GNE 肌病功能活动量表的编制及初步心理测量学特性证据。
Development and preliminary evidence of the psychometric properties of the GNE myopathy functional activity scale.
机构信息
Ultragenyx Pharmaceutical, 60 Leveroni Court, Novato, CA 94949, USA.
Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
出版信息
J Comp Eff Res. 2018 Apr;7(4):381-395. doi: 10.2217/cer-2017-0062. Epub 2017 Nov 15.
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.
目的
GNE 肌病是一种罕见的严重进行性肌病，表现为下肢远端肌肉无力。GNE 肌病功能活动量表（GNEM-FAS）评估 GNE 肌病对成人功能的影响。本文介绍了 GNEM-FAS 的心理测量学验证。
患者和方法
使用随机、双盲、安慰剂对照 II 期研究（n = 46）的数据进行 GNEM-FAS 的验证。
结果
域评分分布可接受。中等的项目间相关性（典型范围，0.40-0.70）、较强的项目收敛和判别有效性以及较高的内部一致性可靠性（α = 0.88-0.92）支持了该工具的结构。测试-重测信度较强（ICC 范围：0.87-0.95）。量表评分可区分不同疾病严重程度的受试者（p < 0.05）。
结论
本研究初步证明了 GNEM-FAS 是一种有效且可靠的评估方法。
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