Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Am J Kidney Dis. 2014 Oct;64(4):633-7. doi: 10.1053/j.ajkd.2014.01.434. Epub 2014 Mar 19.
Atypical hemolytic uremic syndrome (aHUS) is a life-threatening thrombotic microangiopathy, and as many as 70% of patients with aHUS have mutations in the genes encoding complement regulatory proteins. Eculizumab, a humanized recombinant monoclonal antibody targeting C5, has been used successfully in patients with aHUS since 2009. The standard maintenance treatment requires life-long eculizumab therapy, but the possibility of discontinuation has not yet been tested systematically. We report the safety of discontinuing eculizumab treatment in 10 patients who stopped treatment with the aim of minimizing the risk of adverse reactions, reducing the risk of meningitis, and improving quality of life while also reducing the considerable treatment costs. Disease activity was monitored closely at home by means of urine dipstick testing for hemoglobin. During the cumulative observation period of 95 months, 3 of the 10 patients experienced relapse within 6 weeks of discontinuation, but then immediately resumed treatment and completely recovered. Our experience supports the possibility of discontinuing eculizumab therapy with strict home monitoring for early signs of relapse in patients with aHUS who achieve stable remission.
非典型溶血尿毒症综合征（aHUS）是一种危及生命的血栓性微血管病，多达 70%的 aHUS 患者有补体调节蛋白编码基因的突变。自 2009 年以来，针对 C5 的人源化重组单克隆抗体依库珠单抗已成功用于 aHUS 患者。标准维持治疗需要终生依库珠单抗治疗，但尚未系统地测试停药的可能性。我们报告了 10 名患者停止依库珠单抗治疗的安全性，这些患者的停药目的是最大限度地降低不良反应风险、降低脑膜炎风险、提高生活质量，同时降低相当大的治疗费用。通过尿液潜血试验监测血红蛋白，患者在家中密切监测疾病活动情况。在 95 个月的累积观察期内，有 3 名患者在停药后 6 周内复发，但随后立即恢复治疗并完全康复。我们的经验支持在 aHUS 患者达到稳定缓解后，通过严格的家庭监测，对可能出现的复发迹象进行早期发现，从而停止依库珠单抗治疗。
