TY - JOUR
T1 - 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
T2 - A prospective validation study
AU - Ruperto, Nicolino
AU - Ravelli, Angelo
AU - Pistorio, Angela
AU - Ferriani, Virginia
AU - Calvo, Immaculada
AU - Ganser, Gerd
AU - Brunner, Jurgen
AU - Dannecker, Guenther
AU - Silva, Clovis Arthur
AU - Stanevicha, Valda
AU - Ten Cate, Rebecca
AU - Van Suijlekom-Smit, Lisette W.A.
AU - Voygioyka, Olga
AU - Fischbach, Michel
AU - Foeldvari, Ivan
AU - Hilario, Odete
AU - Modesto, Consuelo
AU - Saurenmann, Rotraud K.
AU - Sauvain, Marie Josephe
AU - Scheibel, Iloite
AU - Sommelet, Danièle
AU - Tambic-Bukovac, Lana
AU - Barcellona, Roberto
AU - Brik, Riva
AU - Ehl, Stephan
AU - Jovanovic, Mirjana
AU - Rovensky, Jozef
AU - Bagnasco, Francesca
AU - Lovell, Daniel J.
AU - Martini, Alberto
PY - 2008/1/15
Y1 - 2008/1/15
N2 - 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. Conclusion. 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.
AB - 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. Conclusion. 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.
UR - http://www.scopus.com/inward/record.url?scp=38649141175&partnerID=8YFLogxK
U2 - 10.1002/art.23248
DO - 10.1002/art.23248
M3 - Article
C2 - 18163404
AN - SCOPUS:38649141175
SN - 2151-4658
VL - 59
SP - 4
EP - 13
JO - Arthritis Care and Research
JF - Arthritis Care and Research
IS - 1
ER -