Mycophenolate mofetil for induction and maintenance of remission in microscopic polyangiitis with mild to moderate renal involvement--a prospective, open-label pilot trial
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Francisco Silva1, Ulrich Specks, Sanjay Kalra
1Division of Nephrology and Hypertension, Mayo Clinic College of Medicine, 200 First Street, SW, Rochester, MN 55905, USA.
Background And Objectives:
Microscopic polyangiitis (MPA) is a systemic small-vessel vasculitis associated with anti-neutrophil cytoplasmic antibodies (ANCA), often targeting myeloperoxidase (MPO). Cyclophosphamide (CYC) plus corticosteroids (CS) is considered standard therapy for patients with renal involvement, but treatment response is not satisfactory in all patients and CYC has well recognized toxicity. This prospective pilot trial explored whether mycophenolate mofetil (MMF) represents an effective alternative to CYC for induction and maintenance of remission in MPA with mild to moderate renal involvement.
Design, Setting, Participants, & Measurements:
Seventeen P-ANCA/MPO-ANCA-positive patients with MPA with mild to moderate renal involvement received MMF (1000 mg orally, twice daily) and CS (intravenous methylprednisolone, 1 to 3 g, followed by oral prednisone at 1 mg/kg per day). Oral CS were discontinued by month 6; MMF was continued through month 18. The primary outcome measure was remission by month 6 and stable renal function. Secondary endpoints included major relapses necessitating a switch to CYC plus CS, minor relapses requiring an increase in CS dosage, and adverse events.
Results:
Thirteen of 17 patients enrolled achieved the primary outcome, and 4 failed because of insufficient response, relapse, or MMF intolerance. Twelve patients remained in remission through month 18, renal function remained stable, and proteinuria improved. Side effects of MMF were mild, transient, and responsive to dose adjustments in all patients except one.
Conclusions:
MMF represents an alternative to CYC for induction and maintenance of remission in patients with MPO-ANCA-associated MPA with mild to moderate renal disease.
Insights
Mycophenolate mofetil (MMF) offers a promising alternative to cyclophosphamide (CYC) for treating microscopic polyangiitis (MPA). This study found MMF effective for inducing and maintaining remission in MPA patients with kidney involvement.
Area of Science:
- Nephrology
- Rheumatology
- Immunology
Background:
- Microscopic polyangiitis (MPA) is a small-vessel vasculitis often linked to anti-neutrophil cytoplasmic antibodies (ANCA) targeting myeloperoxidase (MPO).
- Current standard therapy for MPA with renal involvement, cyclophosphamide (CYC) plus corticosteroids (CS), has limitations in efficacy and toxicity.
- This study investigated mycophenolate mofetil (MMF) as a potential alternative induction and maintenance therapy.
Purpose of the Study:
- To evaluate mycophenolate mofetil (MMF) as an alternative to cyclophosphamide (CYC) for remission induction and maintenance in microscopic polyangiitis (MPA).
- To assess the efficacy and safety of MMF in patients with MPA and mild to moderate renal involvement.
Main Methods:
- A prospective pilot trial involving 17 patients with P-ANCA/MPO-ANCA-positive MPA and mild to moderate renal disease.
- Patients received MMF (2000 mg/day) and corticosteroids (CS).
- Primary outcome was remission by month 6 with stable renal function; secondary outcomes included relapse rates and adverse events.
Main Results:
- Thirteen of 17 patients achieved remission by month 6; 12 remained in remission through month 18 with stable renal function and improved proteinuria.
- MMF demonstrated a favorable safety profile with mild, transient side effects in most patients.
- Four patients did not achieve the primary outcome due to insufficient response, relapse, or intolerance.
Conclusions:
- Mycophenolate mofetil (MMF) is a viable alternative to cyclophosphamide (CYC) for inducing and maintaining remission in MPO-ANCA-associated MPA.
- MMF offers a potentially safer and effective treatment option for MPA patients with mild to moderate renal involvement.