Department of Onco-Hematology, AORN San Giuseppe Moscati Avellino, Avellino, Italy.
Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples, Italy.
Br J Haematol. 2022 Jan;196(2):288-303. doi: 10.1111/bjh.17753. Epub 2021 Aug 5.
Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement-mediated intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients with bone marrow failure the treatment follows that of immune-mediated aplastic anaemia, that of classic, haemolytic PNH is based on anti-complement medication. The anti-C5 monoclonal antibody eculizumab has revolutionized treatment, resulting in control of intravascular haemolysis and thromboembolic risk, with improved long-term survival. Novel strategies of complement inhibition are emerging. New anti-C5 agents reproduce the safety and efficacy of eculizumab, with improved patient convenience. Proximal complement inhibitors have been developed to address C3-mediated extra-vascular haemolysis and seem to improve haematological response.
阵发性睡眠性血红蛋白尿症（PNH）的特征是补体介导的血管内溶血、严重的血栓形成倾向和骨髓衰竭。对于骨髓衰竭的患者，治疗遵循免疫介导的再生障碍性贫血的治疗方法，而经典的溶血性 PNH 的治疗则基于抗补体药物。抗 C5 单克隆抗体依库珠单抗（eculizumab）彻底改变了治疗方法，控制了血管内溶血和血栓栓塞风险，改善了长期生存。新的补体抑制策略正在出现。新型抗 C5 药物再现了依库珠单抗的安全性和疗效，提高了患者的便利性。已开发出近端补体抑制剂来解决 C3 介导的血管外溶血，并似乎改善了血液学反应。
