Annals of clinical and translational neurology · 2026
Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points.
СтатьяМногоцентровое исследованиеНаблюдательное исследованиеНазвание и аннотация: Перевод в очереди
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Grobe-Einsler Marcus, Maas Vivian, Amin Arian Taheri, Faber Jennifer, Schaprian Tamara, Hill Katharina, Synofzik Matthis, Graf Lisa H, Jacobi Heike, Lima Manuela, Ferreira Ana F, van de Warrenburg Bart P, Willemse Ilse H J, Timmann Dagmar, Thieme Andreas, Giunti Paola, Garcia-Moreno Hector, Santana Magda M, Hübener-Schmid Jeannette, Davies Elin H, Klockgether Thomas
Аннотация
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.
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.