Mycophenolate mofetil for maintenance of remission in autoimmune hepatitis in patients resistant to or intolerant of azathioprine

J Hepatol. 2000 Sep;33(3):371-5. doi: 10.1016/s0168-8278(00)80271-8.

Abstract

Background/aim: Azathioprine is standard therapy for maintenance of remission in patients with autoimmune hepatitis. However, approximately 15% of patients are intolerant of therapy and 10% do not respond to it. There is a need for alternative therapies. We describe here the results of mycophenolate mofetil therapy in patients with autoimmune hepatitis.

Patients: We studied seven patients with type 1 AIH (six female). Three were intolerant of azathioprine and had elevated transaminases and liver histology showing active disease despite prednisolone therapy. Four had been on a dose of 2 mg per kg of azathioprine without complete normalisation of ALT, and had liver biopsies showing active disease. All were treated with mycophenolate 1 g bd and were followed for a median of 46 months (21-59). End points were improvement in histological inflammation, ALT and prednisolone dose.

Results: Five of the seven (71%) patients had normal transaminases after 3 months of treatment. The steroid dose fell from a median of 20 mg per day to 2 mg per day at 9 months (p=0.0001) and the hepatic activity index fell from median 11 to 3 (p=0.001) after 7 months of therapy. One patient required dose reduction because of a fall in white cell count. No other adverse effects were seen.

Conclusions: Mycophenolate mofetil is effective and well tolerated in patients with type 1 AIH who are intolerant of, or do not respond to, azathioprine.

MeSH terms

  • Adolescent
  • Adult
  • Alanine Transaminase / blood
  • Anti-Inflammatory Agents / administration & dosage
  • Anti-Inflammatory Agents / therapeutic use
  • Azathioprine / adverse effects*
  • Azathioprine / therapeutic use*
  • Dose-Response Relationship, Drug
  • Drug Resistance
  • Hepatitis, Autoimmune / drug therapy*
  • Hepatitis, Autoimmune / pathology
  • Humans
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Leukocyte Count
  • Liver / pathology
  • Mycophenolic Acid / adverse effects
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use*
  • Prednisone / administration & dosage
  • Prednisone / therapeutic use

Substances

  • Anti-Inflammatory Agents
  • Immunosuppressive Agents
  • Alanine Transaminase
  • Mycophenolic Acid
  • Azathioprine
  • Prednisone