Wegener granulomatosis in childhood and adolescence

Abstract

Wegener granulomatosis (WG) is a necrotising granulomatous small vessel vasculitis with a clinical predilection for involvement of the upper airways, lungs and kidneys, which occurs at all ages. The aetiology of WG, like other primary systemic vasculitides, remains unknown. Existing evidence suggests an autoimmune inflammatory process, characterised by early lesions with neutrophils and endothelial cells as active participants and involvement of antineutrophil cytoplasmatic antibodies (ANCA) directed against proteinase-3 (PR3). However, other concomitant factors like infections and environmental factors also appear to be necessary for the development of WG. In addition, multiple genetic factors seem to be involved in disease susceptibility. Whereas the first publications on WG in childhood were based on case reports, some studies in recent years allow to compare clinical findings, disease course, morbidity and mortality rates for childhood and adult onset patients. Whereas most aspects of WG are similar at all ages, some features appear to be significantly different. WG in childhood is more frequently complicated by subglottic stenosis and nasal deformity while treatment-related morbidity and malignancies are less common compared to adults. Introduction of combined treatment with cyclophosphamide and glucocorticoids resulted in a dramatic improvement of patient outcome; however, commonly occurring disease relapses and the risk of chronic organ damage at all ages make long-term follow-up of all patients and the establishment of new therapeutic regimens necessary.