title: Efficacy and safety of ursodeoxycholic acid in primary, type IIa or IIb hypercholesterolemia: a multicenter, randomized, double-blind clinical trial.
abstract: BACKGROUND
  Ursodeoxycholic acid (UDCA) is a therapeutic bile acid used in dissolution of gallstones and treatment of several cholestatic liver diseases. Results obtained from primary biliary cirrhosis patients treated with UDCA suggested that this agent exerts significant cholesterol-lowering effects and justifies evaluation in primary hypercholesterolemic patients without liver disease. Purpose of this study was to determine whether UDCA had potential to be an effective, safe cholesterol-lowering agent in primary type IIa or IIb hypercholesterolemia.
  
  
  METHODS
  This was a multicenter randomized, double blind, placebo-controlled trial. After a 6-week placebo lead-in period during which two qualifying lipid profiles were obtained, patients with a mean serum LDL-cholesterol (LDL-C) between 130 and 190mg/dL, triglycerides <400mg/dL and HDL-cholesterol >30mg/dL were randomized to UDCA or matching placebo for 24 weeks.
  
  
  RESULTS
  Seven sites screened 200 patients with 134 patients meeting the entry criteria who were randomized to the two treatments. There were 125 patients meeting the efficacy evaluation criteria, 57 on UDCA and 68 on placebo. LDL-C change from weeks 0 to 24 showed no significant difference between groups. No significant differences in changes for total cholesterol, HDL-cholesterol and triglycerides were observed. Both groups had similar adverse event profiles.
  
  
  CONCLUSIONS
  UDCA did not show intrinsic cholesterol-lowering properties and therefore is not a useful therapy in treating type IIa or type IIb hypercholesterolemic patients. UDCA was confirmed as a well tolerated and safe drug in this population.
authors.affiliation: St. Michael's Hospital, University of Toronto, ON, Canada.
authors.forename: Manoela F B
authors.initials: MF
authors.lastname: Braga
authors.forename: Michael G A
authors.initials: MG
authors.lastname: Grace
authors.forename: Jacques
authors.initials: J
authors.lastname: Lenis
authors.forename: Frank P
authors.initials: FP
authors.lastname: Kennedy
authors.forename: Avery L
authors.initials: AL
authors.lastname: Teplinsky
authors.forename: Ghislaine
authors.initials: G
authors.lastname: Roederer
authors.forename: Pasquale J
authors.initials: PJ
authors.lastname: Palumbo
authors.forename: Patrick
authors.initials: P
authors.lastname: Colin
authors.forename: Lawrence A
authors.initials: LA
authors.lastname: Leiter
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002784:Cholesterol / Q000378:metabolism; D008078:Cholesterol, LDL / Q000378:metabolism; D015243:Cholesterol, VLDL / Q000378:metabolism; D004311:Double-Blind Method; D006801:Humans; D006937:Hypercholesterolemia / Q000188:drug therapy*; D008875:Middle Aged; D010919:Placebos; D013997:Time Factors; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use*
chemical_list: D008078:Cholesterol, LDL; D015243:Cholesterol, VLDL; D010919:Placebos; D014580:Ursodeoxycholic Acid; D002784:Cholesterol