Development, validation, and evaluation of the PBC-40, a disease specific health related quality of life measure for primary biliary cirrhosis

• Published in: Gut Vol. 54; no. 11; pp. 1622 - 1629
• Main Authors: Jacoby, A, Rannard, A, Buck, D, Bhala, N, Newton, J L, James, O F W, Jones, D E J
• Format: Journal Article
• Language: English
• Published: London BMJ Publishing Group Ltd and British Society of Gastroenterology 01.11.2005; BMJ; BMJ Publishing Group LTD; Copyright 2005 by Gut
• Subjects: Adult; Aged; Biological and medical sciences; Chronic illnesses; chronic liver disease questionnaire; CLDQ; Clinical trials; Employment; epilepsy surgery inventory-55; ESI-55; fatigue impact scale; Female; FIS; Gastroenterology. Liver. Pancreas. Abdomen; health related quality of life; Health Status Indicators; HRQOL; Humans; ICC; Interviews; intraclass correlation coefficient; Liver; Liver Cirrhosis, Biliary - psychology; Liver Cirrhosis, Biliary - rehabilitation; Liver diseases; Liver. Biliary tract. Portal circulation. Exocrine pancreas; Male; Medical sciences; Middle Aged; Other diseases. Semiology; patient based measure; PBC; primary biliary cirrhosis; Psychometrics; Qualitative research; Quality of Life; Reproducibility of Results; SF-36; short form-36; Studies; Surveys and Questionnaires; Validity; Primary biliary cirrhosis; Digestive diseases; Hepatic disease; Gastroenterology; Quality of life
• ISSN: 0017-5749; 1468-3288; 1458-3288
• DOI: 10.1136/gut.2005.065862

Background and aims: Study of health related quality of life (HRQOL) and the factors responsible for its impairment in primary biliary cirrhosis (PBC) has, to date, been limited. There is increasing need for a HRQOL questionnaire which is specific to PBC. The aim of this study was to develop, validate, and evaluate a patient based PBC specific HRQOL measure. Subjects and methods: A pool of potential questions was derived from thematic analysis of indepth interviews carried out with 30 PBC patients selected to represent demographically the PBC patient population as a whole. This pool was systematically reduced, pretested, and cross validated with other HRQOL measures in national surveys involving a total of 900 PBC patients, to produce a quality of life profile measure, the PBC-40, consisting of 40 questions distributed across six domains. The PBC-40 was then evaluated in a blinded comparison with other HRQOL measures in a further cohort of 40 PBC patients. Results: The six domains of PBC-40 relate to fatigue, emotional, social, and cognitive function, general symptoms, and itch. The highest mean domain score was seen for fatigue and the lowest for itch. The measure has been fully validated for use in PBC and shown to be scientifically sound. PBC patient satisfaction, measured in terms of the extent to which a questionnaire addresses the problems that they experience, was significantly higher for the PBC-40 than for other HRQOL measures. Conclusion: The PBC-40 is a short easy to complete measure which is acceptable to PBC patients and has significantly greater relevance to their problems than other frequently used HRQOL measures. Its scientific soundness, shown in extensive testing, makes it a valuable instrument for future use in clinical and research settings.