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Development, validation, and evaluation of a pediatric ulcerative colitis activity index: a prospective multicenter
Dan Turner1, Anthony R Otley, David Mack
1Division of Gastroenterology, Hepatology and Nutrition, the Hospital for Sick Children, University of Toronto, Toronto, Canada.
Insights
A new noninvasive index, the Pediatric Ulcerative Colitis Activity Index (PUCAI), was developed to assess disease activity in children. This validated tool offers a reliable and responsive alternative to invasive colonoscopies for pediatric ulcerative colitis management.
Area of Science:
- Pediatric Gastroenterology
- Inflammatory Bowel Disease Research
- Clinical Assessment Tools
Background:
- Colonoscopy is the standard for assessing ulcerative colitis activity in adults but is poorly tolerated by children.
- There is a need for a noninvasive method to evaluate disease activity in pediatric ulcerative colitis.
Purpose of the Study:
- To develop and validate a noninvasive disease activity index specifically for pediatric ulcerative colitis.
- To create a reliable and responsive tool for assessing ulcerative colitis in children.
Main Methods:
- Expert consensus (Delphi group) and regression modeling were used for item selection and weighting.
- A prospective cohort of 157 pediatric patients with ulcerative colitis was used for weighting the index.
- Validation was performed on a separate cohort of 48 children, with responsiveness assessed in 75 children.
Main Results:
- The Pediatric Ulcerative Colitis Activity Index (PUCAI) was developed, comprising 11 items, with six clinical items being significant.
- The PUCAI demonstrated high correlations with established measures (Physician's Global Assessment, Mayo score, colonoscopy) and superior performance to adult noninvasive indices.
- Excellent interobserver and test-retest reliability (ICC = 0.95) and responsiveness (effect size = 1.9) were observed.
Conclusions:
- The rigorously developed PUCAI is a noninvasive, valid, highly reliable, and responsive index.
- PUCAI provides an effective tool for assessing disease activity in pediatric ulcerative colitis, improving patient tolerance.
- This index facilitates better management of ulcerative colitis in pediatric populations.
Background And Aims:
Colonoscopic appearance, the primary measure of disease activity in adult ulcerative colitis, is less acceptable to children. Our aim was to develop a noninvasive activity index of pediatric ulcerative colitis.
Methods:
Item selection was performed judgmentally using a Delphi group of 36 experts in pediatric inflammatory bowel disease. Item weighting was performed by regression modeling using a prospective cohort of 157 pediatric ulcerative colitis patients. Validation was assessed on a separate prospective cohort of 48 children with ulcerative colitis undergoing complete colonoscopy. Responsiveness was evaluated at a follow-up visit of 75 children using effect size statistics and diagnostic utility approaches.
Results:
A list of 41 items was generated and reduced to 11 by rank order. Two physicians completed the Pediatric Ulcerative Colitis Activity Index (PUCAI) on each of the patients in the weighting cohort. Six clinical items were significant in the regression analysis; the laboratory items and an endoscopic appearance item did not improve the PUCAI performance. In the validation cohort, the PUCAI was highly correlated with the Physician's Global Assessment (r = 0.91, P < .001), Mayo score (r = 0.95, P < .001), and colonoscopic appearance (r = 0.77, P < .001). Correlations were higher than 2 noninvasive adult indices calculated concurrently. Interobserver and test-retest reliability were excellent (intraclass correlation coefficient = 0.95; 95% CI: 0.93-0.97). Cut-off points were established using receiver operator characteristic curves on the full cohort. Excellent responsiveness was found at repeated visits (effect size = 1.9, area under the receiver operator characteristic curve = 0.97).
Conclusions:
The rigorously developed PUCAI is a noninvasive, valid, highly reliable, and responsive index with which to assess disease activity in pediatric ulcerative colitis.
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