Fecal Microbiome Transplant National Registry

项目概要 迅速积累的证据表明，改变肠道微生物群可以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）是首个成功的临床应用 这个概念。鉴于 CDI 发病率不断上升且缺乏针对复发性 CDI 的有效治疗方法，使用 尽管缺乏临床数据，并且存在肠道转移的理论上的风险，但 FMT 仍在迅速增长。 微生物群可能诱发慢性疾病。 FMT 在临床实践中的早期采用和扩展 绕过了标准的研究途径，并先于通常进行的大型随机对照试验 评估疗效并提供重要的短期和长期安全数据。为了克服这一不足 知识和经验，我们建议建立一个国家 FMT 登记处。该提案的目标是 出于以下目的从 FMT 捐赠者和接受者收集临床数据： 1) 评估短期和 长期安全； 2）收集美国实践的信息并评估其有效性 干预； 3）促进科学研究； 4) 帮助从业者和试验申办者满足 监管要求。该登记处将前瞻性地招募在整个地区接受 FMT 的患者。 美国有关 FMT 方法、FMT 适应症以及捐赠者和接受者基线数据的信息将在 从各个网站收集。 FMT 后将定期对患者进行长达 10 年的随访，以达到预先确定的效果 并自发报告不良事件和预定的有效性结果。注册表将 促进信号检测的统计分析、描述性数据的生成以及比较的进行 有效性研究。由 4,000 名 FMT 患者组成的前瞻性队列将提供发病率的精确估计 现实世界实践中的安全性和有效性终点，并排除具有高风险的不常见不良事件 信心。该注册表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 份粪便样本，为研究操纵提供了丰富的资源 人类肠道微生物群。美国胃肠病学协会 (AGA) 的领导及其 伙伴组织共同努力制定了该登记处的计划，这将是一个极其重要的 评估短期安全性、识别潜在长期后果、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，并评估潜在后果 操纵肠道微生物组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.