Operating Properties of Disease Activity Indices in Pediatric Inflammatory Bowel Disease: A Systematic Review

Ruben J Colman; Virginia Solitano; John K MacDonald; Christopher Ma; Anne M Griffiths; Vipul Jairath; Eileen Crowley

doi:10.1093/ibd/izae060

Operating Properties of Disease Activity Indices in Pediatric Inflammatory Bowel Disease: A Systematic Review

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

Authors

Ruben J Colman¹, Virginia Solitano^{2

3

4}, John K MacDonald⁴, Christopher Ma^{4

5

6}, Anne M Griffiths⁷, Vipul Jairath^{2

4

8}, Eileen Crowley^{2

9}

Affiliations

¹ Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Stanford University School of Medicine, USA.
² Department of Medicine, Division of Gastroenterology, Schulich School of Medicine, Western University, Canada.
³ Gastroenterology and Endoscopy, IRCCS Ospedale San Raffaele and University Vita-Salute San Raffaele, Milan, Italy.
⁴ Alimentiv Inc, London, ON, Canada.
⁵ Division of Gastroenterology and Hepatology, Department of Medicine, University of Calgary, Calgary, AB, Canada.
⁶ Department of Community Health Sciences, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
⁷ Division of Gastroenterology, Hepatology & Nutrition, Department of Pediatrics and IBD Centre, SickKids Hospital, University of Toronto, Toronto, ON, Canada.
⁸ Department of Epidemiology and Biostatistics, Western University, London, ON, Canada.
⁹ Department of Pediatrics, Division of Pediatric Gastroenterology, Children's Hospital Western Ontario, Western University, London Health Sciences Centre, London, Ontario, Canada.

PMID: 38547511
DOI: 10.1093/ibd/izae060

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.

Keywords: children; disease activity indices; operating properties; outcomes; quality of life.

Plain language summary

The operating properties of pediatric inflammatory bowel disease clinical trial end points varied widely. These results highlight the need for further validation and development of novel indices in this population.