Mycophenolate mofetil for autoimmune hepatitis: a single practice experience.

Autoimmune hepatitis (AIH) is refractory to standard therapy with prednisone and azathioprine in 20% of patients. Investigators are exploring alternative immunosuppressant treatments, including mycophenolate mofetil (MMF). We assessed the outcome of MMF therapy in patients with AIH and related disorders. A retrospective analysis was performed in 16 patients with AIH, immune cholangitis, and overlap syndromes between AIH, primary biliary cirrhosis, and primary sclerosing cholangitis. MMF was used in lieu of azathioprine on account of patients' intolerance to azathioprine, disease that was refractory to prednisone plus azathioprine, or the perceived potency of MMF. With initiation of MMF, median ALT decreased from 81.5 U/l to 42.5 U/l (P = 0.03). Median prednisone dose decreased from 10 mg to 2.5 mg (P = 0.01). Prednisone was completely withdrawn in three patients. Five of 16 patients (31%) achieved biochemical remission, defined as a reduction in ALT from greater than to less than twice normal. Seven additional patients (44%) were maintained in biochemical remission. Two patients had an incomplete response to MMF and two patients experienced treatment failure. MMF was tolerated well in all but one patient, who discontinued the drug on account of paresthesias. MMF may be a safe and effective alternative to azathioprine in patients with AIH and related disorders.