文摘
摘要目的:评估的可行性和实用程序管理不良事件单元(AEU)作为衡量治疗副反应(AE)负担在重症肌无力(MG)诊所。
背景:我们开发了一个病人,医生加权共识单位,类似于货币(美元),称为AEU,根据不良事件的常见术语标准的优势(CTCAE)版本4。1、2不同于以往的措施,AEU量化AE负担,独立于任何疾病或治疗,为了便于比较不同药物之间。收集MG疾病的特定结果指标是标准的做法在许多MG诊所提供更精确的理解MG病人的状态。3 - 5然而,目前MG指标没有量化治疗AE负担。我们预料AEU分数将提供新颖的信息更好的通知个别病人治疗决策和治疗MG种群的范例。
设计/方法:MG-ADL, MG-QOL15r, MG-Composite通常管理病人在佛蒙特大学(UVM)重症肌无力临床访问。在这项研究中,AEU分数也被从MG患者在获得人在UVM和远程医疗。AEU政府将被认为是可行的,如果完成比例等于或优于MG-ADL完成6个月期间。6、7参与的医生和病人也被调查了AEU的满意程度和效用。我们也比较时间管理AEU和其他MG度量,并评估如果AEU分数与决策改变治疗。
结果:前瞻性的分析AEU UVM神经肌肉诊所正在进行。
结论:本研究将数据管理的可行性和效用AEU MG患者在常规临床访问一个毫克专科诊所。最后将呈现研究结果。
披露:Hehir博士已经收到个人薪酬在5000 - 9999美元的范围为Argenx担任顾问。Hehir博士已经收到个人薪酬在10000 - 49999美元的范围为Alexion担任顾问。Hehir博士已经收到个人薪酬在500 - 4999美元的范围为CSL贝林担任顾问。马克Conaway没有披露。圣Sauveur女士没有披露。科尔布博士已经收到个人薪酬在500 - 4999美元的范围作为鲍鱼医疗顾问服务。科尔布博士已经收到个人薪酬在500 - 4999美元的范围为解除疗法作为顾问。科尔布博士已经收到个人薪酬在500 - 4999美元的范围为alexion担任顾问。科尔布博士已经收到个人薪酬在500 - 4999美元的范围为锁法作为专家证人。科尔布博士已经收到个人薪酬在500 - 4999美元的范围作为走律师事务所的专家证人。 Dr. Kolb has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for lambert coffin. Dr. Kolb has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for bma law firm. Dr. Kolb has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for ralston, pope, diehl. The institution of Dr. Kolb has received research support from National Cancer Institute. Dr. Waheed has nothing to disclose. Dr. McNeish has nothing to disclose. Dr. Tweedy has nothing to disclose. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Accordant Health Services. Dr. Sanders has received personal compensation in the range of $0-$499 for serving as a Consultant for Cabaletta BIO. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kashiv Biosciences. Dr. Sanders has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Momenta Pharmaceuticals. Dr. Sanders has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen RnD. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi-aventis. Dr. Sanders has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for VielaBio. Dr. Sanders has received publishing royalties from a publication relating to health care. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson and Johnson. Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving as a Consultant for Sarepta. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ARGENX US Inc. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle and Nerve. Dr. Narayanaswami has received stock or an ownership interest from Moderna. Dr. Narayanaswami has received stock or an ownership interest from Dr. Reddys laboratories. Dr. Narayanaswami has received stock or an ownership interest from Pfizer. Dr. Narayanaswami has received stock or an ownership interest from Viatris. Dr. Narayanaswami has received stock or an ownership interest from Doximity. The institution of Dr. Narayanaswami has received research support from PCORI. The institution of Dr. Narayanaswami has received research support from Johnson and Johnson. The institution of Dr. Narayanaswami has received research support from Alexion. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Member, Pharmacy and Therapeutics Committee with Blue Cross Blue Shield of MA. Dr. Narayanaswami has a non-compensated relationship as a Member, Medical and Scientific Advisory Board with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities. Dr. Burns has nothing to disclose.
