Journal
NATURE REVIEWS GASTROENTEROLOGY & HEPATOLOGY
Volume 12, Issue 3, Pages 147-158Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nrgastro.2015.12
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Funding
- UK Medical Research Council [L001489]
- Medical Research Council [G0500020, MR/L001489/1, MR/K001949/1, MR/K501037/1, MC_PC_12039] Funding Source: researchfish
- National Institute for Health Research [NF-SI-0513-10091, IS-BRC-0211-10046, NF-SI-0512-10080] Funding Source: researchfish
- NIHR Newcastle Biomedical Research Centre [BH141402, BH134330, BH121729, BH137357] Funding Source: researchfish
- MRC [MC_PC_12039, G0500020, MR/K001949/1, MR/L001489/1] Funding Source: UKRI
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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.
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