Evidence-based treatment of primary biliary cirrhosis.

The natural history of primary biliary cirrhosis (PBC) has improved greatly during the past two decades because of its diagnosis at earlier stages and the widespread use of ursodeoxycholic acid as first-line treatment. As a result, far fewer patients require liver transplantation and patients diagnosed with early-stage disease appear to have a normal life expectancy. The evidence-based treatment of PBC is reviewed here. The challenge of designing and executing high-quality clinical trials as well as lessons from past clinical trials are discussed. The rationales for the use of budesonide and new investigational therapies (PPAR and FXR agonists) as well as their therapeutic potential in light of recent available data are also reviewed. An algorithm of the management of PBC patients responding or not to ursodeoxycholic acid is proposed.