Dataset Information

Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.

ABSTRACT:

Purpose

This single-arm prospective cohort study aimed to evaluate the feasibility and utility of in-home body weight support harness system (BWSS) use in children treated for spinal muscular atrophy (SMA).

Methods

Individuals with 2 or 3 copies of SMN2 who received pharmacotherapeutic treatment, had head control, and weight <50lbs were enrolled. Families were provided a BWSS and documented use. Motor outcome assessments were completed at baseline, month 3 and month 6. Families provided feedback in an end of study survey.

Results

All 32 participants (2.9 (SD 1.9) yrs), improved or remained stable on all outcomes. Average reported frequency of use was 4.1(2.3) hrs/week. Controlling for other covariates, frequency of use explained over 70% of the variability in change scores. Family feedback was overwhelmingly positive.

Conclusion

Use of in-home BWSS is a safe, feasible and useful option to increase exercise dosage after treatment in SMA and may help optimize motor abilities.

Trial registration

Study registered with: Clinicaltrials.gov Clinicaltrials.gov identifier: NCT05715749.

SUBMITTER: Iammarino MA

PROVIDER: S-EPMC10950233 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature

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Publications

Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.

Iammarino Megan A MA Alfano Lindsay N LN Reash Natalie F NF Sabo Brenna B Conroy Sara S Noritz Garey G Wendland Madalynn M Lowes Linda P LP

PloS one 20240319 3

<h4>Purpose</h4>This single-arm prospective cohort study aimed to evaluate the feasibility and utility of in-home body weight support harness system (BWSS) use in children treated for spinal muscular atrophy (SMA).<h4>Methods</h4>Individuals with 2 or 3 copies of SMN2 who received pharmacotherapeutic treatment, had head control, and weight <50lbs were enrolled. Families were provided a BWSS and documented use. Motor outcome assessments were completed at baseline, month 3 and month 6. Families pr ...[more]

PMID: 38502672

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Project description:BackgroundNeuromuscular disorders (NMD) commonly affect the upper extremity. Due to muscle weakness, performance of daily activities becomes increasingly difficult, which leads to reduced independence and quality of life. In order to support the performance of upper extremity tasks, dynamic arm supports may be used. The Yumen Arm is a novel dynamic arm support specially developed for people with NMD. The aim of this study is to evaluate the feasibility and effectiveness of the Yumen Arm in persons with Duchenne Muscular Dystrophy (DMD) and persons with Spinal Muscular Atrophy (SMA).MethodsThree persons with DMD and three persons with SMA participated in this study. All participants conducted a set of measures with and without the Yumen Arm. Outcome measures were: active range of motion of the arm and trunk (i.e. Reachable Workspace, Functional Workspace, and trunk movement), fatigue (OMNI-RPE), Performance of Upper Limb (PUL) scale and some additional activities of daily living. User experiences were collected using a questionnaire.ResultsThe Yumen Arm could be used by all participants. Results showed a median increase in active range of motion (4% relative surface area), and a median increase of function ability (> 11% PUL score) when using the Yumen Arm. In addition, three out of four (data from 2 participants was missing) participants indicated that activity performance was less fatiguing when using the Yumen Arm. Four out of five (data from 1 participant was missing) participants indicated that they would like to use the Yumen Arm in their daily lives.ConclusionThis study is one of the first studies describing a range of objective measures to examine the effectiveness of a dynamic arm support. Based on these measurements we can conclude that the Yumen Arm effectively improves arm function in NMD patients, however the effectiveness varies a lot between individual subjects. We provided detailed recommendations for the improvement of the Yumen Arm, and possible also for the development of other dynamic arm supports. This study showed a lot of variability between individual subjects, which emphasizes the importance of tuning dynamic arm supports based on individual user characteristics, such as scoliosis, functional capacity and muscle strength.

Dataset Information

Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.

Purpose

Methods

Results

Conclusion

Trial registration

Publications

Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.

Similar Datasets

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