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

Nicolino Ruperto, Angelo Ravelli, Angela Pistorio, Virginia Ferriani, Immaculada Calvo, Gerd Ganser, Jurgen Brunner, Guenther Dannecker, Clovis Arthur Silva, Valda Stanevicha, Rebecca Ten Cate, Lisette W A Van Suijlekom-Smit, Olga Voygioyka, Michel Fischbach, Ivan Foeldvari, Odete Hilario, Consuelo Modesto, Rotraud K. Saurenmann, Marie Josephe Sauvain, Iloite Scheibel & 10 others Danièle Sommelet, Lana Tambic-Bukovac, Roberto Barcellona, Riva Brik, Stephan Ehl, Mirjana Jovanovic, Jozef Rovensky, Francesca Bagnasco, Daniel J. Lovell, Alberto Martini

Research output: Contribution to journalArticle

97 Citations (Scopus)

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. 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.

Original languageEnglish
Pages (from-to)4-13
Number of pages10
JournalArthritis Care and Research
Volume59
Issue number1
DOIs
Publication statusPublished - Jan 15 2008

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Validation Studies
Rheumatology
Aptitude
Organizations
Prospective Studies
Pediatrics
Therapeutics
Parents
Physicians
Muscle Strength
Juvenile dermatomyositis
Consensus
Quality of Life
Outcome Assessment (Health Care)
Clinical Trials

ASJC Scopus subject areas

  • Rheumatology

Cite this

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. / Ruperto, Nicolino; Ravelli, Angelo; Pistorio, Angela; Ferriani, Virginia; Calvo, Immaculada; Ganser, Gerd; Brunner, Jurgen; Dannecker, Guenther; Silva, Clovis Arthur; Stanevicha, Valda; Ten Cate, Rebecca; Van Suijlekom-Smit, Lisette W A; Voygioyka, Olga; Fischbach, Michel; Foeldvari, Ivan; Hilario, Odete; Modesto, Consuelo; Saurenmann, Rotraud K.; Sauvain, Marie Josephe; Scheibel, Iloite; Sommelet, Danièle; Tambic-Bukovac, Lana; Barcellona, Roberto; Brik, Riva; Ehl, Stephan; Jovanovic, Mirjana; Rovensky, Jozef; Bagnasco, Francesca; Lovell, Daniel J.; Martini, Alberto.

In: Arthritis Care and Research, Vol. 59, No. 1, 15.01.2008, p. 4-13.

Research output: Contribution to journalArticle

Ruperto, N, Ravelli, A, Pistorio, A, Ferriani, V, Calvo, I, Ganser, G, Brunner, J, Dannecker, G, Silva, CA, Stanevicha, V, Ten Cate, R, Van Suijlekom-Smit, LWA, Voygioyka, O, Fischbach, M, Foeldvari, I, Hilario, O, Modesto, C, Saurenmann, RK, Sauvain, MJ, Scheibel, I, Sommelet, D, Tambic-Bukovac, L, Barcellona, R, Brik, R, Ehl, S, Jovanovic, M, Rovensky, J, Bagnasco, F, Lovell, DJ & Martini, A 2008, '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', Arthritis Care and Research, vol. 59, no. 1, pp. 4-13. https://doi.org/10.1002/art.23248
Ruperto, Nicolino ; Ravelli, Angelo ; Pistorio, Angela ; Ferriani, Virginia ; Calvo, Immaculada ; Ganser, Gerd ; Brunner, Jurgen ; Dannecker, Guenther ; Silva, Clovis Arthur ; Stanevicha, Valda ; Ten Cate, Rebecca ; Van Suijlekom-Smit, Lisette W A ; Voygioyka, Olga ; Fischbach, Michel ; Foeldvari, Ivan ; Hilario, Odete ; Modesto, Consuelo ; Saurenmann, Rotraud K. ; Sauvain, Marie Josephe ; Scheibel, Iloite ; Sommelet, Danièle ; Tambic-Bukovac, Lana ; Barcellona, Roberto ; Brik, Riva ; Ehl, Stephan ; Jovanovic, Mirjana ; Rovensky, Jozef ; Bagnasco, Francesca ; Lovell, Daniel J. ; Martini, Alberto. / 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. In: Arthritis Care and Research. 2008 ; Vol. 59, No. 1. pp. 4-13.
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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. 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.",
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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

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AU - Modesto, Consuelo

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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

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