Duchene Muscular Dystrophy
Conditions
Keywords
Duchene Muscular Dystrophy, Tele-rehabilitation, Respiratory Function, Home-based Exercise, Caregiver Education
Brief summary
This study evaluates the feasibility and preliminary clinical effects of a home-based tele-rehabilitation program aimed at improving respiratory function in children with Duchenne Muscular Dystrophy (DMD).
Detailed description
This is a prospective single-arm interventional study evaluating a 6-week home-based tele-rehabilitation program for respiratory function in children with Duchenne Muscular Dystrophy. The intervention includes weekly remote education sessions and home-based exercises, with assessments conducted before and after the intervention and a 2-week follow-up for adherence monitoring.
Interventions
BEHAVIORALHome-Based Respiratory Telerehabilitation Program
A 6-week caregiver-assisted home-based respiratory telerehabilitation program for children with Duchenne muscular dystrophy. The program includes weekly remote education sessions, respiratory muscle exercises, airway clearance training, relaxation positioning, and self-directed home exercises performed at least 5 times per week. Outcomes are assessed before and after the intervention and at 2-week follow-up.
Sponsors
Samsung Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Children aged 3 years or older with genetically confirmed Duchenne muscular dystrophy * Participants who are able to understand the study procedures and appropriately participate in the study
Exclusion criteria
* Requirement for daytime ventilator support or invasive mechanical ventilation via tracheostomy (nighttime non-invasive ventilation such as positive airway pressure ventilation is allowed) * History of peripheral nerve injury * History of major surgery within 12 weeks prior to enrollment or expected major surgery during the study period * History of central nervous system disorders (e.g., cerebral infarction or spinal cord injury) * Severe cognitive impairment or other conditions that may interfere with participation in the study procedures
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility of the Home-Based Respiratory Tele-rehabilitation Program | Baseline to immediately after the 6-week intervention | Feasibility of the home-based respiratory telerehabilitation program will be assessed using exercise adherence rate, session attendance rate, and exercise log completion during the 6-week intervention period. These measures will be summarized descriptively as indicators of program feasibility. Higher percentages indicate better feasibility. |
| Acceptability of the Home-Based Respiratory Tele-rehabilitation Program | Immediately after the 6-week intervention and at 2-week follow-up | Acceptability of the home-based respiratory telerehabilitation program will be assessed using caregiver-reported satisfaction, perceived exercise difficulty, caregiver burden, and child participation questionnaires during the intervention period. Higher satisfaction and participation scores indicate greater acceptability, whereas higher perceived difficulty and caregiver burden scores indicate lower acceptability. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Inspiratory Muscle Strength | Baseline and immediately after the 6-week intervention | Inspiratory muscle strength will be assessed using maximal inspiratory pressure (MIP). Measurements will be recorded in centimeters of water pressure (cmH2O). Higher values indicate greater inspiratory muscle strength. |
| Expiratory Muscle Strength | Baseline and immediately after the 6-week intervention | Expiratory muscle strength will be assessed using maximal expiratory pressure (MEP). Measurements will be recorded in centimeters of water pressure (cmH2O). Higher values indicate greater expiratory muscle strength. |
| Respiratory Muscle Function | Baseline and immediately after the 6-week intervention | Respiratory muscle function will be assessed using inspiratory muscle endurance time, surface electromyography (sEMG) of the sternocleidomastoid muscle, and respiratory muscle ultrasonography. Inspiratory muscle endurance will be measured in seconds, and respiratory muscle thickness will be measured in millimeters (mm). Higher endurance time and greater muscle thickness indicate improved respiratory muscle function. |
| Physical Functional Performance | Baseline and immediately after the 6-week intervention | Physical functional performance will be assessed using the Vignos Scale, Brooke Upper Extremity Functional Rating Scale, and North Star Ambulatory Assessment (NSAA). The Vignos Scale ranges from 1 to 10, with higher scores indicating greater lower extremity disability. The Brooke Scale ranges from 1 to 6, with higher scores indicating greater upper extremity functional limitation. The NSAA ranges from 0 to 34, with higher scores indicating better motor function. |
| Functional Exercise Capacity | Baseline and immediately after the 6-week intervention | Functional exercise capacity will be assessed using the 6-Minute Walk Test (6MWT). Total walking distance will be recorded in meters. Greater walking distance indicates better functional exercise capacity. |
Countries
South Korea
Outcome results
None listed