title: The provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism Disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: a prospective validation study.
abstract: OBJECTIVE
  To validate a core set of outcome measures for the evaluation of response to treatment in patients with juvenile dermatomyositis (DM).
  
  
  METHODS
  In 2001, a preliminary consensus-derived core set for evaluating response to therapy in juvenile DM was established. In the present study, the core set was validated through an evidence-based, large-scale data collection that led to the enrollment of 294 patients from 36 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, concordance 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) muscle strength, 3) global disease activity measure, 4) parent's global assessment of patient's well-being, 5) functional ability, and 6) health-related quality of life.
  
  
  CONCLUSIONS
  The members of the Paediatric Rheumatology International Trials Organisation, with the endorsement of the American College of Rheumatology and the European League Against Rheumatism, 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 child with juvenile DM has responded adequately to therapy.
authors.affiliation: IRCCS G. 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: Angela
authors.initials: A
authors.lastname: Pistorio
authors.forename: Virginia
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authors.forename: Immaculada
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authors.lastname: Calvo
authors.forename: Gerd
authors.initials: G
authors.lastname: Ganser
authors.forename: Jurgen
authors.initials: J
authors.lastname: Brunner
authors.forename: Guenther
authors.initials: G
authors.lastname: Dannecker
authors.forename: Clovis Arthur
authors.initials: CA
authors.lastname: Silva
authors.forename: Valda
authors.initials: V
authors.lastname: Stanevicha
authors.forename: Rebecca Ten
authors.initials: RT
authors.lastname: Cate
authors.forename: Lisette W A
authors.initials: LW
authors.lastname: van Suijlekom-Smit
authors.forename: Olga
authors.initials: O
authors.lastname: Voygioyka
authors.forename: Michel
authors.initials: M
authors.lastname: Fischbach
authors.forename: Ivan
authors.initials: I
authors.lastname: Foeldvari
authors.forename: Odete
authors.initials: O
authors.lastname: Hilario
authors.forename: Consuelo
authors.initials: C
authors.lastname: Modesto
authors.forename: Rotraud K
authors.initials: RK
authors.lastname: Saurenmann
authors.forename: Marie-Josephe
authors.initials: MJ
authors.lastname: Sauvain
authors.forename: Iloite
authors.initials: I
authors.lastname: Scheibel
authors.forename: Danièle
authors.initials: D
authors.lastname: Sommelet
authors.forename: Lana
authors.initials: L
authors.lastname: Tambic-Bukovac
authors.forename: Roberto
authors.initials: R
authors.lastname: Barcellona
authors.forename: Riva
authors.initials: R
authors.lastname: Brik
authors.forename: Stephan
authors.initials: S
authors.lastname: Ehl
authors.forename: Mirjana
authors.initials: M
authors.lastname: Jovanovic
authors.forename: Jozef
authors.initials: J
authors.lastname: Rovensky
authors.forename: Francesca
authors.initials: F
authors.lastname: Bagnasco
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Lovell
authors.forename: Alberto
authors.initials: A
authors.lastname: Martini
mesh_terms: D002648:Child; D003882:Dermatomyositis / Q000175:diagnosis*; D005260:Female; D006801:Humans; D008297:Male; D017410:Practice Guidelines as Topic; D011446:Prospective Studies