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The Paediatric Rheumatology International Trials Organisation provisional criteria for the evaluation of response to therapy in juvenile dermatomyositis
Author(s) -
Ruperto Nicolino,
Pistorio Angela,
Ravelli Angelo,
Rider Lisa G.,
Pilkington Clarissa,
Oliveira Sheila,
Wulffraat Nico,
Espada Graciela,
Garay Stella,
Cuttica Ruben,
Hofer Michael,
Quartier Pierre,
MeloGomes Jose,
Reed Ann M.,
Wierzbowska Malgorzata,
Feldman Brian M.,
Harjacek Miroslav,
Huppertz HansIko,
Nielsen Susan,
Flato Berit,
Lahdenne Pekka,
Michels Harmut,
Murray Kevin J.,
Punaro Lynn,
Rennebohm Robert,
Russo Ricardo,
Balogh Zsolt,
Rooney Madeleine,
Pachman Lauren M.,
Wallace Carol,
Hashkes Philip,
Lovell Daniel J.,
Giannini Edward H.,
Gare Boel Andersson,
Martini Alberto
Publication year - 2010
Publication title -
arthritis care and research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.032
H-Index - 163
eISSN - 2151-4658
pISSN - 2151-464X
DOI - 10.1002/acr.20280
Subject(s) - juvenile dermatomyositis , medicine , physical therapy , kappa , rheumatology , dermatomyositis , gold standard (test) , set (abstract data type) , clinical trial , receiver operating characteristic , medical physics , mathematics , computer science , programming language , geometry
Objective 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. Methods 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. Results 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. Conclusion 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.