Duchenne Muscular Dystrophy
Conditions
Keywords
Walking, Muscle Strength, Energy Cost, Quality of Life
Brief summary
The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.
Detailed description
Duchenne muscular dystrophy (DMD) is an X-linked recessive disease of muscle characterized by a progressive loss of functional muscle mass, which is replaced with fibrofatty tissue. Historically, boys with DMD lose the ability to walk between the ages of 8-12 years, due to progressive weakness of the quadriceps coupled with the development of contractures at the hip, knee and ankle. This progressive loss in function necessitates individuals with DMD to spend less time walking and more time in wheelchairs, leading to the development of spinal deformities. Recently, corticosteroids have been shown to reduce the expected loss of muscle strength, extend the time that ambulation and standing are maintained, and minimize or eliminate spinal deformity in individuals with DMD; yet, the side effects of such treatment preclude use in some patients. To date, differences in gait patterns and other markers of disease progression between boys on corticosteroids and those not utilizing such treatment have not been objectively quantified. This lack of knowledge is a major obstacle to determining the most effective treatment for subsets of boys with DMD.
Interventions
None listed
Sponsors
Shriners Hospitals for Children
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
4 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of DMD * Male. * Four years of age or older. * Ability to walk independently for five minutes to 10 minutes at self-selected speed. * Ability to cognitively understand directions for testing procedures.
Exclusion criteria
* Female * Nonambulatory
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Gait pattern | every six months (2x/year) | computerized assessment of walking |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| muscle strength | every six months (2x/year) | quantitative assessment of strength with a Biodex |
| energy cost of walking | every six months (2x/year) | assessment of how much energy it takes to walk, assessed with a Cosmed K4b2 |
| gross motor functional skills | every six months (2x/year) | assessment of gross motor skills, ie getting up off the floor, ascending/descending stairs |
| Step activity Monitor-participation | one week every six months | measurement of the number of steps taken in the community/home environment during weekdays and weekends |
Countries
United States
Outcome results
None listed