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Partial Bile Duct Ligation in the Mouse: A Controlled Model of Localized Obstructive Cholestasis
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Emerging therapies for PBC.

David Maxwell Hunter Chascsa1,2, Keith Douglas Lindor3

  • 1Department of Gastroenterology and Hepatology, Mayo Clinic, MD 5777 E. Mayo Blvd, Phoenix, AZ, 85054, USA. chascsa.david@mayo.edu.

Journal of Gastroenterology
|January 24, 2020
PubMed
Summary
This summary is machine-generated.

New therapies are needed for primary biliary cholangitis, a serious liver disease. Fibrates show promise, improving surrogate and clinical endpoints for patients with this uncommon condition.

Keywords:
FibrateLiver diseaseObeticholic acidPrimary biliary cholangitisUrsodeoxycholic acid

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Area of Science:

  • Hepatology
  • Cholestatic Liver Diseases
  • Drug Development

Background:

  • Primary biliary cholangitis (PBC) is a rare cholestatic liver disease primarily affecting middle-aged women.
  • Untreated PBC can lead to end-stage liver disease, with limited therapeutic options currently available.
  • Ursodeoxycholic acid (UDCA) and obeticholic acid (OCA) are approved treatments, but many patients lack adequate response.

Purpose of the Study:

  • To evaluate the potential of new therapeutic agents for primary biliary cholangitis.
  • To address the challenges in evaluating PBC therapies, including disease heterogeneity and lengthy endpoint development.

Main Methods:

  • Review of current and emerging treatment strategies for PBC.
  • Analysis of clinical trial data assessing surrogate and clinical endpoints for PBC therapies.
  • Focus on the evaluation of fibrates as a novel therapeutic class.

Main Results:

  • Fibrates have demonstrated beneficial effects on surrogate endpoints in PBC.
  • Emerging data suggest that fibrates are beginning to show improvements in clinical endpoints.
  • Challenges in PBC trial evaluation include disease heterogeneity and the need for standardized response criteria.

Conclusions:

  • Fibrates represent a promising new therapeutic avenue for primary biliary cholangitis.
  • Further research and long-term data are necessary to fully establish the role of fibrates in PBC management.
  • Addressing evaluation challenges is crucial for accelerating the development of effective PBC treatments.