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

Efe, Cumali; Taii, Haider Al; Ytting, Henriette; Aehling, Niklas; Bhanji, Rahima A.; Hagström, Hannes; Purnak, Tugrul; Muratori, Luigi; Werner, Mårten and Muratori, Paolo, et al. (2018) In Digestive Diseases and Sciences
Abstract

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... (More)

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.

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publication status
epub
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keywords
Autoimmune hepatitis, Cirrhosis, Liver transplantation, Mycophenolate mofetil, Pediatric, Second-line, Tacrolimus
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Digestive Diseases and Sciences
pages
7 pages
publisher
Springer
external identifiers
  • scopus:85044335493
ISSN
0163-2116
DOI
10.1007/s10620-018-5011-x
language
English
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no
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a7fced60-125f-4e5b-a4da-bd35614be312
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2018-04-04 13:45:07
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2019-02-10 05:04:51
@article{a7fced60-125f-4e5b-a4da-bd35614be312,
  abstract     = {<p>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 (&lt; 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.</p>},
  author       = {Efe, Cumali and Taii, Haider Al and Ytting, Henriette and Aehling, Niklas and Bhanji, Rahima A. and Hagström, Hannes and Purnak, Tugrul and Muratori, Luigi and Werner, Mårten and Muratori, Paolo and Klintman, Daniel and Schiano, Thomas D. and Montano-Loza, Aldo J. and Berg, Thomas and Larsen, Fin Stolze and Alkhouri, Naim and Ozaslan, Ersan and Heneghan, Michael A. and Yoshida, Eric M. and Wahlin, Staffan},
  issn         = {0163-2116},
  keyword      = {Autoimmune hepatitis,Cirrhosis,Liver transplantation,Mycophenolate mofetil,Pediatric,Second-line,Tacrolimus},
  language     = {eng},
  month        = {03},
  pages        = {7},
  publisher    = {Springer},
  series       = {Digestive Diseases and Sciences},
  title        = {Tacrolimus and Mycophenolate Mofetil as Second-Line Therapies for Pediatric Patients with Autoimmune Hepatitis},
  url          = {http://dx.doi.org/10.1007/s10620-018-5011-x},
  year         = {2018},
}