A real‐world study focused on the long‐term efficacy of mycophenolate mofetil as first‐line treatment of autoimmune hepatitis

Summary Background Front‐line therapy with mycophenolate mofetil ( MMF ) in autoimmune hepatitis ( AIH ) has shown high on‐treatment remission rates. Aim To study prospectively in a real‐world fashion the long‐term outcome of a large group of consecutive treatment‐naïve AIH patients. Methods 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. Results 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. Conclusions 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.