杂志文章%@ 1448 -8871 I Gunther Eysenbach %V 13% N 1 %P 6 %T患者报告的结果作为超适应症处方的证据来源:分析数据从PatientsLikeMe %霜,Jeana %奥肯,莎莉%沃恩,盖%海伍德,詹姆斯%威克斯,保罗% + VU阿姆斯特丹,KankerNL, 1081年德Boelelaan,阿姆斯特丹,1081高压,荷兰,31日20 598 8636,jeana.frost@gmail.com % K标示外% K互联网% K研究% K病人平台% K方法% K在线社区% D原始论文7 21.01.2011 % 9 2011% % J J互联网Res % G英语% X背景:评估一个新的使用现有的药物可以是昂贵和费时的。医生和病人往往都必须依靠自己个人层面的经验来为临床实践提供信息，而这只能产生轶事和非结构化的数据。虽然学术主导的临床试验偶尔会进行，以测试专利过期药物的适应症外使用，但这是相对罕见的。在这项工作中，我们探索了一个以患者为中心的在线研究平台如何通过高效聚合结构化的患者报告数据来补充传统试验，从而更丰富地了解医疗产品的后市场。PatientsLikeMe是一个为患者、研究人员和护理人员(目前有来自11个基于病情的社区的82,000名成员)提供帮助的工具，可以帮助用户做出治疗决定、管理症状和改善结果。成员输入人口统计信息、纵向治疗、症状、结果数据和治疗评估。这些反映为纵向健康概况和汇总报告。在过去的3年里，患者已经输入了数千种医疗产品的治疗历史和评估。这些数据可以帮助评估一些治疗的有效性和安全性，比传统试验更有效和更长的时间。 Objective: The objective of our study was to examine the illustrative cases of amitriptyline and modafinil – drugs commonly used off-label. Methods: We analyzed patient-reported treatment histories and drug evaluations for each drug, examining prevalence, treatment purpose, and evaluations of effectiveness, side effects, and burden. Results: There were 1948 treatment histories for modafinil and 1394 treatment reports for amitriptyline reported across five PatientsLikeMe communities (multiple sclerosis, Parkinson's disease, mood conditions, fibromyalgia/chronic fatigue syndrome, and amyotrophic lateral sclerosis). In these reports, the majority of members reported taking the drug for off-label uses. Only 34 of the 1755 (1%) reporting purpose used modafinil for an approved purpose (narcolepsy or sleep apnea). Only 104 out of 1197 members (9%) reported taking amitriptyline for its approved indication, depression. Members taking amitriptyline for off-label purposes rated the drug as more effective than those who were taking it for its approved indication. While dry mouth is a commonly reported side effect of amitriptyline for most patients, 88 of 220 (40%) of people with amyotrophic lateral sclerosis on the drug reported taking advantage of this side effect to treat their symptom of excess saliva. Conclusions: Patient-reported outcomes, like those entered within PatientsLikeMe, offer a unique real-time approach to understand utilization and performance of treatments across many conditions. These patient-reported data can provide a new source of evidence about secondary uses and potentially identify targets for treatments to be studied systematically in traditional efficacy trials. %M 21252034 %R 10.2196/jmir.1643 %U //www.mybigtv.com/2011/1/e6/ %U https://doi.org/10.2196/jmir.1643 %U http://www.ncbi.nlm.nih.gov/pubmed/21252034