Primary biliary cirrhosis in 2014.

PURPOSE OF REVIEW: Primary biliary cirrhosis (PBC) was first described in the 1950s as a clinical syndrome of progressive cholestatic liver disease resulting from chronic inflammatory destruction of the intrahepatic bile ducts. In the 1980s, the autoimmune nature of the disease was appreciated with the discovery of disease-specific loss of immune tolerance to the pyruvate dehydrogenase complex and subsequent development of antimitochondrial antibodies and autoreactive T cells. Then, in the 1990s, multiple clinical trials demonstrating the efficacy of ursodiol as a treatment for PBC were published, although it has been clear that ursodiol is not a cure and only delays progression in some patients.

RECENT FINDINGS: The study of PBC in the 2000s has been buoyed by two basic science advances: rapid sequencing technologies that have led to genome wide association studies, and elucidation of the role of nuclear hormone receptors in the regulation of bile salt metabolism, which has led to novel therapies under study for cholestatic diseases.

SUMMARY: Today's clinician should be able to determine which patients with PBC are likely to progress despite treatment with ursodiol and understand the putative new bile acid and immunosuppressant treatment strategies under development, as well as be aware of the recently described genetic factors at play in the development of PBC.