PBC-10: a short quality of life measure for clinical screening in primary biliary cholangitis.

Abstract

Current guidelines in primary biliary cholangitis ( PBC) recommend routine screening for symptoms. However, at present there are no validated practical tools suitable for screening use in practice. To develop a short quality of life questionnaire for PBC METHODS: The short PBC HRQL questionnaire was derived and validated by analysing the PBC-40 questionnaires from the UK-PBC Research Cohort. Construct validity was assessed using the European Quality of Life Five Dimensions (EQ5D) questionnaire. Test-retest analysis was done by asking a subgroup of patients to complete the questionnaire twice within 2-4weeks. A total of 2219 patients completed PBC-40 questionnaire in 2013. Stepwise regression identified 10 questions that contributed to more than 95% of the PBC-40 score variance and covered the main domains of PBC. The short HRQL questionnaire, PBC-10, had good internal consistency (Cronbach's α 0.905) and item-total correlations. PBC-10 demonstrated no ceiling effects but a floor effect was noted. Further validation on 2502 patients who completed the PBC questionnaire in 2017 confirmed the psychometric properties of PBC-10. Further analysis on 186 patients showed that PBC-10 demonstrated good internal consistency (Cronbach's α=0.936), had good reproducibility (intra-class correlation coefficient=0.945), good correlation with the EQ5D (r=.736), and was responsive to change. A change of 4 points in the PBC-10 score would be considered clinically important. PBC-10 is a short and valid questionnaire for assessing the HRQL in patients with PBC in clinical practice.