Primary biliary cholangitis: Old and novel therapy

Eur J Intern Med. 2018 Jan:47:1-5. doi: 10.1016/j.ejim.2017.06.020. Epub 2017 Jun 29.

Abstract

Primary biliary cholangitis (PBC), formerly called primary biliary cirrhosis, is a chronic cholestatic liver disease that progresses slowly to end-stage liver disease. The first Food and Drug Administration (FDA)-approved treatment for PBC was ursodeoxycholic acid (UDCA). This treatment slows the progress of the disease, but approximatively 30-40% of patients fail to respond to UDCA. A number of options are under investigation as second line treatment. Obeticholic acid (OCA), a Farnesoid X Receptor agonist, has been approved in May 2017 by FDA for patients non responders or intolerant to UDCA. The results of a randomized, double blind, phase 3 study of OCA (mg or 10mg) compared to placebo, showed that approximatively 50% of patients reached a significant reduction in serum alkaline phosphatase, a marker predictive of disease progression, liver transplantation or death. Other emerging therapies include: agents targeting fibrosis, inflammation, or immunological response. Indeed, after 30years of UDCA therapy as unique choice for PBC patients, a number of targets, derived from a deeper knowledge of the pathophysiology of the disease, has been discovered and they offer different and new therapeutic approaches that are now under evaluation.

Keywords: Budesonide; Fibrates; Obeticholic acid; Primary biliary cholangitis; Primary biliary cirrhosis; Rituximab; Treatment; Ursodeoxycholic acid; Ustekinumab.

Publication types

  • Review

MeSH terms

  • Budesonide / therapeutic use*
  • Chenodeoxycholic Acid / adverse effects
  • Chenodeoxycholic Acid / analogs & derivatives*
  • Chenodeoxycholic Acid / therapeutic use
  • Disease Progression*
  • End Stage Liver Disease / etiology
  • Humans
  • Liver Cirrhosis, Biliary / complications
  • Liver Cirrhosis, Biliary / therapy*
  • Liver Transplantation
  • Randomized Controlled Trials as Topic
  • Ursodeoxycholic Acid / adverse effects
  • Ursodeoxycholic Acid / therapeutic use*

Substances

  • obeticholic acid
  • Chenodeoxycholic Acid
  • Budesonide
  • Ursodeoxycholic Acid