A staged approach to the treatment of Wegener's granulomatosis - Inductionof remission with glucocorticoids and daily cyclophosphamide switching to methotrexate for remission maintenance

Objective. To determine the efficacy of a daily cyclophosphamide (CYC) and glucocorticoid induction and methotrexate (MTX) remission-maintenance regim en for the treatment of Wegener's granulomatosis (WG), Methods. An open-label, prospective, standardized trial for the treatment o f WG was performed using CYC and glucocorticoids for remission induction an d MTX for remission maintenance, Thirty-one patients were enrolled in this study. Outcome was assessed using predetermined definitions based on clinic al characteristics and pathologic, laboratory, and radiographic findings. Results, The use of CYC and glucocorticoids for remission induction and MTX for remission maintenance resulted in disease remission for all 31 patient s. The median time to remission was 3 months and the median time to discont inuation of glucocorticoids was 8 months. No patients have died, and 5 pati ents (16%) have had disease relapses at a median of 13 months after achievi ng remission. Only 2 patients (6%) have had to withdraw from the trial as a result of medication toxicity. Conclusion. The use of CYC and glucocorticoids for remission induction and MTX for remission maintenance was shown by this study to be an acceptable a lternative therapy for patients with active WG, including those with severe disease at onset.