Year: 2026
Type: article
Abstract: ABSTRACT 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 SARA home , a video‐based, self‐administered tool for high‐frequency, remote ataxia assessment. Methods We assessed feasibility and validity of SARA home in 65 SCA3 patients from seven centers. Participants recorded SARA home 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 SARA home 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, SARA home showed higher sensitivity to change than conventional SARA (SRM: 0.67 vs. 0.37). Interpretation SARA home 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.
DOI: 10.1002/acn3.70316
Authors: Marcus Grobe‐Einsler, Vivian Maas, Arian Taheri Amin, Jennifer Faber, Tamara Schaprian, Katharina Hill, Matthis Synofzik, Lisa H. Graf, Heike Jacobi, Manuela Lima, Ana F. Ferreira, Bart P. van de Warrenburg, Ilse H. J. Willemse, Dagmar Timmann, Andreas Thieme, Paola Giunti, Hector Garcia‐Moreno, Magda M. Santana, Jeannette Hübener‐Schmid, Elin H. Davies, Thomas Klockgether
Institutions: University Hospital Bonn, German Center for Neurodegenerative Diseases, Heinrich Heine University Düsseldorf, Hertie Institute for Clinical Brain Research, University of Tübingen, University Hospital Heidelberg, Universidade dos Açores, Universidade do Porto, Instituto de Ciências Biomédicas Albel Salazar, Radboud University Nijmegen, Radboud University Medical Center, Essen University Hospital, University of Duisburg-Essen, UCL Queen Square Institute of Neurology, University College London, Centro de Neurociências e Biologia Celular, Centro de Inovação em Biomedicina e Biotecnologia, University of Coimbra, Aparito Limited (United Kingdom)
Language: English
Subfield: Cellular and Molecular Neuroscience
Field: Neuroscience
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $4,100
OpenAlex ID: W7125396993