Background

Childhood-onset hyperkinetic movement disorders (HMD), including dystonia are notoriously difficult to treat and there are limited studies showing successful medical, surgical or non-pharmacological interventions.

Methods

This prospective study used grouped data (n = 22) from two studies of the Cognitive Orientation to daily Occupational Performance (CO-OP) Approach for patient-selected goals. Eligibility included aged 6–21 years, deep brain stimulation in place, with manual ability classification system level I-IV. Outcome was assessed on a range of patient-reported and clinician-rated measures across the International Classification of Function at end-treatment (10 weekly sessions) (series 1 and 2) and 3-month follow-up (series 1). Feasibility of outcomes to be used in a full trial were explored.

Findings

Nineteen participants completed the intervention and were included in the analysis. Of the primary outcome measures, the self-reported Canadian Occupational Performance Measure showed improvement in goal performance (mean change 4.08, 95% CI [3.37,4.79] post-; 4.18 [5.10,5.26] follow-up), and satisfaction (4.03 [3.04,5.03) post-; 4.44 [3.07,5.82] follow-up]. The Assessment of Motor and Process Skills showed improved motor score (0.52 [0.01,1.03] at follow-up only, while the process score did not change. Objective blind-rated pooled data using the Performance Quality Rating Scale-individualized indicated significant change for trained goals (3.79 [3.37,4.21] post-; (4.01,5.10) follow-up] and untrained goals (1.90 [1.24,2.55] post 1.91 [0.23,3.60] follow-up]. Motor impairment assessed by the Burke-Fahn Motor Disability Rating Scale was unchanged (−3.26 [-6.62,0.09] post-; −1.11 [-8.05,5.82] follow-up). Improvement was also observed in self-efficacy (0.97 [0.47,1.47] post-; 1.37 [1.91–0.83] follow-up) and Quality of Life (0.12 [0.03–0.22] follow-up).

Goal improvement; self-efficacy and quality of life captured significant change post-intervention. This improvement was shown despite no change on impairment-related measures and were shown to be feasible measures to use in a larger study of CO-OP for this population.

中文翻译：

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儿童期多动性运动障碍（包括肌张力障碍）的康复：ICF 结果的治疗变化以及完整试验评估结果的可行性

背景

众所周知，儿童期发病的多动性运动障碍 (HMD) 包括肌张力障碍难以治疗，而且很少有研究表明药物、手术或非药物干预是成功的。

方法

这项前瞻性研究使用了来自认知定向到日常职业表现 (CO-OP) 方法的两项研究的分组数据 (n = 22)，用于患者选择的目标。资格包括 6-21 岁，深部脑刺激到位，手动能力分类系统 I-IV 级。在结束治疗（10 周疗程）（系列 1 和 2）和 3 个月随访（系列 1）时，根据一系列患者报告和临床医生评级的措施评估结果。探讨了用于完整试验的结果的可行性。

发现

19 名参与者完成了干预并被纳入分析。在主要结果测量中，自我报告的加拿大职业绩效测量显示目标绩效（平均变化 4.08，95% CI [3.37,4.79] 后；4.18 [5.10,5.26] 后续）和满意度（4.03 [3.04,5.03) 后; 4.44 [3.07,5.82] 随访]。运动和过程技能评估显示运动得分提高（仅在随访时为 0.52 [0.01,1.03]，而过程得分没有变化。使用绩效质量评定量表-个体化的客观盲评汇总数据表明训练后的目标（3.79 [3.37,4.21] 后；（4.01,5.10）随访]和未训练的目标（1.90 [1.24,2.55] 后 1.91 [0.23,3.60] 随访]。由 Burke 评估的运动障碍Fahn 运动残疾评定量表没有变化 (-3. 26 [-6.62,0.09] 后；−1.11 [-8.05,5.82] 随访）。自我效能（术后 0.97 [0.47,1.47]；随访 1.37 [1.91-0.83]）和生活质量（0.12 [0.03-0.22] 随访）也有改善。

目标改进；自我效能和生活质量在干预后发生了重大变化。尽管与损伤相关的措施没有变化，但仍显示出这种改善，并且证明是在针对该人群的更大规模 CO-OP 研究中使用的可行措施。