Discontinuation of Eculizumab Maintenance Treatment for Atypical Hemolytic Uremic Syndrome: A Report of 10 Cases
Discontinuation of Eculizumab Maintenance Treatment for Atypical Hemolytic Uremic Syndrome: A Report of 10 Cases
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DOI：
10.1053/j.ajkd.2014.01.434
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发表时间：
2014-10-01
影响因子：
13.2
通讯作者：
Cugno, Massimo
中科院分区：
文献类型：
--
作者：
Ardissino, Gianluigi;Testa, Sara;Cugno, Massimo
非典型溶血性尿毒综合征（阿胡斯）是一种危及生命的血栓性微血管病，多达70%的阿胡斯患者编码补体调节蛋白的基因发生突变。Eculizumab是一种靶向C5的人源化重组单克隆抗体，自2009年以来已成功用于阿胡斯患者。标准的维持治疗需要终生的依库珠单抗治疗，但停药的可能性尚未得到系统性的测试。我们报告了10例停止治疗的患者停止依库珠单抗治疗的安全性，目的是最大限度地减少不良反应的风险，降低脑膜炎的风险，提高生活质量，同时降低可观的治疗费用。通过尿试纸检测血红蛋白，在家中密切监测疾病活动。在95个月的累积观察期内，10例患者中有3例在停药后6周内复发，但随后立即恢复治疗并完全康复。我们的经验支持停止依库珠单抗治疗的可能性，并对达到稳定缓解的阿胡斯患者进行严格的家庭监测，以发现早期复发迹象。(C)2014年，美国国家肾脏基金会（National Kidney Foundation，Inc.）
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. (C) 2014 by the National Kidney Foundation, Inc.