Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond

NCT ID: NCT03567460

Last Updated: 2020-07-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-01
• Study Completion Date: 2019-12-31

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.

Conditions

Marfan Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

10,000 Steps/day for Pediatric Marfan Patients

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.

Group Type: EXPERIMENTAL

Exercise

Intervention Type: 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.

Interventions

Exercise

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.

Intervention Type: BEHAVIORAL

Eligibility Criteria

Inclusion Criteria

1. 10-19 years of age,

2. MFS by revised Ghent criteria,

3. Cardiac clearance to exercise by the primary cardiologist.

Exclusion Criteria

1. Ventricular dysfunction,

2. Prior history of aortic surgery.

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Midwestern University

OTHER

Sponsor Role: collaborator

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Seda Tierney

Assistant Professor in Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Seda Tierney, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford Children's Health

Palo Alto, California, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 36453629 (View on PubMed)

Other Identifiers

37176

Identifier Type: -

Identifier Source: org_study_id