The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort

Greta Pells; George F Mells; Marco Carbone; Julia L Newton; Andrew J Bathgate; Andrew K Burroughs; Michael A Heneghan; James M Neuberger; Darren B Day; Samantha J Ducker; Richard N Sandford; Graeme J Alexander; David E J Jones; UK Primary Biliary Cirrhosis (PBC) Consortium

doi:10.1016/j.jhep.2013.02.019

The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort

Greta Pells, George F Mells, Marco Carbone, Julia L Newton, Andrew J Bathgate, Andrew K Burroughs, Michael A Heneghan, James M Neuberger, Darren B Day, Samantha J Ducker, Richard N Sandford, Graeme J Alexander, David E J Jones, UK Primary Biliary Cirrhosis (PBC) Consortium

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Abstract

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.

Original language	English
Pages (from-to)	67-73
Number of pages	7
Journal	Journal of Hepatology
Volume	59
Issue number	1
DOIs	https://doi.org/10.1016/j.jhep.2013.02.019
Publication status	Published - Jul 2013

Keywords

Adult
Age Factors
Aged
Cohort Studies
Cross-Sectional Studies
Fatigue
Female
Great Britain
Humans
Liver Cirrhosis, Biliary
Liver Transplantation
Male
Middle Aged
Phenotype
Quality of Life
Risk Factors
Sex Factors

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10.1016/j.jhep.2013.02.019

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Pells, G., Mells, G. F., Carbone, M., Newton, J. L., Bathgate, A. J., Burroughs, A. K., Heneghan, M. A., Neuberger, J. M., Day, D. B., Ducker, S. J., Sandford, R. N., Alexander, G. J., Jones, D. E. J., & UK Primary Biliary Cirrhosis (PBC) Consortium (2013). The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort. Journal of Hepatology, 59(1), 67-73. https://doi.org/10.1016/j.jhep.2013.02.019

@article{eb39757389154f9faa3791d455aaa5d8,

title = "The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort",

abstract = "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.",

keywords = "Adult, Age Factors, Aged, Cohort Studies, Cross-Sectional Studies, Fatigue, Female, Great Britain, Humans, Liver Cirrhosis, Biliary, Liver Transplantation, Male, Middle Aged, Phenotype, Quality of Life, Risk Factors, Sex Factors",

author = "Greta Pells and Mells, {George F} and Marco Carbone and Newton, {Julia L} and Bathgate, {Andrew J} and Burroughs, {Andrew K} and Heneghan, {Michael A} and Neuberger, {James M} and Day, {Darren B} and Ducker, {Samantha J} and Sandford, {Richard N} and Alexander, {Graeme J} and Jones, {David E J} and {UK Primary Biliary Cirrhosis (PBC) Consortium}",

note = "Copyright {\textcopyright} 2013. Published by Elsevier B.V.",

year = "2013",

month = jul,

doi = "10.1016/j.jhep.2013.02.019",

language = "English",

volume = "59",

pages = "67--73",

journal = "Journal of Hepatology",

issn = "0168-8278",

publisher = "Elsevier",

number = "1",

}

Pells, G, Mells, GF, Carbone, M, Newton, JL, Bathgate, AJ, Burroughs, AK, Heneghan, MA, Neuberger, JM, Day, DB, Ducker, SJ, Sandford, RN, Alexander, GJ, Jones, DEJ & UK Primary Biliary Cirrhosis (PBC) Consortium 2013, 'The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort', Journal of Hepatology, vol. 59, no. 1, pp. 67-73. https://doi.org/10.1016/j.jhep.2013.02.019

TY - JOUR

T1 - The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort

AU - Pells, Greta

AU - Mells, George F

AU - Carbone, Marco

AU - Newton, Julia L

AU - Bathgate, Andrew J

AU - Burroughs, Andrew K

AU - Heneghan, Michael A

AU - Neuberger, James M

AU - Day, Darren B

AU - Ducker, Samantha J

AU - Sandford, Richard N

AU - Alexander, Graeme J

AU - Jones, David E J

AU - UK Primary Biliary Cirrhosis (PBC) Consortium

PY - 2013/7

Y1 - 2013/7

N2 - 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.

AB - 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.

KW - Adult

KW - Age Factors

KW - Aged

KW - Cohort Studies

KW - Cross-Sectional Studies

KW - Fatigue

KW - Female

KW - Great Britain

KW - Humans

KW - Liver Cirrhosis, Biliary

KW - Liver Transplantation

KW - Male

KW - Middle Aged

KW - Phenotype

KW - Quality of Life

KW - Risk Factors

KW - Sex Factors

U2 - 10.1016/j.jhep.2013.02.019

DO - 10.1016/j.jhep.2013.02.019

M3 - Article

C2 - 23466308

SN - 0168-8278

VL - 59

SP - 67

EP - 73

JO - Journal of Hepatology

JF - Journal of Hepatology

IS - 1

ER -

The impact of liver transplantation on the phenotype of primary biliary cirrhosis patients in the UK-PBC cohort

Abstract

Keywords

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