To develop response criteria for adult dermatomyositis (DM) and polymyositis (PM). Expert surveys, logistic regression, and conjoint analysis were used to develop 287 definitions using core set measures. Myositis experts rated greater improvement among multiple pairwise scenarios in conjoint analysis surveys, where different levels of improvement in 2 core set measures were presented. The PAPRIKA (Potentially All Pairwise Rankings of All Possible Alternatives) method determined the relative weights of core set measures and conjoint analysis definitions. The performance characteristics of the definitions were evaluated on patient profiles using expert consensus (gold standard) and were validated using data from a clinical trial. The nominal group technique was used to reach consensus. Consensus was reached for a conjoint analysis-based continuous model using absolute per cent change in core set measures (physician, patient, and extramuscular global activity, muscle strength, Health Assessment Questionnaire, and muscle enzyme levels). A total improvement score (range 0-100), determined by summing scores for each core set measure, was based on improvement in and relative weight of each core set measure. Thresholds for minimal, moderate, and major improvement were ≥20, ≥40, and ≥60 points in the total improvement score. The same criteria were chosen for juvenile DM, with different improvement thresholds. Sensitivity and specificity in DM/PM patient cohorts were 85% and 92%, 90% and 96%, and 92% and 98% for minimal, moderate, and major improvement, respectively. Definitions were validated in the clinical trial analysis for differentiating the physician rating of improvement (p<0.001). The response criteria for adult DM/PM consisted of the conjoint analysis model based on absolute per cent change in 6 core set measures, with thresholds for minimal, moderate, and major improvement.

Academic Medical Center Amsterdam The Netherlands

Brigham and Women's Hospital and Harvard Medical School Boston Massachusetts USA

Central Manchester University Hospitals NHS Foundation Trust University of Manchester Manchester UK

Charles University Prague Czech Republic

Graduate School of Convergence Science and Technology and Seoul National University Hospital Seoul Korea

Hospital General de Occidente de la Secretaría de Salud and University of Guadalajara Guadalajara México

Istituto Giannina Gaslini Pediatria 2 Rheumatologia PRINTO Genoa Italy

Johns Hopkins University School of Medicine Baltimore Maryland USA

Karolinska University Hospital Karolinska Institute Stockholm Sweden

Mayo Clinic Rochester Minnesota USA

NIEHS NIH Bethesda Maryland USA

Social and Scientific Systems Inc Durham North Carolina USA

Stanford University Redwood City California USA

The Hospital for Sick Children Toronto Ontario Canada

University College London London UK

University of Debrecen Debrecen Hungary

University of Liverpool Liverpool UK

University of Pittsburgh Pittsburgh Pennsylvania USA

Komentář v

Nat Rev Rheumatol. 2017 Jun;13(6):321. doi: 10.1038/nrrheum.2017.61. PubMed

Zobrazit více v PubMed

Rider LG, Miller FW. Deciphering the clinical presentations, pathogenesis, and treatment of the idiopathic inflammatory myopathies. JAMA. 2011;305(2):183–90. PubMed PMC

Moghadam-Kia S, Aggarwal R, Oddis CV. Treatment of inflammatory myopathy: emerging therapies and therapeutic targets. Expert Rev Clin Immunol. 2015;11(11):1265–75. PubMed PMC

Oddis CV, Reed AM, Aggarwal R, Rider LG, Ascherman DP, Levesque MC, et al. Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: A randomized, placebo-phase trial. Arthritis Rheum. 2013;65(2):314–24. PubMed PMC

Muscle Study Group. A randomized, pilot trial of etanercept in dermatomyositis. Ann Neurol. 2011;70(3):427–36. PubMed PMC

Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, et al. Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial. Lancet. 2016;387(10019):671–8. PubMed

Rider LG, Werth VP, Huber AM, Alexanderson H, Rao AP, Ruperto N, 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 (Hoboken ) 2011;63(Suppl 11):S118–S157. PubMed PMC

Miller FW, Rider LG, Chung YL, Cooper R, Danko K, Farewell V, et al. Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies. Rheumatology (Oxford) 2001;40(11):1262–73. PubMed

Rider LG, Giannini EH, Harris-Love M, Joe G, Isenberg D, Pilkington C, et al. Defining Clinical Improvement in Adult and Juvenile Myositis. J Rheumatol. 2003;30(3):603–17. PubMed

Rider LG, Giannini EH, Brunner HI, Ruperto N, James-Newton L, Reed AM, et al. International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis Rheum. 2004;50(7):2281–90. PubMed

American College of Rheumatology Committee to Reevaluate Improvement Criteria. A proposed revision to the ACR20: the hybrid measure of American College of Rheumatology response. Arthritis Rheum. 2007;57(2):193–202. PubMed

de Lautour H, Taylor WJ, Adebajo A, Alten R, Burgos-Vargas R, Chapman P, et al. Development of Preliminary Remission Criteria for Gout Using Delphi and 1000Minds(R) Consensus Exercises. Arthritis Care Res (Hoboken ) 2016;68(5):667–72. PubMed

Johnson SR, Naden RP, Fransen J, Van Den Hoogen F, Pope JE, Baron M, et al. Multicriteria decision analysis methods with 1000Minds for developing systemic sclerosis classification criteria. J Clin Epidemiol. 2014;67(6):706–14. PubMed PMC

Hansen P, Ombler F. A new method for scoring additive multi-attribute value models using pairwise rankings of alternatives. J Multi-Crit Decis Anal. 2008;15:87–107.

Amaya-Amaya M, Gerard K, Ryan M. Discrete choice experiments in a nutshell. In: Ryan M, Gerard K, Amaya-Amaya M, editors. Using Discrete Choice Experiments to Value Health and Health Care. Dordrecht: Springer; 2008.

Ruperto N, Martini A. Networking in paediatrics: the example of the Paediatric Rheumatology International Trials Organisation (PRINTO) Arch Dis Child. 2011;96(6):596–601. PubMed

Taylor WJ, Singh JA, Saag KG, Dalbeth N, MacDonald PA, Edwards NL, et al. Bringing it all together: a novel approach to the development of response criteria for chronic gout clinical trials. J Rheumatol. 2011;38(7):1467–70. PubMed

Taylor WJ, Brown M, Aati O, Weatherall M, Dalbeth N. Do patient preferences for core outcome domains for chronic gout studies support the validity of composite response criteria? Arthritis Care Res (Hoboken) 2013;65(8):1259–64. PubMed

Utz KS, Hoog J, Wentrup A, Berg S, Lammer A, Jainsch B, et al. Patient preferences for disease-modifying drugs in multiple sclerosis therapy: a choice-based conjoint analysis. Ther Adv Neurol Disord. 2014;7(6):263–75. PubMed PMC

de Bekker-Grob E, Ryan M, Gerard K. Discrete choice experiments in health economics: a review of the literature. Health Econ. 2012;21:145–72. PubMed

Rider LG, Aggarwal R, Pistorio A, Bayat N, Erman B, Feldman BM, et al. 2016 American College of Rheumatology (ACR) - European League Against Rheumatism (EULAR) Criteria for Minimal, Moderate and Major Clinical Response for Juvenile Dermatomyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative. Arthritis Rheumatol. 2016;00(00):000. PubMed

Rider LG. Outcome assessment in the adult and juvenile idiopathic inflammatory myopathies. Rheum Dis Clin North Am. 2002;28(4):935–77. PubMed

Aggarwal R, Rider LG, Ruperto N, Bayat N, Erman B, Feldman BM, et al. A consensus hybrid definition using a conjoint analysis is the proposed response criteria for minimal and moderate improvement for adult polymyositis and dermatomyositis clinical trials. Arthritis Rheumatol. 2014;66(S11):S404.

Rider LG, Ruperto N, Pistorio A, Erman B, Bayat N, Lachenbruch PA, et al. Development of Adult Dermatomyositis and Polymyositis and Juvenile Dermatomyositis Response Criteria--Methodological Aspects: An American College of Rheumatology/European League Against Rheumatism/International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative. Rheumatology (Oxford) 2016;2016 Submitted. PubMed

Bohan A, Peter JB. Polymyositis and dermatomyositis. Parts 1 and 2. N Engl J Med. 1975;292:344–7. 3403–407. PubMed

Rider LG, Lee J, Jansen A, Ruperto N, Huber AM, Oddis CV, et al. Defining clinically relevant changes in core set activity measures for adult and juvenile idiopathic inflammatory myopathies (IIM) Arthritis Rheum. 2011;63(Suppl):S89.

Metz CE. Basic principles of ROC analysis. Semin Nucl Med. 1978;8(4):283–98. PubMed

Ruperto N, Meiorin S, Iusan SM, Ravelli A, Pistorio A, Martini A. Consensus procedures and their role in pediatric rheumatology. Curr Rheumatol Rep. 2008;10(2):142–6. PubMed

Delbecq A, Van de Ven A, Gustafson D. A guide to nominal group and delphi processes. Glenview, IL: Scott, Foresman and Company; 1975. Group techniques for program planning.

Nair R, Aggarwal R, Khanna D. Methods of formal consensus in classification/diagnostic criteria and guideline development. Semin Arthritis Rheum. 2011;41(2):95–105. PubMed PMC

Ruperto N, Ozen S, Pistorio A, Dolezalova P, Brogan P, Cabral DA, et al. EULAR/PRINTO/PRES criteria for Henoch-Schonlein purpura, childhood polyarteritis nodosa, childhood Wegener granulomatosis and childhood Takayasu arteritis: Ankara 2008. Part I: Overall methodology and clinical characterisation. Ann Rheum Dis. 2010;69(5):790–7. PubMed

Piram M, Kone-Paut I, Lachmann HJ, Frenkel J, Ozen S, Kuemmerle-Deschner J, et al. Validation of the auto-inflammatory diseases activity index (AIDAI) for hereditary recurrent fever syndromes. Ann Rheum Dis. 2014;73(12):2168–73. PubMed PMC

Ruperto N, Ravelli A, Pistorio A, Ferriani V, Calvo I, Ganser G, 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(1):4–13. PubMed

Ruperto N, Ravelli A, Pistorio A, Malattia C, Cavuto S, Gado-West L, et al. Cross-cultural adaptation and psychometric evaluation of the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ) in 32 countries. Review of the general methodology. Clin Exp Rheumatol. 2001;19(Suppl 23):S1–S9. PubMed

Apaz MT, Saad-Magalhaes C, Pistorio A, Ravelli A, de Oliveira SJ, Marcantoni MB, et al. Health-related quality of life of patients with juvenile dermatomyositis: results from the Pediatric Rheumatology International Trials Organisation multinational quality of life cohort study. Arthritis Rheum. 2009;61(4):509–17. PubMed

Van Den Hoogen F, Khanna D, Fransen J, Johnson SR, Baron M, Tyndall A, et al. 2013 classification criteria for systemic sclerosis: an American College of Rheumatology/European League against Rheumatism collaborative initiative. Arthritis Rheum. 2013;65(11):2737–47. PubMed PMC

Neogi T, Aletaha D, Silman AJ, Naden RL, Felson DT, Aggarwal R, et al. The 2010 American College of Rheumatology/European League Against Rheumatism classification criteria for rheumatoid arthritis: Phase 2 methodological report. Arthritis Rheum. 2010;62(9):2582–91. PubMed PMC

Neogi T, Jansen TL, Dalbeth N, Fransen J, Schumacher HR, Berendsen D, et al. 2015 Gout Classification Criteria: an American College of Rheumatology/European League Against Rheumatism collaborative initiative. Arthritis Rheumatol. 2015;67(10):2557–68. PubMed PMC

Felson DT, Anderson JJ, Boers M, Bombardier C, Furst D, Goldsmith C, et al. American College of Rheumatology. Preliminary definition of improvement in rheumatoid arthritis. Arthritis Rheum. 1995;38(6):727–35. PubMed

Felson DT, Smolen JS, Wells G, Zhang B, van Tuyl LH, Funovits J, et al. American College of Rheumatology/European League Against Rheumatism provisional definition of remission in rheumatoid arthritis for clinical trials. Arthritis Rheum. 2011;63(3):573–86. PubMed PMC

Aggarwal R, Cassidy E, Fertig N, Koontz DC, Lucas M, Ascherman DP, et al. Patients with non-Jo-1 anti-tRNA-synthetase autoantibodies have worse survival than Jo-1 positive patients. Ann Rheum Dis. 2014;73(1):227–32. PubMed PMC

Hengstman GJ, ter Laak HJ, Vree Egberts WT, Lundberg IE, Moutsopoulos HM, Vencovsky J, et al. Anti-signal recognition particle autoantibodies: marker of a necrotising myopathy. Ann Rheum Dis. 2006;65(12):1635–8. PubMed PMC

Tjarnlund A, Bottai M, Rider LG, Werth VP, Pilkington C, de Visser M, et al. Progress report on development of classification criteria for adult and juvenile idiopathic inflammatory myopathies. Arthritis & Rheumatism. 2012;64(Suppl):S323–S324.

Alexanderson H, Del GM, Bingham CO, III, Orbai AM, Sarver C, Clegg-Smith K, et al. Patient-reported outcomes and adult patients’ disease experience in the idiopathic inflammatory myopathies. report from the OMERACT 11 Myositis Special Interest Group. J Rheumatol. 2014;41(3):581–92. PubMed PMC

Volochayev R, Csako G, Wesley R, Rider LG, Miller FW. Laboratory Test Abnormalities are Common in Polymyositis and Dermatomyositis and Differ Among Clinical and Demographic Groups. Open Rheumatol J. 2012;6:54–63. PubMed PMC

Performance of the 2016 ACR-EULAR Myositis Response Criteria in adult dermatomyositis/polymyositis therapeutic trials and consensus profiles

Idiopathic inflammatory myopathies

The effect of a 24-week training focused on activities of daily living, muscle strengthening, and stability in idiopathic inflammatory myopathies: a monocentric controlled study with follow-up

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

Opportunities and challenges in rheumatology research in Central Europe