A single center review of the use of mycophenolate mofetil in the treatment of autoimmune hepatitis.

Background & Aims: Standard treatment for autoimmune hepatitis (AIH) involves immune suppression by using prednisone alone or in combination with azathioprine (AZA). Although this regimen achieves remission in approximately 80%, some patients are intolerant or do not respond. Mycophenolate mofetil (MMF) is a potent immunosuppressant. However, its utility in AIH is not well-defined.

Methods: We performed a retrospective longitudinal analysis of patients with AIH.

Results: We identified 128 patients with AIH: mean age, 42.8 years; 83% female; 69% white. At presentation, median AST and ALT were 227 and 261 U/L, respectively, and bridging fibrosis and cirrhosis were present in 38% and 22%, respectively. Overall, 29 patients received MMF; 12 were switched to MMF after intolerance or nonresponse to prednisone +/- AZA, whereas 17 received MMF +/- prednisone as initial therapy. The main reasons for switching to MMF were nausea/vomiting (n = 4) and failure to normalize liver enzymes (n = 3). Ten of the 29 patients who received MMF therapy (34%) discontinued MMF as a result of side effects. Sixteen (84%) of the remaining 19 patients on MMF achieved remission, which closely matched the remission rate of those who remained on prednisone +/- AZA (82%). The only independent clinical factor that predicted the eventual need for the use of MMF was absence of cirrhosis (P = .0067).

Conclusions: (1) MMF was associated with a high rate of intolerance (34%). (2) In those who could tolerate it, it was associated with a high rate of remission (84%). (3) Absence of cirrhosis on presentation was the only independent factor associated with eventual need for MMF.