Background: Cystic fibrosis-related liver disease peaks in adolescence with up to 20% of patients developing chronic liver disease. Early changes in the liver may ultimately result in end stage liver disease with patients needing transplantation. One therapeutic option currently used is ursodeoxycholic acid.
Objectives: To assess the effectiveness of ursodeoxycholic acid in cystic fibrosis liver disease.
Search strategy: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group trials register, and contacted drug companies. Date of the most recent search of the Group's specialised register: June 1999.
Selection criteria: Randomised and quasi-randomised trials of use of ursodeoxycholic acid for at least three months compared with placebo or no additional treatment in people with cystic fibrosis.
Data collection and analysis: Two reviewers independently assessed trial eligibility and quality.
Main results: Three trials involving 118 patients were included. The complex study design used in two trials meant that data could only be analysed for subsets of patients. There was no difference in weight change (weighted mean difference -0.496, 95% confidence interval -1.545 to +0.553) based on 30 patients from two trials. Improvement in biliary excretion was reported in only one study and no significant change after treatment was shown. Long term outcomes such as death or need for liver transplantation were not reported.
Reviewer's conclusions: There are few trials assessing the effectiveness of ursodeoxycholic acid. There is insufficient evidence to justify is its routine use in cystic fibrosis.