title: Wegener granulomatosis in children and adolescents: clinical presentation and outcome.
abstract: We prospectively studied and compared clinical features, treatment, course of illness, and long-term morbidity and mortality rates for Wegener granulomatosis in 23 childhood-onset patients with those of 135 adult-onset patients who were studied concurrently. Treatment was usually provided with glucocorticoids and cyclophosphamide. The mean follow-up period was 8.7 years for childhood-onset and 7.6 years for adult-onset Wegener granulomatosis. Most aspects of Wegener granulomatosis were similar in childhood-onset and adult-onset patients. Permanent morbidity from disease occurred in 86% of both groups. However, some features were significantly different. Wegener granulomatosis in childhood-onset patients was complicated five times more often by subglottic stenosis and twice as often by nasal deformity. Treatment-related permanent morbidity occurred in 22% of childhood-onset patients and 45% of adult-onset patients. After similar periods of cyclophosphamide therapy and follow-up, cyclophosphamide-related malignancies were less likely (0% vs 11%) to have developed in childhood-onset patients. Although 89% of patients treated with glucocorticoids and cyclophosphamide had remission, prolonged delay in achieving remission and relapses led in both patient groups to freedom from active disease for approximately 50% of the total patient-years. As a result, morbidity was substantial and has led to comparative studies of alternative therapies.
authors.affiliation: Laboratory of Clinical Investigation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland 20892.
authors.forename: M
authors.initials: M
authors.lastname: Rottem
authors.forename: A S
authors.initials: AS
authors.lastname: Fauci
authors.forename: C W
authors.initials: CW
authors.lastname: Hallahan
authors.forename: G S
authors.initials: GS
authors.lastname: Kerr
authors.forename: R
authors.initials: R
authors.lastname: Lebovics
authors.forename: R Y
authors.initials: RY
authors.lastname: Leavitt
authors.forename: G S
authors.initials: GS
authors.lastname: Hoffman
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D002648:Child; D003520:Cyclophosphamide / Q000009:adverse effects / Q000627:therapeutic use; D005128:Eye Diseases / Q000503:physiopathology; D005260:Female; D005500:Follow-Up Studies; D005921:Glomerulonephritis / Q000503:physiopathology; D014890:Granulomatosis with Polyangiitis / Q000150:complications / Q000188:drug therapy / Q000503:physiopathology*; D006801:Humans; D007829:Laryngostenosis / Q000503:physiopathology; D008171:Lung Diseases / Q000503:physiopathology; D008297:Male; D009668:Nose Diseases / Q000503:physiopathology; D009894:Opportunistic Infections; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use; D011446:Prospective Studies; D012074:Remission Induction; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D003520:Cyclophosphamide; D011241:Prednisone