Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Stanford University
- Enrollment
- 24
- Locations
- 2
- Primary Endpoint
- To determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients.
Study Overview
Brief Summary
Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.
There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.
For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health in adolescent Marfan patients.
Detailed Description
Marfan syndrome is an inherited disorder of the connective tissue, which provides material and support for the skeleton, muscles, and blood vessels. Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta). The wall of the aorta weakens and dilates which can rupture, leading to death, and sometimes during intense exercise.
Typically, the weakening process starts when elastin fibers in the aorta become fragmented. There is some evidence in Marfan patients that a stiffer aorta increases risk for rupture.
For some time, clinical care has focused on what type of exercise these patients should avoid due to risk for aortic dissection. Little clinical emphasis has been placed on encouraging patients to engage in routine and safe exercise such as walking. These young patients also frequently choose sedentary lifestyles, most likely due to limitations imposed by parents as well as adolescents' own perceptions of what is safe for them and their physical capabilities. Another complicating factor is that these patients often experience difficulty coping with their diagnosis. Informed by this evidence, the investigators propose to collaboratively investigate whether regular exercise improves aortic health and coping skills in adolescent Marfan patients.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Prevention
- Masking
- None
Eligibility Criteria
- Ages
- 10 Years to 19 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •10-19 years of age,
- •MFS by revised Ghent criteria,
- •Cardiac clearance to exercise by the primary cardiologist.
Exclusion Criteria
- •Ventricular dysfunction,
- •Prior history of aortic surgery.
Outcomes
Primary Outcomes
To determine if 6 months of regular physical activity improves aortic stiffness in MFS patients. exercise, a decrease in expressed biomarkers, and an increase in COPE scores of MFS patients.
Time Frame: 6 months of intervention
The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity).
Secondary Outcomes
- To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice.(6 months of intervention)
- To determine if 6 months of regular physical activity improves coping skills in Marfan patients.(6 months of intervention)
- To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients.(6 months of intervention)
Investigators
Seda Tierney
Assistant Professor in Pediatrics
Stanford University