title: New therapies for primary biliary cirrhosis.
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.
authors.affiliation: Department of Surgery, Oncology and Gastroenterology, University of Padova, Padova, Italy, annarosa.floreani@unipd.it.
authors.forename: Annarosa
authors.initials: A
authors.lastname: Floreani
authors.forename: Irene
authors.initials: I
authors.lastname: Franceschet
authors.forename: Lisa
authors.initials: L
authors.lastname: Perini
authors.forename: Nora
authors.initials: N
authors.lastname: Cazzagon
authors.forename: M Eric
authors.initials: ME
authors.lastname: Gershwin
authors.forename: Christopher L
authors.initials: CL
authors.lastname: Bowlus
mesh_terms: D001685:Biological Factors / Q000627:therapeutic use; D019819:Budesonide / Q000627:therapeutic use; D002635:Chenodeoxycholic Acid / Q000031:analogs & derivatives / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D058607:Fibric Acids / Q000627:therapeutic use; D006801:Humans; D008105:Liver Cirrhosis, Biliary / Q000188:drug therapy / Q000628:therapy*; D016896:Treatment Outcome
chemical_list: D001685:Biological Factors; D058607:Fibric Acids; C464660:obeticholic acid; D002635:Chenodeoxycholic Acid; D019819:Budesonide