Item Generation and Reduction Toward Developing a Patient‐reported Outcome for Pediatric Ulcerative Colitis (TUMMY‐UC)
ABSTRACT Background: The Pediatric Ulcerative Colitis Activity Index (PUCAI) is a noninvasive clinician‐based index, which reflects disease severity in pediatric ulcerative colitis (UC) when no endoscopy is performed. Here, we aimed to explore signs and symptoms important to children with UC and the...
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| Published in: | Journal of pediatric gastroenterology and nutrition Vol. 64; no. 3; pp. 373 - 377 |
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| Main Authors: | , , , , , , , , , , |
| Format: | Journal Article |
| Language: | English |
| Published: |
United States
by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology
01-03-2017
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| Subjects: | |
| Online Access: | Get full text |
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| Summary: | ABSTRACT
Background:
The Pediatric Ulcerative Colitis Activity Index (PUCAI) is a noninvasive clinician‐based index, which reflects disease severity in pediatric ulcerative colitis (UC) when no endoscopy is performed. Here, we aimed to explore signs and symptoms important to children with UC and their caregivers as the first stage of developing a patient‐reported outcome (PRO) measure for pediatric UC (ie, the TUMMY‐UC index) to supplement endoscopic assessment.
Methods:
Concept elicitation qualitative interviews were performed with children who have UC and their caregivers in 6 centers. Items were rank‐ordered by the interviewees according to the frequency of endorsement and importance, graded on a 1 to 5 scale.
Results:
A total of 46 children (ages 12.5 ± 3.3 years, range 7–18, 48% boys, 83% with pancolitis, 24% with moderate–severe disease) and 33 caregivers were interviewed (ie, 79 interviews). The following items were identified by the children, in decreasing order of weights: abdominal pain (importance × frequency weight 3.9), rectal bleeding (3.6), stool frequency (3.0), stool consistency (3.0), general well‐being/fatigue (2.9), urgency (1.9), and nocturnal stools (1.6). Two other items were scored lower: lack of appetite (1.1) and weight loss (0.6). Children 13 to 18 years comprehended adult vocabulary, children 8 to 12 years comprehended simple vocabulary, and younger children had poor understanding in completing the questions.
Conclusions:
In this first stage of the TUMMY‐UC development, items were generated and ranked by input from patients. These items are now being explored for optimal vocabulary and response options. The TUMMY‐UC will supplement the PUCAI in clinical trial outcome assessment. |
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| Bibliography: | L.M., A.N., A.M.G., and D.T. contributed equally to this article. Supplemental digital content is available for this article. Direct URL citations appear in the printed text, and links to the digital files are provided in the HTML text of this article on the journal's Web site www.jpgn.org D.T. received consultation fee, research grant, royalties, or honorarium from Janssen, Pfizer, Hospital for Sick Children, Ferring, MegaPharm, AstraZeneca, Abbvie, MSD, and BMS. D.T. and A.O. are one of the inventors of the Pediatric Ulcerative Colitis Activity Index, which is copyrighted to the Hospital for Sick Children. D.M. received education grant from Abbvie. A.O. received consultation fee, research grant, education grant, or honorarium from Janssen, AbbVie, Shire, Astellas. NMC's institution has received consultation fee, research grant, royalties, or honorarium from Abbvie, Abbot, Shire, Norgine, Ferring, Johnson and Johnson, Dr Falk, MSD, Schering‐Plough, and GSK. The remaining authors report no conflicts of interest. . This study was funded by an academic grant from Janssen, which was not involved in any part of the study design, conduct and analyses, and manuscript preparation. It was performed as part of L.M.'s MD degree requirements from the Medical School of the Hebrew University of Jerusalem. ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-1 content type line 23 |
| ISSN: | 0277-2116 1536-4801 |
| DOI: | 10.1097/MPG.0000000000001259 |