Search

Background, & Aims: Studies of primary biliary cirrhosis (PBC) phenotypes largely have been performed using small and selected populations. Study size has precluded investigation of important disease subgroups, such as men and young patients. We used a national patient cohort to obtain a better picture of PBC phenotypes. Methods: We performed a cross-sectional study using the United Kingdom-PBC, patient cohort. Comprehensive data were collected for 2353 patients on diagnosis reports, response to therapy with ursodeoxycholic acid (UDCA), laboratory results, and symptom impact (assessed using the PBC-40 and other related measures). Results: Seventy-nine percent of the patients reported current UDCA, therapy, with 80% meeting Paris response criteria. Men were significantly less likely to have responded to UDCA than women (72% vs 80% response rate; P

Background & Aims: Liver transplantation improves survival in end-stage primary biliary cirrhosis (PBC), but the benefit for systemic symptoms including fatigue is less clear. The aim of this study was to utilise the comprehensive UK-PBC Research Cohort, including 380 post-transplant patients and 2300 non-transplanted patients, to answer key questions regarding transplantation for PBC. Methods: Cross-sectional study of post-transplant PBC patients and case-matched non-transplanted patients. Detailed clinical information was collected, together with patient systemic symptom impact data using validated assessment tools. Results: Over 25% of patients in the transplant cohort were grafted within 2 years of PBC diagnosis suggesting advanced disease at presentation. Transplanted patients were significantly younger at presentation than non-transplanted (mean 7 years) and >35% of all patients in the UK-PBC cohort who presented under 50 years had already undergone liver transplantation at the study censor point (>50% were treatment failures (post-transplant or unresponsive to UDCA)). Systemic symptom severity (fatigue and cognitive symptoms) was identical in female post-transplant patients and matched non-transplanted controls and unrelated to disease recurrence or immunosuppression type. In males, symptoms were worse in transplanted than in non-transplanted patients. Conclusions: Age at presentation is a major risk factor for progression to transplant (as well as UDCA non-response) in PBC. Although both confirmatory longitudinal studies, and studies utilising objective as well as subjective measures of function, are needed if we are to address the question definitively, we found no evidence of improved systemic symptoms after liver transplantation in PBC and patients should be advised accordingly. Consideration needs to be given to enhancing rehabilitation approaches to improve function and life quality after liver transplant for PBC. © 2013 European Association for the Study of the Liver. Published

Primary biliary cirrhosis (PBC) has a complex clinical phenotype, with debate about the extent and specificity of frequently described systemic symptoms such as fatigue. The aim of this study was to use a national patient cohort of 2,353 patients recruited from all clinical centers in the UK to explore the impact of disease on perceived life quality. Clinical data regarding diagnosis, therapy, and biochemical status were collected and have been reported previously. Detailed symptom phenotyping using recognized and validated symptom assessment tools including the PBC-40 was also undertaken and is reported here. Perception of poor quality of life and impaired health status was common in PBC patients (35% and 46%, respectively) and more common than in an age-matched and sex-matched community control group (6% and 15%, P < 0.0001 for both). Fatigue and symptoms of social dysfunction were associated with impaired perceived quality of life using multivariate analysis. Fatigue was the symptom with the greatest impact. Depression was a significant factor, but appeared to be a manifestation of complex symptom burden rather than a primary event. Fatigue had its greatest impact on perceived quality of life when accompanied by symptoms of social dysfunction, suggesting that maintenance of social networks is critical for minimizing the impact of fatigue. Conclusion: The symptom burden in PBC, which is unrelated to disease severity or ursodeoxycholic acid response, is significant and complex and results in significant quality of life deficit. The complexity of symptom burden, and its lack of relation to disease severity and treatment response, suggest that specific approaches to symptom management are warranted that address both symptom biology and social impact. (Hepatology 2013;58:-)

G. Mells, M. Carbone, G. Pells, J. Newton, A. Bathgate, A. Burroughs, M. Heneghan, J. Neuberger, D. Day, S. Ducker, R. Sandford, G. Alexander, D. Jones, The UK-PBC Consortium. Academic Department of Medical Genetics, Cambridge University, Department of Hepatology, Cambridge University Hospitals, Cambridge, Institute of Cellular Medicine, NIHR Biomedical Research Centre in Ageing and Chronic Disease, Newcastle University, Newcastle-upon-Tyne, Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh, Edinburgh, Hepatology Dept, Royal Free Campus, University College London, Institute of Liver Studies, King’s College Hospital NHS Foundation Trust, London, The Liver Unit, Queen Elizabeth Hospital, Birmingham, UK E-mail: greta.pells@ncl.ac.uk

925 FACTORS ASSOCIATED WITH POOR OUTCOME IN PRIMARY BILIARY CIRRHOSIS M. Carbone, G. Mells, H.J. Cordell, M.A. Heneghan, J.M. Neuberger, P.T. Donaldson, A.J. Bathgate, A.K. Burroughs, M.H. Davies, D.B. Day, S. Rushton, M.F. Dawwas, S. Thomson, G.J. Alexander, D.E. Jones, R.N. Sandford, UK Primary Biliary Cirrhosis (PBC) Consortium. Academic Department of Medical Genetics, University of Cambridge, Department of Medicine, Division of Gastroenterology and Hepatology, Cambridge University Hospitals, Cambridge, Institute of Genetic Medicine, Newcastle University, Newcastle Upon Tyne, Institute of Liver Studies, King’s College Hospital, London, Liver Unit, Queen Elizabeth Hospital, Birmingham, Institute of Cellular Medicine, Newcastle University, Newcastle Upon Tyne, Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh, Edinburgh, Liver Transplantation and Hepato-Biliary Medicine Unit, Royal Free Hospital, London, Liver Unit, St James’s University Hospital, Leeds, Organ Donation and Transplantation, NHS Blood and Transplant, Bristol, UK E-mail: marco.carbone@uniroma2.it