Siegel Corey A, Rai Victoria, Outtier An, Vermeire Severine, Law Cindy C W, Sands Bruce E, Abdulhamid Asim, Gearry Richard, McGuire Josh, Lindsay James O, Panaccione Remo, Schweistein Hagai, Dotan Iris, Scarallo Luca, Griffiths Anne, Dubinsky Marla C
Inflammatory Bowel Disease Program, Section of Gastroenterology and Hepatology, Dartmouth Hitchcock Medical Center, Lebanon, NH.
Department of Cellular and Molecular Physiology, Yale University, New Haven, CT.
J Crohns Colitis. 2024 Jun 22. doi: 10.1093/ecco-jcc/jjae097.
Clinical trial recruitment for patients with inflammatory bowel disease (IBD) has become more challenging over time. We aimed to develop recommendations for broadening IBD clinical trial eligibility to improve the inclusion of a more representative patient population in a more efficient timeline.
We applied the RAND/UCLA Appropriateness Method focused on broadening IBD clinical trial eligibility. A literature review was performed for 7 domains, each representing a different area related to trial recruitment. Based on these domains, 32 statements were developed. A questionnaire was sent to IBD specialists to anonymously vote on each statement with regards to its appropriateness and feasibility. After the first round of voting, participants met for a moderated discussion to review all statements. At the end of the discussion a second round of anonymous voting led to the final recommendations.
The final round of voting resulted in 26 statements. All were rated as feasible and 25 of 26 rated as appropriate. Recommendations generally are to be more inclusive of complicated disease phenotypes, more liberal around safety criteria, to recognize the importance of non-invasive imaging and biomarkers, to minimize the washout period and to not enforce a minimum or maximum number of prior medications, to allow a recently recorded colonoscopy to count as a baseline study, and to be less restrictive of age.
Recommendations to broaden clinical trial eligibility were found to be both appropriate and feasible with a high degree of agreement amongst an international group of IBD specialists.
随着时间的推移，炎症性肠病（IBD）患者的临床试验招募变得更具挑战性。我们旨在制定相关建议，以扩大IBD临床试验的入选标准，从而在更有效的时间范围内纳入更具代表性的患者群体。
我们应用了兰德/加州大学洛杉矶分校适宜性方法，重点是扩大IBD临床试验的入选标准。针对7个领域进行了文献综述，每个领域代表与试验招募相关的不同方面。基于这些领域，制定了32条陈述。向IBD专家发送了一份问卷，让他们就每条陈述的适宜性和可行性进行匿名投票。第一轮投票后，参与者进行了一次有主持人的讨论，以审查所有陈述。讨论结束时，第二轮匿名投票产生了最终建议。
最后一轮投票产生了26条陈述。所有陈述均被评为可行，26条中有25条被评为适宜。建议通常包括更全面地纳入复杂的疾病表型，在安全标准方面更加宽松，认识到非侵入性成像和生物标志物的重要性，尽量缩短洗脱期，不强制规定既往用药的最小或最大数量，允许最近记录的结肠镜检查作为基线研究，以及放宽对年龄的限制。
国际IBD专家小组高度一致地认为，扩大临床试验入选标准的建议既适宜又可行。
