To assess the benefit of the UDCA–budesonide combination in association with mycophenolate mofetil (MMF) in patients with primary biliary cirrhosis (PBC) at high risk of developing cirrhosis or liver failure.

Inclusion criteria for this three-year open study were: 1) suboptimal biochemical response to one-year UDCA therapy at 13–15mg/kg/d; 2) significant interface hepatitis without cirrhosis at liver biopsy. Treatment regimen included UDCA (13–15mg/kg/d), budesonide (6mg/d) and MMF (1.5g/d). All patients underwent a control biopsy at three years.

Fifteen patients fulfilled the inclusion criteria. Six patients (41%) normalized biochemistries and seven (47%) had a partial but significant biochemical response, as defined by a serum bilirubin less than 17μmol/L, alanine aminotransferase less than 70UI/L and alkaline phosphatase less than 250UI/L. Histological activity and fibrosis were markedly improved. Side effects were minimal or absent.

Triple therapy with UDCA, budesonide and MMF may provide benefit in non-cirrhotic PBC patients with features of severe disease not responding to UDCA.