Wearable Device Use for Heart Rate Variability Measurement for Objective Assessment of Hyperphagia

Funding Summary

Directly measured, objective outcomes are needed for Prader-Willi Syndrome (PWS) clinical trials for accurate assessment of treatment efficacy. We will conduct in person focus groups of primary caregivers of children between 8-17 years with PWS to review potential options for wearable devices and options for daily diaries/data entry, and use this data to study a wearable device within the natural patient environment.  The first and second phases of the study will provide critical information about the feasibility of using emerging technology to assess heart rate variability (HRV) as an objective marker of behaviors in PWS such as outbursts, food seeking, and anxiety-related behaviors. 

Lay Abstract

Directly measured, objective outcomes are needed for Prader-Willi Syndrome (PWS) clinical trials for accurate assessment of treatment efficacy. Although met with excitement and mechanistic relevance to PWS, multiple drug studies have failed to show clinically meaningful change in trial outcomes despite anecdotal evidence of impact. To date, trials have relied on parent-reported outcomes, which summarize parents’ observance of specific behaviors. These reports are dependent on caretaker-regulated controls across environments and fail to discern the severity of the patient’s features in the context of level of support in place. Moreover, questionnaires index stable individual differences between children and may not be optimal for measuring change.

To address the need for directly measured, quantitative, objective outcomes, we will conduct in person focus groups of primary caregivers of children between 8-17 years with Prader-Willi Syndrome (English (PWSAUSA Conference) and Spanish-speaking (recruited to attend in person in Denver as satellite during FPWR conference)) to review potential options for wearable devices and options for daily diaries/data entry). We also plan to recruit high functioning English-speaking adults with PWS for virtual focus groups to elicit feedback regarding wearable device options. Wearable devices will be available for inspection during the in person focus groups with videos of the device options available during the virtual groups. Data gathered from the focus groups will be recorded and coded using 2 different coders for common themes with analyses using qualitative methods. We will evaluate comparator groups between English and Spanish groups along with virtual groups and in person groups.   We will plan to use data from multiple focus groups to prepare for the second study phase in which a wearable is studied within the natural patient environment. The first and second phases of the study will provide critical information about the feasibility of using emerging technology to assess heart rate variability (HRV) as an objective marker of behaviors in PWS such as outbursts, food seeking, and anxiety-related behaviors.

Research Outcomes: Public Summary

We have completed an in person focus group of English- speaking families of children with Prader-Willi Syndrome during the Prader-Willi Syndrome Association annual scientific conference in Orlando, FL. We have also completed a virtual focus group of high functioning adults with Prader-Willi Syndrome. Dr. Hwu's team at Baylor College of Medicine/Texas Children's Hospital is responding to IRB queries in preparation to conduct an in person focus group of Spanish- speaking families of children with Prader-Willi Syndrome in the early Spring 2025. An abstract with preliminary data from the focus groups was accepted for poster presentation during the 2024 FPWR Annual Conference in Atlanta. Another abstract has been submitted entitled "Use of Wearable Devices in PWS Clinical Trials: Formative Data to Inform a Feasibility Pilot” and accepted for poster presentation on April 10, 2025 at the 57th Gatlinburg Conference on Research and Theory in Intellectual and Developmental Disabilities. We plan to submit an abstract for presentation during the 2025 IPWSO annual meeting.