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Lookup NU author(s): Dr Lin Lee Wong, Dr Vinod Hegade, Professor David Jones
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© 2017 S. Karger AG, Basel. Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterized by chronic cholestasis. Treatment with the accepted primary therapy ursodeoxycholic acid (UDCA) has been shown to be associated with delayed disease progression probably through reduced impact of cholestatic injury on the target biliary epithelial cells. Patients with inadequate response to UDCA (which can be identified through validated biochemical criteria) are at increased risk of disease progression, need for liver transplantation, and death. Obeticholic acid (OCA) is a farnesoid X receptor (FXR) agonist which has been evaluated as a second-line therapy in PBC and has been recently licensed by the Food and Drug Administration and European Medicines Agency for use in patients showing an inadequate response to UDCA or who are unable to tolerate it. Although evidence for biochemical improvement by OCA is compelling, there is, as yet, no evidence that OCA improves hard clinical outcomes or quality of life. In addition, OCA may not be suitable for PBC patients with pruritus as it can worsen the symptom. Other novel agents currently in clinical development may have better side-effect profile. Fibrates have the potential but currently lack high quality evidence to support their routine clinical use in PBC. Symptom management of PBC is challenging and ASBT inhibitors and rituximab are being evaluated for pruritus and fatigue, respectively.
Author(s): Wong LL, Hegade VS, Jones DEJ
Publication type: Article
Publication status: Published
Journal: Digestive Diseases
Year: 2017
Volume: 35
Issue: 4
Pages: 359-366
Online publication date: 03/05/2017
Acceptance date: 02/04/2016
ISSN (print): 0257-2753
ISSN (electronic): 1421-9875
Publisher: S. Karger AG
URL: https://doi.org/10.1159/000467547
DOI: 10.1159/000467547
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