The gross motor function measure is a valid and sensitive outcome measure for spinal muscular atrophy

Title

The gross motor function measure is a valid and sensitive outcome measure for spinal muscular atrophy

Creator

Nelson L; Owens H; Hynan L S; Iannaccone S T

Publisher

Neuromuscular Disorders

Date

2006

Subject

Clinical Trials as Topic; Male; Sensitivity and Specificity; Severity of Illness Index; Child; Humans; Adolescent; Female; Child Preschool; Psychomotor Performance/physiology; Endpoint Determination; Motor Activity/physiology; Outcome Assessment (Health Care)/methods; Spinal Muscular Atrophies of Childhood/drug therapy/physiopathology; tone and motor problems; SMA1; tool development; scale development; GMFM

Description

Spinal muscular atrophy is a genetic disease of the anterior horn cell with high morbidity rate in childhood. Certain drugs may be of benefit and are in or under consideration for Phase II trials. Outcome measures that are age appropriate and representative of disease activity remain under study. Several have not yet been validated for spinal muscular atrophy. The Gross Motor Function Measure is a measure of motor function. We showed previously that the Gross Motor Function Measure is a reliable outcome measure to assess motor function in children with spinal muscular atrophy. By collating our data from 40 spinal muscular atrophy patients, ages 5 through 17 years, we now show the validity of the Gross Motor Function Measure when compared to Quantitative Muscle Testing and ambulatory status in children with spinal muscular atrophy. The median for Gross Motor Function Measure total scores for walkers was 237 (range: 197-261) and for non-walkers, 64 (range: 4-177; P<0.0001) with no distributional overlap. We conclude that the Gross Motor Function Measure is valid and sensitive as an outcome measure for clinical trials in pediatric spinal muscular atrophy.

Rights

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Citation

Nelson L; Owens H; Hynan L S; Iannaccone S T, “The gross motor function measure is a valid and sensitive outcome measure for spinal muscular atrophy,” Pediatric Palliative Care Library, accessed May 17, 2022, https://pedpalascnetlibrary.omeka.net/items/show/16766.

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