Background
Clinical Outcome Assessments are important for measuring disease severity and evaluating the effectiveness of therapeutic interventions however are only a snapshot of a person’s physical ability and are subject to how the disease is affecting the individual on any given day. Daily activity measures are a critical unmet need to assess the effect of interventions. The aim of this study was to prospectively measure physical activity in children and adults with Charcot-Marie-Tooth disease (CMT) in their home environment and correlate with clinical outcome measures.
Methods
46 Participants (19-70 years, 80% female) were recruited for this study at annual Hereditary Neuropathy Foundation Summits. All participants completed the CMT-FOM. Daily activity was monitored continuously for 14 days at home using PAMSysTM pendant sensors (BioSensics, Newton MA). Sensor-derived metrics were compared with the CMT-FOM. One-year follow up data collection is underway.
Results
Participants ranged from mildly to severely affected as measured by the CMT-FOM (48.8 ± 13.9, range: 25 – 84). Compliance with PAMSys pendant and wrist sensors was excellent with mean non-compliance of 3.2% (46.7 minutes per day). Disease severity, measured by the CMT-FOM was significantly correlated with daily step counts (ρ=-0.50, p=0.002), cadence (steps/min) (ρ=-0.39, p=0.018) and percentage of time spent standing (ρ=-0.356, p=0.031) and walking (ρ=-0.476, p=0.003).
Conclusion
Daily activity of adults with CMT is significantly correlated with clinical outcome assessment data. Wearable sensors provide real-world evidence that reflects patients’ daily activity in their natural environments.