Fecal Microbiome Transplant National Registry

项目摘要 迅速积累的证据表明，修改肠道微生物区系可能会促进健康或治疗疾病， 粪便微生物移植(FMT)治疗艰难梭菌感染(CDI)是首次成功的临床应用 对这个概念的理解。鉴于CDI的发病率不断上升，以及对复发的CDI缺乏有效的治疗方法，使用 尽管缺乏临床数据，尽管理论上肠道移植存在风险，但FMT仍在迅速增长。 微生物区系可能会引发慢性病。FMT在临床上的早期采用和推广已经 绕过了标准的研究途径，在大型随机对照试验之前进行，这些试验通常 评估疗效并提供重要的短期和长期安全数据。为了克服这一逆差， 根据我们的知识和经验，我们建议建立一个国家《禁产条约》登记册。这项提议的目标是 为以下目的从FMT捐赠者和接受者那里收集临床数据：1)评估短期和 长期安全；2)收集美国实践的信息，并评估 干预；3)促进科学调查；4)帮助从业者和试验发起人满足 监管要求。这一登记将前瞻性地登记在全国各地接受FMT的患者 美国将提供关于FMT方法、FMT适应症以及捐赠者和接受者的基线数据的信息 从每个站点收集的。患者将在FMT后定期接受长达10年的跟踪，以进行预先定义的 并自发报告不良事件和预定义的有效性结果。注册处将 促进对信号检测、描述性数据的生成和进行比较的统计分析 有效性研究。4000名FMT患者的前瞻性队列将提供对发病率的准确估计 在现实世界实践中的安全性和有效性终点，并排除不常见的不良事件 自信。这个登记处将链接到一个独立的生物库，该生物库将收集、存档和分析大约 来自捐赠者和接受者的3,000个粪便样本，为研究操纵提供了丰富的来源 人类的肠道微生物区系。美国胃肠病学会(AGA)的领导及其 伙伴组织已共同努力为这一登记册制定计划，这将是一个极其重要的 评估短期安全性、确定潜在的长期后果、确定有效性的重要工具 在现实世界的实践中，帮助标准化最佳FMT实践，并评估 操纵肠道微生物群对人类健康和疾病的影响。

Project Summary Rapidly accumulating evidence suggests that modifying gut microbiota may promote health or treat disease, with fecal microbiota transplantation (FMT) for C. difficile infection (CDI) being the first successful clinical application of this concept. Given the rising incidence of CDI and the lack of effective therapies for recurrent CDI, the use of FMT is growing rapidly, despite a paucity of clinical data and despite the theoretical risk that transfer of gut microbiota may induce chronic diseases. The early adoption and expansion of FMT in clinical practice has bypassed the standard investigatory pathway and preceded large randomized controlled trials that typically evaluate efficacy and contribute important short- and long-term safety data. In order to overcome this deficit in knowledge and experience, we propose to develop a national FMT registry. The goal of this proposal is to collect clinical data from both FMT donors and recipients for the following purposes: 1) To assess short- and long-term safety; 2) to gather information on practice in the U.S. and assess effectiveness of the intervention; 3) to promote scientific investigation; and 4) to aid practitioners and trial sponsors in satisfying regulatory requirements. This registry will prospectively enroll patients who undergo FMT at sites throughout the U.S. Information on FMT methodology, FMT indication, as well as baseline data on donors and recipients will be collected from each site. Patients will be followed at regular intervals for up to 10 years after FMT for pre-defined and spontaneously reported adverse events and for pre-defined outcomes of effectiveness. The registry will facilitate statistical analyses for signal detection, generation of descriptive data, and the conduct of comparative effectiveness studies. A prospective cohort of 4,000 FMT patients will provide precise estimates of the incidence of safety and effectiveness endpoints in real-world practice and rule out uncommon adverse events with high confidence. This registry will link to an independent biobank that will collect, archive, and analyze approximately 3,000 fecal samples from the donors as well as recipients, providing a rich source for studying manipulation of the gut microbiota in humans. The leadership of the American Gastroenterological Association (AGA) and its partner organizations have worked together to develop the plans for this registry, which will be an extremely important tool to assess short-term safety, identify potential long-term consequences, determine effectiveness in real-world practice, help standardize best FMT practice, and gauge the potential consequences of manipulating the gut microbiome in human health and disease.