脆性X相关震颤共济失调综合征评定量表的临床测量特性
Clinimetric Properties of the Fragile X-associated Tremor Ataxia Syndrome Rating Scale.
作者信息
Hall Deborah A, Stebbins Glenn T, Jacquemont Sebastien, Berry-Kravis Elizabeth, Goetz Christopher G, Hagerman Randi, Zhang Lin, Leehey Maureen A
机构信息
Department of Neurological Sciences and Pediatrics Rush University Chicago Illinois United States.
Department of Pediatrics University of Montreal Montreal Canada.
出版信息
Mov Disord Clin Pract. 2019 Jan 22;6(2):120-124. doi: 10.1002/mdc3.12708. eCollection 2019 Feb.
BACKGROUND
There are currently no proven treatments for fragile X-associated tremor and ataxia syndrome (FXTAS). Validated outcome measures are needed in order to plan and conduct clinical trials to aid in the development of therapy.
METHODS
This study examined the reliability and construct validity of the FXTAS Rating Scale. The study was conducted by using ratings from movement disorder specialists, who were blinded to gene status, on the FXTAS Rating Scale.
RESULTS
In 295 premutation carriers with and without FXTAS, 33 scale items showed a high level of overall reliability, adequate item-to-total correlations and construct validity. Factor analysis revealed four components.
CONCLUSIONS
The result demonstrates that many items in the scale meet standard clinimetric criteria, but modification of the scale improved the overall utility.
背景
目前对于脆性X相关震颤共济失调综合征（FXTAS）尚无经证实的治疗方法。为了规划和开展有助于治疗方法开发的临床试验，需要经过验证的结果测量指标。
方法
本研究检验了FXTAS评定量表的信度和结构效度。研究通过让对基因状态不知情的运动障碍专家依据FXTAS评定量表进行评分来开展。
结果
在295名携带前突变基因且有或无FXTAS的个体中，33个量表项目显示出高度的总体信度、足够的项目与总分相关性以及结构效度。因子分析揭示了四个组成部分。
结论
结果表明该量表中的许多项目符合标准的临床测量学标准，但对量表的修改提高了其总体效用。
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