title: A real-world study focused on the long-term efficacy of mycophenolate mofetil as first-line treatment of autoimmune hepatitis.
abstract: BACKGROUND
  Front-line therapy with mycophenolate mofetil (MMF) in autoimmune hepatitis (AIH) has shown high on-treatment remission rates.
  
  
  OBJECTIVE
  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.
authors.affiliation: Department of Medicine and Research Laboratory of Internal Medicine, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: K
authors.initials: K
authors.lastname: Zachou
authors.affiliation: Department of Medicine and Research Laboratory of Internal Medicine, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: N K
authors.initials: NK
authors.lastname: Gatselis
authors.affiliation: Department of Medicine and Research Laboratory of Internal Medicine, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: P
authors.initials: P
authors.lastname: Arvaniti
authors.affiliation: Department of Medicine and Research Laboratory of Internal Medicine, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: S
authors.initials: S
authors.lastname: Gabeta
authors.affiliation: Department of Medicine and Research Laboratory of Internal Medicine, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: E I
authors.initials: EI
authors.lastname: Rigopoulou
authors.affiliation: Department of Pathology, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: G K
authors.initials: GK
authors.lastname: Koukoulis
authors.affiliation: Department of Medicine and Research Laboratory of Internal Medicine, Medical School, University of Thessaly, Larissa, Greece.
authors.forename: G N
authors.initials: GN
authors.lastname: Dalekos
mesh_terms: D000328:Adult; D001379:Azathioprine / Q000008:administration & dosage; D004359:Drug Therapy, Combination; D005260:Female; D019693:Hepatitis, Autoimmune / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D008103:Liver Cirrhosis / Q000188:drug therapy / Q000209:etiology; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011239:Prednisolone / Q000008:administration & dosage; D011446:Prospective Studies; D012074:Remission Induction; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D011239:Prednisolone; D009173:Mycophenolic Acid; D001379:Azathioprine