The impact of early powered mobility training in families and children with Spinal Muscular Atrophy Type I

This study examined the effects of early powered mobility training using modified ride-on toy cars in young children with Spinal Muscular Atrophy type I (SMA I). A randomized, waitlist-controlled, single-blinded trial was conducted in Spain with 16 children aged 10 months to 5 years. The intervention consisted of 12 weeks of powered mobility training in natural environments, followed by 4 weeks of follow-up. Primary outcomes included participation (YC-PEM), functional ability (PEDI-CAT), and quality of life (PedsQL-NM), assessed at five time points. Parental stress (PSI-SF) was a secondary outcome. Children in the experimental group demonstrated clinically meaningful gains in Daily Activities and Mobility from week 4 onward (z = 0.41–0.72; g = 0.29–0.53), while Social/Cognitive improvements exceeded SEMm by weeks 8–16. Quality of life showed significant differences at week 4 (<i>p</i> = 0.03; g = 0.93), though below the SEMm threshold. Modest increases were observed in home participation, while Desired Change remained stable. Parental stress reductions reached statistical significance at weeks 4, 8, and 16 (<i>p</i> &lt; 0.05), though within SEM_m margins. Findings support early powered mobility as an assistive technology that enhances functional independence in SMA I. Complementary psychosocial and environmental supports may maximize broader participation and quality-of-life outcomes.

本研究针对1型脊髓性肌萎缩症（Spinal Muscular Atrophy type I, SMA I）幼儿群体，探讨采用改良式骑乘玩具车开展早期动力移动训练的干预效果。本研究在西班牙开展一项随机、等候组对照、单盲试验，共纳入16名年龄介于10个月至5岁的幼儿。干预方案包含12周的自然环境动力移动训练，后续辅以4周的随访评估。主要结局指标包括参与度（YC-PEM）、功能能力（PEDI-CAT）与生活质量（PedsQL-NM），共设置5个时间点进行评估；次要结局指标为父母压力（PSI-SF）。试验组幼儿自第4周起，在日常活动与移动能力维度展现出具有临床意义的提升（z=0.41~0.72；g=0.29~0.53）；社交/认知能力的改善在第8周至第16周时超过SEMm阈值。生活质量在第4周时出现显著组间差异（*p*=0.03；g=0.93），但仍低于SEMm阈值。家庭参与度出现小幅提升，而“期望改变”维度得分保持稳定。父母压力评分在第4、8、16周时达到统计学显著性降低（*p*<0.05），但变化幅度处于SEM_m区间内。研究结果证实，早期动力移动训练作为一种辅助技术，可有效提升1型脊髓性肌萎缩症幼儿的功能独立性。辅以针对性的社会心理与环境支持，或可进一步拓展其社会参与范围并优化生活质量结局。