The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure.

OBJECTIVE: We sought to develop and validate the Crohn's Disease-Health Index (CD-HI), a disease-specific, patient-reported outcome measure that serially measures Crohn's disease (CD) symptomatic burden in adults with CD. BACKGROUND: As therapeutic interventions are tested among patients with CD, responsive outcome measures are needed to track disease progression and therapeutic gain during clinical trials. PATIENTS AND METHODS: We conducted a national cross-sectional study of individuals with CD to identify the most prevalent and impactful symptoms of CD. The most relevant symptoms were included in the CD-HI. We used factor analysis, qualitative patient interviews, test-retest reliability evaluation, and known group validity testing to evaluate and optimize the CD-HI. RESULTS: The CD-HI contains 12 subscales that comprehensively measure CD burden using the patient's perspective. Fifteen adults with CD beta tested the CD-HI and found the instrument to be clear, easy to use, and relevant to them. Twenty-three adults with CD participated in an assessment of test-retest reliability, which indicated high reliability of individual questions, subscales, and the full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument). The CD-HI distinguished between groups of individuals with CD known to differ in disease severity. CONCLUSIONS: This research supports the use of the CD-HI as a valid, sensitive, reliable, and relevant patient-reported outcome to determine the multifactorial disease burden of those with CD, assess the relevance and merit of future CD therapies, and support drug labeling claims.

Patient-Reported Impact of Symptoms in Crohn's Disease.

INTRODUCTION: Patients with Crohn's disease (CD) experience a variety of symptoms that significantly affect their lives. In this study, we (i) ascertain the most prevalent and impactful symptoms in CD and (ii) identify modifying factors that are associated with a higher disease burden in CD. METHODS: We conducted semistructured interviews with adult participants with CD to determine what issues have the greatest impact on their lives. Next, we conducted a large cross-sectional study of individuals with CD to determine the prevalence and relative importance of those symptoms and themes and to identify the demographic features that are associated with a higher disease burden. RESULTS: Sixteen individuals with CD provided 792 direct quotes regarding their symptomatic burden. Four hundred three people with CD participated in our cross-sectional study. The symptomatic themes with the highest prevalence in CD were gastrointestinal issues (93.0%), fatigue (86.4%), dietary restrictions (77.9%), and impaired sleep or daytime sleepiness (75.6%). The symptomatic themes that had the greatest impact on patients' lives (0-4 scale) related to fatigue (1.82), impaired sleep or daytime sleepiness (1.71), gastrointestinal issues (1.66), and dietary restrictions (1.61). Symptomatic theme prevalence was strongly associated with a higher number of soft stools per day, greater number of bowel movements per day, missed work, employment and disability status, and having perianal disease. DISCUSSION: Patients with CD experience numerous symptoms that affect their daily life. These symptoms, some underrecognized, vary based on disease and demographic characteristics and represent potential targets for future therapeutic interventions.

P047 The Crohn's Disease-Health Index (CD-HI): Development and Validation of a Novel, Disease-Specific Patient Reported Outcome Measure for Clinical Trials.

BACKGROUND: In preparation for upcoming clinical trials involving patients with Crohn's disease (CD), we examine the validity, reliability and usability of the Crohn's Disease-Health Index (CD-HI). The CD-HI is a multifaceted, disease-specific patient reported outcome measure (PROM) designed to measure CD symptomatic disease burden during clinical trials. As promising therapeutic interventions are being tested among CD patients, there is a clear need for researchers to have access to a valid, sensitive, and reliable patient reported outcome tool to track disease burden. This research describes the development and validation of the CD-HI as an efficient mechanism to quantify how CD patients both feel and function. METHODS: We conducted semi-structured, qualitative interviews with CD patients to identify potential symptoms of importance in CD. We then conducted a large, cross-sectional survey study with CD patients to identify the prevalence and importance of symptoms identified during the prior interviews. Symptom questions in the first version of the CD-HI were selected based on overall frequency and impact in a large population of CD patients, generalizability, and potential to respond to therapeutic intervention. Questions which measured a similar concept were grouped into subscales using factor analysis. The first version of the CD-HI was beta tested to explore the usability and relevance of the instrument to patients. We then performed test-retest reliability of each question and subscale. Lastly, we determined the internal consistency for each subscale and the overall instrument. The CD-HI is now finalized and available for use in upcoming clinical trials. RESULTS: Sixteen adults with CD participated in semi-structured qualitative interviews, providing 792 quotes regarding the symptomatic burden of CD. Four hundred and three adults with CD completed an online survey to determine the prevalence and relative importance of 148 patient identified symptoms. Questions were selected for the CD-HI based on their prevalence and relative importance to CD patients. Sixteen adults with CD participated in beta interview testing to address the usability and relevance of the instrument. Patients found the CD-HI to be clear, highly relevant, and easy to use. Test-retest reliability was conducted with twenty-three adults with CD, where participants completed the CD-HI at baseline and fourteen days later. One question was removed to optimize the overall reliability of the instrument. The final version of the CD-HI contains subscales that measure the following granular areas of CD health: 1) fatigue; 2) dietary restrictions; 3) gastrointestinal health; 4) sleep and daytime sleepiness; 5) bowel and bladder function; 6) emotional health; 7) joint health; 8) pain; 9) neck and back health; 10) activity participation; 11) social health; and 12) skin health. Total CD disease burden is measured using a weighted composite of these subscale scores. CONCLUSION: This research successfully demonstrates the ability of the CD-HI to report valid, reliable, and patient-relevant data as a disease-specific PROM. The CD-HI provides researchers and clinicians with an optimal mechanism to record relevant changes in CD health using the patient's perspective.