Abstract
BackgroundAs part of a late onset GM2 gangliosidosis natural history study, digital health technology was utilized to monitor a group of patients remotely between hospital visits. This approach was explored as a means of capturing continuous data and moving away from focusing only on episodic data captured in traditional study designs. A strong emphasis was placed on real-time capture of symptoms and mobile Patient Reported Outcomes (mPROs) to identify the disease impact important to the patients themselves; an impact that may not always correlate with the measured clinical outcomes assessed during patient visits. This was supported by passive, continuous data capture from a wearable device.ResultsAdherence rate for wearing the device and completing the mPROs was 84 and 91%, respectively, resulting in a rich multidimensional dataset. As expected for a six-month proof-of-concept study in a disease that progresses slowly, statistically significant changes were not expected or observed in the clinical, mPROs, or wearable device data.ConclusionsThe study demonstrated that patients were very enthusiastic and motivated to engage with the technology as demonstrated by excellent compliance. The combination of mPROs and wearables generates feature-rich datasets that could be a useful and feasible way to capture remote, real-time insight into disease burden.
Highlights
The GM2 gangliosidoses, Tay-Sachs (TSD) and Sandhoff (SD) diseases, are neurodegenerative disorders, caused by a deficiency of the lysosomal enzyme beta- hexosaminidase A (Hex A)
In contrast to infantile Tay-Sachs disease (TSD) or SD disease the late-onset forms have symptom onset in adolescence or early adulthood, with ataxia, selective and progressive muscular atrophy leading to increased falls and difficulty rising from a chair or the floor, and for TSD patients, dysarthria
A “very good” overall impression was reported by two out of eight patients, one reported their overall impression as “good”, and four as “ok”. This feedback was instrumental in the redevelopment of the app and the introduction of a new wearable. This feasibility study demonstrated that utilizing mHealth with wearable technology was well accepted by patients over a six-month natural history study
Summary
The GM2 gangliosidoses, Tay-Sachs (TSD) and Sandhoff (SD) diseases, are neurodegenerative disorders, caused by a deficiency of the lysosomal enzyme beta- hexosaminidase A (Hex A). As part of a late onset GM2 gangliosidosis natural history study, digital health technology was utilized to monitor a group of patients remotely between hospital visits. This approach was explored as a means of capturing continuous data and moving away from focusing only on episodic data captured in traditional study designs. A strong emphasis was placed on real-time capture of symptoms and mobile Patient Reported Outcomes (mPROs) to identify the disease impact important to the patients themselves; an impact that may not always correlate with the measured clinical outcomes assessed during patient visits This was supported by passive, continuous data capture from a wearable device
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