Primary sclerosing cholangitis is a chronic, progressive cholangiopathy that frequently affects men and is associated with inflammatory bowel disease. Although the cause of the disease is still debated, a genetic association and link to immune-mediated disease triggered by environmental factors are thought to contribute. The disease can present as isolated imaging abnormalities, biochemical changes, cholangiocarcinoma, or end-stage complications such as cirrhosis. Symptoms of primary sclerosing cholangitis include fatigue, jaundice, pruritus, or steatorrhoea. Differentiation of primary sclerosing cholangitis can be challenging because other chronic cholangiopathies can present similarly; however, the distinction is necessary to optimise disease surveillance. Management involves assessment for comorbid inflammatory bowel disease and exclusion of other associated cholangiopathic disorders. Patients with primary sclerosing cholangitis have a poor prognosis; progression to liver cirrhosis is common, and an increased risk of hepatobiliary and colorectal cancers is present in those with inflammatory bowel disease. Although much research involves locating an active therapy that can alter the disease course, the only available treatment is liver transplantation, and risk for disease recurrence remains. Use of ursodeoxycholic acid can improve alkaline phosphatase and bilirubin concentrations but does not alter the disease course. In this Review, we summarise aetiological theories, provide an update on hepatobiliary malignancies that require surveillance, and discuss exciting areas of investigation for potential treatment.
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