title: Early Outcomes in Children With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis.
abstract: To characterize the early disease course in childhood-onset antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) and the 12-month outcomes in children with AAV.
  
  
  
  Eligible subjects were children entered into the Pediatric Vasculitis Initiative study who were diagnosed before their eighteenth birthday as having granulomatosis with polyangiitis (Wegener's), microscopic polyangiitis, eosinophilic granulomatosis with polyangiitis (Churg-Strauss), or ANCA-positive pauci-immune glomerulonephritis. The primary outcome measure was achievement of disease remission (Pediatric Vasculitis Activity Score [PVAS] of 0) at 12 months with a corticosteroid dosage of <0.2 mg/kg/day. Secondary outcome measures included the rates of inactive disease (PVAS of 0, with any corticosteroid dosage) and rates of improvement at postinduction (4-6 months after diagnosis) and at 12 months, presence of damage at 12 months (measured by a modified Pediatric Vasculitis Damage Index [PVDI]; score 0 = no damage, score 1 = one damage item present), and relapse rates at 12 months.
  
  
  
  In total, 105 children with AAV were included in the study. The median age at diagnosis was 13.8 years (interquartile range 10.9-15.8 years). Among the study cohort, 42% of patients achieved remission at 12 months, 49% had inactive disease at postinduction (4-6 months), and 61% had inactive disease at 12 months. The majority of patients improved, even if they did not achieve inactive disease. An improvement in the PVAS score of at least 50% from time of diagnosis to postinduction was seen in 92% of patients. Minor relapses occurred in 12 (24%) of 51 patients after inactive disease had been achieved postinduction. The median PVDI damage score at 12 months was 1 (range 0-6), and 63% of patients had ≥1 PVDI damage item scored as present at 12 months.
  
  
  
  This is the largest study to date to assess disease outcomes in pediatric AAV. Although the study showed that a significant proportion of patients did not achieve remission, the majority of patients responded to treatment. Unfortunately, more than one-half of this patient cohort experienced damage to various organ systems early in their disease course.
authors.affiliation: Kimberly A. Morishita, MD, MHSc, David A. Cabral, MBBS: British Columbia Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
authors.forename: Kimberly A
authors.initials: KA
authors.lastname: Morishita
authors.affiliation: Rutgers-Robert Wood Johnson Medical School, New Brunswick, New Jersey.
authors.forename: Lakshmi N
authors.initials: LN
authors.lastname: Moorthy
authors.affiliation: Simon Fraser University, Burnaby, British Columbia, Canada.
authors.forename: Joanna M
authors.initials: JM
authors.lastname: Lubieniecka
authors.affiliation: Marinka Twilt, MD, PhD, Susanne M. Benseler, MD, PhD: Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
authors.forename: Marinka
authors.initials: M
authors.lastname: Twilt
authors.affiliation: Rae S. M. Yeung, MD, PhD: The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Rae S M
authors.initials: RSM
authors.lastname: Yeung
authors.affiliation: Akron Children's Hospital, Akron, Ohio.
authors.forename: Mary B
authors.initials: MB
authors.lastname: Toth
authors.affiliation: Seattle Children's Hospital, Seattle, Washington.
authors.forename: Susan
authors.initials: S
authors.lastname: Shenoi
authors.affiliation: Mother and Child Health Care Institute of Serbia, Belgrade, Serbia.
authors.forename: Goran
authors.initials: G
authors.lastname: Ristic
authors.affiliation: Rigshospitalet, Copenhagen, Denmark.
authors.forename: Susan M
authors.initials: SM
authors.lastname: Nielsen
authors.affiliation: Nuffield Orthopaedic Centre, University of Oxford, Oxford, UK.
authors.forename: Raashid A
authors.initials: RA
authors.lastname: Luqmani
authors.affiliation: Joseph M. Sanzari Children's Hospital, Hackensack, New Jersey.
authors.forename: Suzanne C
authors.initials: SC
authors.lastname: Li
authors.affiliation: Tzielan Lee, MD: Stanford Children's Health, Stanford University School of Medicine, Stanford, California.
authors.forename: Tzielan
authors.initials: T
authors.lastname: Lee
authors.affiliation: University of California at San Francisco.
authors.forename: Erica F
authors.initials: EF
authors.lastname: Lawson
authors.affiliation: Saint-Petersburg State Pediatric Medical University, Saint Petersburg, Russia.
authors.forename: Mikhail M
authors.initials: MM
authors.lastname: Kostik
authors.affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois.
authors.forename: Marisa
authors.initials: M
authors.lastname: Klein-Gitelman
authors.affiliation: IWK Health Centre and Dalhousie University, Halifax, Nova Scotia, Canada.
authors.forename: Adam M
authors.initials: AM
authors.lastname: Huber
authors.affiliation: University of Utah, Salt Lake City.
authors.forename: Aimee O
authors.initials: AO
authors.lastname: Hersh
authors.affiliation: University Children's Hospital, Muenster, Germany.
authors.forename: Dirk
authors.initials: D
authors.lastname: Foell
authors.affiliation: University of Florida, Gainesville.
authors.forename: Melissa E
authors.initials: ME
authors.lastname: Elder
authors.affiliation: Cohen Children's Medical Center of New York, New Hyde Park, New York.
authors.forename: Barbara A
authors.initials: BA
authors.lastname: Eberhard
authors.affiliation: Janeway Children's Health and Rehabilitation Centre, St. John's, Newfoundland, Canada.
authors.forename: Paul
authors.initials: P
authors.lastname: Dancey
authors.affiliation: Sirirat Charuvanij, MD: Siriraj Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: Sirirat
authors.initials: S
authors.lastname: Charuvanij
authors.affiliation: Marinka Twilt, MD, PhD, Susanne M. Benseler, MD, PhD: Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
authors.forename: Susanne M
authors.initials: SM
authors.lastname: Benseler
authors.affiliation: Kimberly A. Morishita, MD, MHSc, David A. Cabral, MBBS: British Columbia Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
authors.forename: David A
authors.initials: DA
authors.lastname: Cabral
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D056648:Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis / Q000150:complications / Q000188:drug therapy*; D001379:Azathioprine / Q000627:therapeutic use; D002648:Child; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007674:Kidney Diseases / Q000188:drug therapy* / Q000209:etiology; D008171:Lung Diseases / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D009173:Mycophenolic Acid / Q000627:therapeutic use; D011446:Prospective Studies; D012008:Recurrence; D012042:Registries; D012074:Remission Induction; D012189:Retrospective Studies; D000069283:Rituximab / Q000627:therapeutic use
chemical_list: D000305:Adrenal Cortex Hormones; D007166:Immunosuppressive Agents; D000069283:Rituximab; D003520:Cyclophosphamide; D009173:Mycophenolic Acid; D001379:Azathioprine; D008727:Methotrexate