Treatment options for primary sclerosing cholangitis.
Primary sclerosing cholangitis is a cholestatic liver disease characterized by inflammation and fibrosis of intra-/extrahepatic bile ducts, leading to multifocal strictures. Primary sclerosing cholangitis exhibits a progressive course resulting in cirrhosis and the need for liver transplantation over a median period of 12 years. The disease is frequently associated with inflammatory bowel disease and carries an increased risk of colorectal cancer and cholangiocarcinoma. Despite extensive research, there is currently no effective medical treatment. Multiple drugs are shown to be ineffective in halting disease progression, including ursodeoxycholic acid, the most widely evaluated drug. High-dose ursodeoxycholic acid (28-30 mg/kg/day) was recently shown to increase the adverse events rate. Endoscopic or radiological dilatation of a 'dominant' stricture may lead to symptomatic and biochemical improvement. However, liver transplantation is the only life-prolonging treatment for patients with end-stage disease. Studies with promising drugs, such as antibiotics, antifibrotic agents and bile acid derivatives, are eagerly awaited.
Sinakos
Emmanouil
E
Division of Gastroenterology and Hepatology, Mayo Clinic, 200 First Street, SW, Rochester, MN 55905, USA.
Lindor
Keith
K
D001662:Biliary Tract Surgical Procedures; D002756:Cholagogues and Choleretics / Q000627:therapeutic use; D015209:Cholangitis, Sclerosing / Q000150:complications / Q000628:therapy*; D004106:Dilatation; D018450:Disease Progression; D016145:Endoscopy, Digestive System; D005765:Gastrointestinal Agents / Q000627:therapeutic use*; D006801:Humans; D016031:Liver Transplantation; D013997:Time Factors; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use
D002756:Cholagogues and Choleretics; D005765:Gastrointestinal Agents; D014580:Ursodeoxycholic Acid