The Inflammatory Bowel Disease Questionnaire in Randomized Controlled Trials of Treatment for Ulcerative Colitis: Systematic Review and Meta-Analysis (original) (raw)

Psychometric validation of the SF-36® Health Survey in ulcerative colitis: results from a systematic literature review

Quality of Life Research, 2017

Purpose To conduct a systematic literature review of the reliability, construct validity, and responsiveness of the SF-36 Ò Health Survey (SF-36) in patients with ulcerative colitis (UC). Methods We performed a systematic search of electronic medical databases to identify published peer-reviewed studies which reported scores from the eight scales and/or two summary measures of the SF-36 collected from adult patients with UC. Study findings relevant to reliability, construct validity, and responsiveness were reviewed. Results Data were extracted and summarized from 43 articles meeting inclusion criteria. Convergent validity was supported by findings that 83% (197/236) of correlations between SF-36 scales and measures of disease symptoms, disease activity, and functioning exceeded the prespecified threshold (r C |0.40|). Known-groups validity was supported by findings of clinically meaningful differences in SF-36 scores between subgroups of patients when classified by disease activity (i.e., active versus inactive), symptom status, and comorbidity status. Responsiveness was supported by findings of clinically meaningful changes in SF-36 scores following treatment in non-comparative trials, and by meaningfully larger improvements in SF-36 scores in treatment arms relative to controls in randomized controlled trials. The sole study of SF-36 reliability found evidence supporting internal consistency (Cronbach's a C 0.70) for all SF-36 scales and test-retest reliability (intraclass correlation coefficient C0.70) for six of eight scales. Conclusions Evidence from this systematic literature review indicates that the SF-36 is reliable, valid, and responsive when used with UC patients, supporting the inclusion of the SF-36 as an endpoint in clinical trials for this patient population. Keywords SF-36 Á Psychometric validation Á Ulcerative colitis Á Systematic review Ulcerative colitis (UC), a subtype of inflammatory bowel disease (IBD), is a chronic, recurrent disease characterized by inflammation and ulceration of tissue lining the colon. Prevalence of UC in the United States (US) has been estimated as 28.63 with an annual incidence of 1.22 (both per 10,000) [1]. During periods of active disease, UC patients experience clinical symptoms including abdominal pain, fatigue, diarrhea, bloody stool, and unpredictable urges to evacuate the bowels. Effective treatments for UC have been defined by the likelihood that they produce a meaningful reduction of clinical symptoms and improvements in mucosal health [2-6].

Further Validation of the Crohn’s and Ulcerative Colitis Ques-tionnaire-32 (CUCQ-32) to Measure the Quality of Life in Pa-tients Treated with Biologics Therapy

Background: Crohn’s and Ulcerative Colitis Questionnaire-32 (CUCQ-32) is a validated questionnaire to measure the quality of life (QoL) in inflammatory bowel disease (IBD). However, it does not have stoma specific questions and can be lengthy. This study aimed to validate a subset of the CUCQ-32 that would be suitable for patients with a stoma. Methods: Baseline data were collected from a cohort of patients with acute ulcerative colitis who were participating in the CONSTRUCT multi-centre clinical trial. A subset of the CUCQ-32 questions was selected by stepwise regression. Further validation was examined using data from the UK IBD biological therapies audit. Construct validity was carried out using the EuroQol 5 dimensions (EQ5D) questionnaire, Simple Clinical Colitis Activity Index (SCCAI) and the Harvey-Bradshaw Index (HBI). Literature review and an expert focus group identified supplementary questions to cover patients with a stoma. Test-retest analysis was done during the patie...

Clinical Activity and Quality of Life Indices Are Valid Across Ulcerative Colitis But Not Crohn's Disease Phenotypes

Digestive diseases and sciences, 2016

Clinical activity and quality of life (QOL) indices assess disease activity in Crohn's disease (CD) and ulcerative colitis (UC). However, a paucity of data exists on the validity of these indices according to disease characteristics. To examine the correlation between QOL and clinical activity indices and endoscopic disease activity according to disease characteristics. We used a prospective registry to identify CD and UC patients ≥18 years old with available information on Short Inflammatory Bowel Disease Questionnaire scores (SIBDQ), Harvey-Bradshaw Index (HBI) and simple endoscopic scores for CD (SES-CD), and Simple Clinical Colitis Activity Index (SCCAI) and Mayo endoscopic score for UC. We used Spearman rank correlations to calculate correlations between indices and Fisher transformation to compare correlations across disease characteristics. Among 282 CD patients, we observed poor correlation between clinical activity and QOL indices to SES-CD with no differences in correl...

Health-Related Quality of Life in Patients with Ulcerative Colitis After a 10-year Disease Course: Results from the IBSEN Study

Inflammatory Bowel Diseases, 2012

for the IBSEN Study Group Background: Ulcerative colitis (UC) negatively affects health-related quality of life (HRQoL), but population-based and long-term data on this topic are scarce. Our aim was to determine the HRQoL in UC patients after a 10-year disease duration. Methods: UC patients from a population-based inception cohort met at a prescheduled 10-year follow-up visit. In addition to a clinical examination, interview, and blood samples, the patients completed the Short Form 36 (SF-36) and the Norwegian Inflammatory Bowel Disease Questionnaire (N-IBDQ). The SF-36 scores were compared to scores from a general population sample using one-sample t-tests. Standardized scores were calculated and interpreted according to Cohen's effect size index. The associations between relevant clinical and demographic factors and HRQoL were examined through linear regression analyses. Results: A total of 196 patients completed the HRQoL questionnaires (response rate: 80%), of whom 54% were women; the mean age of all patients was 48 years (range: 22-86). The SF-36 scores were comparable to those of the general population except for lower scores in the General Health dimension. The SF-36 scores were significantly lower in the presence of current symptoms, in patients who had used corticosteroids, and in patients who reported not working. Overall N-IBDQ scores were equivalent to scores of patients in remission. Female gender, work status (not working), current symptoms, and smoking were associated with significantly lower N-IBDQ scores. Conclusions: SF-36 scores were not reduced compared to the general population sample. The presence of current symptoms, the use of corticosteroids, work status (not working), female gender, and smoking had a negative impact on HRQoL.

Disease Burden and Patient-Reported Outcomes Among Ulcerative Colitis Patients According to Therapy at Enrollment Into CorEvitas’ Inflammatory Bowel Disease Registry

Crohn's & Colitis 360, 2022

Background To evaluate disease burden and patient-reported outcomes (PROs) of ulcerative colitis (UC) patients at enrollment into CorEvitas’ Inflammatory Bowel Disease Registry by therapy class. Methods Between May 3, 2017 and September 3, 2019, 773 UC registry patients were categorized by therapy class at enrollment: patients on 5-aminosalicylic acids (5-ASAs) only (n = 290), and patients on biologics/Janus kinase inhibitors (JAKi) alone or in combination with 5-ASAs or immunosuppressant therapies (BIO/JAKi) (n = 315). To quantify between group differences, the mean/proportional differences and corresponding 95% CIs were calculated. Results Among 605 UC patients at enrollment, BIO/JAKi patients were younger (44.1 vs. 50.9 years) more were female (58.0% vs. 49.7%), had lower remission (45.4% vs. 60.0%), had more moderate/severe disease (16.5% vs. 7.1%), experienced less proctitis (10.5% vs. 22.1%), but more pancolitis (54.6% vs. 34.1%), more corticosteroid experience (70.8% vs. 44.5...

Burden of disease and patient-reported outcomes in patients with moderate to severe ulcerative colitis in the last 12 months - Multicenter European cohort study

Digestive and liver disease : official journal of the Italian Society of Gastroenterology and the Italian Association for the Study of the Liver, 2016

Treatment of ulcerative colitis (UC) is aimed at maintaining corticosteroid-free remission and improving quality of life (QoL). Assess patients' perception of disease burden and unmet clinical needs in moderate/severe UC patients. Adults surgery-free conventionally treated patients with Mayo score ≥6 were enrolled in an observational, cross-sectional, retrospective study in 11 European countries. Disease control was defined as Mayo score ≤2 with no sub-score >1. No corticosteroid was used the previous two months. Unmet clinical needs were defined as: non-controlled disease, self-perception of 'moderate'/'severe' disease, and dissatisfaction with treatments. Disease burden on QoL and work productivity were assessed (EuroQol-5D-5L, Short Inflammatory Bowel Disease Questionnaire (SIBDQ) and Work Productivity and Activity Impairment (WPAI) in UC questionnaire). UC patients (n=253) with mean Mayo score at enrolment of 4.9, 44.3% of patients had Mayo score ≥6. Main ...

Ulcerative Colitis-Symptom Questionnaire: Valid for Use in Adults with Moderately to Severely Active Ulcerative Colitis

Digestive Diseases and Sciences

Background Due to wide-ranging impacts of Ulcerative Colitis (UC), regulatory authorities emphasize the importance of including validated patient-reported symptom severity measures in clinical trials. Aim To describe the development and validation of the Ulcerative Colitis-Symptom Questionnaire (UC-SQ). Methods The UC-SQ was developed in a qualitative study involving a targeted literature review, semi-structured concept elicitation interviews, and combined concept elicitation/cognitive interviews. Measurement properties, including item-level analyses, factor structure, reliability, validity, responsiveness, and clinically meaningful change were evaluated using data from a phase 2b, randomized trial in adults with UC (N = 113). Results Fourteen symptom concepts were elicited across 22 interviews, with saturation at the fifth interview. Twenty-two items were unmodified as cognitive interview participants interpreted underlying concepts correctly. Instructions were clear and items were relevant, with appropriate response options and recall periods. Reduction to 17 items was completed prior to psychometric testing. Two items (joint pain/constipation) did not contribute to reliability in initial testing and were included as non-scored items. The 15-item UC-SQ showed evidence of internal consistency (α = 0.86) and test-retest reliability (intraclass correlation coefficient = 0.88). The UC-SQ discriminated by disease severity as defined by Mayo and Inflammatory Bowel Disease Questionnaire scores (p < 0.0001). Convergent validity was supported by strong correlations with criterion measures. The UC-SQ was responsive in patients indicating change in other measures. A 10-point decrease from baseline indicated within-patient meaningful improvement. Conclusions The UC-SQ is reliable, valid and responsive, with a 10-point improvement estimating within-patient clinically meaningful improvement. The tool is fit-for-purpose as a key endpoint in pivotal UC trials.