Safety and effectiveness of eculizumab for adult patients with atypical hemolytic-uremic syndrome in Japan: interim analysis of post-marketing surveillance.
Safety and effectiveness of eculizumab for adult patients with atypical hemolytic-uremic syndrome in Japan: interim analysis of post-marketing surveillance.
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DOI：
10.1007/s10157-018-1609-8
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发表时间：
2019-01
影响因子：
2.3
通讯作者：
Okada H
中科院分区：
文献类型：
--
作者：
Kato H;Miyakawa Y;Hidaka Y;Inoue N;Ito S;Kagami S;Kaname S;Matsumoto M;Mizuno M;Matsuda T;Shimono A;Maruyama S;Fujimura Y;Nangaku M;Okada H
自2013年以来，依库珠单抗已在日本用于治疗非典型溶血性尿毒症综合征（阿胡斯）。为了评估依库珠单抗在现实生活中成人阿胡斯患者中的安全性和有效性，我们对日本法规要求的上市后监测进行了中期分析。本研究入组了根据2013年发表的日本临床指南诊断为TMA（不包括产滋贺毒素大肠埃希菌-HUS或血栓性血小板减少性紫癜）并接受依库珠单抗治疗的任何患者作为入选标准。虽然在2016年修订的指南中，术语阿胡斯被重新定义为仅表示补体介导的HUS，但包括其他原因引起的TMA（继发性TMA）患者。在6个月、12个月和此后每年评价患者结局和安全性。入组了33例阿胡斯患者和27例继发性TMA患者。阿胡斯的中位治疗持续时间为24周。18例阿胡斯患者中11例（61.1%）检测到补体基因变异。在具有可用基线数据的29名阿胡斯患者中，血小板计数（PLT）、乳酸脱氢酶和血清肌酐（SCr）在依库珠单抗开始后1个月内改善。TMA无事件状态、TMA完全缓解、PLT正常化和SCr降低的患者比例分别为67.9%（19/28）、27.8%（5/18）、56.5%（13/23）和57.1%（16/28）。在阿胡斯（13/33例患者）和继发性TMA（6/27例患者）患者中分别观察到33例和11例不良反应。该中期分析证实了依库珠单抗在真实世界环境中用于日本成人阿胡斯患者的可接受的安全性特征和有效性。本文的在线版本（10.1007/s10157-018-1609-8）包含补充材料，可供授权用户使用。
Eculizumab has been available for the treatment of atypical hemolytic–uremic syndrome (aHUS) in Japan since 2013. To assess safety and effectiveness of eculizumab in adult aHUS patients in the real-life setting, we performed interim analysis of a post-marketing surveillance mandated by Japanese regulations. This study enrolled any patient who was diagnosed with TMA excluding Shiga toxin-producing Escherichia coli-HUS or thrombotic thrombocytopenic purpura based on Japanese clinical guide published in 2013 as inclusion criteria and treated with eculizumab. Although the term aHUS was redefined to denote only complement-mediated HUS in the guide revised in 2016, the patients with TMA caused by other causes (secondary TMA) were included. Patient outcomes and safety were evaluated at 6 months, 12 months, and annually thereafter. Thirty-three patients with aHUS and 27 patients with secondary TMA were enrolled. Median treatment duration of aHUS was 24weeks. Complement genes variants were detected in 11 of 18 patients with aHUS (61.1%). Among the 29 aHUS patients with available baseline data, platelet count (PLT), lactic dehydrogenase and serum creatinine (SCr) improved within 1-month after eculizumab initiation. TMA event-free status, complete TMA response, PLT normalization, and SCr decrease were achieved in 67.9% (19/28), 27.8% (5/18), 56.5% (13/23), and 57.1% (16/28) of patients, respectively. Thirty-three and 11 adverse reactions were observed in patients with aHUS (13/33 patients) and secondary TMA (6/27 patients), respectively. This interim analysis confirmed the acceptable safety profile and effectiveness of eculizumab for Japanese adult aHUS patients in real-world settings. The online version of this article (10.1007/s10157-018-1609-8) contains supplementary material, which is available to authorized users.
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