Patient stratification and therapy in atypical haemolytic uraemic syndrome (aHUS)
Patient stratification and therapy in atypical haemolytic uraemic syndrome (aHUS)
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DOI：
10.1016/j.imbio.2015.05.002
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发表时间：
2016-06-01
期刊：
影响因子：
2.8
通讯作者：
Goodship, Timothy H. J.
中科院分区：
文献类型：
--
作者：
Wong, Edwin;Challis, Rachel;Goodship, Timothy H. J.
大约50%的aHUS患者有潜在的遗传和/或获得性补体异常，这容易导致替代途径的过度激活。因此，使用补体抑制剂如eculizumab治疗aHUS是合乎逻辑的。轶事报道和随后的开放标签试验证明了eculizumab治疗aHUS的疗效，并获得了FDA和EMA的批准。2013年，英国国家医疗服务体系为aHUS建立了临时国家服务，包括为新患者和接受移植的患者提供eculizumab资金。NICE的指导现在也建议eculizumab在专家中心的协调下在英国的NHS内获得资助。该队列的调查和治疗反应为患者分层提供了独特的资源。(C) 2015 Elsevier GmbH版权所有。
Approximately 50% of aHUS patients have an underlying inherited and/or acquired abnormality of complement which predisposes to excessive activation of the alternative pathway. Use of complement inhibitors such as eculizumab to treat aHUS is therefore logical. Anecdotal reports and subsequent open-label trials demonstrated the efficacy of eculizumab in aHUS leading to approval by both the FDA and EMA. NHS England established in 2013 an interim national service for aHUS including funding for eculizumab for both new patients and those undergoing transplantation. NICE guidance now also recommends eculizumab for funding within the NHS in England under the coordination of an expert centre. The investigation and response to treatment in this cohort provides a unique resource for patient stratification. (C) 2015 Elsevier GmbH. All rights reserved.