Barbuto Scott, Lee Seonjoo, Stein Joel, Kuo Sheng-Han, Quinn Lori, Spinner Michael, Stern Yaakov
Department of Physical Medicine and Rehabilitation, College of Physicians and Surgeons, Columbia University Medical Center, New York, New York.
Departments of Psychiatry and Biostatistics, College of Physicians and Surgeons, Columbia University Medical Center, New York, New York.
JAMA Neurol. 2025 Sep 14. doi: 10.1001/jamaneurol.2025.3421.
Clinical practice guidelines advise balance training for cerebellar ataxia, but little is known regarding high-intensity aerobic exercise.
To compare home high-intensity aerobic training to home balance training on improvements of ataxia symptoms using the Scale for the Assessment and Rating of Ataxia (SARA).
DESIGN, SETTING, AND PARTICIPANTS: This assessor-masked randomized clinical trial was conducted between January 1, 2021, and September 1, 2024, through home training, with in-person assessments at a single ataxia care center in a large urban US city. Individuals with various cerebellar ataxia types were eligible for inclusion. Data analysis was performed from November 2024 to February 2025.
Individuals in the aerobic group trained for 30 minutes/session, 5 times/week, at up to 85% predicted maximum heart rate. Individuals in the balance group performed 30 minutes of balance exercises of varying difficulty 5 times/week. Participants were given study support of biweekly phone calls for only the first 6 months of this 12-month study.
The primary outcome was SARA score to measure ataxia symptoms (range, 0-40 points, with higher scores indicating more ataxia). Secondary outcomes included number of adverse events, training adherence, balance measures, gait speed, quality of life, fatigue, and fitness levels (assessed via V̇o2max). Assessments were conducted at baseline and at 6, 9, and 12 months.
A total of 114 individuals with various cerebellar ataxia types were approached: 52 individuals declined participation or did not meet inclusion criteria, while 62 individuals were enrolled. The 62 participants included 29 women (46.8%), with a mean (SD) age of 54.4 (12.9) years and mean (SD) SARA score of 12.1 (4.1) points. Linear mixed-effects model analysis revealed that the home aerobic group had significantly larger improvement in outcomes than the balance group, particularly for SARA score (β, -1.53; 95% CI, -2.44 to -0.61; P = .001), fatigue (β, -9.38; 95% CI, -15.1 to -3.7; P = .001), and Vo2max (β, 4.26; 95% CI, 2.1-6.4; P < .001). At 6 months, the aerobic and balance groups had changes in SARA scores of -2.4 points (95% CI, -3.1 to -1.80) and -0.9 points (95% CI, -1.5 to -0.2), respectively. For the aerobic group, individuals who continued training maintained benefits in SARA score (change from baseline, -3.81 points; 95% CI, -2.2 to -5.4), whereas those who limited or stopped training had benefits trend back to baseline levels (change from baseline, 0.4 points; 95% CI, -0.4 to 1.2) at 1 year.
In this randomized clinical trial, home high-intensity aerobic training improved ataxia symptoms, fatigue, and aerobic fitness more than dose-matched home balance training among individuals with cerebellar ataxias. Individuals in the aerobic group who continued to train regularly maintained benefits at 1 year.
ClinicalTrials.gov Identifier: NCT05002218.
临床实践指南建议对小脑性共济失调进行平衡训练，但对于高强度有氧运动知之甚少。
使用共济失调评估与分级量表（SARA）比较家庭高强度有氧运动训练与家庭平衡训练对共济失调症状改善情况的影响。
设计、地点和参与者：这项评估者盲法随机临床试验于2021年1月1日至2024年9月1日进行，通过家庭训练，并在美国一个大城市的单一共济失调护理中心进行面对面评估。各种类型的小脑性共济失调患者均符合纳入标准。数据分析于2024年11月至2025年2月进行。
有氧运动组的个体每次训练30分钟，每周5次，运动强度最高达到预测最大心率的85%。平衡组的个体每周进行5次，每次30分钟难度各异的平衡练习。在这项为期12个月的研究中，仅在前6个月为参与者提供每两周一次的电话研究支持。
主要结局是用SARA评分来衡量共济失调症状（范围为0 - 40分，分数越高表明共济失调越严重）。次要结局包括不良事件数量、训练依从性、平衡测量、步态速度、生活质量、疲劳和体能水平（通过最大摄氧量评估）。在基线以及6、9和12个月时进行评估。
共接触了114名各种类型的小脑性共济失调患者：52名患者拒绝参与或不符合纳入标准，62名患者被纳入研究。这62名参与者包括29名女性（46.8%），平均（标准差）年龄为54.4（12.9）岁，平均（标准差）SARA评分为12.1（4.1）分。线性混合效应模型分析显示，家庭有氧运动组在结局改善方面显著大于平衡组，特别是在SARA评分（β， - 1.53；95%置信区间， - 2.44至 - 0.61；P = 0.001）、疲劳（β， - 9.38；95%置信区间， - 15.1至 - 3.7；P = 0.001）和最大摄氧量（β，4.26；95%置信区间，2.1 - 6.4；P < 0.001）方面。在6个月时，有氧运动组和平衡组的SARA评分变化分别为 - 2.4分（95%置信区间， - 3.1至 - 1.80）和 - 0.9分（95%置信区间， - 1.5至 - 0.2）。对于有氧运动组，继续训练的个体在SARA评分方面保持获益（相对于基线变化 - 3.81分；95%置信区间， - 2.2至 - 5.4），而那些限制或停止训练的个体在1年时获益趋势回到基线水平（相对于基线变化0.4分；95%置信区间， - 0.4至1.2）。
在这项随机临床试验中，对于患有小脑性共济失调的个体，家庭高强度有氧运动训练在改善共济失调症状、疲劳和有氧适能方面比剂量匹配的家庭平衡训练更有效。有氧运动组中继续定期训练的个体在1年时保持获益。
ClinicalTrials.gov标识符：NCT05002218。
