Validity and Responsiveness of the Patient-reported Outcomes Measurement Information System in Children With Ulcerative Colitis

Abstract

Objectives: Patient-reported outcome measures allow children to directly report on their health and well-being. We assessed the construct validity and responsiveness of the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric measures in children and adolescents with ulcerative colitis (UC).

Methods: Through the Inflammatory Bowel Disease Partners Kids & Teens' Internet-based cohort, children with UC reported symptoms related to disease activity (Pediatric Ulcerative Colitis Activity Index), IMPACT-III health-related quality of life measure, and 5 PROMIS Pediatric measures (anxiety, depressive symptoms, pain interference, fatigue, and peer relationships). We included participants aged 9 to 17 years and conducted cross-sectional and longitudinal, mixed-linear regression analyses to examine the extent to which PROMIS Pediatric scores are associated with and respond to changes in Pediatric Ulcerative Colitis Activity Index and IMPACT-III.

Results: We evaluated 91 participants with UC (mean age 13 years, 57% girls). Better PROMIS Pediatric scores were associated with lower disease activity, in both cross-sectional and longitudinal analyses. For a change from moderate/severe to remission, observed effect estimates were -5.1 points for anxiety, -5.0 for depressive symptoms, -14.7 for pain interference, -13.7 for fatigue, and 5.3 for peer relationships (P < 0.05 for all domains). Better PROMIS Pediatric scores were associated with improved IMPACT-III scores (P values <0.01), and changes in scores were moderately correlated with changes in IMPACT-III over time (adjusted P values <0.01).

Conclusions: This study provides evidence for the construct validity and longitudinal responsiveness of the PROMIS Pediatric measures in pediatric patients with UC, thus supporting their use in clinical research and patient care.

Figure 1.

Predicted mean change in PROMIS^® Pediatric domains according to change in ulcerative colitis disease activity* as measured by the Pediatric Ulcerative Colitis Activity Index (PUCAI)** *Disease activity categories as follows: Remission = PUCAI score <10; Mild = PUCAI score 10–34; Moderate/Severe = PUCAI score 35–85. **The sign of change for peer relationships was inversed for this figure, such that a higher score indicates worsened peer relationships. For all other domains, higher scores indicate more of the domain being measured. The threshold used to indicate change in disease activity was a change in PUCAI category.

Figure 2.

Mean change in PROMIS^® Pediatric scores according to change in IMPACT-III: *IMPACT-III is a 35 item health-related quality of life (HRQOL) measure. Possible scores range from 35–175, with higher scores indicating better functioning. ^#The sign of change for peer relationships was inversed for this figure, such that a higher score indicates worsened peer relationships. For all other domains, higher scores indicate more of the domain being measured.