• Nicolino Ruperto, Angelo Ravelli, Sheila Oliveira, Maria Alessio, Dimitrina Mihaylova, Srdjan Pasic, Elisabetta Cortis, Maria Apaz, Ruben Burgos-Vargas, Florence Kanakoudi-Tsakalidou, Ximena Norambuena, Fabrizia Corona, Valeria Gerloni, Stefan Hagelberg, Amita Aggarwal, Pavla Dolezalova, Claudia Magalhaes Saad, Sang-Cheol Bae, Richard Vesely, Tadej Avcin, Helen Foster, Carolina Duarte, Troels Herlin, Gerd Horneff, Loredana Lepore, Marion van Rossum, Lucia Trail, Angela Pistorio, Boel Andersson-Gäre, Edward H Giannini, Alberto Martini, and Pediatric Rheumatology International Trials Organization.
• Pediatric Rheumatology International Trials Organization, IRCCS G. Gaslini, Università di Genova Pediatria II-Reumatologia, 5 Largo Gaslini, 16147 Genoa, Italy. nicolaruperto@ospedale-gaslini.ge.it
• Arthritis Rheum. 2006 Jun 15;55(3):355-63.

ObjectiveTo use the Pediatric Rheumatology International Trials Organization (PRINTO) core set of outcome measures to develop a validated definition of improvement for the evaluation of response to therapy in juvenile systemic lupus erythematosus (SLE).MethodsThirty-seven experienced pediatric rheumatologists from 27 countries, each of whom had specific experience in the assessment of juvenile SLE patients, achieved consensus on 128 patient profiles as being clinically improved or not improved. Using the physicians' consensus ratings as the gold standard measure, the chi-square, sensitivity, specificity, false-positive and false-negative rates, area under the receiver operating characteristic curve, and kappa level of agreement for 597 candidate definitions of improvement were calculated. Only definitions with a kappa value greater than 0.7 were retained. The top definitions were selected based on the product of the content validity score multiplied by its kappa statistic.ResultsThe definition of improvement with the highest final score was at least 50% improvement from baseline in any 2 of the 5 core set measures, with no more than 1 of the remaining worsening by more than 30%.ConclusionPRINTO proposes a valid and reproducible definition of improvement that reflects well the consensus rating of experienced clinicians and that incorporates clinically meaningful change in core set measures in a composite end point for the evaluation of global response to therapy in patients with juvenile SLE. The definition is now proposed for use in juvenile SLE clinical trials and may help physicians to decide whether a child with SLE responded adequately to therapy.

25 keywords...

hide…