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. Conclusion. 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.
| Original language | English |
|---|---|
| Pages (from-to) | 2854-2864 |
| Number of pages | 11 |
| Journal | Arthritis and Rheumatism |
| Volume | 52 |
| Issue number | 9 |
| DOIs | |
| Publication status | Published - Sep 2005 |
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ASJC Scopus subject areas
- Immunology
- Rheumatology
Cite this
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. / Ruperto, Nicolino; Ravelli, Angelo; Cuttica, Ruben; Espada, Graciela; Ozen, Seza; Porras, Oscar; Sztajnbok, Flavio; Falcini, Fernanda; Kasapcopur, Ozgur; Venning, Helen; Bica, Bianca; Merino, Rosa; Coto, Cecilia; Ros, Joan; Susic, Gordana; Gamir, Mária Luz; Minden, Kirsten; See, Yvonne; Uziel, Yosef; Mukamel, Masha; Riley, Phil; Zulian, Francesco; Olivieri, Alma Nunzia; Cimaz, Rolando; Girschick, Hermann; Rumba, Ingrida; Cavuto, Silvio; Pistorio, Angela; Lovell, Daniel J.; Martini, Alberto.
In: Arthritis and Rheumatism, Vol. 52, No. 9, 09.2005, p. 2854-2864.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - The pediatric rheumatology international trials organization criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus
T2 - Prospective validation of the disease activity core set
AU - Ruperto, Nicolino
AU - Ravelli, Angelo
AU - Cuttica, Ruben
AU - Espada, Graciela
AU - Ozen, Seza
AU - Porras, Oscar
AU - Sztajnbok, Flavio
AU - Falcini, Fernanda
AU - Kasapcopur, Ozgur
AU - Venning, Helen
AU - Bica, Bianca
AU - Merino, Rosa
AU - Coto, Cecilia
AU - Ros, Joan
AU - Susic, Gordana
AU - Gamir, Mária Luz
AU - Minden, Kirsten
AU - See, Yvonne
AU - Uziel, Yosef
AU - Mukamel, Masha
AU - Riley, Phil
AU - Zulian, Francesco
AU - Olivieri, Alma Nunzia
AU - Cimaz, Rolando
AU - Girschick, Hermann
AU - Rumba, Ingrida
AU - Cavuto, Silvio
AU - Pistorio, Angela
AU - Lovell, Daniel J.
AU - Martini, Alberto
PY - 2005/9
Y1 - 2005/9
N2 - 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. Conclusion. 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.
AB - 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. Conclusion. 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.
UR - http://www.scopus.com/inward/record.url?scp=25444439493&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=25444439493&partnerID=8YFLogxK
U2 - 10.1002/art.21230
DO - 10.1002/art.21230
M3 - Article
C2 - 16142708
AN - SCOPUS:25444439493
VL - 52
SP - 2854
EP - 2864
JO - Arthritis care and research : the official journal of the Arthritis Health Professions Association
JF - Arthritis care and research : the official journal of the Arthritis Health Professions Association
SN - 0893-7524
IS - 9
ER -