Development and content validation of patient-reported outcomes tools for ulcerative colitis and Crohn’s disease in adults with moderate-to-severe disease

Abstract

Background Ulcerative colitis (UC) and Crohn’s disease (CD) are associated with a range of symptoms that adversely affect health-related quality of life. This research aimed to develop and validate two patient-reported outcome (PRO) tools to assess signs and symptoms in patients with moderate-to-severe UC or CD. Methods PRO-UC and PRO-CD Diaries were developed in accordance with US Food and Drug Administration (FDA) recommendations. Data were collected from concept elicitation interviews (in which patients described their symptoms and experience of the disease) and further refined through cognitive interviews (in which patients assessed the relevance and clarity of questions in the tools). Results Interviews were conducted with 12 patients for each indication. Five symptoms (urgent bowel movements, abdominal pain, frequent bowel movements, bloody stools, diarrhea/watery stools) were reported by 83–100% of participants with UC and were included in the final 6-item PRO-UC Diary: stool frequency, rectal bleeding (2 items), diarrhea, rectal urgency, and abdominal pain. For CD, seven symptoms (abdominal pain, diarrhea/loose stools, urgent bowel movements, fatigue/tiredness/weakness, frequent bowel movements, bloody stools, nausea) were reported by 50–100% of participants. These, together with vomiting and incontinence (reported by 42% and 33% of participants, respectively), were included in the final 10-item PRO-CD Diary, covering abdominal pain (2 items), stool frequency, liquid/very soft stool frequency, rectal bleeding, rectal urgency, nausea, vomiting, bowel incontinence, and general well-being. Symptoms were consistently cited across both indications to have an impact on quality of life, with frequent complaints being the need to always be near a toilet and inability to leave home, as well as general pain, discomfort, and nausea. For both tools, questions were accurately interpreted, with at least 67% of participants in both indications stating that items were easy to answer/relevant to their condition and symptoms were easy to recall over the last 24 h. Conclusions Both the PRO-UC and PRO-CD Diaries were developed and validated in accordance with FDA recommendations, providing two new tools for use in clinical trials to assess response to treatment in patients with UC or CD. Psychometric analyses are warranted to fully evaluate their properties and value for use in clinical trials.

Modified Mayo score versus Mayo score for evaluation of treatment efficacy in patients with ulcerative colitis: data from the tofacitinib OCTAVE program

“…One disadvantage of both the mMayo and total Mayo scores, however, is that they only focus on two patient-reported outcomes (rectal bleeding, stool frequency). Other patient-reported symptoms, such as stool urgency, and pain, have been perceived as most important by patients with UC 19 , 20 and therefore should also be considered in clinical trial data to fully capture disease activity pertinent to patients.…”

Bowel Urgency in Ulcerative Colitis: Current Perspectives and Future Directions

“…Patients with UC prefer a BU scale that distinguishes levels of severity instead of a binary “yes/no” response ( 85 ). Consequently, several PRO tools have been developed, including the 29-item Symptoms and Impacts Questionnaire for Ulcerative Colitis, the Ulcerative Colitis Patient-Reported Outcomes Signs and Symptoms measure, the Patient-Reported Outcome-Ulcerative Colitis diary, and the Urgency NRS ( 6 , 39 , 58 , 86 ). Table 3 provides an overview of PROs developed in accordance with Food and Drug Administration guidance that include BU.…”

“…BU is a multidimensional symptom, and its severity is associated with varied characteristics such as deferral time, context (e.g., bathroom accessibility), frequency of BU, previous experience, and the extent or burden of adaptive behaviors, such as carrying clean-up equipment ( 6 , 66 , 68 , 86 , 89 , 90 ). Deferral time is the period during which it is possible to defer defecation after the desire to evacuate ( 8 ).…”

A Novel Remote Patient and Medication Monitoring Solution to Improve Adherence and Persistence With Inflammatory Bowel Disease Therapy (ASSIST Study): Protocol for a Randomized Controlled Trial

“…To assess disease activity using the Tappt system, we will use the PRO-2 assessments for CD [31] and UC [32]. These measures of PROs have been validated in IBD and demonstrate an association between improvements in patient-reported symptoms and clinical remission and response [37,38]. For CD, the PRO-2 assessment comprises questions regarding the frequency of bowel movements in the prior week and the degree of abdominal pain (Table 5).…”