Journal Article

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

Melanie S. Joy, Susan L. Hogan, J. Charles Jennette, Ronald J. Falk and Patrick H. Nachman

in Nephrology Dialysis Transplantation

Published on behalf of European Renal Association - European Dialysis and Transplant Assoc

Volume 20, issue 12, pages 2725-2732
Published in print December 2005 | ISSN: 0931-0509
Published online September 2005 | e-ISSN: 1460-2385 | DOI:
A pilot study using mycophenolate mofetil in relapsing or resistant ANCA small vessel vasculitis

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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.

Keywords: ANCA; antineutrophil cytoplasmic autoantibodies; microscopic polyangiitis; mycophenolate mofetil; Wegener's granulomatosis; vasculitis

Journal Article.  5456 words.  Illustrated.

Subjects: Nephrology

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