Pediatric Primary Sclerosing Cholangitis

Abstract

Primary sclerosing cholangitis (PSC) is a rare disorder of the hepatobiliary system characterized by chronic diffuse inflammation and obliterative fibrosis of the intrahepatic and/or extrahepatic bile ducts that subsequently progresses to liver cirrhosis and end-stage liver disease in the majority of patients. The estimated incidence and prevalence of pediatric PSC in the United States is 0.2 and 1.5 cases per 100,000 children. The majority of cases of pediatric PSC are symptomatic at presentation and common manifestations include fatigue, abdominal pain, anorexia, and pruritus. Approximately 30–40 % of pediatric PSC patients will suffer from consequences of chronic biliary disease, including significant pruritus, recurrent bacterial cholangitis, and complications of portal hypertension. Unique aspects of pediatric PSC include a high incidence of autoimmune sclerosing cholangitis (PSC-autoimmune hepatitis overlap syndrome) and small duct PSC. Cholangiocarcinoma is an extremely rare complication of pediatric PSC. There is no known therapy to prevent progression of pediatric PSC; however further research on the effectiveness of ursodeoxycholic acid and oral vancomycin therapies is warranted. Liver transplantation is a viable option for end-stage liver disease secondary to pediatric PSC and accounts for ~2 % of all pediatric liver transplants in the United States.