High dose ursodeoxycholic acid for the treatment of primary sclerosing cholangitis is safe and effective

J Hepatol. 2008 May;48(5):792-800. doi: 10.1016/j.jhep.2007.12.023. Epub 2008 Feb 14.


Background/aims: Ursodeoxycholic acid (UDCA) has been shown to improve serum liver tests in primary sclerosing cholangitis (PSC), but controlled trials have shown inconsistent effects on liver histology, and did not reveal a survival benefit. This pilot, randomised dose-ranging trial attempted to determine whether further enrichment of the bile acid pool with UDCA would lead to an improvement in outcome for PSC patients.

Methods: Thirty-one patients with PSC were randomised to treatment with either 10 mg/kg (low dose), 20 mg/kg (standard dose) or 30 mg/kg (high dose) daily of UDCA for 2 years. Patients were assessed every 12 weeks and underwent liver biopsy at the beginning and end of the trial.

Results: Serum liver tests improved in all groups taking UDCA. Survival probability at 1-4 years as evaluated by the Mayo risk score tended to improve for all patients and significantly improved for the high dose group (p<0.02). Only 3 (10%) of all patients had a Ludwig score showing histological deterioration over the trial period.

Conclusions: High dose UDCA is well-tolerated and is associated with an improvement in survival probability. A trend towards stability/improvement in histological stage was also observed. This treatment appears to be effective for PSC and deserves further evaluation.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Cholangitis, Sclerosing / drug therapy*
  • Cholangitis, Sclerosing / mortality
  • Cholangitis, Sclerosing / pathology
  • Dose-Response Relationship, Drug
  • Female
  • Humans
  • Male
  • Middle Aged
  • Survival Rate
  • Ursodeoxycholic Acid / adverse effects
  • Ursodeoxycholic Acid / therapeutic use*


  • Ursodeoxycholic Acid