Burton-Murray Helen, Guadagnoli Livia, Kamp Kendra, Trindade Inês A, Powell Lynda H, Simrén Magnus, Ljótsson Brjánn, Keefer Laurie
Division of Gastroenterology, Massachusetts General Hospital, Boston, Massachusetts, USA.
Harvard Medical School, Boston, Massachusetts, USA.
Aliment Pharmacol Ther. 2025 Mar;61(5):787-802. doi: 10.1111/apt.18482. Epub 2025 Jan 21.
BACKGROUND: Brain-gut behaviour therapies (BGBT) have gained widespread acceptance as therapeutic modalities for the management of disorders of gut-brain interaction (DGBI). However, existing treatment evaluation methods in the medical field fail to capture the specific elements of scientific rigour unique to behavioural trial evaluation. AIMS: To offer the first consensus on the development and testing of BGBT in DGBI. METHODS: An international, interdisciplinary team of experts developed a consensus statement heavily informed by best practice recommendations for behavioural clinical trials for chronic diseases, organised by a selected treatment development model. RESULTS: We suggest an existing behavioural treatment development model that has an iterative progression aligned with the drug development model with nuances specific to BGBT. We describe the iterative phases through initial discovery and experimental work, assembly of a mechanistic pathway and candidate treatment components, treatment refinement and optimisation, initial proof-of-concept, feasibility of clinical trials and, finally, confirmatory efficacy and effectiveness testing. We delineate recommendations for and provide examples that lend themselves to gastroenterologists planning to develop or test BGBT, reviewing proposals for or results from BGBT studies or writing management guidelines for DGBI. CONCLUSIONS: This working team report facilitates a shared understanding of the elements of scientific rigour necessary for BGBT development and could support future standards on which BGBT are evaluated in gastroenterology.
背景:脑-肠行为疗法(BGBT)作为治疗肠-脑相互作用障碍(DGBI)的治疗方式已获得广泛认可。然而,医学领域现有的治疗评估方法未能涵盖行为试验评估所特有的科学严谨性的具体要素。 目的:就DGBI中BGBT的开发和测试达成首个共识。 方法:一个国际跨学科专家团队制定了一份共识声明,该声明主要参考了针对慢性病行为临床试验的最佳实践建议,并由选定的治疗开发模型进行组织。 结果:我们提出了一种现有的行为治疗开发模型,该模型具有与药物开发模型一致的迭代进展,并具有BGBT特有的细微差别。我们描述了迭代阶段,包括初始发现和实验工作、机制途径和候选治疗组件的组装、治疗改进和优化、初步概念验证、临床试验的可行性,以及最终的验证性疗效和有效性测试。我们为计划开发或测试BGBT的胃肠病学家、审查BGBT研究的提案或结果或撰写DGBI管理指南的人员划定了建议并提供了示例。 结论:这份工作团队报告有助于对BGBT开发所需的科学严谨性要素形成共同理解,并可为未来胃肠病学中评估BGBT的标准提供支持。
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