Медицинская библиотека · Neurology · 2017

Motor, cognitive, and functional declines contribute to a single progressive factor in early HD.

Schobel Scott A, Palermo Giuseppe, Auinger Peggy, Long Jeffrey D, Ma Shiyang, Khwaja Omar S, Trundell Dylan, Cudkowicz Merit, Hersch Steven, Sampaio Cristina, Dorsey E Ray, Leavitt Blair R, Kieburtz Karl D, Sevigny Jeffrey J, Langbehn Douglas R, Tabrizi Sarah J, TRACK-HD, COHORT, CARE-HD, and 2CARE Huntington Study Group Investigators

СтатьяНаблюдательное исследованиеПеревод пока не подготовлен

Аннотация

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.

Для цитирования: Schobel Scott A, Palermo Giuseppe, Auinger Peggy, Long Jeffrey D, Ma Shiyang, Khwaja Omar S, Trundell Dylan, Cudkowicz Merit, Hersch Steven, Sampaio Cristina, Dorsey E Ray, Leavitt Blair R, Kieburtz Karl D, Sevigny Jeffrey J, Langbehn Douglas R, Tabrizi Sarah J, TRACK-HD, COHORT, CARE-HD, and 2CARE Huntington Study Group Investigators. Motor, cognitive, and functional declines contribute to a single progressive factor in early HD.. Neurology. 2017. DOI: 10.1212/wnl.0000000000004743.

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