TY - JOUR
T1 - The Paediatric Rheumatology International Trials Organisation provisional criteria for the evaluation of response to therapy in juvenile dermatomyositis.
AU - Ruperto, Nicolino
AU - Pistorio, Angela
AU - Ravelli, Angelo
AU - Rider, Lisa G.
AU - Pilkington, Clarissa
AU - Oliveira, Sheila
AU - Wulffraat, Nico
AU - Espada, Graciela
AU - Garay, Stella
AU - Cuttica, Ruben
AU - Hofer, Michael
AU - Quartier, Pierre
AU - Melo-Gomes, Jose
AU - Reed, Ann M.
AU - Wierzbowska, Malgorzata
AU - Feldman, Brian M.
AU - Harjacek, Miroslav
AU - Huppertz, Hans Iko
AU - Nielsen, Susan
AU - Flato, Berit
AU - Lahdenne, Pekka
AU - Michels, Harmut
AU - Murray, Kevin J.
AU - Punaro, Lynn
AU - Rennebohm, Robert
AU - Russo, Ricardo
AU - Balogh, Zsolt
AU - Rooney, Madeleine
AU - Pachman, Lauren M.
AU - Wallace, Carol
AU - Hashkes, Philip
AU - Lovell, Daniel J.
AU - Giannini, Edward H.
AU - Gare, Boel Andersson
AU - Martini, Alberto
AU - Paediatric Rheumatology International Trials Organisation (PRINTO), Rheumatology International Trials Organisation (PRINTO)
AU - Pediatric Rheumatology Collaborative Study Group (PRCSG), Rheumatology Collaborative Study Group (PRCSG)
PY - 2010/11
Y1 - 2010/11
N2 - To develop a provisional definition for the evaluation of response to therapy in juvenile dermatomyositis (DM) based on the Paediatric Rheumatology International Trials Organisation juvenile DM core set of variables. Thirty-seven experienced pediatric rheumatologists from 27 countries achieved consensus on 128 difficult patient profiles as clinically improved or not improved using a stepwise approach (patient's rating, statistical analysis, definition selection). Using the physicians' consensus ratings as the "gold standard measure," chi-square, sensitivity, specificity, false-positive and-negative rates, area under the receiver operating characteristic curve, and kappa agreement for candidate definitions of improvement were calculated. Definitions with kappa values >0.8 were multiplied by the face validity score to select the top definitions. The top definition of improvement was at least 20% improvement from baseline in 3 of 6 core set variables with no more than 1 of the remaining worsening by more than 30%, which cannot be muscle strength. The second-highest scoring definition was at least 20% improvement from baseline in 3 of 6 core set variables with no more than 2 of the remaining worsening by more than 25%, which cannot be muscle strength (definition P1 selected by the International Myositis Assessment and Clinical Studies group). The third is similar to the second with the maximum amount of worsening set to 30%. This indicates convergent validity of the process. We propose a provisional data-driven definition of improvement that reflects well the consensus rating of experienced clinicians, which incorporates clinically meaningful change in core set variables in a composite end point for the evaluation of global response to therapy in juvenile DM.
AB - To develop a provisional definition for the evaluation of response to therapy in juvenile dermatomyositis (DM) based on the Paediatric Rheumatology International Trials Organisation juvenile DM core set of variables. Thirty-seven experienced pediatric rheumatologists from 27 countries achieved consensus on 128 difficult patient profiles as clinically improved or not improved using a stepwise approach (patient's rating, statistical analysis, definition selection). Using the physicians' consensus ratings as the "gold standard measure," chi-square, sensitivity, specificity, false-positive and-negative rates, area under the receiver operating characteristic curve, and kappa agreement for candidate definitions of improvement were calculated. Definitions with kappa values >0.8 were multiplied by the face validity score to select the top definitions. The top definition of improvement was at least 20% improvement from baseline in 3 of 6 core set variables with no more than 1 of the remaining worsening by more than 30%, which cannot be muscle strength. The second-highest scoring definition was at least 20% improvement from baseline in 3 of 6 core set variables with no more than 2 of the remaining worsening by more than 25%, which cannot be muscle strength (definition P1 selected by the International Myositis Assessment and Clinical Studies group). The third is similar to the second with the maximum amount of worsening set to 30%. This indicates convergent validity of the process. We propose a provisional data-driven definition of improvement that reflects well the consensus rating of experienced clinicians, which incorporates clinically meaningful change in core set variables in a composite end point for the evaluation of global response to therapy in juvenile DM.
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U2 - 10.1002/acr.20280
DO - 10.1002/acr.20280
M3 - Article
C2 - 20583105
AN - SCOPUS:79952111683
SN - 2151-464X
VL - 62
SP - 1533
EP - 1541
JO - Arthritis and Rheumatism
JF - Arthritis and Rheumatism
IS - 11
ER -