Use of a cyclophosphamide-induction methotrexate-maintenance regimen for the treatment of Wegener's granulomatosis: extended follow-up and rate of relapse

Am J Med. 2003 Apr 15;114(6):463-9. doi: 10.1016/s0002-9343(03)00077-9.


Purpose: To determine the relapse rate and outcome in patients with Wegener's granulomatosis treated with daily cyclophosphamide and glucocorticoids to induce remission followed by methotrexate for remission maintenance.

Methods: We performed an open-label prospective study in 42 patients with active Wegener's granulomatosis. All patients were treated with a standardized regimen. Outcomes were assessed using predetermined definitions based on clinical characteristics and pathologic, laboratory, and radiographic findings.

Results: All patients achieved disease remission. The median time to remission was 3 months, and the median time to discontinuation of glucocorticoids was 8 months. During a median of 32 months of follow-up, 1 patient died (of a myocardial infarction not related to vasculitis). Two patients (5%) had to withdraw from the study because of medication toxicity. Twenty-two patients (52%) relapsed, with glomerulonephritis occurring in 16 patients. Of these 16 patients, 4 had an increase of >0.2 mg/dL in serum creatinine level. All 4 patients returned to their prior level of renal function with treatment. None of the 22 relapses met the criteria for severe disease.

Conclusion: The use of cyclophosphamide and glucocorticoids for induction and methotrexate for maintaining remission is an effective and well-tolerated therapeutic approach in patients with active Wegener's granulomatosis.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antibodies, Antineutrophil Cytoplasmic / analysis
  • Antimetabolites, Antineoplastic / adverse effects
  • Antimetabolites, Antineoplastic / therapeutic use*
  • Antineoplastic Agents, Alkylating / adverse effects
  • Antineoplastic Agents, Alkylating / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Child
  • Cyclophosphamide / adverse effects
  • Cyclophosphamide / therapeutic use*
  • Female
  • Follow-Up Studies
  • Glucocorticoids / therapeutic use
  • Granulomatosis with Polyangiitis / drug therapy*
  • Granulomatosis with Polyangiitis / immunology
  • Humans
  • Male
  • Methotrexate / adverse effects
  • Methotrexate / therapeutic use*
  • Prednisone / therapeutic use
  • Prospective Studies
  • Recurrence
  • Remission Induction


  • Antibodies, Antineutrophil Cytoplasmic
  • Antimetabolites, Antineoplastic
  • Antineoplastic Agents, Alkylating
  • Glucocorticoids
  • Cyclophosphamide
  • Prednisone
  • Methotrexate