2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects

Lisa G Rider¹, Nicolino Ruperto², Angela Pistorio³, Brian Erman⁴, Nastaran Bayat¹, Peter A Lachenbruch¹, Howard Rockette⁵, Brian M Feldman⁶, Adam M Huber⁷, Paul Hansen⁸, Chester V Oddis⁹, Ingrid E Lundberg¹⁰, Anthony A Amato¹¹, Hector Chinoy^{12

13}, Robert G Cooper¹⁴, Lorinda Chung¹⁵, Katalin Danko¹⁶, David Fiorentino¹⁷, Ignacio García-De la Torre¹⁸, Ann M Reed¹⁹, Yeong Wook Song²⁰, Rolando Cimaz²¹, Rubén J Cuttica²², Clarissa A Pilkington²³, Alberto Martini^{2

24}, Janjaap van der Net²⁵, Susan Maillard²³, Frederick W Miller¹, Jiri Vencovsky²⁶, Rohit Aggarwal⁹; International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation

Affiliations

¹ Environmental Autoimmunity Group, NIEHS, National Institutes of Health, Bethesda, MD, USA.
² Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO.
³ Istituto Giannina Gaslini, Servizio di Epidemiologia e Biostatistica, Genoa, Italy.
⁴ Social and Scientific Systems, Inc., Durham, NC.
⁵ Department of Biostatistics, University of Pittsburgh, Pittsburgh, PA, USA.
⁶ Division of Rheumatology, Hospital for Sick Children, Toronto, Ontario.
⁷ Rheumatology Department, Izaak Walton Killam Health Centre, Halifax, Nova Scotia, Canada.
⁸ Department of Economics, University of Otago, Dunedin, New Zealand.
⁹ Department of Medicine, Division of Rheumatology and Clinical Immunology, University of Pittsburgh, Pittsburgh, PA, USA.
¹⁰ Rheumatology Unit, Karolinska University Hospital, Karolinska Institute, Stockholm, Sweden.
¹¹ Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA.
¹² National Institute of Health Research Manchester Musculoskeletal Biomedical Research Unit, Central Manchester University Hospitals NHS Foundation Trust.
¹³ Manchester Academic Health Science Centre, The University of Manchester, Manchester.
¹⁴ Institute of Ageing and Chronic Disease, University of Liverpool, Liverpool, UK.
¹⁵ Division of Rheumatology, Stanford University, Redwood City, CA, USA.
¹⁶ 3rd Department of Internal Medicine, Division of Immunology, University of Debrecen, Debrecen, Hungary.
¹⁷ Department of Dermatology, Stanford University, Redwood City, CA, USA.
¹⁸ Hospital General de Occidente de la Secretaría de Salud, Guadalajara, Jalisco, Mexico.
¹⁹ Department of Pediatrics, Duke University, Durham, NC, USA.
²⁰ Department of Molecular Medicine and Biopharmaceutical Sciences, Medical Research Center, Seoul National University, Seoul, Korea.
²¹ Pediatric Rheumatology, Azienda Ospedaliero Universitaria Meyer, University of Florence, Florence, Italy.
²² Department of Pediatric Rheumatology, Hospital de Niños Pedro de Elizalde, University of Buenos Aires, Buenos Aires, Argentina.
²³ Department of Paediatric Rheumatology, Great Ormond Street Hospital for Children NHS Trust, London, UK.
²⁴ Università degli Studi di Genova, Dipartimento di Pediatria, Genoa, Italy.
²⁵ Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands.
²⁶ Institute of Rheumatology and Department of Rheumatology, Charles University, Prague, Czech Republic.

PMID: 28977549
PMCID: PMC5850656
DOI: 10.1093/rheumatology/kex226

2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects

Lisa G Rider et al. Rheumatology (Oxford). 2017.

. 2017 Nov 1;56(11):1884-1893.

doi: 10.1093/rheumatology/kex226.

Authors

Affiliations

¹ Environmental Autoimmunity Group, NIEHS, National Institutes of Health, Bethesda, MD, USA.
² Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO.
³ Istituto Giannina Gaslini, Servizio di Epidemiologia e Biostatistica, Genoa, Italy.
⁴ Social and Scientific Systems, Inc., Durham, NC.
⁵ Department of Biostatistics, University of Pittsburgh, Pittsburgh, PA, USA.
⁶ Division of Rheumatology, Hospital for Sick Children, Toronto, Ontario.
⁷ Rheumatology Department, Izaak Walton Killam Health Centre, Halifax, Nova Scotia, Canada.
⁸ Department of Economics, University of Otago, Dunedin, New Zealand.
⁹ Department of Medicine, Division of Rheumatology and Clinical Immunology, University of Pittsburgh, Pittsburgh, PA, USA.
¹⁰ Rheumatology Unit, Karolinska University Hospital, Karolinska Institute, Stockholm, Sweden.
¹¹ Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA.
¹² National Institute of Health Research Manchester Musculoskeletal Biomedical Research Unit, Central Manchester University Hospitals NHS Foundation Trust.
¹³ Manchester Academic Health Science Centre, The University of Manchester, Manchester.
¹⁴ Institute of Ageing and Chronic Disease, University of Liverpool, Liverpool, UK.
¹⁵ Division of Rheumatology, Stanford University, Redwood City, CA, USA.
¹⁶ 3rd Department of Internal Medicine, Division of Immunology, University of Debrecen, Debrecen, Hungary.
¹⁷ Department of Dermatology, Stanford University, Redwood City, CA, USA.
¹⁸ Hospital General de Occidente de la Secretaría de Salud, Guadalajara, Jalisco, Mexico.
¹⁹ Department of Pediatrics, Duke University, Durham, NC, USA.
²⁰ Department of Molecular Medicine and Biopharmaceutical Sciences, Medical Research Center, Seoul National University, Seoul, Korea.
²¹ Pediatric Rheumatology, Azienda Ospedaliero Universitaria Meyer, University of Florence, Florence, Italy.
²² Department of Pediatric Rheumatology, Hospital de Niños Pedro de Elizalde, University of Buenos Aires, Buenos Aires, Argentina.
²³ Department of Paediatric Rheumatology, Great Ormond Street Hospital for Children NHS Trust, London, UK.
²⁴ Università degli Studi di Genova, Dipartimento di Pediatria, Genoa, Italy.
²⁵ Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands.
²⁶ Institute of Rheumatology and Department of Rheumatology, Charles University, Prague, Czech Republic.

PMID: 28977549
PMCID: PMC5850656
DOI: 10.1093/rheumatology/kex226

Abstract

Objective: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM.

Methods: Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data.

Results: Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference.

Conclusion: Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

Keywords: 1000Minds software; conjoint analysis; dermatomyositis; hybrid measure; juvenile dermatomyositis; outcome assessment; polymyositis; response criteria.

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Figures

F<sc>ig</sc>. 1 — **Fig. 1**
Overview of the development of the new response criteria

See this image and copyright information in PMC

References

1. Rider LG, Werth VP, Huber AM. et al. Measures of adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis: Physician and Patient/Parent Global Activity, Manual Muscle Testing (MMT), Health Assessment Questionnaire (HAQ)/Childhood Health Assessment Questionnaire (C-HAQ), Childhood Myositis Assessment Scale (CMAS), Myositis Disease Activity Assessment Tool (MDAAT), Disease Activity Score (DAS), Short Form 36 (SF-36), Child Health Questionnaire (CHQ), Physician Global Damage, Myositis Damage Index (MDI), Quantitative Muscle Testing (QMT), Myositis Functional Index-2 (FI-2), Myositis Activities Profile (MAP), Inclusion Body Myositis Functional Rating Scale (IBMFRS), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), Cutaneous Assessment Tool (CAT), Dermatomyositis Skin Severity Index (DSSI), Skindex, and Dermatology Life Quality Index (DLQI). Arthritis Care Res 2011;63 (Suppl 11):S118–57. - PMC - PubMed
1. Ruperto N, Martini A.. Networking in paediatrics: the example of the Paediatric Rheumatology International Trials Organisation (PRINTO). Arch Dis Child 2011;96:596–601. - PubMed
1. Miller FW, Rider LG, Chung YL. et al. Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. Rheumatology 2001;40:1262–73. - PubMed
1. Ruperto N, Ravelli A, Pistorio A. et al. The provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism Disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: a prospective validation study. Arthritis Rheum 2008;59:4–13. - PubMed
1. Ruperto N, Ravelli A, Murray KJ. et al. Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis. Rheumatology 2003;42:1452–9. - PubMed

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2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects

Affiliations

2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects

Authors

Affiliations

Abstract

Figures

References

Publication types

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

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