Development and validation of a primary sclerosing cholangitis-specific health-related quality of life instrument: CLDQ-PSC

Abstract

Objectives: To understand the full impact of primary sclerosing cholangitis (PSC) on patients’ health, it is important to assess their health-related quality of life (HRQL). Using the Chronic Liver Disease Questionnaire (CLDQ), we aimed to develop and validate a PSC-specific HRQL instrument. Methods: Previously collected clinical and patient-reported outcome data from PSC patients were used. The original CLDQ with 29 items was subjected to item reduction, followed by factor analysis. A standard HRQL instrument validation pipeline was then applied to the new CLDQ-PSC. Results: There were 100 PSC patients (44±13 y, 32% male, 79% college educated, 39% cirrhosis, 67% inflammatory bowel disease, 66% ulcerative colitis, and 50% on ursodeoxycholic acid After item reduction and exploratory factor analysis, there were 24 items and 5 factors left; based on factor loadings, the factors were named emotional function, fatigue, symptoms, worry, and sleep. Internal consistency assessment returned Cronbach alpha 0.85–0.94, item-to-own domain correlations >0.66 for 22/24 items. Known-groups validity suggests discrimination between PSC patients with and without cirrhosis or its complications, obesity, history of depression, weight loss, and PSC patients on versus not on ursodeoxycholic acid (p<0.05 for all or select CLDQ-PSC domains). Relevant items of Short Form-36 and CLDQ-PSC were highly correlated (all p<0.0001). Matching with items of another PSC-specific instrument (PSC-patient-reported outcome; 42 items) for relevance and redundancy suggests that CLDQ-PSC is a relevant, comprehensive, and short HRQL instrument, which can be used for patients with PSC. Conclusions: The CLDQ-PSC is a PSC-specific HRQL instrument that was developed using an established methodology and demonstrated good psychometric characteristics.

Protocol for the development of a core outcome set for clinical trials in primary sclerosing cholangitis

“… 18 20 Additionally, in recognising the need to accurately capture the patient experience, regulatory agencies have advocated for the inclusion of patient-reported outcome measures (PROMs). While a variety of PROMs have been used, 22–24 many lack international validation, or study in patient populations with diverse demographics, comorbidities and at different stages of disease. Moreover, there are limited data to show how concomitant IBD activity (which is present in up to 80% of patients) may affect the readout of a ‘PSC-specific’ PROM.…”