Motor, cognitive, and functional declines contribute to a single progressive factor in early HD
Motor, cognitive, and functional declines contribute to a single progressive factor in early HD
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DOI：
10.1212/wnl.0000000000004743
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发表时间：
2017-12-12
期刊：
影响因子：
9.9
通讯作者：
Tabrizi, Sarah J.
中科院分区：
文献类型：
--
作者：
Schobel, Scott A.;Palermo, Giuseppe;Tabrizi, Sarah J.
目的：使用前瞻性观察性队列研究的数据和随机双盲临床试验的安慰剂组数据，确定早期亨廷顿病（HD）临床进展的改进指标。方法：我们研究了统一亨廷顿病评定量表（UHDRS）和非UHDRS临床指标以及1例进行性萎缩的脑指标，668例早期HD患者前瞻性随访长达30至36个月的纵向临床随访。结果表明，运动，认知，和整体功能下降最好地表征了临床进展，并且与进行性皮质纹状体萎缩的脑测量最强相关。在HD中使用复合运动、认知和整体功能临床结局指标提供了与进行性脑萎缩指标更相关的临床进展指标，并提供了相对于目前使用的个体运动、认知和功能结局指标提高临床试验效率的机会。
Objective: To identify an improved measure of clinical progression in early Huntington disease (HD) using data from prospective observational cohort studies and placebo group data from randomized double-blind clinical trials.Methods: We studied Unified Huntington Disease Rating Scale (UHDRS) and non-UHDRS clinical measures and brain measures of progressive atrophy in 1,668 individuals with early HD followed up prospectively for up to 30 to 36 months of longitudinal clinical follow-up.Results: The results demonstrated that a composite measure of motor, cognitive, and global functional decline best characterized clinical progression and was most strongly associated with brain measures of progressive corticostriatal atrophy.Conclusions: Use of a composite motor, cognitive, and global functional clinical outcome measure in HD provides an improved measure of clinical progression more related to measures of progressive brain atrophy and provides an opportunity for enhanced clinical trial efficiency relative to currently used individual motor, cognitive, and functional outcome measures.