Primary biliary cirrhosis in adults

Abstract

Primary biliary cirrhosis (PBC) is a chronic, autoimmune, cholestatic liver disease. It is characterized by slow destruction of small intrahepatic bile ducts, impaired biliary secretion and stasis of toxic endogenous bile acids within the liver with progression to liver fibrosis and cirrhosis. It has an increasing prevalence worldwide. It occurs more commonly in women than men at a ratio of 10:1. In most cases, diagnosis relies on a positive antimitochondrial antibody in the context of chronic cholestasis, without the need for a liver biopsy. Ursodeoxycholic acid improves survival even in patients with advanced liver disease. Certain findings such as fatigue, anti-nuclear antibodies, anti-centromere antibodies and the GP210 antinuclear antibody predict a poor outcome. Up to 40% of patients do not respond satisfactorily to ursodeoxycholic acid therapy and should be considered for adjunctive therapies. Several adjunctive and newer therapies are being tested and some appear promising. We provide a review of PBC with a focus on advances in therapies that may impact the management of PBC in the near future.

Keywords::

• adjunctive therapy
• farnesoid X receptor agonist
• obeticholic acid
• primary biliary disease
• ursodeoxycholic acid

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

Key issues

• A better understanding of the pathogenesis of primary biliary cirrhosis (PBC) is needed to devise new therapies.

• Although Ursodeoxycholic acid is effective in most patients, there are still many left with a suboptimal response.

• Newer therapies are being considered on an empirical basis because we do not have a firm understanding of the cause of PBC.

• Several newer approaches hold promise as therapy for these patients and we await the results of trials underway to help establish their value for PBC patients.