title: The Pediatric Rheumatology International Trials Organization/American College of Rheumatology provisional criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus: prospective validation of the definition of improvement.
abstract: OBJECTIVE
  To use the Pediatric Rheumatology International Trials Organization (PRINTO) core set of outcome measures to develop a validated definition of improvement for the evaluation of response to therapy in juvenile systemic lupus erythematosus (SLE).
  
  
  METHODS
  Thirty-seven experienced pediatric rheumatologists from 27 countries, each of whom had specific experience in the assessment of juvenile SLE patients, achieved consensus on 128 patient profiles as being clinically improved or not improved. Using the physicians' consensus ratings as the gold standard measure, the chi-square, sensitivity, specificity, false-positive and false-negative rates, area under the receiver operating characteristic curve, and kappa level of agreement for 597 candidate definitions of improvement were calculated. Only definitions with a kappa value greater than 0.7 were retained. The top definitions were selected based on the product of the content validity score multiplied by its kappa statistic.
  
  
  RESULTS
  The definition of improvement with the highest final score was at least 50% improvement from baseline in any 2 of the 5 core set measures, with no more than 1 of the remaining worsening by more than 30%.
  
  
  CONCLUSIONS
  PRINTO proposes a valid and reproducible definition of improvement that reflects well the consensus rating of experienced clinicians and that incorporates clinically meaningful change in core set measures in a composite end point for the evaluation of global response to therapy in patients with juvenile SLE. The definition is now proposed for use in juvenile SLE clinical trials and may help physicians to decide whether a child with SLE responded adequately to therapy.
authors.affiliation: Pediatric Rheumatology International Trials Organization, IRCCS G. Gaslini, Università di Genova Pediatria II-Reumatologia, 5 Largo Gaslini, 16147 Genoa, 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: Sheila
authors.initials: S
authors.lastname: Oliveira
authors.forename: Maria
authors.initials: M
authors.lastname: Alessio
authors.forename: Dimitrina
authors.initials: D
authors.lastname: Mihaylova
authors.forename: Srdjan
authors.initials: S
authors.lastname: Pasic
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Cortis
authors.forename: Maria
authors.initials: M
authors.lastname: Apaz
authors.forename: Ruben
authors.initials: R
authors.lastname: Burgos-Vargas
authors.forename: Florence
authors.initials: F
authors.lastname: Kanakoudi-Tsakalidou
authors.forename: Ximena
authors.initials: X
authors.lastname: Norambuena
authors.forename: Fabrizia
authors.initials: F
authors.lastname: Corona
authors.forename: Valeria
authors.initials: V
authors.lastname: Gerloni
authors.forename: Stefan
authors.initials: S
authors.lastname: Hagelberg
authors.forename: Amita
authors.initials: A
authors.lastname: Aggarwal
authors.forename: Pavla
authors.initials: P
authors.lastname: Dolezalova
authors.forename: Claudia Magalhaes
authors.initials: CM
authors.lastname: Saad
authors.forename: Sang-Cheol
authors.initials: SC
authors.lastname: Bae
authors.forename: Richard
authors.initials: R
authors.lastname: Vesely
authors.forename: Tadej
authors.initials: T
authors.lastname: Avcin
authors.forename: Helen
authors.initials: H
authors.lastname: Foster
authors.forename: Carolina
authors.initials: C
authors.lastname: Duarte
authors.forename: Troels
authors.initials: T
authors.lastname: Herlin
authors.forename: Gerd
authors.initials: G
authors.lastname: Horneff
authors.forename: Loredana
authors.initials: L
authors.lastname: Lepore
authors.forename: Marion
authors.initials: M
authors.lastname: van Rossum
authors.forename: Lucia
authors.initials: L
authors.lastname: Trail
authors.forename: Angela
authors.initials: A
authors.lastname: Pistorio
authors.forename: Boel
authors.initials: B
authors.lastname: Andersson-Gäre
authors.forename: Edward H
authors.initials: EH
authors.lastname: Giannini
authors.forename: Alberto
authors.initials: A
authors.lastname: Martini
mesh_terms: D002648:Child; D032921:Consensus; D023381:Endpoint Determination / Q000379:methods* / Q000592:standards; D005260:Female; D006304:Health Status; D006801:Humans; D007391:International Cooperation; D008180:Lupus Erythematosus, Systemic / Q000175:diagnosis* / Q000503:physiopathology / Q000628:therapy; D008297:Male; D017063:Outcome Assessment, Health Care; D010372:Pediatrics / Q000379:methods*; D015203:Reproducibility of Results; D012219:Rheumatology / Q000379:methods*; D012720:Severity of Illness Index; D012955:Societies, Medical; D016896:Treatment Outcome