Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn’s Disease

Miller, Talya L.; Schuchard, Julia; Carle, Adam C.; Forrest, Christopher B.; Kappelman, Michael D.; Adler, Jeremy; Ammoury, Rana; Bass, Dorsey; Bass, Julie; Benkov, Keith; Bousvaros, Athos; Boyle, Brendan; Cabrera, José; Colletti, Richard B.; Dorsey, Jill M.; Ebach, Dawn R.; Firestine, Ann; Gulati, Ajay; Hoffenberg, Edward J.; Jester, Traci; Kaplan, Jess L.; Kugathasan, Subra; Kusek, Mark E.; Leibowitz, Ian; Linville, Tiffany; Margolis, Peter A.; Minar, Phillip; Rios, Zarela Molle; Moses, Jonathan; Palomo, Pablo J.; Pappa, Helen; Pashankar, Dinesh S.; Saeed, Shehzad Ahmed; Samson, Charles M.; Sandberg, Kelly C.; Steiner, Steven J.; Strople, J; Sullivan, Jillian S.; Tung, Jeanne; Wali, Prateek

doi:10.1016/j.jpeds.2021.10.053

Cited by 7 publications

(10 citation statements)

References 26 publications

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“…Other published studies have reported the results of additional measures and cohort-specific analyses for the PEPR cohorts, including cross-sectional and longitudinal differences in PROs by disease activity, controlling for demographic and other potential confounding variables. [32][33][34][35] One of the strengths of the PROMIS Pediatric measures is that they are useful for assessing chronic disease outcomes across care settings in both clinical care and research. Importantly, we are able to compare the burden of different diseases both against each other and relative to a US general population.…”

Section: Discussionmentioning

confidence: 99%

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases

Forrest

Schuchard

Bruno

et al. 2022

The Journal of Pediatrics

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Section: Discussionmentioning

confidence: 99%

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases

Forrest

Schuchard

Bruno

et al. 2022

The Journal of Pediatrics

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“…Thus, 14 studies reporting on fatigue in PIBD were eventually included, of which the primary outcome was fatigue in 10 studies. 3,5,[20][21][22][23][24][25][26][27] Nine of the studies used PROs completed by patients, [19][20][21][22][25][26][27][28][29][30] while four used a combination of PRO and parent proxy-reported outcome 3,5,23,31 (Table 2). No study reported fatigue rates in children younger than 6 years of age.…”

Section: Literature Review In Childrenmentioning

confidence: 99%

Fatigue in pediatric inflammatory bowel diseases: A systematic review and a single center experience

Turner,

Focht,

Orlanski‐Meyer

et al. 2023

J. pediatr. gastroenterol. nutr.

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ObjectivesWe aimed to review the literature on fatigue in pediatric inflammatory bowel diseases (PIBD), to explore how it is measured, and approximate its rate in an inception pediatric cohort.MethodsStudies on fatigue were systematically reviewed and selected by two authors. Next, we retrieved the two fatigue‐related questions of the IMPACT‐III questionnaire at 4 and 12 months after diagnosis from a prospectively maintained cohort of PIBD patients, each scoring 0‐100 (lower scores imply more fatigue), and 44 healthy controls.ResultsThe systematic review identified 14 studies reporting fatigue in children, of which nine had fatigue as the primary outcome and only two provided rates of fatigue. No standalone index was identified for measuring fatigue specifically for PIBD. Of 80 children included in the inception cohort, 62 (78%) scored an average of ≤75 on the two IMPACT‐III questions (approximating at least mild fatigue), 26 (33%) scored ≤50 (at least moderate fatigue) and nine (11%) scored ≤25 (severe fatigue). In comparison, only four (9%) healthy children scored at least moderate fatigue (p = 0.007). Fatigue rates at 12 months were only slightly and nonsignificantly lower. Fatigue of any severity was reported in 92% children with active disease versus 63% of those in clinical remission (p = 0.01).ConclusionLiterature reporting on fatigue in PIBD is scarce, and no PIBD‐specific tool is available to measure fatigue. In our cohort, fatigue‐related questions were frequently scored low in children with IBD, mainly among children with active disease but also during clinical remission.

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“…implemented in pediatric IBD (17)(18)(19), and adapted from originally created paper-versions for pediatric liver transplant recipients.…”

Section: What Is Newmentioning

confidence: 99%

“…Adaptation of PROMs to electronic (e-)formats further increases accessibility (14–16). E-PROMs have been implemented in pediatric IBD (17–19), and adapted from originally created paper-versions for pediatric liver transplant recipients.…”

mentioning

confidence: 99%

Equivalence of Paper and Electronic-Based Patient Reported Outcome Measures for Children: A Systematic Review

Kortbeek

Pawaria²,

2022

Journal of Pediatric Gastroenterology &Amp; Nutrition

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Patient-reported outcome measures (PROMs) exist for a variety of chronic gastrointestinal disorders in children. The availability of electronic (e-)formats of PROMs enhance the accessibility of these tools. The International Society for Pharmacoeconomic and Outcomes Research (ISPOR) defines measurement equivalence (ME) as “comparability of the psychometric properties of data” obtained from the administration of original and adapted versions of PROMs. Consideration of proxy PROM versions is unique to pediatrics and must be included in ME evaluations. We conducted a systematic review (SR) of the literature evaluating ME of e-versions adapted from pediatric paper-based PROMs. A literature search was conducted through Medline, Embase, APA PsychInfo, and the Cochrane Library. Titles, abstracts, and manuscripts were reviewed by 2 independent reviewers. The search yielded 19 studies meeting pre-defined criteria. Just over half (52.6%) of 19 PROMs were disease-specific ones. ME between paper- and e-PROM versions was reported as present in all 19 studies evaluating 5653 participants under the age of 18 years. However, only 6 (31.6%) studies evaluated ME in proxy reported e-versions. Despite the use of PROMs for children with a variety of chronic gastrointestinal disorders, only 1 study evaluated a PROM in this population (IMPACT III for inflammatory bowel disease). Findings from this SR highlight strategic opportunities for the pediatric gastroenterologist to broaden the clinical and research armamentarium to include e-PROMs.

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Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn’s Disease

Cited by 7 publications

References 26 publications

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases

Fatigue in pediatric inflammatory bowel diseases: A systematic review and a single center experience

Equivalence of Paper and Electronic-Based Patient Reported Outcome Measures for Children: A Systematic Review

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