Inductivo-Yu I et al. (2007) Mycophenolate mofetil in autoimmune hepatitis patients not responsive or intolerant to standard immunosuppressive therapy. Clin Gastroenterol Hepatol 5: 799–802

Standard immunosuppressive therapy, consisting of prednisone and azathioprine, is ineffective in 10–20% of patients with autoimmune hepatitis. Inductivo-Yu and colleagues, therefore, investigated the biochemical, histological and hematological effects of mycophenolate mofetil (MMF) in autoimmune hepatitis patients who were refractory to standard treatment.

This retrospective study included 15 patients (mean age 60 years, 73% women) who had discontinued treatment with prednisone (with or without azathioprine) because of lack of response (73%) or adverse effects (95%), and were subsequently treated with MMF for at least 1 year. MMF, at an initial dose of 1 g twice daily, either replaced prednisone monotherapy (n = 3) or was administered alongside prednisone instead of azathioprine in patients who initially received combination therapy (n = 12).

At the end of MMF treatment (mean duration 41 months), the mean alanine aminotransferase level decreased significantly (P = 0.03); normal levels were reached in 11 patients. The mean inflammatory score also decreased significantly (P = 0.02), as did the mean Ishak fibrosis score (P = 0.02). There were no significant adverse changes in hematological parameters including white cell count, platelet count and hemoglobin level. There were no differences between patients receiving MMF monotherapy and those receiving MMF plus prednisone, and no infection-related adverse events occurred in any patients during MMF treatment.

The authors conclude that MMF (with or without prednisone) improves histological and biochemical parameters in patients with autoimmune hepatitis who are unresponsive or intolerant to standard therapy, and call for prospective studies to validate their findings.