title: Clinical characteristics and outcomes of childhood-onset ANCA-associated vasculitis: a French nationwide study.
abstract: BACKGROUND
  Data on anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis are scarce in children. The current study is aimed at describing the clinical features and outcomes of childhood-onset ANCA-associated vasculitis (AAV).
  
  
  METHODS
  We conducted a retrospective French multicentre study involving patients in whom AAV was diagnosed before the age of 18 years. Inclusion criteria were (i) granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) according to classification criteria of the European League Against Rheumatism/Paediatric Rheumatology European Society, and (ii) ANCA positivity. Patient and renal survival were analysed.
  
  
  RESULTS
  Among 66 children included, 80% were female, 42% had GPA and 58% MPA including renal-limited vasculitis, 67% were pANCA+ and 33% cANCA+. The mean incidence of reported cases increased to 0.45 per million children/year in the period 2006-10. Median age at diagnosis was 11.5 years, and median time to diagnosis was 1 month. Initial symptoms included fever and fatigue (79%), skin lesions (41%), arthritis (42%), pulmonary (45%) and renal involvement (88%). Clinical features were similar between GPA and MPA with the exception of upper airway impairment (28%) specific of GPA. Ninety percent of the patients achieved remission after induction treatment. After a median follow-up of 5.2 years, 4 patients (6%) died, corresponding to a mortality rate of 1.2 per 100 person-years, and 22 patients (34%) developed end-stage renal disease (ESRD). Renal survival was 74, 70 and 59% at 1, 5 and 10 years, respectively. In a multivariable Cox regression model, baseline glomerular filtration rate, ethnic origin, histopathological classification and era of treatment were associated with the occurrence of ESRD. Relapse-free survival was 57% at 5 years and 34% at 10 years of follow-up. Patient and renal outcome did not significantly differ between GPA and MPA.
  
  
  CONCLUSIONS
  Childhood-onset AAV is a rare disease characterized by female predominance, delayed diagnosis, frequent renal impairment and a high remission rate. Baseline GFR and new histopathological classification system are strong predictors of ESRD. Renal survival in childhood AAV has improved over time.
authors: Service de Pédiatrie, Hôpital Pellegrin-Enfants, CHU de Bordeaux, Bordeaux, France.
authors: Anne-Sylvia
authors: AS
authors: Sacri
authors: Service de Néphrologie, Hôpital Dupuytren, CHU de Limoges, Limoges, France.
authors: Tristan
authors: T
authors: Chambaraud
authors: Service de Néphrologie et Rhumatologie Pédiatriques, Hôpital Femme Mère Enfant, CHU de Lyon, Lyon, France.
authors: Bruno
authors: B
authors: Ranchin
authors: Unité d'Immunologie, Hématologie et Rhumatologie Pédiatriques, Hôpital Necker-Enfants-Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Benoît
authors: B
authors: Florkin
authors: Service de Néphrologie Pédiatrique, Hôpital Robert Debré, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Hélène
authors: H
authors: Sée
authors: Service de Néphrologie et Rhumatologie Pédiatriques, Hôpital des Enfants, CHU de Toulouse, Toulouse, France.
authors: Stéphane
authors: S
authors: Decramer
authors: Service de Pédiatrie, Groupe Hospitalier Saint-Pierre, CHU La Réunion, Saint Pierre, France.
authors: Hugues
authors: H
authors: Flodrops
authors: Service de Néphrologie Pédiatrique, Hôpital Trousseau, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Tim
authors: T
authors: Ulinski
authors: Service de Néphrologie et Rhumatologie Pédiatriques, Hôpital Femme Enfant Adolescent, CHU de Nantes, Nantes, France.
authors: Emma
authors: E
authors: Allain-Launay
authors: Service de Néphrologie Pédiatrique, Hôpital Necker-Enfants-Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Olivia
authors: O
authors: Boyer
authors: Service de Pédiatrie, Hôpital Félix Guyon, CHU La Réunion, Saint-Denis, La Réunion, France.
authors: Olivier
authors: O
authors: Dunand
authors: Service de Pédiatrie, Hôpital de Hautepierre, CHU de Strasbourg, Strasbourg, France.
authors: Michel
authors: M
authors: Fischbach
authors: Service de Médecine Interne, Hôpital Huriez, CHU de Lille, Lille, France.
authors: Eric
authors: E
authors: Hachulla
authors: Service de Pédiatrie, Hôpital Américain, CHU de Reims, Reims, France.
authors: Christine
authors: C
authors: Pietrement
authors: Service de Néphrologie, Hôpital Pontchaillou, CHU de Rennes, Rennes, France.
authors: Patrick
authors: P
authors: Le Pogamp
authors: Service de Pédiatrie, Hôpital Nord, CHU de Saint-Etienne, Saint Etienne, France.
authors: Jean-Louis
authors: JL
authors: Stephan
authors: Service de Néphrologie et Rhumatologie Pédiatriques, Hôpital Femme Mère Enfant, CHU de Lyon, Lyon, France.
authors: Alexandre
authors: A
authors: Belot
authors: Service de Néphrologie et Immunologie Clinique, Hôpital Bretonneau, CHU de Tours, Tours, France.
authors: Hubert
authors: H
authors: Nivet
authors: Service de Pédiatrie, Hôpital Jean Minjoz, CHU de Besançon, Besançon, France.
authors: François
authors: F
authors: Nobili
authors: Service de Médecine Interne, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Loic
authors: L
authors: Guillevin
authors: Unité d'Immunologie, Hématologie et Rhumatologie Pédiatriques, Hôpital Necker-Enfants-Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Pierre
authors: P
authors: Quartier
authors: Service de Néphrologie Pédiatrique, Hôpital Robert Debré, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Georges
authors: G
authors: Deschênes
authors: Service de Néphrologie Pédiatrique, Hôpital Necker-Enfants-Malades, Assistance Publique-Hôpitaux de Paris, Paris, France.
authors: Rémi
authors: R
authors: Salomon
authors: Service de Néphrologie, Hôpital Dupuytren, CHU de Limoges, Limoges, France.
authors: Marie
authors: M
authors: Essig
authors: Service de Pédiatrie, Hôpital Pellegrin-Enfants, CHU de Bordeaux, Bordeaux, France.
authors: Jérôme
authors: J
authors: Harambat
mesh_terms: D000293:Adolescent; D056648:Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis / Q000150:complications* / Q000401:mortality / Q000628:therapy; D019268:Antibodies, Antineutrophil Cytoplasmic / Q000276:immunology; D002648:Child; D005006:Ethnicity; D005260:Female; D005602:France / Q000453:epidemiology; D005919:Glomerular Filtration Rate; D006801:Humans; D015994:Incidence; D007674:Kidney Diseases / Q000453:epidemiology / Q000209:etiology* / Q000401:mortality; D008297:Male; D055953:Microscopic Polyangiitis / Q000150:complications* / Q000401:mortality / Q000628:therapy; D011379:Prognosis; D012008:Recurrence; D012189:Retrospective Studies; D015996:Survival Rate
chemical_list: D019268:Antibodies, Antineutrophil Cytoplasmic
keywords: ANCA-associated vasculitis; ESRD; children; outcome; relapse