Physical Activity a Useful End Point for Charcot-Marie-Tooth Disease Trials

Data from a recent study suggest that physical activity measured by wearable devices is associated with in-person measures of strength and function in Charcot-Marie-Tooth Disease Type 1A (CMT1A) and may be useful as an end point in clinical trials.

Researchers from the University of Rochester found that physical activity as measured by the number of steps was significantly correlated with the CMT functional outcome measure (CMT-FOM; ρ = -0.71; P = .003) and overall lower extremity strength (ρ = 0.54; P = .04).

These findings were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference 2021, March 15-18, by Katy J. Eichinger, PhD, DPT, assistant professor, department of neurology, University of Rochester Medical Center.

“Physical activity has been reported to be reduced in individuals with CMT1A and as a measure of function, it may serve as an important endpoint for clinical trials. Physical activity has been measured using many different wearable devices that produce variables such as the time spent in various positions as well as the number of steps taken per day,” Eichinger and colleagues wrote.

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Eichinger and colleagues analyzed data from 15 participants in order to examine physical activity and the relationships to measures of function and disease severity in individuals with CMT1A. The patients were 67% female (n = 10) with a mean age of 37.3 years (range, 18-64) and a mean CMT Exam Score (CMTES) of 8.5 (range, 3-17).

The participants wore BioStamp MC-10 sensors applied with adhesives at the chest, thigh, and lower leg during in-person mobility assessments and the following 24 hours. The sensors recorded data on time spent resting, moving, and sleeping, as well as number of steps taken. Researchers then compared these physical activity variables to the CMT-FOM and measures of disease severity such as strength and CMTES.

Eichinger and colleagues found that participants spent a mean of 150 minutes per day moving and a mean of 781 minutes per day resting. They took an average of 4416 steps per day. The number of steps taken per day was found to be significantly correlated with the CMT-FOM (ρ = -0.71; P = .003) and overall lower extremity strength (ρ = 0.54; P = .04).

“Physical activity, as measured by the number of steps taken, using adhesive wearable devices was associated with in-person measures of strength and function. Future studies of longer duration are needed to further examine physical activity as an endpoint for future clinical trials,” Eichinger and colleagues concluded.

NeurologyLive previously spoke with K. Florian P. Thomas, MD, MA, PhD, MS, Director, Multiple Sclerosis and Hereditary Neuropathy Centers, Seton Hall-Hackensack Meridian School of Medicine, about the state of CMT at the time, a conversation during which he noted the importance of devices in CMT care and their benefits.

“I think it's important to realize that a lot can be done in terms of rehabilitative treatment of both arm and leg weakness, specifically with ankle braces which can correct foot drop and allow people to walk much more normally than they would without those ankle braces,” Thomas told NeurologyLive.

“There are all kinds of tools that occupational therapists are familiar with that can help with daily activities. It's important to provide patients with appropriate consultations so they can benefit from these devices,” he said.

For more coverage of MDA 2021, click here.

REFERENCE

Echinger K, Baker L, Behrens-Spraggins S, et al. A pilot study using a novel wearable sensor to examine physical activity in individuals with Charcot Marie Tooth Disease 1A (CMT1A). Presented at: MDA Clinical and Scientific Conference 2021; March 15-18.