Development and preliminary evidence of the psychometric properties of the GNE myopathy functional activity scale
Abstract
Aim: GNE myopathy, a rare, severe, progressive myopathy, presents with lower extremity distal muscle weakness. The GNE myopathy functional activity scale (GNEM-FAS) evaluates the impact of GNE myopathy on functioning in adults. This paper presents the psychometric validation of the GNEM-FAS. Patients & methods: Validation of the GNEM-FAS was performed using data from a randomized, double-blind, placebo-controlled Phase-II study (n = 46). Results: Domain score distributions were acceptable. Moderate inter-item correlations (typical range, 0.40–0.70), strong item convergent and discriminant validity and high internal consistency reliability (α = 0.88–0.92) supported the instrument structure. Test–retest reliability was strong (ICC range: 0.87–0.95). Scale scores distinguished among subjects with differing disease severity (p < 0.05). Conclusion: This study provides preliminary evidence of the GNEM-FAS as a valid, reliable assessment.
Supplementary Material
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© 2018 Adelphi Values Ltd.
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Received: 9 August 2017
Accepted: 20 October 2017
Published online: 15 November 2017
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Development and preliminary evidence of the psychometric properties of the GNE myopathy functional activity scale. (2017) Journal of Comparative Effectiveness Research. DOI: 10.2217/cer-2017-0062
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