HEGENER'S GRANULOMATOSIS (WG) IN CHILDHOOD WITH PROLONGED SURVIVAL FOLLOWING CYTOTOXIC THERAPY

Abstract

Wegener's granulomatosis is a systemic disease of unknown etiology involving mainly the kidney and respiratory tract in a necrotizing and granulomatous vasculitis, though other organ systems may be affected. Only 6 cases have been reported in children under age 16 years and each had a rapidly fatal outcome despite supportive therapy and corticosteroids. Although several studies have reported a good response in adults to cytotoxic therapy, this form of therapy has not been tried in children with WG. We have treated 2 girls, aged 10 and 13 years, with a combination of prednisone and cyclophosphamide therapy for generalized WG with renal, ocular, nasopharyngeal, sinus and cutaneous involvement. One patient also had middle ear pulmonary and laryngeal granulomata. Multiple biopsies of involved tissues confirmed the diagnoses. Cyclophosphamide was capable of inducing remission in disease activity initially and during subsequent relapses. Proteinuria decreased in one patient from 5.2 to 0.8 gm/24 h and her serum creatinine concentration fell from 2.1 to 1.3 mg/dl. Pulmonary granuloma regressed in the second patient. Hemorrhagic cystitis was the only complication of therapy, but did not recur with readministration. These patients have survived 2½ and 5 years respectively. Both are in good health and attending school at present. These are the first children of which we are aware to receive this form of therapy for WG.

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