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Primary biliary cholangitis (PBC) is a chronic, progressive liver disease in which the bile ducts become inflamed and damaged. This leads to the buildup of bile and causes liver problems such as scarring, cirrhosis (scarring and poor liver function), and eventual liver failure. PBC is more common in women. Many people do not have symptoms when they are first diagnosed and may not develop symptoms for several years. Early symptoms may include fatigue (the most common symptom), itchy skin ( pruritus), and abdominal pain. As the disease progresses, people with PBC may develop weakness, nausea, diarrhea, swelling in the legs and feet (edema), bone and joint pain, jaundice, dark urine, and xanthomas. The symptoms of PBC can significantly impair quality of life.
The diagnosis of PBC may involve blood tests, imaging studies (such as X-ray or ultrasound), and sometimes, a liver biopsy. Blood tests may include tests for anti-mitochondrial antibodies (which may confirm the diagnosis), liver function tests, and cholesterol tests. Abnormal blood test results commonly lead to the diagnosis in people with PBC who do not have symptoms.
PBC is considered an autoimmune disease in which the immune system malfunctions and mistakenly attacks a person’s healthy bile duct cells, causing the inflammation and damage. It is thought to be caused by a combination of genetic susceptibility and environmental triggers ( multifactorial inheritance).
The first treatment recommended for people with PBC is ursodiol, also called ursodeoxycholic acid (UDCA), which has been shown to slow disease progression and reduce the need for a liver transplant. Obeticholic acid (OCA) is available as a second-line treatment either in combination with UDCA (in those with an inadequate response to UDCA), or by itself (in those who are not able to tolerate UDCA). The symptoms of PBC typically do not improve with UDCA or OCA, so individual symptoms are treated separately. A liver transplant may be needed when PBC leads to liver failure.
The rate of progression varies greatly among people with PBC, and the disease may progress over many decades before resulting in end-stage liver disease and its complications.
Source: GARD Last updated on 05-01-20
Treatment for primary biliary cholangitis (PBC) is life-long and aims to slow the disease progression and improve the symptoms that reduce quality of life. The first-line treatment for PBC is long-term use of ursodiol, also called ursodeoxycholic acid (UDCA). This medication, which is approved by the Food and Drug Administration (FDA) for the treatment of PBC, has been shown to slow disease progression and reduce the need for a liver transplant. Liver function should be monitored periodically to help detect people who do not adequately respond to UDCA. Because UDCA therapy does not improve fatigue, itching (pruritus), bone problems, and many other signs and symptoms of PBC, these should be addressed separately.
Obeticholic acid (OCA), a second-line treatment for PBC, is also approved by the FDA. OCA may be used along with UDCA in people who have an inadequate response after at least a year of treatment. It may also be used as a single treatment in people who cannot tolerate UDCA. OCA is used to increase the flow of bile from the liver. Studies investigating the effect that OCA has on survival of people with PBC are ongoing, but data suggest that its use in combination with UDCA may lower the risk for liver complications and liver-related deaths. OCA is not recommended in people with PBC who have advanced liver disease (decompensated cirrhosis). Like UCDA, OCA typically does not improve symptoms of PBC.
Fibrates, medications currently approved by the FDA as lipid-lowering medications, are being studied as a promising new drug for PBC and may improve liver function as well as pruritus. Like OCA, the use of fibrates is also discouraged in people with decompensated liver disease.
Because the symptoms of PBC do not typically improve with UDCA or OCA treatment, they are treated separately. Management of pruritus may include lifestyle modifications (such as avoiding tight clothing and using moisturizers) and/or medications such as cholestyramine.
Liver transplantation may be successful in people who have the transplant before liver failure occurs.
Details regarding treatment guidelines for PBC are available from the American Association for the Study of Liver Diseases.
Last updated on 05-01-20
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