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

NCT03581682 | Completed

• 1 other identifier: 42159
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Marfan Syndrome

Outcome Measures

Primary Outcomes (1)

• Percent of tele-visits with a clinical visit match-score of ≥13/15 points

  This scoring system was determined after surveying pediatric MFS physicians nationwide and includes these sections: Echo, vital signs, height and weight, interim medical history, cardiac auscultation, and determination of follow up time and medication dose/type.

  3-6 months

Secondary Outcomes (4)

• To test if a tele-visit using parental home echo costs less than an on-site clinic visit.

  3-6 months

• To test if a tele-visit using parental home echo costs less than an on-site clinic visit.

  3-6 months

• To determine if this intervention increases parental sense of empowerment.

  3-6 months

• To determine if this intervention increases parental sense of empowerment.

  3-6 months

Study Arms (1)

Tele-Visit Using Parental Home Echo

EXPERIMENTAL

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.

Other: Tele-Clinic Visits Using Parent-Acquired Echos

Interventions

Tele-Clinic Visits Using Parent-Acquired Echos 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.

Tele-Visit Using Parental Home Echo

Eligibility Criteria

• Age: 5 Years - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Stanford University: lead

Study Sites (1)

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

Location

Related Publications (1)

• 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.

  PMID: 33453199 DERIVED

MeSH Terms

Conditions

Marfan Syndrome

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Officials

• Seda Tierney, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Pediatrics

Study Record Dates

• First Submitted: June 18, 2018
• First Posted: July 10, 2018
• Study Start: July 1, 2018
• Primary Completion: August 1, 2020
• Study Completion: August 1, 2020
• Last Updated: March 17, 2021

Record last verified: 2021-03

Locations

US (1)