Ursodeoxycholic acid for cystic fibrosis‐related liver disease
Abstract
Background
Cystic fibrosis‐related liver disease peaks in adolescence with up to 20% of people with cystic fibrosis developing chronic liver disease. Early changes in the liver may ultimately result in end‐stage liver disease with people needing transplantation. One therapeutic option currently used is ursodeoxycholic acid.
Objectives
To analyse evidence that ursodeoxycholic acid improves indices of liver function, reduces the risk of developing chronic liver disease and improves outcomes in general in cystic fibrosis.
Search methods
We searched the Cochrane CF and Genetic Disorders Group Trials Register comprising references identified from comprehensive electronic database searches, handsearches of relevant journals and abstract books of conference proceedings. We also contacted drug companies.
Date of the most recent search of the Group's trials register: 10 July 2012.
Selection criteria
Randomised controlled trials of the 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 authors independently assessed trial eligibility and quality.
Main results
Ten trials have been identified, of which three trials involving 118 participants were included. The complex design used in two trials meant that data could only be analysed for subsets of participants. There was no significant difference in weight change, mean difference ‐0.90 kg (95% confidence interval ‐1.94 to 0.14) based on 30 participants from two trials. Improvement in biliary excretion was reported in only one trial and no significant change after treatment was shown. Long‐term outcomes such as death or need for liver transplantation were not reported.
Authors' conclusions
There are few trials assessing the effectiveness of ursodeoxycholic acid. There is insufficient evidence to justify its routine use in cystic fibrosis.
PICOs
Plain language summary
Ursodeoxycholic acid for liver disease related to cystic fibrosis
Problems of bile composition and flow cause liver disease for up to 20% of young people with cystic fibrosis. Ursodeoxycholic acid is a naturally occurring bile acid given to try and prevent liver disease in cystic fibrosis. We searched for trials of this treatment which lasted for at least three months. We were able to include three trials with 118 participants. The complex design of two trials meant data could not be analysed for all the participants. There were no significant differences in any of the outcomes measured. Only one trial measured long‐term outcomes such as the need for liver transplant or death. The results were not published per treatment group, only for all participants combined, so we can not draw any firm conclusions from the data. The review did not find enough evidence from trials about the effects of using ursodeoxycholic acid. Current research shows that adverse effects are rare, but there are not enough data about long‐term use. As there is no other preventive treatment, more research on ursodeoxycholic acid is needed.
