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.

journal_name

J Comp Eff Res

authors

Mayhew J,Bonner N,Arbuckle R,Turnbull A,Bowden A,Skrinar A

doi

10.2217/cer-2017-0062

subject

Has Abstract

pub_date

2018-04-01 00:00:00

pages

381-395

issue

4

eissn

2042-6305

issn

2042-6313

journal_volume

7

pub_type

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