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THE PEDIATRIC RHEUMATOLOGY INTERNATIONAL TRIALS ORGANIZATION PROVISIONAL CRITERIA FOR THE EVALUATION OF RESPONSE TO THERAPY IN JUVENILE DERMATOMYOSITIS

Identifieur interne : 006E75 ( Main/Exploration ); précédent : 006E74; suivant : 006E76

THE PEDIATRIC RHEUMATOLOGY INTERNATIONAL TRIALS ORGANIZATION PROVISIONAL CRITERIA FOR THE EVALUATION OF RESPONSE TO THERAPY IN JUVENILE DERMATOMYOSITIS

Auteurs : Nicolino Ruperto [Italie] ; Angela Pistorio [Italie] ; Angelo Ravelli [Italie] ; Lisa G. Rider [États-Unis] ; Clarissa Pilkington [Royaume-Uni] ; Sheila Oliveira [Brésil] ; Nico Wulffraat [Pays-Bas] ; Graciela Espada [Argentine] ; Stella Garay [Argentine] ; Ruben Cuttica [Argentine] ; Michael Hofer [Suisse] ; Pierre Quartier [France] ; Jose Melo-Gomes [Portugal] ; Ann M. Reed [États-Unis] ; Malgorzata Wierzbowska [Pologne] ; Brian M. Feldman [Canada] ; Miroslav Harjacek [Croatie] ; Hans-Iko Huppertz [Allemagne] ; Susan Nielsen [Danemark] ; Berit Flato [Norvège] ; Pekka Lahdenne [Finlande] ; Harmut Michels [Allemagne] ; Kevin J. Murray [Australie] ; Lynn Punaro [États-Unis] ; Robert Rennebohm [Canada] ; Ricardo Russo [Argentine] ; Zsolt Balogh [Argentine] ; Madeleine Rooney [Royaume-Uni] ; Lauren M. Pachman [États-Unis] ; Carol Wallace [États-Unis] ; Philip Hashkes [Israël] ; Daniel J. Lovell [États-Unis] ; Edward H. Giannini [États-Unis] ; Alberto Martini [Italie]

Source :

RBID : PMC:2964396

Abstract

Objective

To develop a provisional definition for the evaluation of response to therapy in juvenile dermatomyositis (JDM) based on the PRINTO JDM 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 (patients rating, statistical analysis, definition selection). Using the physicians’ consensus ratings as the “gold-standard measure”, chi-square, sensitivity, specificity, false positive and negative rate, area under the ROC, and kappa agreement for candidate definitions of improvement were calculated. Definitions with kappa >0.8 were multiplied with the face validity score to select the top definitions.

Results

The top definition of improvement was: at least 20% improvement from baseline in 3/6 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 3/6 core set variables with no more than 2 of the remaining worsening by more than 25%, which cannot be muscle strength which is definition P1 selected by the IMACS 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 proposes 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 endpoint for the evaluation of global response to therapy in JDM.


Url:
DOI: 10.1002/acr.20280
PubMed: 20583105
PubMed Central: 2964396


Affiliations:


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<name sortKey="Rooney, Madeleine" sort="Rooney, Madeleine" uniqKey="Rooney M" first="Madeleine" last="Rooney">Madeleine Rooney</name>
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<name sortKey="Pachman, Lauren M" sort="Pachman, Lauren M" uniqKey="Pachman L" first="Lauren M." last="Pachman">Lauren M. Pachman</name>
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<name sortKey="Wallace, Carol" sort="Wallace, Carol" uniqKey="Wallace C" first="Carol" last="Wallace">Carol Wallace</name>
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<name sortKey="Hashkes, Philip" sort="Hashkes, Philip" uniqKey="Hashkes P" first="Philip" last="Hashkes">Philip Hashkes</name>
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<nlm:aff id="A31">Philip Hashkes, MD, Shaare Zedek Medical Center, Jerusalem, Israel</nlm:aff>
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<name sortKey="Lovell, Daniel J" sort="Lovell, Daniel J" uniqKey="Lovell D" first="Daniel J." last="Lovell">Daniel J. Lovell</name>
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<nlm:aff id="A3">Angelo Ravelli, MD, Prof, Alberto Martini, MD, Prof, IRCCS G. Gaslini, Pediatria II, Reumatologia and Dipartimento di Pediatria, Università degli Studi, Genova, Italy</nlm:aff>
<country xml:lang="fr">Italie</country>
<wicri:regionArea>Angelo Ravelli, MD, Prof, Alberto Martini, MD, Prof, IRCCS G. Gaslini, Pediatria II, Reumatologia and Dipartimento di Pediatria, Università degli Studi, Genova</wicri:regionArea>
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<title xml:lang="en" level="a" type="main">THE PEDIATRIC RHEUMATOLOGY INTERNATIONAL TRIALS ORGANIZATION PROVISIONAL CRITERIA FOR THE EVALUATION OF RESPONSE TO THERAPY IN JUVENILE DERMATOMYOSITIS</title>
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<name sortKey="Ruperto, Nicolino" sort="Ruperto, Nicolino" uniqKey="Ruperto N" first="Nicolino" last="Ruperto">Nicolino Ruperto</name>
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<nlm:aff id="A1">Nicolino Ruperto, MD, MPH, IRCCS G Gaslini, Pediatria II, Reumatologia, PRINTO, Genova, Italy</nlm:aff>
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<affiliation wicri:level="1">
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<country xml:lang="fr">Italie</country>
<wicri:regionArea>Angela Pistorio, MD, PhD, IRCCS G Gaslini, Servizio di Epidemiologia e Biostatistica, Genova</wicri:regionArea>
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<author>
<name sortKey="Ravelli, Angelo" sort="Ravelli, Angelo" uniqKey="Ravelli A" first="Angelo" last="Ravelli">Angelo Ravelli</name>
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<country xml:lang="fr">Italie</country>
<wicri:regionArea>Nicolino Ruperto, MD, MPH, IRCCS G Gaslini, Pediatria II, Reumatologia, PRINTO, Genova</wicri:regionArea>
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<affiliation wicri:level="1">
<nlm:aff id="A3">Angelo Ravelli, MD, Prof, Alberto Martini, MD, Prof, IRCCS G. Gaslini, Pediatria II, Reumatologia and Dipartimento di Pediatria, Università degli Studi, Genova, Italy</nlm:aff>
<country xml:lang="fr">Italie</country>
<wicri:regionArea>Angelo Ravelli, MD, Prof, Alberto Martini, MD, Prof, IRCCS G. Gaslini, Pediatria II, Reumatologia and Dipartimento di Pediatria, Università degli Studi, Genova</wicri:regionArea>
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<name sortKey="Rider, Lisa G" sort="Rider, Lisa G" uniqKey="Rider L" first="Lisa G." last="Rider">Lisa G. Rider</name>
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<nlm:aff id="A4">Lisa G. Rider, MD, NIEHS, NIH, Bethesda, MD, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Lisa G. Rider, MD, NIEHS, NIH, Bethesda, MD</wicri:regionArea>
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<region type="state">Maryland</region>
</placeName>
</affiliation>
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<author>
<name sortKey="Pilkington, Clarissa" sort="Pilkington, Clarissa" uniqKey="Pilkington C" first="Clarissa" last="Pilkington">Clarissa Pilkington</name>
<affiliation wicri:level="3">
<nlm:aff id="A5">Clarissa Pilkington, MD, Great Ormond Street Hospital for Sick Children, London, United Kingdom</nlm:aff>
<country xml:lang="fr">Royaume-Uni</country>
<wicri:regionArea>Clarissa Pilkington, MD, Great Ormond Street Hospital for Sick Children, London</wicri:regionArea>
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<settlement type="city">Londres</settlement>
<region type="country">Angleterre</region>
<region type="région" nuts="1">Grand Londres</region>
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<author>
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<affiliation wicri:level="3">
<nlm:aff id="A6">Sheila Oliveira, MD, Prof, Universidade Federal do Rio de Janeiro, Instituto de Puericultura e Pediatria Martagao Gesteira, Rio de Janeiro, Brazil</nlm:aff>
<country xml:lang="fr">Brésil</country>
<wicri:regionArea>Sheila Oliveira, MD, Prof, Universidade Federal do Rio de Janeiro, Instituto de Puericultura e Pediatria Martagao Gesteira, Rio de Janeiro</wicri:regionArea>
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<settlement type="city">Rio de Janeiro</settlement>
<region type="state">État de Rio de Janeiro</region>
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<name sortKey="Wulffraat, Nico" sort="Wulffraat, Nico" uniqKey="Wulffraat N" first="Nico" last="Wulffraat">Nico Wulffraat</name>
<affiliation wicri:level="3">
<nlm:aff id="A7">Nico Wulffraat, MD, Wilhelmina Kinderziekenhuis, Utrecht, the Netherlands</nlm:aff>
<country xml:lang="fr" wicri:curation="lc">Pays-Bas</country>
<wicri:regionArea>Nico Wulffraat, MD, Wilhelmina Kinderziekenhuis, Utrecht</wicri:regionArea>
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<settlement type="city">Utrecht</settlement>
<region nuts="2" type="province">Utrecht (province)</region>
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<name sortKey="Espada, Graciela" sort="Espada, Graciela" uniqKey="Espada G" first="Graciela" last="Espada">Graciela Espada</name>
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<nlm:aff id="A8">Graciela Espada, MD, Hospital de Ninos Ricardo Gutierrez, Buenos Aires, Argentina</nlm:aff>
<country xml:lang="fr">Argentine</country>
<wicri:regionArea>Graciela Espada, MD, Hospital de Ninos Ricardo Gutierrez, Buenos Aires</wicri:regionArea>
<wicri:noRegion>Buenos Aires</wicri:noRegion>
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<author>
<name sortKey="Garay, Stella" sort="Garay, Stella" uniqKey="Garay S" first="Stella" last="Garay">Stella Garay</name>
<affiliation wicri:level="1">
<nlm:aff id="A9">Stella Garay, MD, Hospital Sor Maria Ludovica, La Plata, Argentina</nlm:aff>
<country xml:lang="fr">Argentine</country>
<wicri:regionArea>Stella Garay, MD, Hospital Sor Maria Ludovica, La Plata</wicri:regionArea>
<wicri:noRegion>La Plata</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Cuttica, Ruben" sort="Cuttica, Ruben" uniqKey="Cuttica R" first="Ruben" last="Cuttica">Ruben Cuttica</name>
<affiliation wicri:level="1">
<nlm:aff id="A10">Ruben Cuttica, MD, Hospital General de Ninos Pedro de Elizalde, Buenos Aires, Argentina</nlm:aff>
<country xml:lang="fr">Argentine</country>
<wicri:regionArea>Ruben Cuttica, MD, Hospital General de Ninos Pedro de Elizalde, Buenos Aires</wicri:regionArea>
<wicri:noRegion>Buenos Aires</wicri:noRegion>
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</author>
<author>
<name sortKey="Hofer, Michael" sort="Hofer, Michael" uniqKey="Hofer M" first="Michael" last="Hofer">Michael Hofer</name>
<affiliation wicri:level="3">
<nlm:aff id="A11">Michael Hofer, MD, Centre Multisite Romand de Rhumatologie Pediatrique, Lausanne, Switzerland</nlm:aff>
<country xml:lang="fr">Suisse</country>
<wicri:regionArea>Michael Hofer, MD, Centre Multisite Romand de Rhumatologie Pediatrique, Lausanne</wicri:regionArea>
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<settlement type="city">Lausanne</settlement>
<region nuts="3" type="region">Canton de Vaud</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Quartier, Pierre" sort="Quartier, Pierre" uniqKey="Quartier P" first="Pierre" last="Quartier">Pierre Quartier</name>
<affiliation wicri:level="4">
<nlm:aff id="A12">Pierre Quartier, MD, Universite Paris-Descartes, Hopital Necker-Enfants Malades, Paris, France</nlm:aff>
<country xml:lang="fr">France</country>
<wicri:regionArea>Pierre Quartier, MD, Universite Paris-Descartes, Hopital Necker-Enfants Malades, Paris</wicri:regionArea>
<placeName>
<region type="region">Île-de-France</region>
<region type="old region">Île-de-France</region>
<settlement type="city">Paris</settlement>
<settlement type="city">Paris</settlement>
</placeName>
<orgName type="university">Université Paris-Descartes</orgName>
</affiliation>
</author>
<author>
<name sortKey="Melo Gomes, Jose" sort="Melo Gomes, Jose" uniqKey="Melo Gomes J" first="Jose" last="Melo-Gomes">Jose Melo-Gomes</name>
<affiliation wicri:level="1">
<nlm:aff id="A13">Jose Melo-Gomes, MD, Instituto Portugues de Reumatologia, Lisbon, Portugal</nlm:aff>
<country xml:lang="fr">Portugal</country>
<wicri:regionArea>Jose Melo-Gomes, MD, Instituto Portugues de Reumatologia, Lisbon</wicri:regionArea>
<wicri:noRegion>Lisbon</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Reed, Ann M" sort="Reed, Ann M" uniqKey="Reed A" first="Ann M." last="Reed">Ann M. Reed</name>
<affiliation wicri:level="2">
<nlm:aff id="A14">Ann M. Reed, MD, Mayo Clinic School of Medicine and Mayo Foundation, Rochester, MN, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Ann M. Reed, MD, Mayo Clinic School of Medicine and Mayo Foundation, Rochester, MN</wicri:regionArea>
<placeName>
<region type="state">Minnesota</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Wierzbowska, Malgorzata" sort="Wierzbowska, Malgorzata" uniqKey="Wierzbowska M" first="Malgorzata" last="Wierzbowska">Malgorzata Wierzbowska</name>
<affiliation wicri:level="1">
<nlm:aff id="A15">Malgorzata Wierzbowska, MD, Institute of Rheumatology, Warsaw, Poland</nlm:aff>
<country xml:lang="fr">Pologne</country>
<wicri:regionArea>Malgorzata Wierzbowska, MD, Institute of Rheumatology, Warsaw</wicri:regionArea>
<wicri:noRegion>Warsaw</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Feldman, Brian M" sort="Feldman, Brian M" uniqKey="Feldman B" first="Brian M." last="Feldman">Brian M. Feldman</name>
<affiliation wicri:level="4">
<nlm:aff id="A16">Brian M. Feldman, MD, MSc, FRCPC, University of Toronto, The Hospital for Sick Children, Toronto, Canada</nlm:aff>
<country xml:lang="fr">Canada</country>
<wicri:regionArea>Brian M. Feldman, MD, MSc, FRCPC, University of Toronto, The Hospital for Sick Children, Toronto</wicri:regionArea>
<orgName type="university">Université de Toronto</orgName>
<placeName>
<settlement type="city">Toronto</settlement>
<region type="state">Ontario</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Harjacek, Miroslav" sort="Harjacek, Miroslav" uniqKey="Harjacek M" first="Miroslav" last="Harjacek">Miroslav Harjacek</name>
<affiliation wicri:level="1">
<nlm:aff id="A17">Miroslav Harjacek, MD, Children's Hospital Zagreb, Zagreb, Croatia</nlm:aff>
<country xml:lang="fr">Croatie</country>
<wicri:regionArea>Miroslav Harjacek, MD, Children's Hospital Zagreb, Zagreb</wicri:regionArea>
<wicri:noRegion>Zagreb</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Huppertz, Hans Iko" sort="Huppertz, Hans Iko" uniqKey="Huppertz H" first="Hans-Iko" last="Huppertz">Hans-Iko Huppertz</name>
<affiliation wicri:level="3">
<nlm:aff id="A18">Hans-Iko Huppertz, MD, Klinikum Bremen-Mitte, Bremen, Germany</nlm:aff>
<country xml:lang="fr">Allemagne</country>
<wicri:regionArea>Hans-Iko Huppertz, MD, Klinikum Bremen-Mitte, Bremen</wicri:regionArea>
<placeName>
<region type="land" nuts="3">Brême (Land)</region>
<settlement type="city">Brême</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Nielsen, Susan" sort="Nielsen, Susan" uniqKey="Nielsen S" first="Susan" last="Nielsen">Susan Nielsen</name>
<affiliation wicri:level="3">
<nlm:aff id="A19">Susan Nielsen, MD, Juliane Marie Centret, Rigshospitalet, Copenhagen, Denmark</nlm:aff>
<country xml:lang="fr">Danemark</country>
<wicri:regionArea>Susan Nielsen, MD, Juliane Marie Centret, Rigshospitalet, Copenhagen</wicri:regionArea>
<placeName>
<settlement type="city">Copenhague</settlement>
<region type="région" nuts="2">Hovedstaden</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Flato, Berit" sort="Flato, Berit" uniqKey="Flato B" first="Berit" last="Flato">Berit Flato</name>
<affiliation wicri:level="3">
<nlm:aff id="A20">Berit Flato, MD, Rikshospitalet University Hospital, Oslo, Norway</nlm:aff>
<country xml:lang="fr">Norvège</country>
<wicri:regionArea>Berit Flato, MD, Rikshospitalet University Hospital, Oslo</wicri:regionArea>
<placeName>
<settlement type="city">Oslo</settlement>
<region nuts="2">Østlandet</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Lahdenne, Pekka" sort="Lahdenne, Pekka" uniqKey="Lahdenne P" first="Pekka" last="Lahdenne">Pekka Lahdenne</name>
<affiliation wicri:level="1">
<nlm:aff id="A21">Pekka Lahdenne, MD, Hospital for Children and Adolescents, Helsinki, Finland</nlm:aff>
<country xml:lang="fr">Finlande</country>
<wicri:regionArea>Pekka Lahdenne, MD, Hospital for Children and Adolescents, Helsinki</wicri:regionArea>
<wicri:noRegion>Helsinki</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Michels, Harmut" sort="Michels, Harmut" uniqKey="Michels H" first="Harmut" last="Michels">Harmut Michels</name>
<affiliation wicri:level="1">
<nlm:aff id="A22">Harmut Michels, MD, Rheumakinderklinik Garmisch-Partenkirchen, Germany</nlm:aff>
<country xml:lang="fr">Allemagne</country>
<wicri:regionArea>Harmut Michels, MD, Rheumakinderklinik Garmisch-Partenkirchen</wicri:regionArea>
<wicri:noRegion>Rheumakinderklinik Garmisch-Partenkirchen</wicri:noRegion>
<wicri:noRegion>Rheumakinderklinik Garmisch-Partenkirchen</wicri:noRegion>
<wicri:noRegion>Rheumakinderklinik Garmisch-Partenkirchen</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Murray, Kevin J" sort="Murray, Kevin J" uniqKey="Murray K" first="Kevin J." last="Murray">Kevin J. Murray</name>
<affiliation wicri:level="1">
<nlm:aff id="A23">Kevin J. Murray, MD, Princess Margaret Hospital for Children, Perth, WA, Australia</nlm:aff>
<country xml:lang="fr">Australie</country>
<wicri:regionArea>Kevin J. Murray, MD, Princess Margaret Hospital for Children, Perth, WA</wicri:regionArea>
<wicri:noRegion>WA</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Punaro, Lynn" sort="Punaro, Lynn" uniqKey="Punaro L" first="Lynn" last="Punaro">Lynn Punaro</name>
<affiliation wicri:level="2">
<nlm:aff id="A24">Lynn Punaro, MD, Texas Scottish Rite Hospital, Dallas, TX, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Lynn Punaro, MD, Texas Scottish Rite Hospital, Dallas, TX</wicri:regionArea>
<placeName>
<region type="state">Texas</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Rennebohm, Robert" sort="Rennebohm, Robert" uniqKey="Rennebohm R" first="Robert" last="Rennebohm">Robert Rennebohm</name>
<affiliation wicri:level="1">
<nlm:aff id="A25">Robert Rennebohm, MD, Alberta Children's Hospital, Calgary, Canada</nlm:aff>
<country xml:lang="fr">Canada</country>
<wicri:regionArea>Robert Rennebohm, MD, Alberta Children's Hospital, Calgary</wicri:regionArea>
<wicri:noRegion>Calgary</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Russo, Ricardo" sort="Russo, Ricardo" uniqKey="Russo R" first="Ricardo" last="Russo">Ricardo Russo</name>
<affiliation wicri:level="1">
<nlm:aff id="A26">Ricardo Russo, MD, Hospital de Pediatria Juan P. Garrahan, Buenos Aires, Argentina</nlm:aff>
<country xml:lang="fr">Argentine</country>
<wicri:regionArea>Ricardo Russo, MD, Hospital de Pediatria Juan P. Garrahan, Buenos Aires</wicri:regionArea>
<wicri:noRegion>Buenos Aires</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Balogh, Zsolt" sort="Balogh, Zsolt" uniqKey="Balogh Z" first="Zsolt" last="Balogh">Zsolt Balogh</name>
<affiliation wicri:level="3">
<nlm:aff id="A27">Zsolt Balogh, MD, National Institute of Rheumatology and Physiotherapy, Budapest, Argentina</nlm:aff>
<country xml:lang="fr">Argentine</country>
<wicri:regionArea>Zsolt Balogh, MD, National Institute of Rheumatology and Physiotherapy, Budapest</wicri:regionArea>
<placeName>
<settlement type="city">Budapest</settlement>
<region nuts="2">Hongrie centrale</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Rooney, Madeleine" sort="Rooney, Madeleine" uniqKey="Rooney M" first="Madeleine" last="Rooney">Madeleine Rooney</name>
<affiliation wicri:level="2">
<nlm:aff id="A28">Madeleine Rooney, MD, Musgrave Park Hospital, Belfast, Northern Ireland</nlm:aff>
<country>Royaume-Uni</country>
<placeName>
<region type="country">Irlande du Nord</region>
</placeName>
<wicri:cityArea>Madeleine Rooney, MD, Musgrave Park Hospital, Belfast</wicri:cityArea>
</affiliation>
</author>
<author>
<name sortKey="Pachman, Lauren M" sort="Pachman, Lauren M" uniqKey="Pachman L" first="Lauren M." last="Pachman">Lauren M. Pachman</name>
<affiliation wicri:level="2">
<nlm:aff id="A29">Lauren M. Pachman, MD, Children's Memorial Hospital, Chicago, IL, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Lauren M. Pachman, MD, Children's Memorial Hospital, Chicago, IL</wicri:regionArea>
<placeName>
<region type="state">Illinois</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Wallace, Carol" sort="Wallace, Carol" uniqKey="Wallace C" first="Carol" last="Wallace">Carol Wallace</name>
<affiliation wicri:level="2">
<nlm:aff id="A30">Carol Wallace, MD, University of Washington/Children's Hospital, Seattle, WA, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Carol Wallace, MD, University of Washington/Children's Hospital, Seattle, WA</wicri:regionArea>
<placeName>
<region type="state">Washington (État)</region>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Hashkes, Philip" sort="Hashkes, Philip" uniqKey="Hashkes P" first="Philip" last="Hashkes">Philip Hashkes</name>
<affiliation wicri:level="1">
<nlm:aff id="A31">Philip Hashkes, MD, Shaare Zedek Medical Center, Jerusalem, Israel</nlm:aff>
<country xml:lang="fr">Israël</country>
<wicri:regionArea>Philip Hashkes, MD, Shaare Zedek Medical Center, Jerusalem</wicri:regionArea>
<wicri:noRegion>Jerusalem</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Lovell, Daniel J" sort="Lovell, Daniel J" uniqKey="Lovell D" first="Daniel J." last="Lovell">Daniel J. Lovell</name>
<affiliation wicri:level="2">
<nlm:aff id="A32">Daniel J. Lovell, MD, MPH, Edward H. Giannini, MSc, DrPH, Children's Hospital Medical Center, Cincinnati, OH, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Daniel J. Lovell, MD, MPH, Edward H. Giannini, MSc, DrPH, Children's Hospital Medical Center, Cincinnati, OH</wicri:regionArea>
<placeName>
<region type="state">Ohio</region>
</placeName>
</affiliation>
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<author>
<name sortKey="Giannini, Edward H" sort="Giannini, Edward H" uniqKey="Giannini E" first="Edward H." last="Giannini">Edward H. Giannini</name>
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<nlm:aff id="A32">Daniel J. Lovell, MD, MPH, Edward H. Giannini, MSc, DrPH, Children's Hospital Medical Center, Cincinnati, OH, USA</nlm:aff>
<country xml:lang="fr">États-Unis</country>
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<region type="state">Ohio</region>
</placeName>
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<author>
<name sortKey="Martini, Alberto" sort="Martini, Alberto" uniqKey="Martini A" first="Alberto" last="Martini">Alberto Martini</name>
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<nlm:aff id="A3">Angelo Ravelli, MD, Prof, Alberto Martini, MD, Prof, IRCCS G. Gaslini, Pediatria II, Reumatologia and Dipartimento di Pediatria, Università degli Studi, Genova, Italy</nlm:aff>
<country xml:lang="fr">Italie</country>
<wicri:regionArea>Angelo Ravelli, MD, Prof, Alberto Martini, MD, Prof, IRCCS G. Gaslini, Pediatria II, Reumatologia and Dipartimento di Pediatria, Università degli Studi, Genova</wicri:regionArea>
<wicri:noRegion>Genova</wicri:noRegion>
</affiliation>
</author>
</analytic>
<series>
<title level="j">Arthritis care & research</title>
<idno type="ISSN">2151-464X</idno>
<idno type="eISSN">2151-4658</idno>
<imprint>
<date when="2010">2010</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
<profileDesc>
<textClass></textClass>
</profileDesc>
</teiHeader>
<front>
<div type="abstract" xml:lang="en">
<sec id="S1">
<title>Objective</title>
<p id="P1">To develop a provisional definition for the evaluation of response to therapy in juvenile dermatomyositis (JDM) based on the PRINTO JDM core set of variables.</p>
</sec>
<sec sec-type="methods" id="S2">
<title>Methods</title>
<p id="P2">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 (patients rating, statistical analysis, definition selection). Using the physicians’ consensus ratings as the “gold-standard measure”, chi-square, sensitivity, specificity, false positive and negative rate, area under the ROC, and kappa agreement for candidate definitions of improvement were calculated. Definitions with kappa >0.8 were multiplied with the face validity score to select the top definitions.</p>
</sec>
<sec id="S3">
<title>Results</title>
<p id="P3">The top definition of improvement was: at least 20% improvement from baseline in 3/6 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 3/6 core set variables with no more than 2 of the remaining worsening by more than 25%, which cannot be muscle strength which is definition P1 selected by the IMACS group. The third is similar to the second with the maximum amount of worsening set to 30%. This indicates convergent validity of the process.</p>
</sec>
<sec id="S4">
<title>Conclusion</title>
<p id="P4">we proposes 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 endpoint for the evaluation of global response to therapy in JDM.</p>
</sec>
</div>
</front>
</TEI>
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<name sortKey="Russo, Ricardo" sort="Russo, Ricardo" uniqKey="Russo R" first="Ricardo" last="Russo">Ricardo Russo</name>
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<name sortKey="Hofer, Michael" sort="Hofer, Michael" uniqKey="Hofer M" first="Michael" last="Hofer">Michael Hofer</name>
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<name sortKey="Quartier, Pierre" sort="Quartier, Pierre" uniqKey="Quartier P" first="Pierre" last="Quartier">Pierre Quartier</name>
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<name sortKey="Melo Gomes, Jose" sort="Melo Gomes, Jose" uniqKey="Melo Gomes J" first="Jose" last="Melo-Gomes">Jose Melo-Gomes</name>
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<name sortKey="Michels, Harmut" sort="Michels, Harmut" uniqKey="Michels H" first="Harmut" last="Michels">Harmut Michels</name>
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<name sortKey="Hashkes, Philip" sort="Hashkes, Philip" uniqKey="Hashkes P" first="Philip" last="Hashkes">Philip Hashkes</name>
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</record>

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