The Paediatric Rheumatology International Trials Organisation provisional criteria for the evaluation of response to therapy in juvenile dermatomyositis.

Nicolino Ruperto, Angela Pistorio, Angelo Ravelli, Lisa G. Rider, Clarissa Pilkington, Sheila Oliveira, Nico Wulffraat, Graciela Espada, Stella Garay, Ruben Cuttica, Michael Hofer, Pierre Quartier, Jose Melo-Gomes, Ann M. Reed, Malgorzata Wierzbowska, Brian M. Feldman, Miroslav Harjacek, Hans Iko Huppertz, Susan Nielsen, Berit FlatoPekka Lahdenne, Harmut Michels, Kevin J. Murray, Lynn Punaro, Robert Rennebohm, Ricardo Russo, Zsolt Balogh, Madeleine Rooney, Lauren M. Pachman, Carol Wallace, Philip Hashkes, Daniel J. Lovell, Edward H. Giannini, Boel Andersson Gare, Alberto Martini, Rheumatology International Trials Organisation (PRINTO) Paediatric Rheumatology International Trials Organisation (PRINTO), Rheumatology Collaborative Study Group (PRCSG) Pediatric Rheumatology Collaborative Study Group (PRCSG)

Research output: Contribution to journalArticlepeer-review

54 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1533-1541
Number of pages9
JournalArthritis Care and Research
Volume62
Issue number11
DOIs
StatePublished - Nov 2010

ASJC Scopus subject areas

  • Rheumatology

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