Inflamm Bowel Dis. 2024 Mar 28:izae060.doi: 10.1093/ibd/izae060. Online ahead of print.

Ruben J Colman ¹, Virginia Solitano ^2 3 4, John K MacDonald ⁴, Christopher Ma ^4 5 6, Anne M Griffiths ⁷, Vipul Jairath ^2 4 8, Eileen Crowley ^2 9

Author information

¹Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Stanford University School of Medicine, USA.

²Department of Medicine, Division of Gastroenterology, Schulich School of Medicine, Western University, Canada.

³Gastroenterology and Endoscopy, IRCCS Ospedale San Raffaele and University Vita-Salute San Raffaele, Milan, Italy.

⁴Alimentiv Inc, London, ON, Canada.

⁵Division of Gastroenterology and Hepatology, Department of Medicine, University of Calgary, Calgary, AB, Canada.

⁶Department of Community Health Sciences, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.

⁷Division of Gastroenterology, Hepatology & Nutrition, Department of Pediatrics and IBD Centre, SickKids Hospital, University of Toronto, Toronto, ON, Canada.

⁸Department of Epidemiology and Biostatistics, Western University, London, ON, Canada.

⁹Department of Pediatrics, Division of Pediatric Gastroenterology, Children's Hospital Western Ontario, Western University, London Health Sciences Centre, London, Ontario, Canada.

Abstract

Background: Accurate, reliable, and responsive disease activity indices are important to streamline drug approval and treatment modalities for pediatric inflammatory bowel disease (pIBD). We aimed to identify all scoring indices used in pIBD randomized controlled trials (RCTs) and to evaluate their operating properties.

Methods: MEDLINE, EMBASE, and CENTRAL were searched on December 6, 2022, to identify studies evaluating clinical, endoscopic, imaging, or patient-reported outcome measures (PROMs) in pIBD including Crohn's disease (CD) and ulcerative colitis (UC). Validity, reliability, responsiveness, and feasibility were summarized.

Results: Seventy RCTs evaluating pIBD indices were identified. Forty-one studies reported on the operating properties of 14 eligible indices (n = 9 CD, n = 5 UC). The Pediatric Crohn's Disease Activity Index (PCDAI) varied widely in terms of validity and reliability and was less feasible overall. In contrast, the Mucosal Inflammation Noninvasive Index, which includes fecal calprotectin, had better operating properties than the PCDAI. The Simplified Endoscopic Mucosal Assessment of Crohn's Disease appears more feasible and had similar operating properties than the longer Simple Endoscopic Score for Crohn's Disease. The Pediatric Ulcerative Colitis Activity Index was feasible, valid, and reliable, but responsiveness needs to be evaluated further. The Endoscopic Mayo score and the Ulcerative Colitis Endoscopic Index of Severity were reliable, but validity and responsiveness need to be evaluated further. Imaging and PROMs/quality of life indices need further evaluation.

Conclusions: The operating properties of pIBD clinical trial end points varied widely. These results highlight the need for further validation and development of novel indices.