Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?
Condition: Marfan Syndrome
MeSH terms: Marfan Syndrome; Arachnodactyly
Study type: Interventional

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.
    

Intervention name(s): Tele-Clinic Visits Using Parent-Acquired Echos
Intervention type(s): Other
Intervention description(s): 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 model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Other