Objective: To study the efficacy of methotrexate (MTX) plus low dose corticosteroids for induction of remission in generalized Wegener's granulomatosis (WG) and to possibly identify predictive factors for the outcome under this therapy.
Methods: We conducted a prospective, open label study, including 17 patients with not immediately life threatening, generalized WG. Treatment consisted of intravenous MTX 0.3 mg/kg once weekly plus daily oral low dose prednisone for initial diagnosis of WG in 11 and for a generalized relapse of WG in 6 patients. Interdisciplinary, standardized assessments of disease activity and extent were done 3-monthly.
Results: Within a median treatment period of 24.5 months remission could be achieved in 10/17 patients (59%), their median corticosteroid dose during that time was 1.75 mg/day. Seven patients with a median concomitant prednisone dose of 7.5 mg/day did not respond, among them 4 patients who were treated for a relapse of WG. Signs of de novo glomerulonephritis occurred in 5 of the 7 nonresponders. Significant side effects, including opportunistic infections, did not occur.
Conclusion: Weekly low dose MTX in combination with low dose corticosteroids leads to an acceptable remission rate of almost 60% without significant side effects. Patients treated for a relapse of WG and patients with a need for a higher concomitant prednisone dose seem to be at risk for nonresponse, with a high likelihood of developing de novo glomerulonephritis.