Primary sclerosing cholangitis: epidemiology, natural history, and prognosis

Abstract

Primary sclerosing cholangitis is a progressive disease associated with significant morbidity and mortality that continues to draw attention from investigators worldwide. Epidemiologic studies indicate that its annual incidence remains stable, although more asymptomatic cases are being diagnosed. Nevertheless, even asymptomatic cases can progress to end-stage liver disease, albeit at a highly variable rate. Moreover, patients are predisposed to several malignancies, especially cholangiocarcinoma, which contributes to their decreased survival. On the other hand, a subgroup of patients with the so-called small-duct variant may have a better prognosis, and those with overlap syndromes with autoimmune hepatitis may respond well to immunosuppressants. Otherwise, efficient treatment is not available for patients with primary sclerosing cholangitis. Thus, even though it occurs rarely, this disease is commonly an indication for liver transplantation. Fortunately, transplantation outcomes are excellent, with overall 5-year survival rates of approximately 80%.