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Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points
Marcus Grobe-Einsler1,2,3, Vivian Maas1,2, Arian Taheri Amin1,2,4
1German Center for Neurodgenerative Diseases (DZNE), Bonn, Germany.
Objective:
Spinocerebellar ataxia type 3 (SCA3) is a genetically defined ataxia. The Scale for Assessment and Rating of Ataxia (SARA) is a clinician-reported outcome that measures ataxia severity at a single time point. In its standard application, SARA fails to capture short-term fluctuations, limiting its sensitivity in trials. To overcome this, we employed SARAhome, a video-based, self-administered tool for high-frequency, remote ataxia assessment.
Methods:
We assessed feasibility and validity of SARAhome in 65 SCA3 patients from seven centers. Participants recorded SARAhome twice daily for 14 days using a mobile e-health app. We analyzed adherence, intraindividual fluctuations and their predictors, and evaluated sensitivity to change in a longitudinal substudy of 11 patients.
Results:
Adherence to the study protocol was generally high (80.2%) with valid scores in 79.2% of 1459 recordings. Maximum adherence occurred over a 4-day period (84.8%). Fluctuations ranged 3.0 points between lowest and highest scores (IQR: 2.5-4.5) and 1.0 point based on score IQRs (IQR: 0.5-1.5), corresponding to 10.7% and 3.6% of the maximal SARAhome score. Fluctuations showed rough agreement with patient global impression. Greater disease severity and longer CAG repeats were associated with smaller relative fluctuations. Over a median follow-up of 411 days, SARAhome showed higher sensitivity to change than conventional SARA (SRM: 0.67 vs. 0.37).
Interpretation:
SARAhome is a feasible, innovative video-based tool for remote, high-frequency monitoring of ataxia severity. A 4-day recording effectively captures relevant fluctuations and enhances sensitivity to change, supporting its use in future SCA3 trials.
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