Division of Gastroenterology, Hepatology, and Nutrition, Transplant and Regenerative Center, Group for Improvement of Intestinal Function and Treatment, The Hospital for Sick Children, Toronto, Ontario, Canada.
Registry Data Analyst and Administrator, International Intestinal Rehabilitation and Transplant Association, The Transplantation Society, Montreal, Quebec, Canada.
Eur J Pediatr Surg. 2024 Apr;34(2):172-181. doi: 10.1055/a-2212-6874. Epub 2023 Nov 16.
Pediatric intestinal failure (IF) is a rare disease that represents an evolving field in pediatric gastroenterology and surgery. With only a limited number of multicenter collaborations, much of the research in pediatric IF is often confined to single-center reports with small sample sizes. This has resulted in challenges in data interpretation and left many knowledge gaps unanswered. Over the past two decades, five large multicenter collaborations, primarily from North America and Europe, have published their findings. Apart from one ongoing European adult and pediatric registry, these relatively large-scale efforts have been concluded.In 2018, the International Intestinal Failure Registry (IIFR) was initiated by the International Intestinal Rehabilitation and Transplant Association to continue these efforts and answer some of the knowledge gaps in pediatric IF. The IIFR goals are to prospectively assess the natural history of children diagnosed with IF and creating a worldwide platform to facilitate benchmarking and evidence-based interventions in pediatric IF. A pilot phase involving 204 enrolled patients was initiated in 2018 to assess the feasibility of an international IF registry and refine the study protocol and data collection forms. Following the successful completion of this phase, the current phase of the IIFR was launched in 2021. As of May 2023, the registry includes 362 prospectively followed children from 26 centers worldwide. This review provides an overview of the development, structure, and challenges of the IIFR, as well as the main findings from both the pilot and current phase.
儿科肠衰竭（IF）是一种罕见疾病，代表了儿科胃肠病学和外科学的一个不断发展的领域。由于只有有限的多中心合作，儿科 IF 的大部分研究通常局限于单中心报告，样本量较小。这导致了数据解释的挑战，并留下了许多未解答的知识空白。在过去的二十年中，五个主要来自北美和欧洲的大型多中心合作组织已经公布了他们的研究结果。除了一个正在进行的欧洲成人和儿科登记处外，这些相对大规模的努力已经结束。2018 年，国际肠衰竭登记处（IIFR）由国际肠康复和移植协会发起，旨在继续这些努力并回答儿科 IF 中的一些知识空白。IIFR 的目标是前瞻性评估诊断为 IF 的儿童的自然病史，并创建一个全球平台，以促进儿科 IF 的基准测试和基于证据的干预措施。2018 年启动了一个涉及 204 名入组患者的试点阶段，以评估国际 IF 登记处的可行性，并完善研究方案和数据收集表格。在成功完成这一阶段后，IIFR 的当前阶段于 2021 年启动。截至 2023 年 5 月，该登记处包括来自全球 26 个中心的 362 名前瞻性随访儿童。本文综述了 IIFR 的发展、结构和挑战，以及试点阶段和当前阶段的主要发现。
