Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?

NCT ID: NCT03581682

Last Updated: 2021-03-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-07-01
• Study Completion Date: 2020-08-01

Brief Summary

Marfan syndrome (MFS), a connective tissue disorder seen in 1 in 3,000 individuals, causes progressive aortic root dilation that can result in aortic dissection and sudden death. Clinical care focuses on monitoring the aortic root by serial echocardiography (echo) to guide medical treatment and elective aortic root surgery in a specialized clinic every 6-12 months. This monitoring protocol, coupled with surgical intervention, has doubled the median life expectancy which was previously only 32 years. However, this surveillance carries significant health care costs at >$50 million dollars/year on echos alone (at $3-4K each) in children and adolescents in the US, as well as substantial burden on families residing far from specialized centers. A clinic visit delivered to MFS patients via live-video conferencing at home (tele-visit) could shift this paradigm, if a home echo could be obtained.

Here, the investigator will train parents of Pediatric Marfan patients to take echo images using a hand held device, height, weight, blood pressure, medical history, and listen to the heart of their child. Then, the investigators will ask them to take the equipment home and collect the same data at home during a tele-clinic visit, with further instruction by the study team through secure live-video conferencing.

Detailed Description

In the proposed intervention, every patient (n=60) will have a tele-visit and an on-site clinic visit 1 day apart. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that will be used during the tele-visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo (transferred via Internet for remote interpretation), all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other. Two independent echo readers will analyze home and clinic echos to measure reproducibility.

Conditions

Marfan Syndrome

Study Design

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

Study Groups

Tele-Visit Using Parental Home Echo

All parents will have hands-on echo training. We will test if a tele-visit using parental home echo is clinically reliable compared to an on-site clinic visit, costs less, and improves parental sense of empowerment.

Group Type: EXPERIMENTAL

Tele-Clinic Visits Using Parent-Acquired Echos

Intervention Type: OTHER

Every patient will have a tele-visit and an on-site clinic visit 1 day apart 3-6 months after the training session. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that they will use during the tele-visit. The parents will also have an in-service on how to take weight, height, and blood pressure measurements, and how to use the digital stethoscope.

A tele-visit will be schedule a day prior to the patient's regularly scheduled clinic visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo, all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other.

Interventions

Tele-Clinic Visits Using Parent-Acquired Echos

Every patient will have a tele-visit and an on-site clinic visit 1 day apart 3-6 months after the training session. Parents will have a 1-hour hands-on training session to acquire basic echo images on their children with the same hand-held device that they will use during the tele-visit. The parents will also have an in-service on how to take weight, height, and blood pressure measurements, and how to use the digital stethoscope.

A tele-visit will be schedule a day prior to the patient's regularly scheduled clinic visit. Tele-visit elements will include interim medical history by the parent and patient, and weight, height, vital signs, digital cardiac auscultation, and home echo, all obtained by the parent. Two MFS physicians, following our routine MFS care protocol, will administer either the tele-visit or on-site clinic visit, masked to the findings of the other.

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• 5-19 years of age (patient)
• seen in at least 2 prior clinic visits
• Marfan syndrome by revised Ghent criteria
• presence of parent at home

Exclusion Criteria

• prior aortic surgery
• known cardiomyopathy
• known arrhythmia
• aortic root > 4.5 cm in prior clinic visit
• pregnancy

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Seda Tierney

Associate Professor of Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Seda Tierney, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Lucile Packard Children's Hospital

Palo Alto, California, United States

Countries

United States

References

Chen A, Punn R, Collins RT, Chen JH, Stauffer KJ, Wang R, Alexander S, MacMillen Lechich K, Murphy DJ, Chung S, Selamet Tierney ES. Tele-Clinic Visits in Pediatric Patients with Marfan Syndrome Using Parentally Acquired Echocardiography. J Pediatr. 2021 May;232:140-146. doi: 10.1016/j.jpeds.2021.01.004. Epub 2021 Jan 13.

Reference Type: DERIVED

PMID: 33453199 (View on PubMed)

Other Identifiers

42159

Identifier Type: -

Identifier Source: org_study_id