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Lookup NU author(s): Dr Jess Dyson,
Professor Derek Mann,
Professor David Jones
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Primary biliary cirrhosis (PBC) is a chronic immune-mediated liver disease characterized by progressive cholestasis, biliary fibrosis and eventually cirrhosis. It results in characteristic symptoms with marked effects on life quality. The advent of large patient cohorts has challenged the view of PBC as a benign condition treated effectively by the single licensed therapy-ursodeoxycholic acid ( UDCA). UDCA nonresponse or under-response has a major bearing on outcome, substantially increasing the likelihood that liver transplantation will be required or that patients will die of the disease. In patients with high-risk, treatment-unresponsive or highly symptomatic disease the need for new treatment approaches is clear. Evolution in our understanding of disease mechanisms is rapidly leading to the advent of new and re-purposed therapeutic agents targeting key processes. Notable opportunities are offered by targeting what could be considered as the 'upstream' immune response, 'midstream' biliary injury and 'downstream' fibrotic processes. Combination therapy targeting several pathways or the development of novel agents addressing multiple components of the disease pathway might be required. Ultimately, PBC therapeutics will require a stratified approach to be adopted in practice. This Review provides a current perspective on potential approaches to PBC treatment, and highlights the challenges faced in evaluating and implementing those treatments.
Author(s): Dyson JK, Hirschfield GM, Adams DH, Beuers U, Mann DA, Lindor KD, Jones DEJ
Publication type: Review
Publication status: Published
Journal: Nature Reviews Gastroenterology & Hepatology
Print publication date: 01/03/2015
Online publication date: 03/02/2015
Acceptance date: 01/01/1900
ISSN (print): 1759-5045
ISSN (electronic): 1759-5053
Publisher: NATURE PUBLISHING GROUP