Clinical trial NCT03581682: Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?
Official title: Tele-Clinic Visits in Pediatric Marfan Patients Using Parental Echo: The Future?
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Marfan Syndrome
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2018-07-10
Enrollment: 15
Sex: ALL
Minimum age (years): 5
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

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

Primary outcomes:
measure: Percent of tele-visits with a clinical visit match-score of ≥13/15 points
timeFrame: 3-6 months
Secondary outcomes:
measure: To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
timeFrame: 3-6 months
measure: To test if a tele-visit using parental home echo costs less than an on-site clinic visit.
timeFrame: 3-6 months
measure: To determine if this intervention increases parental sense of empowerment.
timeFrame: 3-6 months
measure: To determine if this intervention increases parental sense of empowerment.
timeFrame: 3-6 months
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital
state: California
Study officials:
affiliation: Stanford University
name: Seda Tierney, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.