Townsend Elise L, Simeone Sarah D, Krosschell Kristin J, Zhang Ren Zhe, Swoboda Kathryn J
Department of Physical Therapy (Dr Townsend), School of Health and Rehabilitation Sciences, MGH Institute of Health Professions, Boston, Massachusetts; Center for Genomic Medicine and Department of Neurology (Drs Townsend, Simeone, and Swoboda and Mr Zhang), Massachusetts General Hospital, Boston, Massachusetts; Department of Physical Therapy and Human Movement Sciences and Department of Pediatrics (Dr Krosschell), Feinberg School of Medicine, Northwestern University, Chicago, Illinois.
Pediatr Phys Ther. 2020 Jul;32(3):235-241. doi: 10.1097/PEP.0000000000000713.
The purpose of this study was to describe stander use in a natural history cohort of drug therapy-naïve children with spinal muscular atrophy (SMA) who are not walking and identify factors associated with consistent stander use.
Data from 397 children with SMA types 1 and 2 characterized the prevalence and frequency of stander use. Predictors of consistent stander use explored were SMA type, survival motor neuron 2 gene (SMN2) copy number, respiratory support, and motor performance.
Prevalence of consistent stander use was 13% in type 1 and 68% in type 2. SMA type, SMN2 copy number, respiratory support, and head rotation control each predicted consistent stander use.
Findings characterize stander use in children with SMA who are not walking, address important safety considerations, identify factors that may inform physical therapists' clinical decision-making related to standing program prescription, and provide guidance for future prospective studies.
本研究旨在描述未行走的未经药物治疗的脊髓性肌萎缩症（SMA）患儿自然病史队列中站立架的使用情况，并确定与持续使用站立架相关的因素。
来自397例1型和2型SMA患儿的数据描述了站立架使用的患病率和频率。探索的持续使用站立架的预测因素包括SMA类型、生存运动神经元2基因（SMN2）拷贝数、呼吸支持和运动表现。
1型患儿持续使用站立架的患病率为13%，2型为68%。SMA类型、SMN2拷贝数、呼吸支持和头部旋转控制各自都可预测持续使用站立架的情况。
研究结果描述了未行走的SMA患儿站立架的使用情况，解决了重要的安全问题，确定了可能为物理治疗师与站立计划处方相关的临床决策提供参考的因素，并为未来的前瞻性研究提供了指导。
