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Updates in Clinical, Endoscopic, and Histologic Composite and Co-primary Endpoints for Clinical Trials in Inflammatory Bowel Disease

  • Irritable Bowel Diseases (G Lichtenstein, Section Editor)
  • Published:
Current Treatment Options in Gastroenterology Aims and scope Submit manuscript

Abstract

Purpose of review

The evolution of endpoints used to assess disease activity and treatment efficacy in inflammatory bowel disease (IBD) has accompanied changes in clinical trial methodology and the therapeutic landscape.

Recent findings

Composite and co-primary endpoints for patient-reported outcomes (without physician’s global assessment) and endoscopic improvement (excluding friability) are used to define clinical remission and clinical response in contemporary clinical trials for ulcerative colitis and Crohn’s disease. Histologic-endoscopic mucosal healing has emerged as a therapeutic goal and validated histological indexes are increasingly integrated into clinical trials. Central reading of endoscopy is recognized as the gold standard for endoscopic assessment in clinical trials to minimize observer bias and inter-observer variability.

Summary

Determination of treatment efficacy in IBD has evolved to incorporate both patient-reported outcome measures and central reading of endoscopy +/- histology as composite endpoints in contemporary clinical trials.

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References and Recommended Reading

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Authors and Affiliations

  1. Division of Gastroenterology, University of California, San Diego, La Jolla, CA, USA

    Chung Sang Tse MD & William J. Sandborn MD

  2. Division of Gastroenterology, Department of Medicine, Western University, London, ON, Canada

    Vipul Jairath MBChB, DPhil, MRCP, FRCPC

  3. Alimentiv Inc. (formerly Robarts Clinical Trials Inc.), London, ON, Canada

    Vipul Jairath MBChB, DPhil, MRCP, FRCPC

  4. Departments of Epidemiology and Biostatistics, Western University, London, ON, Canada

    Vipul Jairath MBChB, DPhil, MRCP, FRCPC

  5. Alimentiv Inc., London, ON, Canada

    Brian G. Feagan MD, FRCPC

  6. Division of Gastroenterology, University of Western Ontario, London, ON, Canada

    Brian G. Feagan MD, FRCPC

  7. Department of Epidemiology and Biostatistics, University of Western Ontario, London, ON, Canada

    Brian G. Feagan MD, FRCPC

  8. Department of Medicine, University of Western Ontario, London, ON, Canada

    Brian G. Feagan MD, FRCPC

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  2. Vipul Jairath MBChB, DPhil, MRCP, FRCPC
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  3. Brian G. Feagan MD, FRCPC
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  4. William J. Sandborn MD
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Correspondence to Chung Sang Tse MD.

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Conflict of Interest

Chung Sang Tse has no conflicts to disclose. Vipul Jairath has received consulting fees from AbbVie, Eli Lilly, GlaxoSmithKline, Arena Pharmaceuticals, Genetech, Pendopharm, Sandoz, Merck, Takeda, Janssen, Robarts Clinical Trials, Topivert, Celltrion; and speaker’s fees from Takeda, Janssen, Shire, Ferring, AbbVie, and Pfizer. Brian Feagan has received grant/research support from Millennium Pharmaceuticals, Merck, Tillotts Pharma AG, AbbVie, Novartis Pharmaceuticals, Centocor Inc, Elan/Biogen, UCB Pharma, Bristol-Myers Squibb, Genentech, ActoGenix, and Wyeth Pharmaceuticals Inc.; consulting fees from Millennium Pharmaceuticals, Merck, Centocor Inc, Elan/Biogen, Janssen-Ortho, Teva Pharmaceuticals, Bristol-Myers Squibb, Celgene, UCB Pharma, AbbVie, Astra Zeneca, Serono, Genentech, Tillotts Pharma AG, Unity Pharmaceuticals, Albireo Pharma, Given Imaging Inc., Salix Pharmaceuticals, Novonordisk, GSK, Actogenix, Prometheus Therapeutics and Diagnostics, Athersys, Axcan, Gilead, Pfizer, Shire, Wyeth, Zealand Pharma, Zyngenia, GiCare Pharma Inc., and Sigmoid Pharma; and speaker’s fees from UCB, AbbVie, and J&J/Janssen. William Sandborn has received research grant support from Atlantic Healthcare Limited, Amgen, Genentech, Gilead Sciences, AbbVie, Janssen, Takeda, Lilly, and Celgene/Receptos; consulting fees from Abbvie, Allergan, Amgen, Boehringer Ingelheim, Celgene, Conatus, Cosmo, Escalier Biosciences, Ferring, Genentech, Gilead, Gossamer Bio, Janssen, Lilly, Miraca Life Sciences, Nivalis Therapeutics, Novartis Nutrition Science Partners, Oppilan Pharma, Otsuka, Paul Hastings, Pfizer, Precision IBD, Progenity, Prometheus Laboratories, Ritter Pharmaceuticals, Robarts Clinical Trials (owned by Health Academic Research Trust or HART), Salix, Shire, Seres Therapeutics, Sigmoid Biotechnologies, Takeda, Tigenix, Tillotts Pharma, UCB Pharma, and Vivelix; and stock options from Ritter Pharmaceuticals, Oppilan Pharma, Escalier Biosciences, Gossamer Bio, Precision IBD, and Progenity.

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Tse, C.S., Jairath, V., Feagan, B.G. et al. Updates in Clinical, Endoscopic, and Histologic Composite and Co-primary Endpoints for Clinical Trials in Inflammatory Bowel Disease. Curr Treat Options Gastro 19, 608–627 (2021). https://doi.org/10.1007/s11938-021-00362-x

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