title: Disease activity assessment in childhood vasculitis: development and preliminary validation of the Paediatric Vasculitis Activity Score (PVAS).
abstract: BACKGROUND
  Rare chronic childhood vasculitides lack a reliable disease activity assessment tool. With emerging new treatment modalities such a tool has become increasingly essential for both clinical practice and therapeutic trials to reproducibly quantify change in disease state.
  
  
  OBJECTIVE
  To develop and validate a paediatric vasculitis activity assessment tool based on modification of the Birmingham Vasculitis Activity Score (BVASv.3).
  
  
  METHODS
  A paediatric vasculitis registry was reviewed to identify clinical features missing in the BVASv.3. A modified nominal group technique was used to develop a working version of the Paediatric Vasculitis Activity Score (PVAS). Prospective validation provided tool reliability, reproducibility and responsiveness to change. Training of assessors was done according to the BVAS principles.
  
  
  RESULTS
  BVAS items were redefined (n=22) and eight paediatric items added in Cutaneous (n=4), Cardiovascular (n=3) and Abdominal (n=1) sections. The final PVAS has 64 active items in nine categories. The principles of new/worse and persistently active disease were retained as were the overall score and weighting of categories. The median PVAS in 63 children with systemic vasculitis was 4/63 (0-38/63). There was a high interobserver agreement for the overall as well as for subsystem scores (linear-weighted-κ ≥0.87). PVAS correlated with physician's global assessment (p<0.01); treatment decision (p=<0.01) and erythrocyte sedimentation rate (ESR) (p=0.01). In response to treatment, 15/19 patients assessed demonstrated a significant fall in PVAS (p=0.002), with good agreement among assessors for this change.
  
  
  CONCLUSIONS
  The PVAS validity in children with systemic vasculitis was demonstrated. Like the BVAS, we anticipate that the PVAS will provide a robust tool to objectively define disease activity for clinical trials and future research.
authors.affiliation: Rheumatology Unit, Department of Paediatrics and Adolescent Medicine, Charles University in Prague, 1st Faculty of Medicine and General University Hospital in Prague, Czech Republic. dolezalova.pavla@vfn.cz
authors.forename: Pavla
authors.initials: P
authors.lastname: Dolezalova
authors.forename: Fiona E
authors.initials: FE
authors.lastname: Price-Kuehne
authors.forename: Seza
authors.initials: S
authors.lastname: Özen
authors.forename: Susanne M
authors.initials: SM
authors.lastname: Benseler
authors.forename: David A
authors.initials: DA
authors.lastname: Cabral
authors.forename: Jordi
authors.initials: J
authors.lastname: Anton
authors.forename: Jürgen
authors.initials: J
authors.lastname: Brunner
authors.forename: Rolando
authors.initials: R
authors.lastname: Cimaz
authors.forename: Katheleen M
authors.initials: KM
authors.lastname: O'Neil
authors.forename: Carol A
authors.initials: CA
authors.lastname: Wallace
authors.forename: Nicholas
authors.initials: N
authors.lastname: Wilkinson
authors.forename: Despina
authors.initials: D
authors.lastname: Eleftheriou
authors.forename: Erkan
authors.initials: E
authors.lastname: Demirkaya
authors.forename: Marek
authors.initials: M
authors.lastname: Böhm
authors.forename: Petra
authors.initials: P
authors.lastname: Krol
authors.forename: Raashid A
authors.initials: RA
authors.lastname: Luqmani
authors.forename: Paul A
authors.initials: PA
authors.lastname: Brogan
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D008297:Male; D015588:Observer Variation; D015203:Reproducibility of Results; D012720:Severity of Illness Index; D016896:Treatment Outcome; D014657:Vasculitis / Q000175:diagnosis* / Q000188:drug therapy
chemical_list: D005938:Glucocorticoids
keywords: Disease Activity; Outcomes research; Systemic vasculitis