Safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome: protocol for a multicentre, open-label, prospective, single-arm study.
Safety and impact of eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome: protocol for a multicentre, open-label, prospective, single-arm study.
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非典型溶血性尿毒症综合征患者的eculizumab戒断的安全性和影响：多中心，开放标签，前瞻性，单臂研究的方案。
DOI：
10.1136/bmjopen-2021-054536
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发表时间：
2022-09-19
期刊：
影响因子：
2.9
通讯作者：
Sheerin, Neil
中科院分区：
文献类型：
--
作者：
Dunn, Sarah;Brocklebank, Victoria;Bryant, Andrew;Carnell, Sonya;Chadwick, Thomas J.;Johnson, Sally;Kavanagh, David;Lecouturier, Jan;Malina, Michal;Moloney, Eoin;Oluboyede, Yemi;Weetman, Christopher;Wong, Edwin Kwan Soon;Woodward, Len;Sheerin, Neil
非典型溶血性尿毒综合征（阿胡斯）是一种罕见的、危及生命的疾病，由称为补体的免疫系统的一部分过度激活引起。依库珠单抗是一种有效的治疗方法，可控制90%的阿胡斯患者。由于复发的风险，目前建议终身治疗。依库珠单抗治疗并非没有问题，最重要的是严重脑膜炎球菌感染的风险，每两周一次静脉注射的负担和高成本。本文描述了在阿胡斯中安全停止依库珠单抗治疗试验的设计，该试验旨在确定安全性监测方案（包括对复发患者重新引入依库珠单抗）是否可能是阿胡斯患者的安全替代治疗策略。这是一项多中心、非随机、开放标签的艾库组单抗停药研究，使用贝叶斯因子单组设计连续监测血栓性微血管病相关严重不良事件。将招募30名患者退出依库珠单抗治疗，并定期进行血液和尿液检查，持续24个月，以监测疾病活动。如果复发，将在出现后24小时内重新开始治疗。20名患者将继续接受治疗，仅填写卫生经济学问卷。一项嵌入式定性研究将探讨与会者的意见。一个有利的伦理意见和批准获得了东北泰恩&穿南研究伦理委员会。结果将通过同行评审的文章和会议演讲传播。EudraCT编号：2017-003916-37和ISRCTN编号：ISRCTN 17503205。
Atypical haemolytic uraemic syndrome (aHUS) is a rare, life-threatening disease caused by excessive activation of part of the immune system called complement. Eculizumab is an effective treatment, controlling aHUS in 90% of patients. Due to the risk of relapse, lifelong treatment is currently recommended. Eculizumab treatment is not without problems, foremost being the risk of severe meningococcal infection, the burden of biweekly intravenous injections and the high cost. This paper describes the design of the Stopping Eculizumab Treatment Safely in aHUS trial that aims to establish whether a safety monitoring protocol, including the reintroduction of eculizumab for those who relapse, could be a safe, alternative treatment strategy for patients with aHUS. This is a multicentre, non-randomised, open-label study of eculizumab withdrawal with continuous monitoring of thrombotic microangiopathy-related serious adverse events using the Bayes factor single-arm design. 30 patients will be recruited to withdraw from eculizumab and have regular blood and urine tests for 24 months, to monitor for disease activity. If relapse occurs, treatment will be restarted within 24 hours of presentation. 20 patients will remain on treatment and complete health economic questionnaires only. An embedded qualitative study will explore the views of participants. A favourable ethical opinion and approval was obtained from the North East-Tyne & Wear South Research Ethics Committee. Outcomes will be disseminated via peer-reviewed articles and conference presentations. EudraCT number: 2017-003916-37 and ISRCTN number: ISRCTN17503205.
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影响因子：
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作者：
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通讯作者：
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