Ursodeoxycholic acid for treatment of primary sclerosing cholangitis: a placebo-controlled trial

Hepatology. 1992 Sep;16(3):707-14. doi: 10.1002/hep.1840160315.


The efficacy and safety of ursodeoxycholic acid for the treatment of primary sclerosing cholangitis were evaluated in a prospective, randomized, double-blind, placebo-controlled trial. Fourteen patients with primary sclerosing cholangitis documented by cholestatic serum enzyme pattern, liver histological appearance and endoscopic retrograde cholangiography were included in the trial. Six patients received ursodeoxycholic acid (13 to 15 mg/kg body wt/day), and eight patients received placebo. Two patients had to be withdrawn from the study, one because of UDCA-related diarrhea and the other because of worsening of the disease during placebo treatment. Patients in the ursodeoxycholic acid group improved significantly during 1 yr of treatment with respect to serum levels of bilirubin (median = -50%), alkaline phosphatase (median = -67%), gamma-glutamyltransferase (median = -53%), AST (median = -54%) and ALT (median = -36%) compared with the placebo group, but not with respect to serum levels of hydrophobic bile acids. During ursodeoxycholic acid treatment, histopathological features also improved significantly, as evaluated by multiparametric score. Expression of human leukocyte antigen class I molecules appeared to be markedly reduced on liver cells after ursodeoxycholic acid treatment. We conclude that ursodeoxycholic acid is beneficial in reducing disease activity in patients with primary sclerosing cholangitis.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Alanine Transaminase / blood
  • Alkaline Phosphatase / blood
  • Aspartate Aminotransferases / blood
  • Chenodeoxycholic Acid / blood
  • Cholangitis, Sclerosing / blood
  • Cholangitis, Sclerosing / drug therapy*
  • Cholangitis, Sclerosing / pathology
  • Cholic Acid
  • Cholic Acids / blood
  • Deoxycholic Acid / blood
  • Diarrhea / chemically induced
  • Double-Blind Method
  • Female
  • Humans
  • Liver / pathology
  • Male
  • Middle Aged
  • Prospective Studies
  • Ursodeoxycholic Acid / therapeutic use*


  • Cholic Acids
  • Deoxycholic Acid
  • Chenodeoxycholic Acid
  • Ursodeoxycholic Acid
  • Aspartate Aminotransferases
  • Alanine Transaminase
  • Alkaline Phosphatase
  • Cholic Acid