Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease of unknown etiology characterized by fibrosing inflammation of the intrahepatic and extrahepatic bile ducts, leading ultimately to cirrhosis and death caused by complications from liver failure if liver transplantation is not peformed. Despite a better understanding of the natural history of the disease, no significant breakthroughs have been made into its pathogenesis. Over the past decade and a half, many clinical trials of medical therapy have been conducted; however, none have demonstrated real efficacy. This reflects our lack of understanding into the basic mechanisms of disease pathogenesis in PSC. Progress has been made in the area of orthotopic liver transplantation for PSC, and to some extent in the diagnosis and management of complications, the most grave of which is the development of cholangiocarcinoma. This review highlights the progress that has been made, describes our current deficiencies, and discusses likely developments in the future. The authors also discuss management controversies and provide current practice guidelines where applicable.
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