Triple therapy with ursodeoxycholic acid, budesonide and mycophenolate mofetil in patients with features of severe primary biliary cirrhosis not responding to ursodeoxycholic acid alone

Background/Aims. - 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. Methods. - Inclusion criteria for this three-year open study were: 1) suboptimal biochemical response to one-year UDCA therapy at 13-15 mg/kg/d; 2) significant interface hepatitis without cirrhosis at liver biopsy. Treatment regimen included UDCA (13-15 mg/kg/d), budesonide (6 mg/d) and MMF (1.5 g/d). All patients underwent a control biopsy at three years. Results. - Fifteen patients fulfilled the inclusion criteria. Six patients (41%) normalized bio-chemistries and seven (47%) had a partial but significant biochemical response, as defined by a serum bilirubin less than 17 mu mol/L, alanine aminotransferase less than 70 Ul/L and alkaline phosphatase less than 250 Ul/L. Histological activity and fibrosis were markedly improved. Side effects were minimal or absent. Conclusions. - Triple therapy with UDCA, budesonide and MMF may provide benefit in noncirrhotic PBC patients with features of severe disease not responding to UDCA. (C) 2010 Elsevier Masson SAS. All rights reserved.