Clinical Outcome Assessments in Pediatric Patients With Ulcerative Colitis and Crohn’s Disease Receiving Biologics: A Retrospective Cohort Study

Objectives To assess disease activity, steroid-free remission, and other clinical outcome assessments among pediatric patients with ulcerative colitis (UC) and Crohn’s disease (CD) in the ImproveCareNow (ICN) registry. Methods Patients aged 2-17 years diagnosed with UC or CD between 1 June 2013 to 31 December 2019 were enrolled if they initiated a biologic after enrollment in the ICN registry and completed at least 12 months follow-up after first maintenance dose. Baseline (at biologic initiation) demographics were summarized using descriptive statistics. PUCAI, partial Mayo score, and PGA were assessed for UC; and the sPCDAI and PGA were assessed for CD at first maintenance dose, 1-year and 3-year time points. Kappa coefficients were used to assess the level of agreement between the outcome measures. Results A total of 1,887 patients (UC=350; CD=1,537) were included. Baseline demographics were similar across groups. For UC patients, mean PUCAI scores decreased and the proportion of patients in steroid free remission, quiescent state based on PGA, and remission based on Partial Mayo score increased from first maintenance dose to 1-year and 3-years. For CD patients, mean sPCDAI score of CD patients decreased and the proportion of patients in steroid-free remission by sPCDAI and in quiescent state based on PGA increased from first maintenance dose to 1-year and 3-years. Kappa coefficients showed only modest correlation between disease activity assessments. Conclusion Disease activity scores improved over time, with more pediatric patients with UC and CD achieving steroid-free remission at 1-year and 3-years after first biologic maintenance dose.