Ursodeoxycholic acid for cystic fibrosis-related liver disease

NICE Quality and Productivity Case Studies
Queen's University Belfast
Publication date:
20 January 2017




NICE summary of Cochrane review conclusions

Evidence does not support the use of ursodeoxycholic acid (UDCA) for the prevention of cystic fibrosis (CF)-related liver disease. Many of the studies performed to date failed to include important outcomes (for example, number of liver transplants), and showed no significant improvement in the normalisation of any single hepatocellular enzyme, increase in weight or improvement in biliary excretion with the use of UDCA. Therefore at present UDCA cannot be recommended.


The ‘Implications for practice’ section of the Cochrane review stated:

‘Evidence of the effectiveness of UDCA is as yet inconclusive. Routine use of UDCA in people with CF cannot, therefore, be recommended. However, in view of these important preliminary results and because of the lack of any other effective intervention to prevent or treat CF-related liver disease, it is essential that a large multicentre RCT of UDCA in people with CF is undertaken.’