Mycophenolate mofetil treatment in patients with autoimmune hepatitis failing standard therapy with prednisolone and azathioprine

Background

Data on rescue treatment of autoimmune hepatitis in patients that fail standard treatment are sparse.

Aims

To report our long-term experience with mycophenolate mofetil.

Methods

Retrospective study in 22 patients with autoimmune hepatitis who failed azathioprine and prednisolone due to adverse events (n?=?14, 64%), lack of remission (n?=?5, 23%) or a combination (n?=?3, 13%).

Results

Mycophenolate mofetil was started at a dose of 20?mg/kg/day and increased to a maximum of 3?g/day. Follow-up was 0–6 months in 7 patients; more than 12 months in 15 (68%) and more than 24 months in 10. Normal aminotransferase levels were obtained (n?=?3) or maintained (n?=?7) in 10 patients (45%) after three to 30 weeks. 12 patients (55%) were withdrawn during the first 6 months, due to adverse events. Three patients were switched to cyclosporine and one underwent liver transplantation. Successful treatment with mycophenolate mofetil continued in 10 patients (45%) for a median of 71 months (range 20–124). Of these, one stopped prednisolone, five have a prednisolone dose <5?mg daily and four patients 5–10?mg.

Conclusion

Approximately one of two patients with autoimmune hepatitis that fail standard treatment benefit from long-term maintenance with mycophenolate mofetil, especially those with previous intolerance to thiopurines, where mycophenolate mofetil is effective in two thirds.