TY - JOUR
T1 - Natural history of 10-meter walk/run test performance in spinal muscular atrophy
T2 - A longitudinal analysis
AU - Krosschell, Kristin J.
AU - Townsend, Elise L.
AU - Kiefer, Michael
AU - Simeone, Sarah D.
AU - Zumpf, Katelyn
AU - Welty, Leah
AU - Swoboda, Kathryn J.
N1 - Funding Information:
We gratefully acknowledge the site clinical coordinators, research nurses, and evaluators who were critical to the success of this study. We thank Cure SMA, the Project Cure Investigators Network and The Eunice Kennedy Shriver National Institutes of Health and Human Development R01-HD054599 award to KJS supported the collection and use of the SMA longitudinal natural history data. We most importantly thank all the children and families who participated in the study.
Publisher Copyright:
© 2021 Elsevier B.V.
PY - 2022/2
Y1 - 2022/2
N2 - As trials and treatments for spinal muscular atrophy (SMA) rapidly evolve, understanding the natural history and potential utility of the 10-meter walk/run test (10MWRT) in ambulant individuals is critical. Study aims were to: 1) establish change over time and across age for 10MWRT time in an untreated natural history cohort of young, ambulatory participants with SMA and 2) identify relations between 10MWRT time and age, SMA type, SMN2 copy number and anthropometrics. Untreated individuals (n = 56) age 2 to 21 years who were enrolled in a long-term natural history study between 2005 and 2014 and met inclusion criteria were included. Linear mixed effects models were used to assess changes in 10MWRT time with age and associations with SMA type, SMN2 copy number, and body mass. SMA type 3b (versus 3a), SMN2 copy number 4 (versus 3) and lower body mass were associated with faster 10MWRT. 10MWRT performance improved between 3 and 8 years of age, was stable between 9 and 10, and gradually declined from 11 to 18. Findings provide the first longitudinal natural history report of 10MWRT time in young individuals with SMA and offer a critical foundation for interpreting childhood change in short distance walking speed with pharmacologic treatment.
AB - As trials and treatments for spinal muscular atrophy (SMA) rapidly evolve, understanding the natural history and potential utility of the 10-meter walk/run test (10MWRT) in ambulant individuals is critical. Study aims were to: 1) establish change over time and across age for 10MWRT time in an untreated natural history cohort of young, ambulatory participants with SMA and 2) identify relations between 10MWRT time and age, SMA type, SMN2 copy number and anthropometrics. Untreated individuals (n = 56) age 2 to 21 years who were enrolled in a long-term natural history study between 2005 and 2014 and met inclusion criteria were included. Linear mixed effects models were used to assess changes in 10MWRT time with age and associations with SMA type, SMN2 copy number, and body mass. SMA type 3b (versus 3a), SMN2 copy number 4 (versus 3) and lower body mass were associated with faster 10MWRT. 10MWRT performance improved between 3 and 8 years of age, was stable between 9 and 10, and gradually declined from 11 to 18. Findings provide the first longitudinal natural history report of 10MWRT time in young individuals with SMA and offer a critical foundation for interpreting childhood change in short distance walking speed with pharmacologic treatment.
KW - 10-meter walk/run test
KW - 10MWRT
KW - Natural history
KW - Outcome measures
KW - Spinal muscular atrophy
KW - Timed function tests
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U2 - 10.1016/j.nmd.2021.08.010
DO - 10.1016/j.nmd.2021.08.010
M3 - Article
C2 - 35063329
AN - SCOPUS:85123098840
SN - 0960-8966
VL - 32
SP - 125
EP - 134
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 2
ER -