Primary biliary cirrhosis: Lessons learned from an organ-specific disease

A. Nishio, E. B. Keeffe, M. Eric Gershwin

Research output: Contribution to journalArticlepeer-review

3 Scopus citations


Primary biliary cirrhosis is an autoimmune liver disease that predominantly affects women and is characterized by chronic progressive destruction of small intrahepatic bile ducts with portal inflammation and subsequent fibrosis. The serological hallmark is the presence of antimitochondrial antibodies, which are found in 95% of patients. These antibodies are directed against the 2-oxo-acid dehydrogenase complexes located on the inner membrane of mitochondria. Although the role of antimitochondrial antibodies in the pathogenesis is unknown, the presence of antibodies has allowed detailed immunological definition of the antigenic epitopes, the autoantibodies, and the T-cell response. Theories have been proposed regarding the mechanism of immune-mediated bile duct damage in primary biliary cirrhosis, including the possible role of T-cell-mediated cyto-toxicity and molecular mimicry. Primary biliary cirrhosis is usually diagnosed based on the triad of elevated alkaline phosphatase, antimitochondrial antibodies, and characteristic histological changes on liver biopsy. Biochemical liver abnormalities are consistent with the presence of cholestasis and include an elevation of both serum alkaline phosphatase and Υ-glutamyl transpeptidase, with or without elevation of aminotransferase levels. Ursodeoxycholic acid, a dihydroxy bile acid, appears to be the only effective therapy in preventing or delaying the need for liver transplantation. However, a number of patients receiving ursodeoxycholic acid still develop progressive disease and require transplantation; at present, liver transplantation is the only effective therapy for end-stage primary biliary cirrhosis.

Original languageEnglish (US)
Pages (from-to)165-178
Number of pages14
JournalClinical and Experimental Medicine
Issue number4
StatePublished - 2001


  • Antimitochondrial antibodies
  • Autoimmunity
  • Cholestatic liver disease
  • Primary biliary cirrhosis

ASJC Scopus subject areas

  • Medicine(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Clinical Biochemistry


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