期刊
MUSCLE & NERVE
卷 58, 期 1, 页码 72-78出版社
WILEY
DOI: 10.1002/mus.26088
关键词
composite measures; facioscapulohumeral muscular dystrophy; functional testing; muscular dystrophy; outcome measures
资金
- FSH Society [FSHS-22013, FSHS-82012]
- National Institute of Arthritis and Musculoskeletal Skin Diseases [U01AR065119]
- University of Rochester CTSA award from National Center for Research Resources [UL1 RR024160]
- National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health
- NCATS grant [KL2TR000119]
Introduction: We developed an evaluator-administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD-COM) comprising patient-identified areas of functional burden for future clinical trials. Methods: We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD-COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test-retest reliability and convergent validity compared to established FSHD disease metrics. Results: The FSHD-COM demonstrated excellent test-retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90-0.94). Cross-sectional associations between the FSHD-COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51-0.92|). Discussion: The FSHD-COM is a disease-relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test-retest reliability and cross-sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD-COM. Muscle Nerve58: 72-78, 2018
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