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

Efe, Cumali; Hagström, Hannes; Ytting, Henriette; Bhanji, Rahima A.; Müller, Niklas F.; Wang, Qixia; Purnak, Tugrul; Muratori, Luigi; Werner, Mårten and Marschall, Hanns Ulrich, et al. (2017) In Clinical Gastroenterology and Hepatology 15(12). p.1-1956
Abstract

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... (More)

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.

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keywords
Autoimmune Liver Disease, Liver Failure, Liver Transplantation, Simplified Criteria
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Clinical Gastroenterology and Hepatology
volume
15
issue
12
pages
1 - 1956
publisher
Elsevier
external identifiers
  • scopus:85029848036
ISSN
1542-3565
DOI
10.1016/j.cgh.2017.06.001
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English
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9053a955-cf5b-4a30-9223-77b1c62e9396
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2017-12-12 10:55:29
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2018-11-11 04:58:31
@article{9053a955-cf5b-4a30-9223-77b1c62e9396,
  abstract     = {<p>Background &amp; 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.</p>},
  author       = {Efe, Cumali and Hagström, Hannes and Ytting, Henriette and Bhanji, Rahima A. and Müller, Niklas F. and Wang, Qixia and Purnak, Tugrul and Muratori, Luigi and Werner, Mårten and Marschall, Hanns Ulrich and Muratori, Paolo and Gunşar, Fulya and Klintman, Daniel and Parés, Albert and Heurgué-Berlot, Alexandra and Schiano, Thomas D. and Cengiz, Mustafa and May-Sien Tana, Michele and Ma, Xiong and Montano-Loza, Aldo J. and Berg, Thomas and Verma, Sumita and Larsen, Fin Stolze and Ozaslan, Ersan and Heneghan, Michael A. and Yoshida, Eric M. and Wahlin, Staffan},
  issn         = {1542-3565},
  keyword      = {Autoimmune Liver Disease,Liver Failure,Liver Transplantation,Simplified Criteria},
  language     = {eng},
  month        = {12},
  number       = {12},
  pages        = {1--1956},
  publisher    = {Elsevier},
  series       = {Clinical Gastroenterology and Hepatology},
  title        = {Efficacy and Safety of Mycophenolate Mofetil and Tacrolimus as Second-line Therapy for Patients With Autoimmune Hepatitis},
  url          = {http://dx.doi.org/10.1016/j.cgh.2017.06.001},
  volume       = {15},
  year         = {2017},
}