Efficacy and Safety of Mycophenolate Mofetil and Tacrolimus as Second-line Therapy for Patients With Autoimmune Hepatitis

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Background & Aims Predniso(lo)ne, alone or in combination with azathioprine, is the standard-of-care (SOC) therapy for autoimmune hepatitis (AIH). However, the SOC therapy is poorly tolerated or does not control disease activity in up to 20% of patients. We assessed the efficacy of mycophenolate mofetil (MMF) and tacrolimus as second-line therapy for patients with AIH. Methods We performed a retrospective study of data (from 19 centers in Europe, the United States, Canada, and China) from 201 patients with AIH who received second-line therapy (121 received MMF and 80 received tacrolimus), for a median of 62 months (range, 6–190 mo). Patients were categorized according to their response to SOC. Patients in group 1 (n = 108) had a complete response to the SOC, but were switched to second-line therapy as a result of side effects of predniso(lo)ne or azathioprine, whereas patients in group 2 (n = 93) had not responded to SOC. Results There was no significant difference in the proportion of patients with a complete response to MMF (69.4%) vs tacrolimus (72.5%) (P =.639). In group 1, MMF and tacrolimus maintained a biochemical remission in 91.9% and 94.1% of patients, respectively (P =.682). Significantly more group 2 patients given tacrolimus compared with MMF had a complete response (56.5% vs 34%, respectively; P =.029) There were similar proportions of liver-related deaths or liver transplantation among patients given MMF (13.2%) vs tacrolimus (10.3%) (log-rank, P =.472). Ten patients receiving MMF (8.3%) and 10 patients receiving tacrolimus (12.5%) developed side effects that required therapy withdrawal. Conclusions Long-term therapy with MMF or tacrolimus generally was well tolerated by patients with AIH. The agents were equally effective in previous complete responders who did not tolerate SOC therapy. Tacrolimus led to a complete response in a greater proportion of previous nonresponder patients compared with MMF.


  • Cumali Efe
  • Hannes Hagström
  • Henriette Ytting
  • Rahima A. Bhanji
  • Niklas F. Müller
  • Qixia Wang
  • Tugrul Purnak
  • Luigi Muratori
  • Mårten Werner
  • Hanns Ulrich Marschall
  • Paolo Muratori
  • Fulya Gunşar
  • Daniel Klintman
  • Albert Parés
  • Alexandra Heurgué-Berlot
  • Thomas D. Schiano
  • Mustafa Cengiz
  • Michele May-Sien Tana
  • Xiong Ma
  • Aldo J. Montano-Loza
  • Och 7 andra
  • Thomas Berg
  • Sumita Verma
  • Fin Stolze Larsen
  • Ersan Ozaslan
  • Michael A. Heneghan
  • Eric M. Yoshida
  • Staffan Wahlin
Externa organisationer
  • Hacettepe University
  • Karolinska University Hospital
  • University of Copenhagen
  • University of Alberta
  • Renji Hospital
  • Umeå University
  • Ege University
  • Skåne University Hospital
  • University of British Columbia
  • University of Barcelona
  • Dr A.Y. Oncology Training and Research Hospital
  • University of Texas Health Science Centre
  • Brighton and Sussex University Hospitals NHS Trust
  • Numune Research and Education Hospital
  • King's College Hospital
  • Karolinska Institute
  • Copenhagen University Hospital
  • Shanghai Jiao Tong University
  • University of Bologna
  • Göteborgs universitet
  • Hopital Robert Debre CHU de Reims
  • Icahn School of Medicine at Mount Sinai
  • University Hospital Leipzig

Ämnesklassifikation (UKÄ) – OBLIGATORISK

  • Gastroenterologi


Sidor (från-till)1950-1956.e1
TidskriftClinical Gastroenterology and Hepatology
Utgåva nummer12
StatusPublished - 2017 dec 1
Peer review utfördJa
Externt publiceradJa