Comparative Efficacy of Mycophenolate Mofetil vs. Azathioprine in Autoimmune Hepatitis: A Systematic Review and

Digestion
|August 28, 2025
PubMed
Abstract

Insights

Mycophenolate mofetil (MMF) shows improved complete biochemical response (CBR) in autoimmune hepatitis (AIH) compared to azathioprine (AZA). This finding is particularly notable in older patients and those with lower IgG levels, suggesting MMF as a promising alternative treatment.

Area of Science:

  • Hepatology
  • Immunology
  • Pharmacology

Background:

  • Azathioprine (AZA) is a standard treatment for autoimmune hepatitis (AIH), but often requires lifelong use and has tolerability concerns.
  • High relapse rates after AZA cessation necessitate exploring alternative therapies.
  • Combination therapy with prednisolone and AZA presents challenges in efficacy and safety.

Purpose of the Study:

  • To systematically review and compare the efficacy and safety of mycophenolate mofetil (MMF) versus AZA in treating AIH.
  • To evaluate treatment outcomes, including complete biochemical response (CBR) and steroid withdrawal, in patients receiving MMF or AZA.
  • To analyze subgroup effects based on age and IgG levels.

Main Methods:

  • A systematic review and meta-analysis of randomized clinical trials and cohort studies.
  • Searched major databases: PubMed, Cochrane, Scopus, and Web of Science.
  • Included four studies (one RCT, three cohort studies) with 505 AIH patients.
  • Performed subgroup analyses by age (>50 years) and IgG levels (<2400 mg/dL).

Main Results:

  • Mycophenolate mofetil (MMF) was significantly associated with increased complete biochemical response (CBR) compared to azathioprine (AZA) (RR = 1.44, p=0.03).
  • No significant difference was observed between MMF and AZA regarding steroid withdrawal.
  • MMF showed increased CBR in patients over 50 years and those with IgG levels <2400 mg/dL.

Conclusions:

  • Mycophenolate mofetil (MMF) demonstrates superior complete biochemical response (CBR) compared to azathioprine (AZA) in autoimmune hepatitis (AIH) patients.
  • MMF offers a potentially effective alternative to AZA, especially in specific patient subgroups.
  • Further research is warranted to define optimal AIH treatment strategies across diverse populations.

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