CORE-IBD: A Multidisciplinary International Consensus Initiative to Develop a Core Outcome Set for Randomized Controlled Trials in Inflammatory Bowel Disease

Christopher Ma*, Jurij Hanzel, The CORE-IBD Collaborators, Remo Panaccione, William J. Sandborn, Geert R. D'Haens, Vineet Ahuja, Raja Atreya, Charles N. Bernstein, Peter Bossuyt, Brian Bressler, Robert V. Bryant, Benjamin Cohen, Jean Frederic Colombel, Silvio Danese, Axel Dignass, Marla C. Dubinsky, Phillip R. Fleshner, Richard B. Gearry, Stephen B. HanauerAilsa Hart, Paulo Gustavo Kotze, Torsten Kucharzik, Peter L. Lakatos, Rupert W. Leong, Fernando Magro, Julian Panés, Laurent Peyrin-Biroulet, Zhihua Ran, Miguel Regueiro, Siddharth Singh, Antonino Spinelli, A. Hillary Steinhart, Simon P. Travis, C. Janneke van der Woude, Bruce Yacyshyn, Takayuki Yamamoto, Matthieu Allez, Willem A. Bemelman, Amy L. Lightner, Edouard Louis, David T. Rubin, Ellen J. Scherl, Corey A. Siegel, Mark S. Silverberg, Severine Vermeire, Claire E. Parker, Stefanie C. McFarlane, Leonardo Guizzetti, Michelle I. Smith, Niels Vande Casteele

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

1 Citation (Scopus)

Abstract

Background & Aims: End points to determine the efficacy and safety of medical therapies for Crohn's disease (CD) and ulcerative colitis (UC) are evolving. Given the heterogeneity in current outcome measures, harmonizing end points in a core outcome set for randomized controlled trials is a priority for drug development in inflammatory bowel disease. Methods: Candidate outcome domains and outcome measures were generated from systematic literature reviews and patient engagement surveys and interviews. An iterative Delphi process was conducted to establish consensus: panelists anonymously voted on items using a 9-point Likert scale, and feedback was incorporated between rounds to refine statements. Consensus meetings were held to ratify the outcome domains and core outcome measures. Stakeholders were recruited internationally, and included gastroenterologists, colorectal surgeons, methodologists, and clinical trialists. Results: A total of 235 patients and 53 experts participated. Patient-reported outcomes, quality of life, endoscopy, biomarkers, and safety were considered core domains; histopathology was an additional domain for UC. In CD, there was consensus to use the 2-item patient-reported outcome (ie, abdominal pain and stool frequency), Crohn's Disease Activity Index, Simple Endoscopic Score for Crohn's Disease, C-reactive protein, fecal calprotectin, and co-primary end points of symptomatic remission and endoscopic response. In UC, there was consensus to use the 9-point Mayo Clinic Score, fecal urgency, Robarts Histopathology Index or Geboes Score, fecal calprotectin, and a composite primary end point including both symptomatic and endoscopic remission. Safety outcomes should be reported using the Medical Dictionary for Regulatory Activities. Conclusions: This multidisciplinary collaboration involving patients and clinical experts has produced the first core outcome set that can be applied to randomized controlled trials of CD and UC.

Original languageEnglish
Pages (from-to)950-964
Number of pages15
JournalGastroenterology
Volume163
Issue number4
DOIs
Publication statusPublished - 1 Oct 2022

Bibliographical note

Funding:
This study was supported by a Canadian Institutes for Health Research Fellowship award.

Publisher Copyright: © 2022 AGA Institute

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