Developing International Consensus Definitions Of Improvement For Adult and Juvenile Dermatomyositis and Polymyositis

Saad Feroz¹, Nicolino. Ruperto², Jiri Vencovsky³, Peter A. Lachenbruch¹, Brian Erman⁴, Adam Huber⁵, Brian M. Feldman⁶, Ingrid E. Lundberg⁷, Angela Pistorio⁸, Howard Rockette⁹, Frederick W. Miller¹, Rohit Aggarwal¹⁰, Lisa G. Rider¹, for The ACR-EULAR Myositis Response Criteria Project Group¹¹, Angelo Ravelli¹², Clarissa Pilkington¹³ and Sheila K. Oliveira¹⁴, ¹Environmental Autoimmunity Group, NIEHS, NIH, Bethesda, MD, ²PRINTO, IRCCS G. Gaslini, Genoa, Italy, ³Institute of Rheumatology, Department of Clinical and Experimental Rheumatology, 1st Faculty of Medicine, Charles University, Prague, Czech Republic, ⁴SRA International, Research Triangle Park, NC, ⁵IWK Health Centre, Halifax, NS, Canada, ⁶Rheumatology, Hospital for Sick Children, Toronto, ON, Canada, ⁷Rheumatology Unit, Karolinska University Hospital, Solna, Karolinska Institutet, Stockholm, Sweden, ⁸PRINTO, Genoa, Italy, ⁹University of Pittsburgh, Pittsburgh, PA, ¹⁰Medicine / Rheumatology, University of Pittsburgh, Pittsburgh, PA, ¹¹NIEHS, NIH, Bethesda, MD, ¹²Paediatric Rheumatology International Trials Organization (PRINTO), Istituto Giannina Gaslini, Genova, Italy, ¹³Rheumatology Department, Great Ormond Street Hospital, London, United Kingdom, ¹⁴Pediatric Rheumatology, Instituto de Pediatria e Puericultura Martagão Gesteira (IPPMG) da Universidade Federal do Rio de Janeiro (UFRJ), Rio de Janeiro, Brazil

Meeting: 2013 ACR/ARHP Annual Meeting

Keywords: Clinical Response, dermatomyositis, juvenile dermatomyositis, Myositis and outcome measures

Session Information

Title: ARHP Imaging of Rheumatic Disease: Research Methodology

Session Type: Abstract Submissions (ARHP)

Background/Purpose:

IMACS and PRINTO have developed preliminary core set activity measures and definitions of improvement (DOIs). However, these were developed from small and partially retrospective data sets. The current project will develop and further validate DOIs for adult and juvenile dermatomyositis (DM) and polymyositis (PM), and the first definition of major clinical improvement for use in therapeutic trials.

Methods: A repository of 8 natural history studies and 8 clinical trials in DM/PM was used to create 270 adult and 299 pediatric patient profiles consisting of core set activity measures on two visits. Clinical experts completed a questionnaire on their experience with the core set measures and care of myositis patients. Experts, randomized into survey groups based on experience, rated the degree of improvement in patient profiles.

Results:

Sixty-six adult specialists (39 rheumatologist, 11 neurologists, 7 dermatologists, 7 other), saw an average of 82 adult DM/PM patients and practiced an average of 18.6 years. Fifty-two percent of adult experts had experience with 5 of 6 core set measures. Sixty-nine pediatric specialists, primarily rheumatologists, saw an average of 28 juvenile DM patients and practiced an average of 19.7 years. Forty-nine percent had experience with all core set measures and 34% had experience with 7 or 8 of 9 measures. Adult and pediatric experts had participated in an average of 1.5 trials utilizing IMACs measures; pediatric experts participated in 1 trial using PRINTO measures. Patient profiles indicated a moderate degree of activity (median MD global activity of 2 and 5, Patient/parent global activity of 5 and 4.9, MMT-8 score of 70 and 54, Extra-Muscular global activity of 1.7 in adult and 5.0 in pediatric profiles). Profiles varied in their degree of improvement based on the IMACS preliminary DOI (52% adult and 77% of pediatric profiles met DOI20%, 18% adult and 54% of pediatric profiles met DOI60%, and 48% adult and 23% of pediatric profiles were not improved by the DOI). Four-hundred-seven DOIs have been drafted to be tested for sensitivity and specificity, based on the number of measures and the degree of change corresponding to consensus clinical improvement, and inclusion of FDA’s recommendation to incorporate patient-reported outcomes. Additional DOIs will be created by logistic regression analysis of the ratings of patient profiles. The top performing DOIs will be externally validated with data from 4 randomized controlled trials.

Conclusion: A DOI for minimal and major clinical response will be developed and fully validated for adult and juvenile DM/PM using large natural history and clinical trial datasets. These internationally accepted consensus DOIs should facilitate new trials and enhance development of future therapies for myositis patients.

Disclosure:

S. Feroz,
None;

N. Ruperto,

ACR-EULAR, The UK Myositis Support Group, Cure JM Foundation, and The Myositis Association,

J. Vencovsky,

ACR-EULAR, the UK Myositis Support Group, Cure JM Foundation, The Myositis Association,

P. A. Lachenbruch,

ACR-EULAR, NIEHS,

B. Erman,

NIEHS,

A. Huber,
None;

B. M. Feldman,
None;

I. E. Lundberg,

Brisol Myers Squibb,

Novartis Pharmaceutical Corporation,

A. Pistorio,

ACR-EULAR,

H. Rockette,

ACR-EULAR, NIEHS,

F. W. Miller,

ACR-EULAR, NIEHS, NIH Office of Rare Diseases, NIAMS, the UK Myositis Support Group, Cure JM Foundation, The Myositis Association,

R. Aggarwal,

ACR-EULAR, the UK Myositis Support Group, Cure JM Foundation, The Myositis Association,

L. G. Rider,

ACR-EULAR, NIEHS, NIH Office of Rare Diseases, NIAMS, the UK Myositis Support Group, Cure JM Foundation, The Myositis Association,

F. T. ACR-EULAR Myositis Response Criteria Project Group,
None;

A. Ravelli,
None;

C. Pilkington,

Great Ormond Street Hospital Charity ,

Great Ormond Street Hospital Trust,

UCB SLE trial,

President of British Society for Paediatric and Adolescent Rheumatology,

S. K. Oliveira,
None.

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