间歇性夜间血红蛋白尿症（PNH）是一种罕见的非恶性克隆性血液系统疾病，其特征是血液系统细胞膜上GPI连接的补体调节因子缺乏，使它们容易受到补体介导的损伤。血管内溶血（IVH），易凝血性和骨髓衰竭是该疾病的主要特征，与高的发病率和死亡率有关。C5 抑制剂的引入彻底改变了该疾病的结果，为PNH患者提供接近正常的预期寿命。然而，在C5抑制剂治疗期间，残留的IVH和细胞外溶血（EVH）仍然会发生，导致相当一部分患者贫血并有些患者仍然需要输血。目前批准使用的C5抑制剂通过静脉注射，对生活质量（QoL）也存在问题。因此，对于不同组成部分的补体级联或采用不同的制剂允许自我管理的新型制剂进行了探索和开发。长效和皮下制剂的C5抑制剂已经证明具有相同的安全性和有效性，而上游补体抑制剂的开发彻底改变了PNH的治疗方案，限制了IVH和EVH，疗效优于C5抑制剂。同时，也进行了组合治疗试验，取得了有希望的结果。本文总结了PNH的当前治疗选项，抗补体治疗的缺点，并讨论了新兴的PNH治疗方法。本文受版权保护。保留所有权利。

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare non-malignant clonal hematological disorder that is characterized by a deficiency of the GPI-linked complement regulators on the membrane of hematopoietic cells, which renders them susceptible to complement-mediated damage. Intravascular hemolysis (IVH), increased tendency for thrombosis and bone marrow failure constitutes hallmark features of the disease and are associated with high morbidity and mortality. The introduction of C5 inhibitors radically changed disease outcomes, offering a near normal life expectancy to PNH patients. However, residual IVH and extravascular hemolysis (EVH) continue to occur during C5-inhibitor treatment, leaving a significant proportion of patients' anemic and some remaining transfusion dependent. Quality of life (QoL) has also been an issue with the regular intravenous (IV) administrations of the currently licensed C5 inhibitors. This has led to the exploration and development of novel agents, targeting different parts of the complement cascade, or having different formulations allowing for self-administration. Longer acting and subcutaneous formulations of C5 inhibitors have shown equal safety and efficiency, whereas the development of proximal complement inhibitors is changing completely the therapeutic landscape of PNH, limiting both IVH and EVH and showing superior efficacy over C5 inhibitors. Combination treatments have also been tested with promising results. This review summarizes the current therapeutic options, gaps in anti-complement therapy and discusses emerging therapeutic approaches for PNH. This article is protected by copyright. All rights reserved.This article is protected by copyright. All rights reserved.