New Therapies for Primary Biliary Cirrhosis

Annarosa Floreani, Irene Franceschet, Lisa Perini, Nora Cazzagon, M. Eric Gershwin, Christopher Bowlus

Research output: Contribution to journalArticle

23 Scopus citations

Abstract

Primary biliary cirrhosis (PBC) is a rare inflammatory liver disease for which ursodeoxycholic acid (UDCA) is the only therapy approved by the U.S. Food and Drug Administration. Patients with a biochemical response to UDCA therapy have a similar survival rate compared to the general population. However, up to 40 % of PBC patients do not achieve a complete response to UDCA, have an increased risk of liver-related death and liver transplantation, and represent a persistent medical need for new therapies. Several novel drugs have recently been studied and show potential efficacy in PBC. Obeticholic acid, a farnesoid X receptor agonist, has been tested in phase II trials and initial results after 1 year in a phase III international trial suggest that it may be effective in achieving a biochemical response in approximately 40 % of patients who do not completely respond to UDCA. Several small studies on fibrates have suggested that they may have efficacy, but larger studies are needed. Surprisingly, results of immunomodulators and biologics have not yet been able to demonstrate efficacy, but new approaches have shown promise in animal models and their translation to human clinical trials are awaited.

Original languageEnglish (US)
Pages (from-to)263-272
Number of pages10
JournalClinical Reviews in Allergy and Immunology
Volume48
Issue number2-3
DOIs
StatePublished - Jun 1 2015

    Fingerprint

Keywords

  • Anti-TNF-α
  • Budesonide
  • CXCL10 monoclonal antibody
  • Fibrates
  • Obeticholic acid
  • Primary biliary cirrhosis
  • Ursodeoxycholic acid

ASJC Scopus subject areas

  • Immunology and Allergy

Cite this