Primary biliary cholangitis

Primary biliary cholangitis (PBC) is characterized by progressive intrahepatic bile duct damage and loss.

PBC is significantly more common in women than in men. Peak incidence is around age 40 years, and median age at diagnosis is 65 years.

The combination of a cholestatic pattern of serum liver tests (elevated alkaline phosphatase, gamma-glutamyl transferase, or both) and a PBC-specific autoantibody (typically antimitochondrial antibody) is sufficient for diagnosis in most patients, with no need for biopsy confirmation.

PBC is progressive in most patients; although, in many people, the rate of progression can be so slow that it may not be clinically relevant. Cirrhosis and its typical complications arise in the end stage.

Symptoms (typically pruritus and fatigue) can significantly lower the quality of life, even in patients with a very slowly progressive disease. These symptoms warrant treatment in their own right using specific regimens.

Progression of the disease can be slowed by therapy with ursodiol.

In patients with an inadequate response to ursodiol, second-line options include elafibranor and seladelpar.

Transplantation is an effective treatment for patients who develop end-stage liver disease with PBC.

Primary biliary cholangitis (PBC) is a chronic disease of the small intrahepatic bile ducts that is characterized by progressive bile duct damage (and eventual loss) occurring in the context of chronic portal tract inflammation. Fibrosis develops as a consequence of the original insult and the secondary effects of toxic bile acids retained in the liver, resulting ultimately in cirrhosis. The almost universal presence of autoantibodies (classically antimitochondrial antibodies) has led to the widely held view that PBC is an autoimmune disease.[1]Jones DE. Pathogenesis of primary biliary cirrhosis. Gut. 2007;56:1615-1624. http://www.ncbi.nlm.nih.gov/pubmed/17641080?tool=bestpractice.com