title: Clinical course and outcomes of childhood-onset granulomatosis with polyangiitis.
abstract: OBJECTIVE
  To characterise the clinical course and outcomes of a cohort of children with granulomatosis with polyangiitis (GPA).
  
  
  METHODS
  Retrospective cohort study of children diagnosed with GPA in a tertiary care facility from 2000-2014. All subjects met the American College of Rheumatology 1990 criteria for GPA or the 2008 European League against Rheumatism/Paediatric Rheumatology International Trials Organisation/Paediatric Rheumatology European Society criteria for GPA. Predictors of readmission were determined using univariate logistic regression. Kaplan-Meier analysis was used to demonstrate the relapse-free survival probability in follow-up.
  
  
  RESULTS
  Twenty-eight children (median age 14.7 years) were diagnosed during the study period. At presentation 14 (50%), 5 (18%), and 4 (14%) children required intensive care unit care, ventilator support, and dialysis, respectively. One-third of the children in our cohort had gastrointestinal involvement, one-quarter of whom were previously diagnosed with inflammatory bowel disease. Two-thirds of children were readmitted. Renal failure and infections accounted for most readmissions. Twenty-three (85%) patients achieved remission, however, 11 subsequently flared (median time to flare 21.5 months). Haematuria at diagnosis was significantly associated with readmission (OR 6.25). At a median follow-up of 3.3 years (range 5 months to 6 years) 10 (37%) children had chronic kidney disease (> stage 2) and none of the children died.
  
  
  CONCLUSIONS
  Children with GPA frequently have severe disease presentations including significant renal, respiratory and gastrointestinal involvement. While most children with GPA achieve remission, nearly half have subsequent relapses.
authors.affiliation: Division of Rheumatology, Department of Paediatrics, Children's Hospital of Philadelphia, PA, USA. karenejames24@gmail.com.
authors.forename: Karen E
authors.initials: KE
authors.lastname: James
authors.affiliation: Department of Biostatistics, Children's Hospital of Philadelphia, and Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Rui
authors.initials: R
authors.lastname: Xiao
authors.affiliation: Department of Biostatistics and Epidemiology, and Division of Rheumatology, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Merkel
authors.affiliation: Division of Rheumatology, Department of Paediatrics, Children's Hospital of Philadelphia; and Department of Paediatrics, Center for Paediatric Clinical Effectiveness, Children's Hospital of Philadelphia, PA, USA.
authors.forename: Pamela F
authors.initials: PF
authors.lastname: Weiss
mesh_terms: D000293:Adolescent; D017668:Age of Onset; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D014890:Granulomatosis with Polyangiitis / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D016015:Logistic Models; D008297:Male; D010359:Patient Readmission; D015143:Philadelphia; D012008:Recurrence; D012074:Remission Induction; D012189:Retrospective Studies; D012307:Risk Factors; D062606:Tertiary Care Centers; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents