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951 THE IMPACT OF LIVER TRANSPLANTATION ON THE PHENOTYPE OF PRIMARY BILIARY CIRRHOSIS (PBC) IN THE UK-PBC COHORT
POSTERS 951 THE IMPACT OF LIVER TRANSPLANTATION ON THE PHENOTYPE OF PRIMARY BILIARY CIRRHOSIS (PBC) IN THE UK-PBC COHORT G. Mells1,2 , M. Carbone2 , G. Pells3 , J. Newton4 , A. Bathgate5 , A. Burroughs6 , M. Heneghan7 , J. Neuberger8 , D. Day1 , S. Ducker3 , R. Sandford1 , G. Alexander1,2 , D. Jones3 , The UK-PBC Consortium. 1 Academic Department of Medical Genetics, Cambridge University, 2 Department of Hepatology, Cambridge University Hospitals, Cambridge, 3 Institute of Cellular Medicine, 4 NIHR Biomedical Research Centre in Ageing and Chronic Disease, Newcastle University, Newcastle-upon-Tyne, 5 Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh, Edinburgh, 6 Hepatology Dept, Royal Free Campus, University College London, 7 Institute of Liver Studies, King’s College Hospital NHS Foundation Trust, London, 8 The Liver Unit, Queen Elizabeth Hospital, Birmingham, UK E-mail: [email protected] PBC is the underlying liver disease in up to 10% of patients listed for liver transplantation in the UK, and although effective in improving survival in end-stage liver disease as a result of PBC, the beneficial impact of transplantation on systemic symptoms of PBC such as fatigue is less clear. This study aimed to use the comprehensive UK-PBC cohort, including 383 post-transplant and 2185 non-transplanted patients, to explore the demographic and symptom phenotype differences between transplanted and nontransplanted patients. A cross-sectional study examining demographic (gender, age at diagnosis, age at study) and treatment (ursodeoxycholic acid[UDCA] treatment and response[Paris criteria], disease years until transplant, immuno-suppression used) data of transplanted and non-transplanted PBC patients from the UK-PBC cohort was undertaken. Transplanted patients were then matched for gender, age at diagnosis, and age at study to a non-transplanted PBC patient for comparison of symptoms assessed in all UK-PBC participants by the following validated measures: PBC-40, Epworth Sleepiness Scale (ESS), Orthostatic Grading Scale (OGS), and Hospital Anxiety and Depression Scale (HADS). Over 25% of transplanted patients were grafted within 2 years of diagnosis. Transplanted patients were significantly younger at presentation than non-transplanted (mean 7 years). >35% of patients presenting under 50-years had already undergone liver transplantation at the study point, and >50% of those diagnosed under 50-years were classed as treatment failures (post-transplant or unresponsive to UDCA). Systemic symptom severity (fatigue, cognitive symptoms) was identical in matched female posttransplant and non-transplant groups and was unrelated to disease recurrence or immune-suppression type. Non-transplanted males had milder systemic symptoms than females, but post-transplant symptoms worsened in males to match levels experienced by both post- and non-transplanted females. Conclusion: Age at PBC presentation (<50 years) is a major risk factor for treatment failure and progression to transplantation. Although confirmatory longitudinal studies are required to assess individual symptom variation post-transplantation, we found no evidence of improved systemic symptoms after liver transplantation in PBC (in fact worsening in males) and patients should be advised accordingly. Consideration needs to be given to enhancing rehabilitation approaches to improve these systemic symptoms known to impact on function and life quality after liver transplant in PBC.
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952 THE IMPACT OF PRIMARY BILIARY CIRRHOSIS (PBC) ON PERCEIVED QUALITY OF LIFE (QoL): THE UK-PBC NATIONAL STUDY G. Mells1,2 , G. Pells3 , J. Newton4 , A. Bathgate5 , A. Burroughs6 , M. Heneghan7 , J. Neuberger8 , D. Day1 , S. Ducker3 , R. Sandford1 , G. Alexander1,2 , D. Jones3,4 , The UK-PBC Consortium. 1 Academic Department of Medical Genetics, Cambridge University, 2 Department of Hepatology, Cambridge University Hospitals, Cambridge, 3 Institute of Cellular Medicine, 4 NIHR Biomedical Research Centre in Ageing and Chronic Disease, Newcastle University, Newcastle-upon-Tyne, 5 Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh, Edinburgh, 6 Hepatology Dept, Royal Free Campus, University College London, 7 Institute of Liver Studies, King’s College Hospital NHS Foundation Trust, London, 8 The Liver Unit, Queen Elizabeth Hospital, Birmingham, UK E-mail: [email protected] PBC is the commonest autoimmune liver disease, and has a complex clinical phenotype. Debate about the extent and specificity of frequently described systemic symptoms such as fatigue has complicated efforts to effectively manage the significant extrahepatic symptom burden seen in PBC. This study aimed to use a national patient cohort recruited from all clinical centres in the UK, to comprehensively quantify and explore the relationships between a variety of systemic PBC symptoms and assess their impact on perceived QoL. A cross-sectional cohort of patients recruited to the UK-PBC Cohort, created to carry out the UK-PBC genetics studies, completed the following established and validated symptom assessment tools: PBC-40, Epworth Sleepiness Scale (ESS), Orthostatic Grading Scale (OGS), and Hospital Anxiety and Depression Score-Depression (HADS-D). Participants were also assessed for perceived quality of life impairment and health status. Controls were recruited via a ‘best-friend‘ method, with Newcastle UK-PBC patients selecting an age- and sex-matched friend to complete a version of the PBC-40 developed and validated for use in controls, along with all other symptom measures. 2353 PBC patients and 196 controls completed all assessment measures, making this the largest study of clinical expression of PBC. Perception of poor QoL was significant in PBC patients vs controls (35% vs 6%, p < 0.0001), as was perceived impaired health status (46% patients vs 6% controls). Fatigue was the symptom with the greatest impact on QoL, with multivariate analysis highlighting that the additional presence of social dysfunction symptoms increased this impact. Depression was a significant factor, but appeared to be a manifestation of complex multi-symptom burden, rather than a primary event. Conclusion: The symptom burden in PBC, which is unrelated to liver disease severity or ursodeoxycholic acid treatment response, is complex and results in significant deficits in quality of life compared to a control population. The complex interaction of symptoms, such as the worsening of fatigue impact when accompanied by social dysfunction, highlights that specific approaches to symptom management are warranted which address both symptom biology and factors modifying social impact.
Journal of Hepatology 2013 vol. 58 | S229–S407
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952 THE IMPACT OF PRIMARY BILIARY CIRRHOSIS (PBC) ON PERCEIVED QUALITY OF LIFE (QoL): THE UK-PBC NATIONAL STUDY G. Mells1,2 , G. Pells3 , J. Newton4 , A. Bathgate5 , A. Burroughs6 , M. Heneghan7 , J. Neuberger8 , D. Day1 , S. Ducker3 , R. Sandford1 , G. Alexander1,2 , D. Jones3,4 , The UK-PBC Consortium. 1 Academic Department of Medical Genetics, Cambridge University, 2 Department of Hepatology, Cambridge University Hospitals, Cambridge, 3 Institute of Cellular Medicine, 4 NIHR Biomedical Research Centre in Ageing and Chronic Disease, Newcastle University, Newcastle-upon-Tyne, 5 Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh, Edinburgh, 6 Hepatology Dept, Royal Free Campus, University College London, 7 Institute of Liver Studies, King’s College Hospital NHS Foundation Trust, London, 8 The Liver Unit, Queen Elizabeth Hospital, Birmingham, UK E-mail: [email protected] PBC is the commonest autoimmune liver disease, and has a complex clinical phenotype. Debate about the extent and specificity of frequently described systemic symptoms such as fatigue has complicated efforts to effectively manage the significant extrahepatic symptom burden seen in PBC. This study aimed to use a national patient cohort recruited from all clinical centres in the UK, to comprehensively quantify and explore the relationships between a variety of systemic PBC symptoms and assess their impact on perceived QoL. A cross-sectional cohort of patients recruited to the UK-PBC Cohort, created to carry out the UK-PBC genetics studies, completed the following established and validated symptom assessment tools: PBC-40, Epworth Sleepiness Scale (ESS), Orthostatic Grading Scale (OGS), and Hospital Anxiety and Depression Score-Depression (HADS-D). Participants were also assessed for perceived quality of life impairment and health status. Controls were recruited via a ‘best-friend‘ method, with Newcastle UK-PBC patients selecting an age- and sex-matched friend to complete a version of the PBC-40 developed and validated for use in controls, along with all other symptom measures. 2353 PBC patients and 196 controls completed all assessment measures, making this the largest study of clinical expression of PBC. Perception of poor QoL was significant in PBC patients vs controls (35% vs 6%, p < 0.0001), as was perceived impaired health status (46% patients vs 6% controls). Fatigue was the symptom with the greatest impact on QoL, with multivariate analysis highlighting that the additional presence of social dysfunction symptoms increased this impact. Depression was a significant factor, but appeared to be a manifestation of complex multi-symptom burden, rather than a primary event. Conclusion: The symptom burden in PBC, which is unrelated to liver disease severity or ursodeoxycholic acid treatment response, is complex and results in significant deficits in quality of life compared to a control population. The complex interaction of symptoms, such as the worsening of fatigue impact when accompanied by social dysfunction, highlights that specific approaches to symptom management are warranted which address both symptom biology and factors modifying social impact.
Journal of Hepatology 2013 vol. 58 | S229–S407