文摘
客观的NA。
背景终端补C5抑制剂eculizumab批准在日本的预防aquaporin-4抗体阳性(AQP4 +)视neuromyelitis谱系障碍(NMOSD)复发和接受强制性的上市后监测(PMS)的实际使用。
设计/方法这项目经理临时分析评估eculizumab的安全性和有效性在日本病人AQP4 + NMOSD病人从批准(2019年11月)临时数据截止(2021年4月1日)。
结果患者数据截止79 eculizumab注册在经前综合症。数据可供20病人提供同意发表,构成了安全数据集;三个病人的第三阶段预防研究被排除在数据集有效性。前两个病人停止数据截止由于医生或病人的决定(分别)。的有效性的数据集,16/17(94%)例患者女性,意味着疾病持续时间为9.2年(标准偏差(SD) 7.5年,范围0.3 - -23.8年)和平均eculizumab起始年龄为48.6岁(SD 11.9年,27 - 77年)。2年eculizumab之前,患者复发,12/17和6/17 = 2复发。重要的是,没有复发报告的有效性数据集(10.7 (PY)治疗白细胞数量);这优于0.65 / PY前2年复发率(34.0 PY)治疗。三个不良事件(不适、眼睑水肿、红斑)和一个严重不良事件(尿路感染(UTI))中观察到的三个病人安全数据集:眼睑水肿和红斑(一个病人)被认为治疗相关的;低迷和泌尿道感染被认为与治疗无关。脑膜炎球菌感染被报道,安全与防止结果是一致的。
结论在真实的环境中,首次eculizumab有效预防复发,在日本AQP4 + NMOSD患者耐受性良好,符合其疗效和安全性在全球第三阶段预防研究。
脚注
披露:中岛美嘉博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会Alexion。中岛美嘉博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会生原体。中岛美嘉博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会诺华。中岛美嘉博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会Chugai。中岛美嘉博士已经收到个人薪酬在10000 - 49999美元的范围服务扬声器Alexion管理局。中岛美嘉博士已经收到个人薪酬在10000 - 49999美元的范围在生原体的演讲人。中岛美嘉博士已经收到个人薪酬在10000 - 49999美元的范围在诺华的演讲人。中岛美嘉博士已经收到个人薪酬在10000 - 49999美元的范围服务扬声器Chugai管理局。中岛美嘉博士的机构已收到从LSI Medience研究支持。Hoshino博士没有披露。 Ms. Okamura has received personal compensation for serving as an employee of Alexion Pharma GK. Ms. Okamura has received stock or an ownership interest from Astra Zeneca Plc. Mr. Kikui has received personal compensation for serving as an employee of Alexion Pharma GK. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck Boipharma. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Japan Tobacco. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubishi-Tanabe. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Fujihara has received research support from Ministry of Health, Welfare and Labor of Japan. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Novartis. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving as a speaker, chair, etc with Biogen. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Mitsubishi-Tanabe. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Chugai. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Alexion. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a chair, speaker with Asahi Kasei Medical. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker with Eisai. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker, chair with Teijin. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving as a speaker with Roche.
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