title: Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis.
abstract: OBJECTIVE
  To identify preliminary core sets of outcome variables for disease activity and damage assessment in juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM).
  
  
  METHODS
  Two questionnaire surveys were mailed to 267 physicians from 46 different countries asking each member to select and rank the response variables used when assessing clinical response in patients with JSLE or JDM. Next, 40 paediatric rheumatologists from 34 countries met and, using the nominal group technique, selected the domains to be included in the disease activity and damage core sets for JSLE and JDM.
  
  
  RESULTS
  A total of 41 response variables for JSLE and 37 response variables for JDM were selected and ranked through the questionnaire surveys. In the consensus conference, domains selected for both JSLE and JDM activity or damage core sets included the physician and parent/patient subjective assessments and a global score tool. Domains specific for JSLE activity were the immunological tests and the kidney function parameters. Concerning JDM, functional ability and muscle strength assessments were indicated for both activity and damage core sets, whereas serum muscle enzymes were included only in the activity core set. A specific paediatric domain called 'growth and development' was introduced in the disease damage core set for both diseases and the evaluation of health-related quality of life was advised in order to capture the influence of the disease on the patient lifestyle.
  
  
  CONCLUSIONS
  We developed preliminary core sets of measures for disease activity and damage assessment in JSLE and JDM. The prospective validation of the core sets is in progress.
authors.affiliation: IRCCS G. Gasalini, Pediatria II, Genova, Italy. nicolaruperto@ospedale-gaslini.ge.it
authors.forename: Nicolino
authors.initials: N
authors.lastname: Ruperto
authors.forename: Angelo
authors.initials: A
authors.lastname: Ravelli
authors.forename: Kevin J
authors.initials: KJ
authors.lastname: Murray
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Lovell
authors.forename: Boel
authors.initials: B
authors.lastname: Andersson-Gare
authors.forename: Brian M
authors.initials: BM
authors.lastname: Feldman
authors.forename: Stella
authors.initials: S
authors.lastname: Garay
authors.forename: Wietse
authors.initials: W
authors.lastname: Kuis
authors.forename: Claudia
authors.initials: C
authors.lastname: Machado
authors.forename: Lauren
authors.initials: L
authors.lastname: Pachman
authors.forename: Anne-Marie
authors.initials: AM
authors.lastname: Prieur
authors.forename: Lisa G
authors.initials: LG
authors.lastname: Rider
authors.forename: Earl
authors.initials: E
authors.lastname: Silverman
authors.forename: Elena
authors.initials: E
authors.lastname: Tsitsami
authors.forename: Pat
authors.initials: P
authors.lastname: Woo
authors.forename: Edward H
authors.initials: EH
authors.lastname: Giannini
authors.forename: Alberto
authors.initials: A
authors.lastname: Martini
mesh_terms: D002648:Child; D002986:Clinical Trials as Topic / Q000379:methods; D003697:Delphi Technique; D003882:Dermatomyositis / Q000175:diagnosis* / Q000628:therapy; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000175:diagnosis* / Q000628:therapy; D011446:Prospective Studies; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires; D016896:Treatment Outcome