Ursodiol is currently the only drug approved by the FDA for the treatment of PBC and it is often the initial therapy for patients. The drug is a “gentler” bile acid than those normally made by the body and is well tolerated. Studies have shown that ursodiol, given in a dose of 13 to 15 mg/kg daily up to four years, delays the time to liver transplantation. In addition to improving blood tests, there are some patients with PBC whose disease appears to become inactive (remission) on ursodiol. Repeat or even serial liver biopsies may be required in order to document remission.
Colchicine is associated with improvement of liver tests in some patients with PBC and improved itching in two recent studies. However, there was no improvement in liver biopsy findings and therefore this medication is not commonly used to treat PBC.
In early studies, methotrexate had appeared to be associated with improvement in symptoms, liver function tests and liver biopsies. However, an 8-year multicenter NIH trial has clearly documented that the use of Methotrexate is not associated with improvements in liver disease and the development of complications of cirrhosis.
Many people with PBC may never develop cirrhosis or liver failure. However, as can be seen in PSC, a late stage of PBC can be liver failure. For those people who reach the late stages of PBC, liver transplantation may be considered. The signs and symptoms of liver failure, which may or (depending on the specific aspects of the symptoms / disease) may not prompt consideration of any potential role for liver transplant, are the following:
- Persistent jaundice
- Fluid retention with swelling of hands, feet and abdomen
- Variceal bleeding (internal bleeding from swollen intestinal blood vessels, commonly in upper stomach and esophagus)
- Bone thinning and/or fractures
- Hepatic coma or encephalopathy
The survival for PBC patients after liver transplantation is excellent although some patients may develop recurrent PBC over time. With better understanding of the nature of PBC and its complications, many patients will have improved outcomes.
Author(s) and Publication Date(s)
Young-Mee Lee, MD, and Marvin Jose Lopez, MD, New England Medical Center, Boston, MA – Published December 2004. Updated January 2010.
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