穿戴式半机械人混合辅助肢体（HAL）的神经控制治疗可改善进展缓慢的罕见神经肌肉疾病患者的步行功能：一项针对疗效和安全性的多中心、随机、对照交叉试验（NCY-3001）。

Cybernic treatment with wearable cyborg Hybrid Assistive Limb (HAL) improves ambulatory function in patients with slowly progressive rare neuromuscular diseases: a multicentre, randomised, controlled crossover trial for efficacy and safety (NCY-3001).

机构信息

Departments of Neurology and Rehabilitation Medicine, National Hospital Organization Niigata National Hospital, 3-52 Akasaka, Kashiwazaki, Niigata, 945-8585, Japan.

Center for Cybernics Research, University of Tsukuba, Tsukuba, Japan.

出版信息

Orphanet J Rare Dis. 2021 Jul 7;16(1):304. doi: 10.1186/s13023-021-01928-9.

DOI:10.1186/s13023-021-01928-9

PMID:34233722

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8261928/

Abstract

BACKGROUND

Rare neuromuscular diseases such as spinal muscular atrophy, spinal bulbar muscular atrophy, muscular dystrophy, Charcot-Marie-Tooth disease, distal myopathy, sporadic inclusion body myositis, congenital myopathy, and amyotrophic lateral sclerosis lead to incurable amyotrophy and consequent loss of ambulation. Thus far, no therapeutic approaches have been successful in recovering the ambulatory ability. Thus, the aim of this trial was to evaluate the efficacy and safety of cybernic treatment with a wearable cyborg Hybrid Assistive Limb (HAL, Lower Limb Type) in improving the ambulatory function in those patients.

RESULTS

We conducted an open-label, randomised, controlled crossover trial to test HAL at nine hospitals between March 6, 2013 and August 8, 2014. Eligible patients were older than 18 years and had a diagnosis of neuromuscular disease as specified above. They were unable to walk for 10 m independently and had neither respiratory failure nor rapid deterioration in gait. The primary endpoint was the distance passed during a two-minute walk test (2MWT). The secondary endpoints were walking speed, cadence, and step length during the 10-m walk test (10MWT), muscle strength by manual muscle testing (MMT), and a series of functional measures. Adverse events and failures/problems/errors with HAL were also evaluated. Thirty patients were randomly assigned to groups A or B, with each group of 15 receiving both treatments in a crossover design. The efficacy of a 40-min walking program performed nine times was compared between HAL plus a hoist and a hoist only. The final analysis included 13 and 11 patients in groups A and B, respectively. Cybernic treatment with HAL resulted in a 10.066% significantly improved distance in 2MWT (95% confidence interval, 0.667-19.464; p = 0.0369) compared with the hoist only treatment. Among the secondary endpoints, the total scores of MMT and cadence at 10MWT were the only ones that showed significant improvement. The only adverse effects were slight to mild myalgia, back pain, and contact skin troubles, which were easily remedied.

CONCLUSIONS

HAL is a new treatment device for walking exercise, proven to be more effective than the conventional method in patients with incurable neuromuscular diseases.

TRIAL REGISTRATION

JMACTR, JMA-IIA00156.

摘要

背景

脊髓性肌萎缩症、脊髓延髓肌萎缩症、肌营养不良症、夏科-马里-图思病、远端肌病、散发性包涵体肌炎、先天性肌病和肌萎缩性侧索硬化症等罕见神经肌肉疾病可导致不可治愈的肌肉萎缩和随之而来的行走能力丧失。迄今为止，尚无治疗方法能够成功恢复行走能力。因此，本试验旨在评估可穿戴外骨骼混合辅助肢体（HAL，下肢型）在改善这些患者的步行功能方面的疗效和安全性。

结果

我们在 2013 年 3 月 6 日至 2014 年 8 月 8 日期间在九家医院进行了一项开放标签、随机、对照交叉试验，以测试 HAL。符合条件的患者年龄大于 18 岁，患有上述神经肌肉疾病的诊断。他们无法独立行走 10 米，没有呼吸衰竭或步态迅速恶化。主要终点是两分钟步行测试（2MWT）期间通过的距离。次要终点是 10 米步行测试（10MWT）期间的步行速度、步频和步长、手动肌肉测试（MMT）的肌肉力量以及一系列功能测量。还评估了不良事件和 HAL 的故障/问题/错误。30 名患者被随机分配到 A 组或 B 组，每组 15 名患者以交叉设计接受两种治疗。比较了 HAL 加吊具与仅用吊具进行的 40 分钟步行方案的疗效，共进行了 9 次。最终分析包括 A 组和 B 组的 13 名和 11 名患者。与仅用吊具治疗相比，HAL 的神经控制治疗可使 2MWT 的距离显著改善 10.066%（95%置信区间，0.667-19.464；p=0.0369）。在次要终点中，只有 10MWT 的 MMT 总分和步频显示出显著改善。唯一的不良影响是轻微到中度的肌痛、背痛和接触性皮肤问题，这些问题很容易得到解决。