BACKGROUND: Abdominal pain (AP) and loose stools are key symptoms of Crohn’s disease (CD). Patient-reported AP and loose stool frequency (SF) items comprise two of the three patient-reported items included in the Crohn’s Disease Activity Index. These AP and SF items are referred to as "the PRO" in Lilly’s clinical trials. This study aimed to elicit in-depth information on how patients understand and respond to the PRO.
METHODS: Cognitive debriefing interviews were conducted in adults and adolescents (aged 15–17 years) with moderately to severely active CD. Eligible participants self-reported disease severity but had to provide proof that they were diagnosed with CD ≥6 months ago. Participants must have received biologic and/or conventional therapy for CD and must not have had surgery to treat CD. Participants provided feedback on the PRO and described what PRO response options or score changes would reflect meaningful improvement in symptoms or denote successful treatment. Interview transcripts and field notes were analysed using a standard thematic analysis approach.
RESULTS: 17 adults and 3 adolescents participated during May–July 2023. Participants had a mean age of 38 (range=17–60) years; 70% (n=14) were female; 70% (n=14) were White. Participants had been diagnosed, on average, 10 (range=0.5–29) years ago. Most participants (n=13, 65%) reported moderate CD severity. All participants interpreted the AP item as intended, found the response options clear and easy to use, and reported that it would be easy to recall their AP severity over 24 hours. Of the 13 participants reporting AP (mild: n=6, 30%; moderate: n=7, 35%), nearly all (n=11, 85%) would consider a 1-level improvement (e.g., ‘mild’ to ‘none’ on the four-level scale) as meaningful. Most participants (n=11, 55%) would consider a treatment successful if their AP was ‘mild’; while many others (n=8, 40%) would need their AP to be completely resolved (i.e., ‘none’). All participants easily understood the SF item, interpreted it as intended, and reported that it would be easy to recall how many very soft or liquid stools they had experienced in a 24-hour period. Participants generally considered a 35.5% decrease in the number of very soft or liquid stools to be a meaningful improvement. On average, participants would consider a treatment successful if they had 1.7 (range=0–5, median=1) very soft or liquid stools per day.
CONCLUSION: Participants understood and easily completed the PRO. These findings support the content validity of the PRO in adults and adolescents with moderately to severely active CD and provide insights on the amount of change in AP and SF that patients would consider meaningful.
