Aparito Limited, Unit 11 Gwenfro, Wrexham Technology Park, Wrexham, Wales, LL13 7YP, UK.
Office of the Clinical Director, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Orphanet J Rare Dis. 2020 Aug 3;15(1):199. doi: 10.1186/s13023-020-01473-x.
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.
Adherence 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.
The 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.
作为晚发性 GM2 神经节苷脂贮积症自然史研究的一部分，利用数字健康技术在医院就诊之间远程监测一组患者。这种方法旨在捕捉连续数据，避免仅关注传统研究设计中捕捉的偶发性数据。强调实时捕捉症状和移动患者报告的结果（mPROs），以确定对患者自身重要的疾病影响；这种影响可能并不总是与患者就诊期间评估的测量临床结果相关。这得到了可穿戴设备的被动、连续数据捕获的支持。
佩戴设备和完成 mPROs 的依从率分别为 84%和 91%，从而产生了丰富的多维数据集。正如在进展缓慢的疾病中进行的为期六个月的概念验证研究所预期的那样，在临床、mPROs 或可穿戴设备数据中未观察到或观察到统计学上显著的变化。
该研究表明，患者非常热衷于使用该技术，并且表现出出色的依从性，这证明了他们的积极性和积极性。mPROs 和可穿戴设备的组合生成了功能丰富的数据集，这可能是一种有用且可行的方法，可以远程实时了解疾病负担。
