Primary Biliary Cholangitis: Overview, Diagnosis, and Treatment Options

Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, provides an overview of primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction. Primary biliary cholangitis affects the bile ducts within the liver. The bile ducts carry a fluid called bile from the liver to the gallbladder, where it is stored. The bile ducts become inflamed and damaged, which causes bile to build up in the liver. This abnormal buildup destroys liver tissue and in later stages results in cirrhosis. Primary biliary cholangitis usually occurs between the ages of 40 and 60 and affects women more often than men. The cause of this condition is unknown, but research suggests that it is an autoimmune disease. Ursodiol (Actigall) and obeticholic acid (Ocaliva) are the only drugs approved by the U.S. Food and Drug Administration for the treatment of primary biliary cholangitis.

It is estimated that 90% of people who are diagnosed with PBC are women. Many people who are diagnosed with PBC are between 40-60 years of age. It is important to note, however, that men and young women can also develop PBC.

The most common symptoms of PBC are pruritus, or itching, and fatigue. Some people with PBC can also experience dry eyes and mouth. For people with more advanced PBC, serious liver damage can cause jaundice, or a yellowing of the skin and eyes.

Many people do not experience any symptoms at the beginning of their PBC. The only sign that they have PBC is that abnormalities appear in their liver function test results.

Doctors can test for PBC by taking blood samples and measuring certain chemicals related to liver function. The tests will show if there are elevated levels of alkaline phosphatase (ALP) and if antimitochondrial antibodies (AMAs) are present, which can be indicators of PBC.