Rheumatology 2001; 40: 1262-1273
© 2001 British Society for Rheumatology
Report |
Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies
Center for Biologics Evaluation and Research, US Food and Drug Administration, Bethesda, Maryland, USA,
1 MRI Unit, Hammersmith Hospital, Du Cane Road, London W12 OHS,
2 Rheumatic Disease Centre, Hope Hospital, Salford M6 8HD, UK,
3 Department of Internal Medicine, Medical University of Debrecen, Moricz Zs KRT 22, Hungary,
4 University College London, Gower Street, London WC1E 6BT, UK,
5 Rheumatology Unit, Department of Medicine, Karolinska Institute, S-17176 Stockholm, Sweden,
6 Department of Rheumatology, Kings College Hospital (Dulwich), East Dulwich Grove, London SE22 8PT,
7 Dermatomyositis and Polymyositis Support Group, 146 Newtown Road, Woolston, Southampton SO19 9HR,
8 Centre for Rheumatology, Middlesex Hospital, London W1P 9PL, UK,
9 Institute of Rheumatology, 12850 Praha 2, Czech Republic,
10 Department of Academic Rheumatology GKT, Weston Education Centre, Cutcombe Road, London SE5 9PJ,
11 King's College Hospital (Dulwich), East Dulwich Grove, London W1P 9PG and
12 The Centre for Rheumatology, University College London Hospital, Arthur Stanley House, 40–50 Tottenham Street, London WIP 9PC, UK
In order to develop a preliminary core set of disease outcome measures for use in clinical trials of idiopathic inflammatory myopathies (IIM), we evaluated those measures used in previous trials, assessed the validation of published instruments and discussed these at an international consensus conference. The initial proposals were further refined by a multidisciplinary group of adult and paediatric specialists experienced in IIM using the Delphi method. The proposed preliminary core set of disease activity measures consists of five domains: physician and patient/parent global assessments of disease activity; muscle strength; physical function; serum activity of muscle enzymes; and an assessment tool to capture extra-skeletal muscle disease activity. The group recommended further development of a core set of disease damage measures for assessment of persistent changes in anatomy, pathology and function of at least 6 months' duration. The group recommended that patient-reported outcomes should include generic health-related quality of life assessments using the Medical Outcomes Study 36-item Short Form (SF-36) health survey in adult IIM patients and a validated quality of life instrument for paediatric patients. We propose the core set of outcome measures as a minimum group of assessments to include in all IIM therapeutic studies. The use of this core set should assist in standardizing outcome measurement and in optimizing therapeutic trials in myositis.
KEY WORDS: Idiopathic inflammatory myopathies, Outcome measures.
* These authors contributed equally to this work.
A complete list of all members can be found in Appendix A.
Correspondence to: F. W. Miller, Environmental Autoimmunity Group, Office of Clinical Research, National Institute of Environmental Health Sciences, National Institutes of Health, 9 Memorial Drive, Room 1W101, MSC 0958, Bethesda, MD 20892, USA.
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