title: Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies.
abstract: 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.
authors.affiliation: Center for Biologics Evaluation and Research, US Food and Drug Administration, Bethesda, Maryland, USA.
authors.forename: F W
authors.initials: FW
authors.lastname: Miller
authors.forename: L G
authors.initials: LG
authors.lastname: Rider
authors.forename: Y L
authors.initials: YL
authors.lastname: Chung
authors.forename: R
authors.initials: R
authors.lastname: Cooper
authors.forename: K
authors.initials: K
authors.lastname: Danko
authors.forename: V
authors.initials: V
authors.lastname: Farewell
authors.forename: I
authors.initials: I
authors.lastname: Lundberg
authors.forename: C
authors.initials: C
authors.lastname: Morrison
authors.forename: L
authors.initials: L
authors.lastname: Oakley
authors.forename: I
authors.initials: I
authors.lastname: Oakley
authors.forename: C
authors.initials: C
authors.lastname: Pilkington
authors.forename: J
authors.initials: J
authors.lastname: Vencovsky
authors.forename: K
authors.initials: K
authors.lastname: Vincent
authors.forename: D L
authors.initials: DL
authors.lastname: Scott
authors.forename: D A
authors.initials: DA
authors.lastname: Isenberg
mesh_terms: D000328:Adult; D002648:Child; D006801:Humans; D009220:Myositis / Q000175:diagnosis / Q000503:physiopathology*; D017063:Outcome Assessment, Health Care