What is the role of mycophenolate mofetil (MMF) in the treatment of granulomatosis with polyangiitis (GPA)?

Updated: Aug 31, 2021
  • Author: Christopher L Tracy, MD; Chief Editor: Herbert S Diamond, MD  more...
  • Print

Mycophenolate mofetil (MMF) (2 g/day) is a derivative of the fungus Penicillium stoloniferum that, similar to azathioprine, limits purine synthesis. MMF is primarily used for immunosuppression in transplant patients and suppresses B and T cells. Because of its favorable side-effect profile and clinical potency, MMF is being increasingly used in the management of systemic connective-tissue disorders and is typically used in combination with prednisone.

A study of microscopic polyangiitis, a myeloperoxidase (MPO)-ANCA ̶ associated vasculitis, suggested that MMF may be an alternative to cyclophosphamide in mild-to-moderate microscopic polyangiitis. [94] MMF has been used in small series of refractory GPA cases, for both induction and maintenance, with varying responses. [95, 96]

Nowack et al reported a good outcome in a study using MMF during the maintenance phase of treatment in 11 adult patients with ANCA-associated systemic vasculitis (9 patients with GPA, 2 patients with microscopic polyangiitis). [97] Patients were started on a regimen of MMF and low-dose oral corticosteroids after a 14-week induction period with steroids and oral cyclophosphamide. Outcome was assessed only to 15 months but was very good. Only 1 patient relapsed 14 months into the maintenance treatment phase. MMF was well tolerated at a dose of 2 g/day, with minimal adverse effects.

Joy et al evaluated MMF in patients with non–life-threatening recurrent or cyclophosphamide-resistant ANCA (+) small-vessel vasculitis. [95] A 24-week treatment period in conjunction with corticosteroids resulted in a marked reduction in disease activity as assessed by the Birmingham Vasculitis Activity Scoring system. Thus, MMF may be useful in this setting to avoid recurrent treatment with alkylating agents.

However, a European Vasculitis Study Group trial (IMPROVE, International Mycophenolate Mofetil to Reduce Outbreaks of Vasculitides) reported that MMF was less effective than azathioprine in the maintenance of remission of AAV. [98] Further studies are needed.

Did this answer your question?
Additional feedback? (Optional)
Thank you for your feedback!