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

Elise L. Townsend; Sarah D. Simeone; Kristin J. Krosschell; Ren Zhe Zhang; Kathryn J. Swoboda

doi:10.1097/PEP.0000000000000713

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

Elise L. Townsend^*, Sarah D. Simeone, Kristin J. Krosschell, Ren Zhe Zhang, Kathryn J. Swoboda

^*Corresponding author for this work

Physical Therapy and Human Movement Sciences

Research output: Contribution to journal › Article › peer-review

6 Scopus citations

Abstract

Purpose: 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. 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, SMN2 copy 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.

Original language	English (US)
Pages (from-to)	235-241
Number of pages	7
Journal	Pediatric Physical Therapy
Volume	32
Issue number	3
DOIs	https://doi.org/10.1097/PEP.0000000000000713
State	Published - Jul 1 2020

Keywords

Functional Motor Scale Extend
Modified Hammersmith Functional Motor Scale-Extend
Test of Infant Motor Performance Screening Items
adverse events
intervention
motor performance
respiratory support
spinal muscular atrophy
stander use
supported standing
weight bearing

ASJC Scopus subject areas

Physical Therapy, Sports Therapy and Rehabilitation
Pediatrics, Perinatology, and Child Health

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10.1097/PEP.0000000000000713

Cite this

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title = "Stander Use in Spinal Muscular Atrophy: Results from a Large Natural History Database",

abstract = "Purpose: The purpose of this study was to describe stander use in a natural history cohort of drug therapy-na{\"i}ve 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, SMN2 copy 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.",

keywords = "Functional Motor Scale Extend, Modified Hammersmith Functional Motor Scale-Extend, Test of Infant Motor Performance Screening Items, adverse events, intervention, motor performance, respiratory support, spinal muscular atrophy, stander use, supported standing, weight bearing",

author = "Townsend, {Elise L.} and Simeone, {Sarah D.} and Krosschell, {Kristin J.} and Zhang, {Ren Zhe} and Swoboda, {Kathryn J.}",

note = "Funding Information: Grant Support: Funding was provided by Cure SMA and The Eunice Kennedy Shriver National Institute of Child Health and Human Development R01-HD054599 to Dr Swoboda. Publisher Copyright: {\textcopyright} 2020 Academy of Pediatric Physical Therapy of the American Physical Therapy Association. Unauthorized reproduction of this article is prohibited.",

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AU - Swoboda, Kathryn J.

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N2 - Purpose: 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. 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, SMN2 copy 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.

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

Abstract

Keywords

ASJC Scopus subject areas

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