Eculizumab for paroxysmal nocturnal haemoglobinuria

伊库利珠单抗 CD59型 阵发性夜间血红蛋白尿 补体系统 溶血 免疫学 补体膜攻击复合物 衰变加速因子 医学 补体成分5 非典型溶血尿毒综合征 内科学 血红蛋白尿 溶血 抗体
作者
Charles J. Parker
出处
期刊:The Lancet [Elsevier BV]
卷期号:373 (9665): 759-767 被引量:150
标识
DOI:10.1016/s0140-6736(09)60001-5
摘要

The complement system plays a central part in both innate and acquired immunity, but the contribution of complement activation to pathobiology is largely ancillary. An exception to the non-dominant role of complement in disease is the haemolytic anaemia of paroxysmal nocturnal haemoglobinuria (PNH). The intravascular haemolysis that is the clinical hallmark of PNH is a consequence of deficiency of the complement inhibitory proteins decay accelerating factor (DAF, CD55) and membrane inhibitor of reactive lysis (MIRL, CD59). Eculizumab is a humanised monoclonal antibody that binds and prevents activation of complement C5 and the subsequent formation of the cytolytic membrane attack complex of complement. Eculizumab inhibits the intravascular haemolysis of PNH, reduces transfusion requirements, stabilises haemoglobin concentration, and improves quality of life. Although chronic treatment with eculizumab increases the risk of infections with Neisseria meningitides, the drug is generally safe and well tolerated. But as is the case with other drugs developed for treatment of ultra-orphan diseases, eculizumab is expensive, and treatment must continue indefinitely because C5 inhibition does not affect the process (ie, clonal proliferation of haemopoietic stem cells with a mutant phosphatidylinositol glycan complementation class A [PIGA] gene) that underlies PNH. Moreover, due to the heterogeneous nature of the disease, treatment with eculizumab is not appropriate for all patients with PNH.
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