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| Name | Class |
|---|---|
| Midwestern University | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 10,000 Steps/day for Pediatric Marfan Patients | Experimental | Participants will be given a Garmin VivoFit and asked to take at least 10,000 steps per day. A study coordinator will reach out at least once per week to check in on progress made and help make weekly goals. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Exercise | Behavioral | Across studies in adolescents in general population, 60 minutes of recommended daily exercise level is achieved, on average, within a total volume of 10,000-11,700 steps. The investigators will assess baseline physical activity quantitatively by using an accelerometer worn on the wrist during waking hours over a 7-day period. Each participant will be given a Garmin device to place on their wrist that will track their steps. Then patients will be asked to complete 10,000 steps daily for 6 months (acclimation over a week), which will be encouraged by daily text messages or e-mail, and weekly phone calls by the intervention team in addition to a Garmin and Facebook peer group. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | The investigators' primary outcome measure is aortic stiffness measured by arterial tonometry (pulse wave velocity). | 6 months of intervention |
| Measure | Description | Time Frame |
|---|---|---|
| To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients. | The secondary outcomes include aortic stiffness at the aortic root at the level of the sinuses of Valsalva and ascending aorta. | 6 months of intervention |
| To determine if 6 months of regular physical activity improves aortic stiffness and the biomarker profile in MFS patients. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Seda Tierney, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Children's Health | Palo Alto | California | 94306 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36453629 | Derived | Selamet Tierney ES, Chung S, Stauffer KJ, Brabender J, Collins RT 2nd, Folk R, Li W, Murthy AK, Murphy DJ, Esfandiarei M. Can 10 000 Healthy Steps a Day Slow Aortic Root Dilation in Pediatric Patients With Marfan Syndrome? J Am Heart Assoc. 2022 Dec 6;11(23):e027598. doi: 10.1161/JAHA.122.027598. Epub 2022 Dec 1. |
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| ID | Term |
|---|---|
| D008382 | Marfan Syndrome |
| ID | Term |
|---|---|
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D006330 | Heart Defects, Congenital |
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| ID | Term |
|---|---|
| D015444 | Exercise |
| ID | Term |
|---|---|
| D009043 | Motor Activity |
| D009068 | Movement |
| D009142 | Musculoskeletal Physiological Phenomena |
| D055687 | Musculoskeletal and Neural Physiological Phenomena |
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Secondary outcomes will be expression levels of TGF-β, Ang-II, MMP-2 & -9, ROS levels. |
| 6 months of intervention |
| To determine if 6 months of regular physical activity decreases aortic stiffness and rate of aortic root dilation in Marfan mice. | Secondary outcomes will be expression levels of TGF-β, MMP-2, MMP-9, and ROS. | 6 months of intervention |
| To determine if 6 months of regular physical activity improves coping skills in Marfan patients. | The outcome measure is the COPE inventory score. | 6 months of intervention |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |