@Article{信息:doi 10.2196 / / jmir。1643年，作者=“Frost, Jeana和Okun, Sally和Vaughan, Timothy和Heywood, James和Wicks, Paul”，标题=“患者报告的结果作为超适应症处方的证据来源:来自PatientsLikeMe的数据分析”，期刊=“J Med Internet Res”，年=“2011”，月=“1”，日=“21”，卷=“13”，数=“1”，页=“e6”，关键词=“超适应症;互联网;研究;病人平台;方法;背景:评估一种现有药物的新用途既昂贵又耗时。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 is a tool for patients, researchers, and caregivers (currently 82,000 members across 11 condition-based communities) that helps users make treatment decisions, manage symptoms, and improve outcomes. Members enter demographic information, longitudinal treatment, symptoms, outcome data, and treatment evaluations. These are reflected back as longitudinal health profiles and aggregated reports. Over the last 3 years, patients have entered treatment histories and evaluations on thousands of medical products. These data may aid in evaluating the effectiveness and safety of some treatments more efficiently and over a longer period of time course than is feasible through traditional trials. 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. ", issn="1438-8871", doi="10.2196/jmir.1643", url="//www.mybigtv.com/2011/1/e6/", url="https://doi.org/10.2196/jmir.1643", url="http://www.ncbi.nlm.nih.gov/pubmed/21252034" }