Tacrolimus and Mycophenolate Mofetil as Second-Line Therapies for Pediatric Patients with Autoimmune Hepatitis

Forskningsoutput: TidskriftsbidragArtikel i vetenskaplig tidskrift


Background: We studied the efficacy and safety of mycophenolate mofetil (MMF) and tacrolimus as second-line therapy in pediatric patients with autoimmune hepatitis (AIH) who were intolerant or non-responders to standard therapy (corticosteroid and azathioprine). Patients and Methods: We performed a retrospective study of data from 13 centers in Europe, USA, and Canada. Thirty-eight patients (< 18 years old) who received second-line therapy (18 MMF and 20 tacrolimus), for a median of 72 months (range 8–182) were evaluated. Patients were categorized into two groups: Group 1 (n = 17) were intolerant to corticosteroid or azathioprine, and group 2 (n = 21) were non-responders to standard therapy. Results: Overall complete response rates were similar in patients treated with MMF and tacrolimus (55.6 vs. 65%, p = 0.552). In group 1, MMF and tacrolimus maintained a biochemical remission in 88.9 and 87.5% of patients, respectively (p = 0.929). More patients in group 2 given tacrolimus compared to MMF had a complete response, but the difference was not statistically significant (50.0 vs. 22.2%, p = 0.195). Biochemical remission was achieved in 71.1% (27/38) of patients by tacrolimus and/or MMF. Decompensated cirrhosis was more commonly seen in MMF and/or tacrolimus non-responders than in responders (45.5 vs. 7.4%, p = 0.006). Five patients who received second-line therapy (2 MMF and 3 tacrolimus) developed side effects that led to therapy withdrawal. Conclusions: Long-term therapy with MMF or tacrolimus was generally well tolerated by pediatric patients with AIH. Both MMF and tacrolimus had excellent efficacy in patients intolerant to corticosteroid or azathioprine. Tacrolimus might be more effective than MMF in patients failing previous therapy.


  • Cumali Efe
  • Haider Al Taii
  • Henriette Ytting
  • Niklas Aehling
  • Rahima A. Bhanji
  • Hannes Hagström
  • Tugrul Purnak
  • Luigi Muratori
  • Mårten Werner
  • Paolo Muratori
  • Daniel Klintman
  • Thomas D. Schiano
  • Aldo J. Montano-Loza
  • Thomas Berg
  • Fin Stolze Larsen
  • Naim Alkhouri
  • Ersan Ozaslan
  • Michael A. Heneghan
  • Eric M. Yoshida
  • Staffan Wahlin
Externa organisationer
  • Hacettepe University
  • Cleveland Clinic
  • University of Copenhagen
  • University of Alberta
  • Karolinska University Hospital
  • Umeå University
  • Skåne University Hospital
  • King's College Hospital
  • The Mount Sinai Medical Center
  • Numune Research and Education Hospital
  • University of British Columbia
  • Copenhagen University Hospital
  • Karolinska Institute
  • University of Bologna
  • Texas Liver Institute
  • Vancouver General Hospital
  • University Hospital Leipzig

Ämnesklassifikation (UKÄ) – OBLIGATORISK

  • Gastroenterologi


Antal sidor7
TidskriftDigestive Diseases and Sciences
StatusE-pub ahead of print - 2018 mar 22
Peer review utfördJa
Externt publiceradJa