文摘
摘要目的:确定临床有意义的数字生物标志物(dBMKs)旨在追踪在广义重症肌无力患者症状和体征(gMG)和准备一个软件的开发医疗设备对病人的无监督数码自我评估。
背景:广义重症肌无力(gMG)的特点是波动的表现和异质性在疲劳和肌肉组织的弱点。文献确定了一个缺乏客观的自我评估测量工具的gMG症状外办公室访问,导致不同的医生和病人之间的感知的影响疾病。
设计/方法:我们使用双钻石感兴趣的方法来识别dBMKs gMG病人和评估他们的相关性的临床医生通过定性访谈。未满足的需求和dBMKs概念中优先技术概念验证(PoC)的研究。数码设备的可接受性评估与prototype-supported采访。
结果:采访27例(法国、美国)证实dBMKs测量gMG-related神经功能的可行性。跟踪症状都感兴趣。9国际MG专家评估的gMG症候学很感兴趣dBMKs办公室访问之间的临床意义。PoC的研究在健康受试者,在不同条件下执行,确认的可行性分析智能手机的传感器收集的原始数据。5数字积极措施受到临床黄金标准和依赖智能手机的传感器选择与他们相关的dBMKs(下垂,呼吸,构音障碍,上、下肢功能)。Patient-reported结果措施(日常活动、疼痛、睡眠、抑郁、生活质量)将合并e-questionnaires。
结论:我们认为需要一个无监督病人自我评估数字解决方案监控症状外办公室访问。ME&MG发展,2023年智能手机解决方案,将开始。它旨在告知和改善病人和他们的护理团队之间的沟通。国际多中心研究比较ME&MG包括定量重症肌无力临床黄金标准分数,将部署。
披露:帕斯卡Laforet没有披露。大卫Orlikowski没有披露。教授尼古拉斯没有披露。Prigent已收到教授研究的机构VLM——病人lysosomial疾病协会的支持。Prigent已收到教授研究的机构支持ADEP援助。berl博士已经收到个人薪酬在10000 - 49999美元的范围为Adscientiam担任顾问。berl博士已经收到个人薪酬在10000 - 49999美元的范围为罗氏公司担任顾问。Ghislain N 'Dah-Sekou没有披露。Gorin博士已经收到个人赔偿作为广告Scientiam的员工。Moreira博士已经收到个人赔偿作为广告Scientiam的员工。 Mrs. Narcy has received personal compensation for serving as an employee of Ad Scientiam. Dr. Plaud has received personal compensation for serving as an employee of Ad Scientiam. Mrs. Touré Cuq has received personal compensation for serving as an employee of Ad Scientiam . Dr. Keller has received personal compensation for serving as an employee of Alexion. Dr. Keller has stock in Alexion, Roche, Novartis, Celgene, Abbvie, Merck, Ionis, Sanofie, Organon, MSD. Mr. Werneburg has received personal compensation for serving as an employee of Alexion. Dr. Aras has received personal compensation for serving as an employee of Alexion Pharmaceuticals Inc. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. The institution of Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Arrowhead. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UniQure. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alector. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Benatar has received intellectual property interests from a discovery or technology relating to health care. Dr. Benatar has received intellectual property interests from a discovery or technology relating to health care.
