Mycophenolate Mofetil Versus Cyclophosphamide for the Induction of Remission in Nonlife-Threatening Relapses of Antineutrophil Cytoplasmic Antibody-Associated Vasculitis: Randomized, Controlled Trial

Janneke Tuin*, Patricia M. Stassen, Daria I. Bogdan, Jan Broekroelofs, Pieter van Paassen, Jan Willem Cohen Tervaert, Jan-Stephan Sanders, Coen A. Stegeman

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Background and objectivesCyclophosphamide has been the mainstay of treatment of ANCA-associated vasculitis. However, cyclophosphamide has unfavorable side effects and alternatives are needed. Evidence suggests that mycophenolate mofetil can induce sustained remission in nonlife-threatening disease. The purpose of this study was to compare the efficacy and safety of mycophenolate mofetil versus cyclophosphamide for the induction treatment of nonlife-threatening relapses of proteinase 3-ANCA- and myeloperoxidase-ANCA-associated vasculitis.Design, setting, participants, & measurementsWe conducted a multicenter randomized, controlled trial. Participants with a first or second relapse of ANCA-associated vasculitis were randomized to induction treatment with cyclophosphamide or mycophenolate mofetil both in combination with glucocorticoids. Maintenance therapy consisted of azathioprine in both arms. Primary outcome was remission at 6 months, and secondary outcomes included disease-free survival at 2 and 4 years.ResultsEighty-four participants were enrolled, of whom 41 received mycophenolate mofetil and 43 received cyclophosphamide. Eighty-nine percent of participants were proteinase 3-ANCA positive. At 6 months, 27 (66%) mycophenolate mofetil-treated participants versus 35 (81%) cyclophosphamide-treated participants were in remission (P=0.11). Disease-free survival rates at 2 and 4 years were 61% and 39% for cyclophosphamide, respectively, and 43% and 32% for mycophenolate mofetil, respectively (at 4 years, log rank test, P=0.17).ConclusionsWe did not demonstrate mycophenolate mofetil to be similarly effective as cyclophosphamide in inducing remission of relapsed ANCA-associated vasculitis. However, mycophenolate mofetil might be an alternative to cyclophosphamide for the treatment of selected patients with nonlife-threatening relapses.

Original languageEnglish
Pages (from-to)1021-1028
Number of pages8
JournalClinical journal of the American Society of Nephrology
Issue number7
Publication statusPublished - 5 Jul 2019


  • ANCA
  • vasculitis
  • mycophenolate mofetil
  • cyclophosphamide
  • randomized controlled trials
  • relapse
  • Mycophenolic Acid
  • Antibodies
  • Antineutrophil Cytoplasmic
  • Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  • Recurrence
  • Remission Induction

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