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亨廷顿病特定任务训练:一项随机对照可行性试验。

Task-specific training in Huntington disease: a randomized controlled feasibility trial.

机构信息

L. Quinn, PT, PhD, School of Healthcare Sciences, Cardiff University, Cardiff, United Kingdom.

K. Debono, BSc AnatomicalScience(Hons), BSc Physiotherapy, MCSP, School of Healthcare Sciences, Cardiff University.

出版信息

Phys Ther. 2014 Nov;94(11):1555-68. doi: 10.2522/ptj.20140123. Epub 2014 Jul 10.

Abstract

BACKGROUND

Task-specific training may be a suitable intervention to address mobility limitations in people with Huntington disease (HD).

OBJECTIVE

The aim of this study was to assess the feasibility and safety of goal-directed, task-specific mobility training for individuals with mid-stage HD.

DESIGN

This study was a randomized, blinded, feasibility trial; participants were randomly assigned to control (usual care) and intervention groups.

SETTING

This multisite study was conducted in 6 sites in the United Kingdom.

PATIENTS

Thirty individuals with mid-stage HD (13 men, 17 women; mean age=57.0 years, SD=10.1) were enrolled and randomly assigned to study groups.

INTERVENTION

Task-specific training was conducted by physical therapists in participants' homes, focusing on walking, sit-to-stand transfers, and standing, twice a week for 8 weeks. Goal attainment scaling was used to individualize the intervention and monitor achievement of personal goals.

MEASUREMENTS

Adherence and adverse events were recorded. Adjusted between-group comparisons on standardized outcome measures were conducted at 8 and 16 weeks to determine effect sizes.

RESULTS

Loss to follow-up was minimal (n=2); adherence in the intervention group was excellent (96.9%). Ninety-two percent of goals were achieved at the end of the intervention; 46% of the participants achieved much better than expected outcomes. Effect sizes on all measures were small.

LIMITATIONS

Measurements of walking endurance were lacking.

CONCLUSIONS

The safety of and excellent adherence to a home-based, task-specific training program, in which most participants exceeded goal expectations, are encouraging given the range of motivational, behavioral, and mobility issues in people with HD. The design of the intervention in terms of frequency (dose), intensity (aerobic versus anaerobic), and specificity (focused training on individual tasks) may not have been sufficient to elicit any systematic effects. Thus, a larger-scale trial of this specific intervention does not seem warranted.

摘要

背景

任务特定训练可能是解决亨廷顿病(HD)患者运动障碍的合适干预措施。

目的

本研究旨在评估针对 HD 中晚期患者的目标导向、任务特定的移动性训练的可行性和安全性。

设计

这是一项随机、双盲、可行性试验;参与者被随机分配到对照组(常规护理)和干预组。

设置

这项多地点研究在英国的 6 个地点进行。

患者

30 名 HD 中晚期患者(13 名男性,17 名女性;平均年龄=57.0 岁,标准差=10.1)被纳入并随机分配到研究组。

干预

物理治疗师在患者家中进行任务特定训练,重点是步行、坐站转移和站立,每周两次,共 8 周。使用目标达成量表对干预进行个体化,并监测个人目标的实现。

测量

记录依从性和不良事件。在 8 周和 16 周时,对标准化结局测量进行调整后的组间比较,以确定效应大小。

结果

随访损失最小(n=2);干预组的依从性极好(96.9%)。干预结束时,92%的目标得以实现;46%的参与者取得了远高于预期的结果。所有测量指标的效应大小均较小。

局限性

缺乏对步行耐力的测量。

结论

考虑到 HD 患者在动机、行为和移动性方面的各种问题,在家中进行基于任务的训练计划的安全性和极好的依从性令人鼓舞,该计划中大多数参与者都超出了预期目标。就频率(剂量)、强度(有氧与无氧)和特异性(针对个别任务的集中训练)而言,该干预措施的设计可能不足以产生任何系统性效果。因此,没有理由进行这种特定干预措施的更大规模试验。

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