TY - JOUR
T1 - Development and Validation of the TUMMY-UC
T2 - A Patient-Reported Outcome for Pediatric Ulcerative Colitis
AU - Marcovitch, Liron
AU - Focht, Gili
AU - Carmon, Natalie
AU - Tersigni, Claudia
AU - Ledder, Oren
AU - Lev-Tzion, Raffi
AU - Church, Peter C.
AU - Hyams, Jeffrey S.
AU - Baldassano, Robert N.
AU - Bousvaros, Athos
AU - Mack, David R.
AU - Hussey, Séamus
AU - Otley, Anthony
AU - Croft, Nicholas M.
AU - Kappelman, Michael D.
AU - Griffiths, Anne M.
AU - Turner, Dan
N1 - Funding Information:
Funding This study was partially funded by an academic grant from Janssen, which was not involved in any part of the design, conduct or analyses of this study, or in manuscript preparation.
Publisher Copyright:
© 2023 AGA Institute
PY - 2023/4
Y1 - 2023/4
N2 - Background & Aims: The TUMMY-UC is a patient-reported outcome measure for pediatric ulcerative colitis (UC) with an observer-reported outcome version for children aged <8 years. It includes eight items selected by concept elicitation interviews. We aimed to finalize the TUMMY-UC by cognitive interviews (stage 2) and to evaluate the index for its psychometric properties (stage 3). Methods: The TUMMY-UC items were first finalized during 129 cognitive debriefing interviews. Then, in a prospective, multicenter validation study, 84 children who underwent colonoscopy or provided stool for calprotectin completed the TUMMY-UC and various measures of disease activity. Assessments were repeated after 7 and 21 days for evaluating reliability and responsiveness. Results: During stage 2, the items were formatted with identical structure to ensure conceptual equivalence and weighted based on ranking of importance. In stage 3, the TUMMY-UC total score had excellent reliability in repeated assessments (intraclass correlation coefficient, 0.90; 95% confidence interval, 0.84–0.94). It also had moderate to strong correlations with all constructs of disease activity: r = 0.70 with UC endoscopic index of severity, r = 0.63 with the IMPACT-III questionnaire, r = 0.43 with calprotectin, r = 0.80 with the Pediatric Ulcerative Colitis Activity Index, r = 0.75 with global assessment of disease activity, and r = 0.46 with C-reactive protein (all P < .015). The index had excellent discrimination of disease activity, with a score of <9 defining remission (area under the receiver operating characteristic curve, 0.95; 95% confidence interval, 0.93–0.99). The ΔTUMMY-UC showed high responsiveness and differentiated well between children who experienced changed from those with no change. Conclusions: The TUMMY-UC, constructed from patient-reported outcome and observer-reported outcome versions, is a reliable, valid and responsive index that can be now used in practice and clinical trials.
AB - Background & Aims: The TUMMY-UC is a patient-reported outcome measure for pediatric ulcerative colitis (UC) with an observer-reported outcome version for children aged <8 years. It includes eight items selected by concept elicitation interviews. We aimed to finalize the TUMMY-UC by cognitive interviews (stage 2) and to evaluate the index for its psychometric properties (stage 3). Methods: The TUMMY-UC items were first finalized during 129 cognitive debriefing interviews. Then, in a prospective, multicenter validation study, 84 children who underwent colonoscopy or provided stool for calprotectin completed the TUMMY-UC and various measures of disease activity. Assessments were repeated after 7 and 21 days for evaluating reliability and responsiveness. Results: During stage 2, the items were formatted with identical structure to ensure conceptual equivalence and weighted based on ranking of importance. In stage 3, the TUMMY-UC total score had excellent reliability in repeated assessments (intraclass correlation coefficient, 0.90; 95% confidence interval, 0.84–0.94). It also had moderate to strong correlations with all constructs of disease activity: r = 0.70 with UC endoscopic index of severity, r = 0.63 with the IMPACT-III questionnaire, r = 0.43 with calprotectin, r = 0.80 with the Pediatric Ulcerative Colitis Activity Index, r = 0.75 with global assessment of disease activity, and r = 0.46 with C-reactive protein (all P < .015). The index had excellent discrimination of disease activity, with a score of <9 defining remission (area under the receiver operating characteristic curve, 0.95; 95% confidence interval, 0.93–0.99). The ΔTUMMY-UC showed high responsiveness and differentiated well between children who experienced changed from those with no change. Conclusions: The TUMMY-UC, constructed from patient-reported outcome and observer-reported outcome versions, is a reliable, valid and responsive index that can be now used in practice and clinical trials.
KW - ObsRO
KW - PRO
KW - PUCAI Scale
KW - Patient-Reported Outcome
KW - Ulcerative Colitis
UR - http://www.scopus.com/inward/record.url?scp=85148913405&partnerID=8YFLogxK
U2 - 10.1053/j.gastro.2022.12.006
DO - 10.1053/j.gastro.2022.12.006
M3 - Article
C2 - 36539016
AN - SCOPUS:85148913405
SN - 0016-5085
VL - 164
SP - 610-618.e4
JO - Gastroenterology
JF - Gastroenterology
IS - 4
ER -