Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies

In order to develop a preliminary core set of disease outcome measures for use in clinical trials of idiopathic inflammatory myopathies (IIM), we eval uated those measures used in previous trials, assessed the validation of pu blished instruments and discussed these at an international consensus confe rence. The initial proposals were further refined by a multidisciplinary gr oup of adult and paediatric specialists experienced in IIM using the Delphi method. The proposed preliminary core set of disease activity measures con sists of five domains: physician and patient parent global assessments of d isease activity; muscle strength: physical function, serum activity of musc le enzymes; and an assessment tool to capture extra-skeletal muscle disease activity. The group recommended further development of a core set of disea se damage measures for assessment of persistent changes in anatomy, patholo gy 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 instr ument 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 measureme nt and in optimizing therapeutic trials in myositis.