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Original research
Efficacy of mycophenolate mofetil as a remission induction therapy in antineutrophil cytoplasmic antibody-associated vasculitis—a meta-analysis
  1. Kentaro Kuzuya1,
  2. Takayoshi Morita1,2 and
  3. Atsushi Kumanogoh1,2,3
  1. 1Department of Respiratory Medicine and Clinical Immunology, Osaka University Graduate School of Medicine, Suita, Japan
  2. 2Laboratory of Immunopathology, World Premier International Immunology Frontier Research Center, Suita, Japan
  3. 3Integrated Frontier Research for Medical Science Division, Institute for Open and Transdisciplinary Research Initiatives, Osaka University, Suita, Japan
  1. Correspondence to Takayoshi Morita; t-morita{at}


Objectives A few studies on antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) treatments have shown the therapeutic efficacy of mycophenolate mofetil (MMF). However, the therapeutic efficacy of MMF compared with that of cyclophosphamide (CYC) in patients with AAV has not been established. We conducted a systematic review and meta-analysis to assess the efficacy of MMF as a remission induction therapy in patients with AAV comparing it with the efficacy of CYC.

Methods We searched randomised controlled trials (RCTs) comparing the efficacy of MMF with that of CYC in patients with AAV on three different websites: PubMed, Cochrane Library and Google Scholar. We compared the difference in the relative risk (RR) of each outcome based on a Mantel-Haenszel random-effects model.

Results We analysed data from four RCTs with 300 patients for the study. The 6-month remission rate (RR 1.09, 95% CI 0.86 to 1.38, p=0.48), the 6-month ANCA negativity (RR 1.31, 95% CI 0.91 to 1.90, p=0.15) and the long-term relapse rate (RR 1.36, 95% CI 0.80 to 2.31, p=0.26) were all similar between the two treatments. The rates of death, infection and leucopenia were also similar between the two groups (RR 1.05, 95% CI 0.40 to 2.74, p=0.93; RR 1.26, 95% CI 0.79 to 2.01, p=0.33; RR 0.45, 95% CI 0.16 to 1.32, p=0.15, respectively).

Conclusions We found no difference between the therapeutic efficacy of MMF and that of CYC in patients with AAV. MMF may be an alternative remission induction therapy in patients with non-life-threatening AAV.

  • ANCA-associated vasculitis
  • meta-analysis
  • randomised control trials
  • mycophenolate mofetil
  • cyclophosphamide

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  • Contributors KK, TM and AK authors were involved in drafting the article or revising it critically for important intellectual content, and all authors approved the final version to be published. TM had full access to all data in the study and takes responsibility for the integrity of the data and the accuracy of the analysis. Study conception and design: KK and TM. Acquisition of data: KK and TM. Analysis and interpretation of data: KK, TM and AK.

  • Funding The authors declare no financial support or other benefits from commercial sources for the work reported on in the manuscript.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Data sharing statement Data are available in a public, open access repository.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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