BACKGROUND: Granulomatosis with polyangiitis (GPA), formerly known as Wegener's granulomatosis (WG), belongs to the group of ANCA-associated necrotizing vasculitides. This study describes the clinical picture of the disease in a large cohort of GPA paediatric patients. Children with age at diagnosis ≤ 18 years, fulfilling the EULAR/PRINTO/PRES GPA/WG classification criteria were extracted from the PRINTO vasculitis database. The clinical signs/symptoms and laboratory features were analysed before or at the time of diagnosis and at least 3 months thereafter and compared with other paediatric and adult case series (>50 patients) derived from the literature. FINDINGS: The 56 children with GPA/WG were predominantly females (68%) and Caucasians (82%) with a median age at disease onset of 11.7 years, and a median delay in diagnosis of 4.2 months. The most frequent organ systems involved before/at the time of diagnosis were ears, nose, throat (91%), constitutional (malaise, fever, weight loss) (89%), respiratory (79%), mucosa and skin (64%), musculoskeletal (59%), and eye (35%), 67% were ANCA-PR3 positive, while haematuria/proteinuria was present in > 50% of the children. In adult series, the frequency of female involvement ranged from 29% to 50% with lower frequencies of constitutional (fever, weight loss), ears, nose, throat (oral/nasal ulceration, otitis/aural discharge), respiratory (tracheal/endobronchial stenosis/obstruction), laboratory involvement and higher frequency of conductive hearing loss than in this paediatric series. CONCLUSIONS: Paediatric patients compared to adults with GPA/WG have similar pattern of clinical manifestations but different frequencies of organ involvement.

Ann and Robert H Lurie Children's Hospital of Chicago Chicago IL USA

Asklepios Klinik Altona Hamburg Germany

Benghazi Children Hospital Benghazi MUB Rheumatology Clinic Benghazi Lybia

Charles University Prague 1st Medical Faculty and General University Hospital Prague Prague Czech Republic

CHU Le Kremlin Bicêtre APHP University of Paris Sud CEREMAI Le Kremlin Bicêtre Paris France

Department of Allergology Rheumatology and Clinical Immunology University Children's Hospital University Medical Centre Ljubljana Ljubljana Slovenia

Department of Pediatric Immunology and Rheumatology Wilhelmina Kinderziekenhuis Utrecht Netherlands

Department of Pediatric Rheumatology and Immunology University Children's Hospital Muenster Germany

Department of Pediatric Rheumatology and Nephrology Hacettepe University Children's Hospital Ankara Turkey

Department of Pediatrics Division of Pneumology and Immunology Charité Universitätsmedizin Berlin Berlin Germany

Department of Pediatrics Emma Children Hospital AMC Amsterdam Netherlands

Department of Pediatrics Skejby Sygehus Aarhus University Hospital Pediatric Rheumatology Clinic Aarhus Denmark

Department of Pediatrics The Queen Silvia Children's Hospital University of Gothenburg Göteborg Sweden

Department of Rheumatology Great Ormond St Hospital NHS Foundation Trust London UK

Dipartimento di Pediatria Università degli Studi di Genova Genova Italy

Hamburger Zentrum für Kinder und Jugendrheumatologie Klinikum Eilbek Hs 6 Hamburg Germany

Hospital Dr Felipe Glasman Rheumatolgy Section Bahía Blanca Buenos Aires Argentina

Istituto Giannina Gaslini Nefrologia Genova Italy

Istituto Giannina Gaslini Pediatria 2 Reumatologia PRINTO Genova Italy

Istituto Giannina Gaslini Pediatria 2 Reumatologia PRINTO Genova Italy ; Charles University Prague 1st Medical Faculty and General University Hospital Prague Prague Czech Republic

Istituto Giannina Gaslini Pediatria 2 Reumatologia PRINTO Genova Italy ; Dipartimento di Pediatria Università degli Studi di Genova Genova Italy

Istituto Giannina Gaslini Servizio di Epidemiologia e Biostatistica Genova Italy

Juliane Marie Centret Rigshospitalet Pediatrisk klinik 2 Copenhagen Denmark

Kinderklinik Garmisch Partenkirchen gGmbH Deutsches Zentrum für Kinder und Jugendrheumatologie Garmisch Partenkirchen Germany

Pediatric Department University Hospital Centre Tirana Albania

Universidade Federal de São Paulo Pediatrics São Paulo Brazil

Université Paris Descartes Institut IMAGINE Hôpital Necker Enfants Malades Centre de référence national pour les Arthrites Juveniles Unité d'Immunologie Hématologie et Rhumatologie Pediatrique Paris France

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