Turkey.
Dokuz Eylul University, Division of Hematology, Department of Internal Medicine, 35340, Inciralti, Izmir, Turkey.
Transfus Apher Sci. 2018 Feb;57(1):31-34. doi: 10.1016/j.transci.2018.02.013. Epub 2018 Feb 21.
Thrombotic microangiopathies (TMAs) are disorders characterized by endothelial cell activation, microangiopathic hemolytic anemia, thrombocytopenia and organ failure of variable intensity. The pathophysiology of various types of TMAs have become an interesting field of study. Alternative complement system activation plays an important role in several pathophysiological conditions. Complement activation is also described in an increasing number of TMAs. Inherited defects in complement regulatory genes and acquired autoantibodies against complement regulatory proteins have been described. Atypical hemolytic uremic synrome (HUS) is caused by uncontrolled activation of the alternative complement system, now called complement-mediated TMAs. Recently, application of a monoclonal antibody that specifically binds to C5 became available to treat patients with complement-mediated TMAs. Eculizumab is a humanized monoclonal antibody that blocks complement C5 activation. Empiric therapeutic apheresis is also recommended in all forms of complement-mediated TMAs. The justification for therapeutic apheresis use in all forms of complement-mediated TMAs is that it can effectively remove the autoantibodies or mutated circulating complement regulators while replacing absent or defective complement regulators. Currently, therapeutic apheresis and eculizumab are the available treatment options for complement-mediated TMAs. In this paper, we review the evidence for the role of therapeutic apheresis in the management of complement-associated TMAs.
血栓性微血管病（TMA）是以内皮细胞活化、微血管病性溶血性贫血、血小板减少以及不同程度的器官功能衰竭为特征的疾病。各类TMA的病理生理学已成为一个有趣的研究领域。替代补体系统激活在多种病理生理状况中发挥重要作用。补体激活在越来越多的TMA中也有描述。已发现补体调节基因的遗传性缺陷以及针对补体调节蛋白的获得性自身抗体。非典型溶血性尿毒症综合征（HUS）由替代补体系统的失控激活所致，现称为补体介导的TMA。最近，一种特异性结合C5的单克隆抗体可用于治疗补体介导的TMA患者。依库珠单抗是一种阻断补体C5激活的人源化单克隆抗体。对于所有形式的补体介导的TMA，经验性治疗性血液成分单采也受到推荐。在所有形式的补体介导的TMA中使用治疗性血液成分单采的理由是，它可以有效去除自身抗体或突变的循环补体调节因子，同时补充缺失或有缺陷的补体调节因子。目前，治疗性血液成分单采和依库珠单抗是补体介导的TMA的可用治疗选择。在本文中，我们综述了治疗性血液成分单采在补体相关TMA管理中作用的证据。
