Primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis, is a chronic liver disease resulting from progressive destruction of the bile ducts in the liver – called the intrahepatic bile ducts.
Bile produced in your liver travels via these ducts to your small intestine where it aids in the digestion of fat and fat-soluble vitamins (A, D, E and K). When the ducts are destroyed, bile builds up in the liver contributing to inflammation and scarring (fibrosis).
Eventually this can lead to cirrhosis and its associated complications, as scar tissue replaces healthy liver tissue and liver function becomes increasingly impaired.
PBC may progress slowly and many people do not have symptoms, particularly in the early stages of the disease. The most common initial symptoms are fatigue and itching of the skin (pruritus). Other symptoms may include:
Individuals may also complain of having dry mouth and eyes, and bone, muscle and joint pain.
As the disease progresses, symptoms of cirrhosis can develop including:
Thinning of the bones (osteoporosis) leading to fractures is another complication of PBC. While this is more common in late stages of the disease, it can occur earlier as well. In addition, people with cirrhosis are at increased risk for liver cancer (hepatocellular carcinoma).
The cause of this disease is unknown. It may be related to problems in the immune system. Although PBC is technically not a hereditary disease, meaning a disease caused by a specific gene or genetic defect that is passed from parent to child, there appears to be some family link. PBC is more common among siblings and in families where one member has been affected.
Because many people with PBC have no symptoms, the disease is often discovered incidentally due to abnormal results on routine liver blood tests. Once PBC is suspected, a blood test to check for antimitochondrial antibody (AMA) is done. This test is positive in nearly all people with PBC. A liver biopsy, where a small sample of liver tissue is removed with a small needle can help confirm the diagnosis. Imaging studies may be used to rule out other diseases, or to further evaluate patients once they have been diagnosed with PBC.
There is no cure for PBC, however, there are medications that can help slow disease progression and manage symptoms. Ursodiol (brand names Actigall, URSO 250, URSO Forte) is a naturally occurring bile acid (ursodeoxycholic acid or UDCA) that helps move bile out of the liver and into the small intestine. If used early enough, Ursodiol can improve liver function and may keep you from needing, or delay the need for a liver transplant. People with PBC must take this medication every day for life.
UDCA is effective in more than 50 percent of patients, but up to 40 percent of patients do not achieve an adequate reduction in alkaline phosphotase (ALP) or bilirubin with UDCA, while 5-10 percent are unable to tolerate UDCA.
In May 2016, obeticholic acid (brand name Ocaliva) was approved for the treatment of PBC in combination with UDCA in adults with an inadequate response to UDCA, or as a single therapy in adults unable to tolerate UDCA. Obeticholic acid increases bile flow from the liver and suppresses bile acid production in the liver, thus reducing the exposure of the liver to toxic levels of bile acids. Side effects of obeticholic acid may include increased itching and elevations in blood lipids.
Other alternative therapies in patients who are incomplete responders to UDCA include fenofibrate. Medications to suppress the immune system may also be prescribed including prednisone or azathioprine in PBC patients with the “overlap syndrome” with autoimmune hepatitis. Liver transplantation is considered when medical treatment no longer sufficiently controls the disease. When a person has end-stage liver disease, a liver transplant is necessary for survival.
Maintaining a healthy lifestyle can help people with PBC feel better, as well as relieve or prevent some symptoms associated with the disease. Upon diagnosis, your doctor may suggest the following:
Keep in mind that PBC usually advances slowly over a period of years. Many people lead normal lives for years without symptoms, depending on how early the diagnosis is made. And while there is no cure, people are having good results slowing disease progression and living longer without complications by adhering to their medication regimen and maintaining a healthy lifestyle.
Women are nine times more likely than men to develop PBC, meaning that women make up about 90% of PBC cases.
Reference: Primary Biliary Cholangitis (PBC, previously Primary Biliary Cirrhosis). (2016, December 6). Retrieved from http://www.liverfoundation.org/abouttheliver/info/pbc/