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

Abstract

Summary Background 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. Aim 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 2‐4 weeks. Results 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. Conclusion PBC‐10 is a short and valid questionnaire for assessing the HRQL in patients with PBC in clinical practice.

A Systematic Review of Patient‐Reported Outcomes in Primary Biliary Cholangitis and Primary Sclerosing Cholangitis

“…Importantly, the instrument has also been adapted into briefer versions (PBC‐27, PBC‐10). ( 23,26 ) In addition to the PBC‐40, the Itch Diary was recently developed based on qualitative interviews with patients with PBC, and thus is another viable option to measure PBC‐induced itch. ( 19 ) Finally, the psychometric properties of the Fatigue Impact Scale and the Fisk Fatigue Severity Scale were evaluated for use in PBC in 2000, with the Fatigue Impact Scale being used frequently thereafter; however, because both were originally developed in other patient populations, neither can claim to be truly representative of PBC‐related fatigue until further qualitative studies are conducted.…”

Review article: pathophysiology and management of primary biliary cholangitis

“…One tool that may be used to assess quality of life and symptom screening is the PBC‐10 questionnaire, a series of 10 questions that can be utilised in clinic, with higher scores indicating worse quality of life and symptom burden (Table 3). 100 …”

Editorial: rapid assessment of health‐related quality of life in primary biliary cholangitis—no excuse not to ask. Authors' reply

“…The authors' declarations of personal and financial interests are unchanged from those in the original article …”

“…We read with great interest Dr Gulamhusein and Dr Jackson's editorial about our paper on the development and validation of PBC‐10 as a short and valid questionnaire that can be used as a simple screening tool of symptom burden in patients with PBC . The PBC‐10 was derived from the original PBC‐40 questionnaire using rigorous statistical analysis of the extensive UK‐PBC Research Cohort database to select the smallest number of items that represent the majority of the PBC‐40 scores.…”