New research indicates that mycophenolate mofetil, a drug that is usually used to prevent rejection after kidney, heart or liver transplant, seems safe and effective in treating autoimmune hepatitis (AIH), a serious chronic liver disease that mainly affects women.

Treatment for AIH is usually based on steroids, which can have very serious side effects when taken long term either alone or in combination with the immunosuppressive drug azathioprine. In this latest real-world study, nearly 94% of patients had an initial complete response to mycophenolate mofetil mostly within 3 months of treatment. A total of 78 of 109 patients (72%) had a complete response on-treatment, and 61 of 78 (78%) maintained remission off steroids. Most importantly, mycophenolate mofetil as front-line treatment for AIH not only accomplished high rates of on-treatment response, but also showed the highest rates of maintenance of complete remission after complete drug withdrawal (75% of patients) ever published, for a median of 2 years.

"As relapse after drug withdrawal in AIH patients is almost universal with conventional therapy, mycophenolate mofetil seems a reasonable, safe, and important alternative first-line treatment of AIH that should seriously and urgently be considered in the future," said Dr. George Dalekos, senior author of the Alimentary Pharmacology & Therapeutics study.

Study: A real-world study focused on the long-term efficacy of mycophenolate mofetil as first-line treatment of autoimmune hepatitis, Zachou, K., Gatselis, N. K., Arvaniti, P., Gabeta, S., Rigopoulou, E. I., Koukoulis, G. K. and Dalekos, G. N., Alimentary Pharmacology & Therapeutics, doi: 10.1111/apt.13584, published online 16 March 2016.