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 study.
Front-line therapy with mycophenolate mofetil (MMF) in autoimmune hepatitis (AIH) has shown high on-treatment remission rates.
To study prospectively in a real-world fashion the long-term outcome of a large group of consecutive treatment-naïve AIH patients.
Between 2000 and 2014, 158 patients were recruited but only 131 were eligible for treatment (109 MMF/prednisolone; 22 prednisolone ± azathioprine). Long-term data on outcome after drug withdrawal were evaluated. Patients stopped treatment after having achieved complete response (normal transaminases and IgG) for at least the last 2 years.
At diagnosis, 31.6% of patients had cirrhosis and 72.8% insidious presentation. A total of 102 of 109 (93.6%) responded initially to MMF within 2 (1–18) months. A total of 78 of 109 (71.6%) had complete response on treatment and 61 of 78 (78.2%) maintained remission off prednisolone. MMF-treated patients had increased probability of complete response compared to those receiving azathioprine (P = 0.03). Independent predictors of complete response were lower ALT at 6 months (P = 0.001) and acute presentation (P = 0.03). So far, treatment withdrawal was feasible in 40/109 patients and 30 (75%) are still in remission after 24 (2–129) months. Remission maintenance was associated with longer MMF treatment (P = 0.005), higher baseline ALT (P < 0.02), lower IgG on 6 months (P = 0.004) and histological improvement.
Mycophenolate mofetil proved to be an efficient first-line treatment for AIH, achieving so far the highest rates of remission maintenance off treatment (75%) ever published for at least a median of 2 years, although the remission criteria used were strict. However, the risk of potential bias and overestimation of intervention benefits from MMF cannot be completely excluded as this is a real world and not a randomised controlled trial.