title: The Pediatric Rheumatology International Trials Organization criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus: prospective validation of the disease activity core set.
abstract: OBJECTIVE
  To validate and promulgate a core set of outcome measures for the evaluation of response to treatment in patients with juvenile systemic lupus erythematosus (SLE).
  
  
  METHODS
  In 2001, a preliminary consensus-derived core set of measures for evaluating the response to therapy in juvenile SLE was established. In the present study, the core set was validated through an evidence-based, large-scale data collection process that led to the enrollment of 557 patients from 39 different countries. Consecutive patients with active disease were assessed at baseline and after 6 months. The validation procedures included assessment of feasibility, responsiveness, discriminant and construct ability, agreement in the evaluation of response to therapy between physicians and parents, redundancy, internal consistency, and ability to predict a therapeutic response.
  
  
  RESULTS
  The following clinical measures were found to be feasible and to have good construct validity, discriminative ability, and internal consistency; furthermore, they were not redundant, proved responsive to clinically important changes in disease activity, and were associated strongly with treatment outcome and thus were included in the final core set: 1) physician's global assessment of disease activity, 2) global disease activity measure, 3) 24-hour proteinuria, 4) parent's global assessment of the patient's overall well-being, and 5) health-related quality of life assessment.
  
  
  CONCLUSIONS
  The members of PRINTO propose a core set of criteria for the evaluation of response to therapy that is scientifically and clinically relevant and statistically validated. The core set will help standardize the conduct and reporting of clinical trials and assist practitioners in deciding whether a patient with juvenile SLE has responded adequately to therapy.
authors.affiliation: Pediatric Rheumatology International Trials Organization (PRINTO), IRCCS G. Gaslini, Università di Genova, Pediatria II-Reumatologia, Largo Gaslini, 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: Ruben
authors.initials: R
authors.lastname: Cuttica
authors.forename: Graciela
authors.initials: G
authors.lastname: Espada
authors.forename: Seza
authors.initials: S
authors.lastname: Ozen
authors.forename: Oscar
authors.initials: O
authors.lastname: Porras
authors.forename: Flavio
authors.initials: F
authors.lastname: Sztajnbok
authors.forename: Fernanda
authors.initials: F
authors.lastname: Falcini
authors.forename: Ozgur
authors.initials: O
authors.lastname: Kasapcopur
authors.forename: Helen
authors.initials: H
authors.lastname: Venning
authors.forename: Blanca
authors.initials: B
authors.lastname: Bica
authors.forename: Rosa
authors.initials: R
authors.lastname: Merino
authors.forename: Cecilia
authors.initials: C
authors.lastname: Coto
authors.forename: Joan
authors.initials: J
authors.lastname: Ros
authors.forename: Gordana
authors.initials: G
authors.lastname: Susic
authors.forename: María Luz
authors.initials: ML
authors.lastname: Gamir
authors.forename: Kirsten
authors.initials: K
authors.lastname: Minden
authors.forename: Yvonne
authors.initials: Y
authors.lastname: See
authors.forename: Yosef
authors.initials: Y
authors.lastname: Uziel
authors.forename: Masha
authors.initials: M
authors.lastname: Mukamel
authors.forename: Phil
authors.initials: P
authors.lastname: Riley
authors.forename: Francesco
authors.initials: F
authors.lastname: Zulian
authors.forename: Alma Nunzia
authors.initials: AN
authors.lastname: Olivieri
authors.forename: Rolando
authors.initials: R
authors.lastname: Cimaz
authors.forename: Hermann
authors.initials: H
authors.lastname: Girschick
authors.forename: Ingrida
authors.initials: I
authors.lastname: Rumba
authors.forename: Silvio
authors.initials: S
authors.lastname: Cavuto
authors.forename: Angela
authors.initials: A
authors.lastname: Pistorio
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Lovell
authors.forename: Alberto
authors.initials: A
authors.lastname: Martini
mesh_terms: D000203:Activities of Daily Living; D002986:Clinical Trials as Topic / Q000379:methods; D019317:Evidence-Based Medicine; 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; D011507:Proteinuria / Q000175:diagnosis; D015203:Reproducibility of Results; D012219:Rheumatology / Q000379:methods* / Q000592:standards; D012720:Severity of Illness Index; D016896:Treatment Outcome