title: The role of mycophenolate mofetil in the management of autoimmune hepatitis and overlap syndromes.
abstract: BACKGROUND
  Treatment failure occurs in 20% of autoimmune hepatitis patients on prednisolone and azathioprine (AZA). There is no established second line treatment.
  
  
  OBJECTIVE
  To assess the efficacy of mycophenolate mofetil as second line treatment after AZA-intolerance or AZA-nonresponse in autoimmune hepatitis and overlap syndromes.
  
  
  METHODS
  Consecutive patients from the Dutch Autoimmune Hepatitis Group cohort, consisting of 661 patients, with autoimmune hepatitis or overlap syndromes, AZA-intolerance or AZA-nonresponse and past or present use of mycophenolate mofetil were included. Primary endpoint of mycophenolate mofetil treatment was biochemical remission. Secondary endpoints were biochemical response (without remission), treatment failure and prevention of disease progression.
  
  
  RESULTS
  Forty-five patients treated with mycophenolate mofetil were included. In autoimmune hepatitis remission or response was achieved in 13% and 27% in the AZA-nonresponse group compared to 67% and 0% in the AZA-intolerance group (P = 0.008). In overlap-syndromes remission or response was reached in 57% and 14% in the AZA-nonresponse group and 63% and 25% of the AZA-intolerance group (N.S.); 33% had side effects and 13% discontinued mycophenolate mofetil. Overall 38% had treatment failure; this was 60% in the autoimmune hepatitis AZA-nonresponse group. Decompensated liver cirrhosis, liver transplantations and death were only seen in the autoimmune hepatitis AZA-nonresponse group (P < 0.001).
  
  
  CONCLUSIONS
  Mycophenolate mofetil induced response or remission in a majority of patients with autoimmune hepatitis and azathioprine-intolerance and with overlap syndromes, irrespective of intolerance or nonresponse for azathioprine. In autoimmune hepatitis with azathioprine nonresponse mycophenolate mofetil is less often effective.
authors.affiliation: Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: A M C
authors.initials: AM
authors.lastname: Baven-Pronk
authors.forename: M J
authors.initials: MJ
authors.lastname: Coenraad
authors.forename: H R
authors.initials: HR
authors.lastname: van Buuren
authors.forename: R A
authors.initials: RA
authors.lastname: de Man
authors.forename: K J
authors.initials: KJ
authors.lastname: van Erpecum
authors.forename: M M H
authors.initials: MM
authors.lastname: Lamers
authors.forename: J P H
authors.initials: JP
authors.lastname: Drenth
authors.forename: A P
authors.initials: AP
authors.lastname: van den Berg
authors.forename: U H
authors.initials: UH
authors.lastname: Beuers
authors.forename: J
authors.initials: J
authors.lastname: den Ouden
authors.forename: G H
authors.initials: GH
authors.lastname: Koek
authors.forename: C M J
authors.initials: CM
authors.lastname: van Nieuwkerk
authors.forename: G
authors.initials: G
authors.lastname: Bouma
authors.forename: J T
authors.initials: JT
authors.lastname: Brouwer
authors.forename: B
authors.initials: B
authors.lastname: van Hoek
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D015331:Cohort Studies; D005260:Female; D019693:Hepatitis, Autoimmune / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D009426:Netherlands; D012189:Retrospective Studies; D012720:Severity of Illness Index; D013577:Syndrome; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid