Application of Wearable Sensors to Assess Disease Severity in Adults with Charcot-Marie-Tooth Disease (S16.007)

To evaluate the feasibility of remote data collection of physical activity and instrumented balance measures in adults with Charcot-Marie-Tooth (CMT).

Reliable, valid, and sensitive clinical outcome assessments (COAs) are essential to monitor disease progression and measure therapeutic interventions. Traditional COAs capture a single day’s performance, which may not reflect a person’s daily abilities. Wearable sensors offer continuous monitoring in a home environment, providing real-world evidence of function and more sensitive assessments of gait and balance.

This cross-sectional cohort study was performed at the Hereditary Neuropathy Foundation CMT Clinical Trial Readiness Summit. Participants completed an in-person assessment (CMT-FOM and instrumented gait and balance assessments using LEGSys™ and BalanSens™ (BioSensics, Newton MA)). Additionally, physical activity was monitored continuously for 14 days at home using PAMSys™ pendant sensor.

26 Participants (19–70yrs, 81% female, 58% CMT1A) participated in this study. Participants ranged from mildly to severely affected as measured by the CMT-FOM (53.2 ± 8.0, range: 37 – 73). Compliance with PAMSys for at home monitoring was excellent with mean non-compliance of 22 minutes per day and 16 patients with 100% compliance. Disease severity, measured by the CMT-FOM, was significantly correlated with daily step count in the community (ρ=−0.608, p=0.001), average cadence (ρ=−0.579, p=0.002) and centre of mass ranges measured by BalanSens (ρ>0.4, p<0.05).

The wearable pendant sensor, due to its ease of use, non-invasive nature, and long battery life, resulted in high patient compliance. These wearables provided real-world evidence by continuously capturing long-term data that accurately reflects patients’ natural environments and daily activities. Sensor-derived metrics were validated against established COAs. In future trials, the sensitivity of these measures to change should be further explored in longitudinal studies.

Disclosure: The institution of Dr. Cornett has received research support from Hereditary Neuropathy Foundation . Ram Kinker Mishra has received personal compensation for serving as an employee of BioSensics LLC. Mrs. CLEM has nothing to disclose. Mr. Estilow has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Applied Therapeutics . Mrs. Aldrich has received personal compensation for serving as an employee of Hereditary Neuropathy Foundation. Mrs. Hollett has nothing to disclose. Mrs. Moore has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD Pharma . Mrs. Moore has received personal compensation in the range of $500-$4,999 for serving as a Consultant for HNF. Mrs. Moore has stock in Miralinc Pharma. Dr. Vaziri has received personal compensation for serving as an employee of Biosensics.