title: Responsiveness of outcome measures in juvenile chronic arthritis. Italian Pediatric Rheumatology Study Group.
abstract: OBJECTIVE
  To examine the responsiveness of the disease activity measures more commonly used in juvenile chronic arthritis (JCA) clinical trials.
  
  
  METHODS
  Data were obtained from an open-label, non-controlled, multicentre trial designed to investigate the efficacy of methotrexate (MTX) in children with JCA. Outcome measures, including physician and parent global assessments, functional ability measures, articular variables, and laboratory indicators of systemic inflammation, were assessed at baseline and after 6 months of MTX treatment in 132 patients. Responsiveness of endpoint variables was evaluated by assessing the effect size (ES) and the standardized response median (SRM).
  
  
  RESULTS
  Physician and parent global assessments were the more responsive instruments, showing ES and SRM above 1.0. Erythrocyte sedimentation rate, C-reactive protein, functional status measures and articular variables showed intermediate responsiveness. Morning stiffness, haemoglobin and platelet count were the least responsive instruments.
  
  
  CONCLUSIONS
  The results of our analysis indicate that subjective estimations of the disease activity, either by the physician or parents, are the most responsive instruments in the assessment of the therapeutic response in children with JCA. The responsiveness of outcome measures in JCA should be further investigated in prospective controlled studies.
authors.affiliation: Laboratorio di Informatica Medica, IRCCS S. Matteo, Pavia, Italy.
authors.forename: N
authors.initials: N
authors.lastname: Ruperto
authors.forename: A
authors.initials: A
authors.lastname: Ravelli
authors.forename: F
authors.initials: F
authors.lastname: Falcini
authors.forename: L
authors.initials: L
authors.lastname: Lepore
authors.forename: A
authors.initials: A
authors.lastname: Buoncompagni
authors.forename: V
authors.initials: V
authors.lastname: Gerloni
authors.forename: M
authors.initials: M
authors.lastname: Bardare
authors.forename: E
authors.initials: E
authors.lastname: Cortis
authors.forename: F
authors.initials: F
authors.lastname: Zulian
authors.forename: M L
authors.initials: ML
authors.lastname: Sardella
authors.forename: C
authors.initials: C
authors.lastname: Giovanni Strano
authors.forename: M
authors.initials: M
authors.lastname: Alessio
authors.forename: M G
authors.initials: MG
authors.lastname: Alpigiani
authors.forename: D
authors.initials: D
authors.lastname: Migliavacca
authors.forename: A
authors.initials: A
authors.lastname: Pistorio
authors.forename: S
authors.initials: S
authors.lastname: Viola
authors.forename: A
authors.initials: A
authors.lastname: Martini
mesh_terms: D000293:Adolescent; D018501:Antirheumatic Agents / Q000627:therapeutic use*; D001171:Arthritis, Juvenile / Q000097:blood / Q000188:drug therapy* / Q000503:physiopathology; D002648:Child; D002675:Child, Preschool; D006801:Humans; D008727:Methotrexate / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D018501:Antirheumatic Agents; D008727:Methotrexate