Primary biliary cholangitis

The prevalence of primary biliary cholangitis (PBC) is up to 35/100,000 in US populations, with a distribution that is heavily skewed towards women (10:1 female-to-male distribution) and those over 45 years of age.

The combination of elevation of serum alkaline phosphatase and a PBC-specific autoantibody (typically anti-mitochondrial 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 ursodeoxycholic acid.

Obeticholic acid is recommended in patients with an inadequate response to ursodeoxycholic acid. The US Food and Drug Administration has restricted the use of obeticholic acid in patients with PBC with advanced cirrhosis because it can cause serious liver injury in this patient population.

Transplantation is an effective treatment for those 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 characterised 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 in PBC patients (classically anti-mitochondrial antibodies) has led to the widely held view that the disease has an autoimmune component to its aetiology.[1]Jones DE. Pathogenesis of primary biliary cirrhosis. Gut. 2007;56:1615-1624. http://www.ncbi.nlm.nih.gov/pubmed/17641080?tool=bestpractice.com