Cyclophosphamide Therapy in Wegener's Granulomatosis

Four adults suffering from Wegener's granulomatosis, each with extensive pulmonary and Central-nervous-system involvement and lesser degrees of renal disease and sinusitis, were treated with cyclophosphamide. Each had previously proved unresponsive to corticosteroid therapy. Cyclophosphamide was given in massive dosages intravenously in one near terminal patient and orally in three others. In all four patients a rapid amelioration of all signs, symptoms and evidence of disease occurred. Pulmonary lesions, even cavitary ones, resolved, as did a diffuse and extensive pulmonary infiltrate in one patient. Neurologic and renal lesions diminished or were completely reversed. In two of the four patients it was possible to omit all therapy for 12 to 20 months, without evidence of recurrence of disease. The other two patients have been asymptomatic for 3 1/2 years on continued low-dose cyclophosphamide. The uniformly good results in this series imply effective immunosuppression, even in an ongoing disease. Further trials appear warranted.