Related Experiment Videos
Pediatric Crohn Disease Activity Index: responsive to short-term change
P S Kundhal1, J N Critch, M Zachos
1Divisions of Gastroenterology and Nutrition, Deparment of Pediatrics, The Hospital for Sick Children, University of Toronto, Canada.
Insights
The Pediatric Crohn's Disease Activity Index (PCDAI) effectively measures short-term improvements in pediatric Crohn's disease. A PCDAI score change of -12.5 indicates meaningful clinical improvement in children with Crohn's disease.
Area of Science:
- Pediatric gastroenterology
- Inflammatory bowel disease research
- Clinical trial methodology
Background:
- The Pediatric Crohn's Disease Activity Index (PCDAI) is validated but its sensitivity to change in short-term trials is unproven.
- Assessing responsiveness is crucial for evaluating acute treatment efficacy in pediatric Crohn's disease.
Purpose of the Study:
- To evaluate the short-term responsiveness of the PCDAI.
- To determine the minimal PCDAI score change indicating clinically meaningful improvement in pediatric Crohn's disease patients.
Main Methods:
- Calculated standardized effect size (SES) and standardized response mean (SRM) in clinical practice and RCT settings.
- Used ROC curves to identify minimal PCDAI change correlated with physician global assessment and adult CDAI changes.
Main Results:
- PCDAI demonstrated strong responsiveness (SES: 1.78-2.10, SRM: 1.41-1.95).
- A PCDAI change of -12.5 was optimal for detecting clinically significant improvement based on physician assessment.
- A PCDAI change of -10 correlated with a change of at least 70 points in the adult CDAI.
Conclusions:
- The PCDAI is a responsive measure of disease activity change in pediatric Crohn's disease over short intervals.
- The PCDAI is suitable for use in pediatric acute treatment trials for Crohn's disease.
Background/Aims:
Despite documented feasibility, reliability, and validity, the Pediatric Crohn's Disease Activity Index (PCDAI) has yet to be demonstrated to be sensitive to change in the time frame of acute treatment trials. We evaluated short-term responsiveness and determined the minimal change in PCDAI score associated with a clinically meaningful improvement in disease activity.
Methods:
Standardized effect size (SES) and standardized response mean (SRM) were calculated as measures of responsiveness among pediatric patients being treated for acute exacerbations of Crohn disease 1) in a regular clinical practice setting and 2) as part of a multicenter, randomized controlled trial (RCT). Receiver operating characteristic (ROC) curves were constructed to determine the minimal PCDAI score change associated with significant clinical improvement used as the gold standard in 1) physician global assessment of change and in 2) change in adult Crohn disease activity index (CDAI).
Results:
Among responders, the SES and SRM of the PCDAI were 1.78 and 1.41 (95% CI: 0.89-1.92) and 2.10 and 1.95 (95% CI: 1.70-2.20) in the clinical practice setting and RCT setting, respectively. The optimal minimal PCDAI change score associated with clinically significant change in physician global assessment was determined to be -12.5 (sensitivity 83.3%, specificity 92.3%). In the RCT setting a change in PCDAI of -10 corresponded to a change in CDAI of not greater-than-or-equal 70 points.
Conclusions:
The PCDAI is responsive to improvement in disease activity in Crohn disease patients over a short interval. As such, the PCDAI is an appropriate instrument to use in pediatric acute treatment trials.