Exploring patient-reported outcome measures to assess symptoms of moderately-to-severely active Crohn's disease in adult and adolescent patients: a qualitative study.

backgroundTo explore patients’ experience of symptoms and impacts of Crohn’s disease (CD) and evaluate content validity of patient-reported outcome (PRO) measures in moderately-to-severely active CD.

methodsQualitative, web-based interviews were conducted to elicit participants’ experiences with CD, and to obtain their feedback on: Patient Global Rating of Severity (PGRS:1 ‘none’ to 6 ‘very severe’), Urgency Numeric Rating Scale (Urgency NRS:0 ‘no urgency’ to 10 ‘worst possible urgency’), patient-reported symptoms from Crohn’s Disease Activity Index (CDAI; abdominal pain [AP] [severe, moderate, mild, none], well-being [WB] [terrible, very poor, poor, slightly under par, generally well], bowel movement [BM] count and stool frequency), and Bristol Stool Chart (type 1 ‘hard’ to type 7 ‘entirely liquid’), including understanding of response options, recall period and examination of meaningful improvement in CD symptoms.

resultsSeventeen adults and 3 adolescents (mean age:37.7 years; 70% female) were interviewed. Overall, 41.2% of adults had a college degree or higher. Most participants reported experiencing fatigue (n = 19), bowel urgency (n = 18), diarrhea (n = 18), AP (n = 17), and/or stool frequency (n = 15). CD impacted participants’ daily activities (n = 20), mood/emotions (n = 19), social activities/relationships (n = 19), productivity/performance at work/school (n = 18), and/or sleep (n = 15). Participants found PRO items and response options easy to understand and use and reported that it was easy to recall their symptoms over 24 h. Participants considered a 1- or 2-point reduction on PGRS as meaningful improvement. Seventeen participants considered 3-point improvement on Urgency NRS as meaningful. Participants with mild/moderate AP (n = 11) and severe AP (n = 13) considered a 1- and 2-point reduction in CDAI-AP score as meaningful improvement. Ten participants reported a 2-point reduction in CDAI-WB score as meaningful improvement if they had ‘terrible’ WB. Generally, participants considered going from 5 to 2 BMs/day as meaningful improvement. Nineteen participants considered improvement to stool type 3 to 5 on Bristol Stool Chart as meaningful.

conclusionsBowel urgency, diarrhea, and AP were the most common CD symptoms. Cognitive debriefing findings support content validity of PRO measures in patients with CD and provide insights on levels of change in PRO measures considered meaningful by patients.