Assessing disease activity using the pediatric Crohn's disease activity index: Can we use subjective or objective parameters alone?

Abstract

Background: The pediatric Crohn's disease activity index (PCDAI) is used as a standard tool to assess disease activity in clinical trials for pediatric Crohn's disease.

Aim: To examine which items on the PCDAI drive assessment of disease activity, and how subgroups of subjective and objective items reflect change in disease state over time.

Methods: Selective raw data from three prospectively collected datasets were combined, including 703 children with full PCDAI data at baseline, at 3-mo (Q1, n = 670), and 1-year (Q4, n = 474). Change in individual PCDAI scores from baseline to Q1 and to Q4 were examined using the non-weighted PCDAI.

Results: Abdominal pain, well-being, weight, and stooling had the highest change scores over time. Objective indicators including albumin, abdominal exam, and height velocity followed. Change scores for well-being and abdominal exam did not explain significant variance at Q1 but were significant predictors at Q4 (P < 0.001 and P < 0.05). Subjective and objective subgroups of items predicted less variance (18% and 22%) on total PCDAI scores at Q1 and Q4 compared to the full PCDAI, or a composite scale (both 32%) containing significant predictors.

Conclusion: Although subjective items on the PCDAI change the most over time, the full PCDAI or a smaller composite of items including a combination of subjective and objective components classifies disease activity better than a subgroup of either subjective or objective items alone. Reliance on subjective or objective items as stand-alone proxies for disease activity measurement could result in misclassification of disease state.

Keywords: Clinical trials; Crohn’s disease; Disease activity; Patient reported outcome measurement; Pediatric; Pediatric Crohn’s disease activity index.