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Interpreting Patient-Reported Outcome Scores: Pediatric Inflammatory Bowel Disease as a Use Case

  • Julia Schuchard
    Correspondence
    Address correspondence to Julia Schuchard, PhD, Department of Pediatrics, Children's Hospital of Philadelphia, 2716 South St. 11th Floor, Philadelphia, PA 19146
    Affiliations
    Department of Pediatrics, Children's Hospital of Philadelphia (J Schuchard and CB Forrest), Philadelphia, Pa
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  • Adam C. Carle
    Affiliations
    Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine (AC Carle), Cincinnati, Ohio

    Department of Pediatrics, University of Cincinnati College of Arts and Sciences (AC Carle), Cincinnati, Ohio

    Department of Psychology, University of Cincinnati College of Arts and Sciences (AC Carle), Cincinnati, Ohio
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  • Michael D. Kappelman
    Affiliations
    Department of Pediatrics, University of North Carolina at Chapel Hill School of Medicine (MD Kappelman), Chapel Hill, NC
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  • Carole A. Tucker
    Affiliations
    Department of Health and Rehabilitation Sciences, Temple University College of Public Health (CA Tucker), Philadelphia, Pa
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  • Christopher B. Forrest
    Affiliations
    Department of Pediatrics, Children's Hospital of Philadelphia (J Schuchard and CB Forrest), Philadelphia, Pa
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  • on behalf of theImproveCareNow Pediatric IBD Learning Health System and COMBINE study team
    Author Footnotes
    1 A complete list of study group members appears in the Acknowledgments.
  • Author Footnotes
    1 A complete list of study group members appears in the Acknowledgments.
Published:January 04, 2022DOI:https://doi.org/10.1016/j.acap.2021.12.029

      Abstract

      Objective

      To demonstrate how to interpret Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome measure (PROM) scores for patients with pediatric inflammatory bowel disease (IBD).

      Methods

      Using data from a prospective cohort study of patients ages 8 to 23 years with IBD (n = 1049), we established disease-specific percentiles and computed the minimal clinically important difference (MCID) change score for 6 pediatric PROMs. We applied these results, general population percentiles, and the reliable change index to interpret PROM scores in a clinical trial sample of patients ages 8 to 20 years with IBD (n = 294) in which PROMIS PROMs were obtained at baseline and 3 months later.

      Results

      Application of general population percentiles showed that the clinical trial sample at baseline had moderately worse self-reported health than the general population (22% of patients at or above the 95th percentile on Fatigue; 21% on Pain Interference). IBD-specific percentiles showed that the sample was somewhat worse than the reference IBD sample (8% of patients at or above the 95th percentile on Fatigue; 11% on Pain Interference). Application of the MCID threshold indicated that among the subgroup of patients that improved by 15 or more on the short Pediatric Crohn's Disease Activity Index (n = 38), 45% also improved on IBD Symptoms, 47% for Fatigue, and 65% for Pain Interference.

      Conclusion

      This study established IBD-specific percentiles for 6 pediatric PROMIS measures and demonstrated the application of percentiles and other methods for interpreting PROM scores.

      Keywords

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