Stander Use in Spinal Muscular Atrophy: Results From a Large Natural History Database

被引：10

作者：

Townsend, Elise L. ^{[1
,2
,3
]}

Simeone, Sarah D. ^{[2
,3
]}

Krosschell, Kristin J. ^{[4
,5
]}

Zhang, Ren Zhe ^{[2
,3
]}

Swoboda, Kathryn J. ^{[2
,3
]}

机构：

[1] MGH Inst Hlth Profess, Sch Hlth & Rehabil Sci, Dept Phys Therapy, 36 1st Ave, Boston, MA 02129 USA

[2] Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA

[3] Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA

[4] Northwestern Univ, Feinberg Sch Med, Dept Phys Therapy & Human Movement Sci, Chicago, IL 60611 USA

[5] Northwestern Univ, Feinberg Sch Med, Dept Pediat, Chicago, IL 60611 USA

来源：

PEDIATRIC PHYSICAL THERAPY | 2020年 / 32卷 / 03期

关键词：

adverse events; Functional Motor Scale Extend; intervention; Modified Hammersmith Functional Motor Scale-Extend; motor performance; respiratory support; spinal muscular atrophy; stander use; supported standing; Test of Infant Motor Performance Screening Items; weight bearing; FUNCTIONAL MOTOR SCALE; SHAM CONTROL; MANAGEMENT; NUSINERSEN; DIAGNOSIS; RECOMMENDATIONS; RELIABILITY; CARE;

D O I：

10.1097/PEP.0000000000000713

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Purpose: The purpose of this study was to describe stander use in a natural history cohort of drug therapy-naive children with spinal muscular atrophy (SMA) who are not walking and identify factors associated with consistent stander use. Methods: 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. Results: Prevalence of consistent stander use was 13% in type 1 and 68% in type 2. SMA type,SMN2copy number, respiratory support, and head rotation control each predicted consistent stander use. Conclusions: 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.

引用

页码：235 / 241

页数：7

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