A pilot study using mycophenolate mofetil in relapsing or resistant ANCA small vessel vasculitis

Nephrol Dial Transplant. 2005 Dec;20(12):2725-32. doi: 10.1093/ndt/gfi117. Epub 2005 Sep 27.


Background: The treatment approaches to antineutrophil cytoplasmic autoantibody (ANCA) small vessel vasculitis expose patients to the risks associated with long-term use of corticosteroids and cytotoxic agents. In an effort to explore approaches to minimize risks, we conducted a pilot efficacy and safety study of mycophenolate mofetil (MMF) in the treatment of subjects with nonlife-threatening recurrent or cyclophosphamide-resistant ANCA-vasculitis.

Methods: MMF was initiated at 500 mg orally twice daily and gradually increased to a target dose of 1000 mg twice daily for a duration of 24 weeks. Concomitant therapy with corticosteroids was allowed. The Birmingham Vasculitis Activity Score (BVAS) was used to assess disease activity and treatment efficacy. ANCA titres, serum creatinine and adverse events were secondary measures of efficacy and/or toxicity.

Results: Twelve subjects were enrolled in the study. Treatment with MMF led to an improvement in disease activity as measured by the BVAS at 24 weeks (P = 0.0013) and 52 weeks (P = 0.0044) as compared to baseline. The BVAS decreased from an average of 9.1+/-3.5 at baseline (range, 3-17) to an average of 2.8+/-1.9 (range, 1-6) at 24 weeks and to 2.8+/-4.3 (range, 0-13) at 52 weeks. Early and sustained reductions in BVAS occurred in subjects initially classified as disease relapses vs those with treatment resistance. Side effect profile was consistent with the mechanism of action and pharmacokinetic disposition of MMF.

Conclusions: MMF is a reasonable option in the treatment of non-life-threatening recurrent or resistant vasculitis and may obviate the immediate need for recurrent use of cytotoxic agents.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Antibodies, Antineutrophil Cytoplasmic / immunology*
  • Disease Progression
  • Feasibility Studies
  • Female
  • Follow-Up Studies
  • Granulomatosis with Polyangiitis / drug therapy*
  • Granulomatosis with Polyangiitis / immunology
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Middle Aged
  • Mycophenolic Acid / administration & dosage
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use
  • Pilot Projects
  • Prodrugs / administration & dosage
  • Prodrugs / therapeutic use*
  • Recurrence
  • Treatment Outcome


  • Antibodies, Antineutrophil Cytoplasmic
  • Immunosuppressive Agents
  • Prodrugs
  • Mycophenolic Acid