Primary sclerosing cholangitis (PSC) is a rare progressive disease of the liver characterized by cholestasis and bile duct fibrosis, leading to decreased quality of life, cirrhosis, and the need for liver transplantation within an average of 12 years. The disease occurs in children and adults, and is closely linked with inflammatory bowel disease (IBD), most often ulcerative colitis (UC). The clinical presentation is variable. Typical symptoms include right upper quadrant abdominal discomfort, fatigue, pruritus, and weight loss. The most frequent abnormal physical exam findings are jaundice, hepatomegaly, and splenomegaly. The pathogenesis of PSC remains unclear but hypotheses include genetic factors lymphocyte recruitment and activation, portal bacteraemia, and bile salt toxicity. Ursodeoxycholic acid (UDCA) is widely used for PSC patients and UDCA can lead to improvements in serum hepatobiliary enzymes. UDCA has been tested alone and in combination with corticosteroids or other immunosuppressant agents. However, there is no medical treatment for PSC with enough evidence to show a delay in disease progression, and new therapy is needed.
Category
Digestive system disease
Brite
Human diseases in ICD-11 classification [BR:br08403]
13 Diseases of the digestive system
Diseases of liver
DB96 Autoimmune liver disease
H01684 Primary sclerosing cholangitis
Comment
See also H01227 Inflammatory bowel disease (IBD) and H01466 ulcerative colitis (UC).
Mizuno S, Hirano K, Tada M, Yamamoto K, Yashima Y, Yagioka H, Kawakubo K, Ito Y, Kogure H, Sasaki T, Arizumi T, Togawa O, Matsubara S, Nakai Y, Sasahira N, Tsujino T, Isayama H, Kawabe T, Omata M, Koike K
Title
Bezafibrate for the treatment of primary sclerosing cholangitis.
