TY  - JOUR
AV  - public
JF  - Movement Disorders
EP  - 12
N1  - This work is licensed under a Creative Commons Attribution 4.0 International License. The images
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ID  - discovery10152453
PB  - WILEY
A1  - Maas, Roderick PPWM
A1  - Teerenstra, Steven
A1  - Lima, Manuela
A1  - Pires, Paula
A1  - Pereira de Almeida, Luis
A1  - van Gaalen, Judith
A1  - Timmann, Dagmar
A1  - Infante, Jon
A1  - Onyike, Chiadi
A1  - Bushara, Khalaf
A1  - Jacobi, Heike
A1  - Reetz, Kathrin
A1  - Santana, Magda M
A1  - Afonso Ribeiro, Joana
A1  - Huebener-Schmid, Jeannette
A1  - de Vries, Jeroen J
A1  - Synofzik, Matthis
A1  - Schoels, Ludger
A1  - Garcia-Moreno, Hector
A1  - Giunti, Paola
A1  - Faber, Jennifer
A1  - Klockgether, Thomas
A1  - van de Warrenburg, Bart PC
Y1  - 2022/07/08/
N2  - Background: Disease severity in spinocerebellar ataxia type 3 (SCA3) is commonly defined by the Scale for the Assessment and Rating of Ataxia (SARA) sum score, but little is known about the contributions and progression patterns of individual items. Objectives: To investigate the temporal dynamics of SARA item scores in SCA3 patients and evaluate if clinical and demographic factors are differentially associated with evolution of axial and appendicular ataxia. Methods: In a prospective, multinational cohort study involving 11 European and 2 US sites, SARA scores were determined longitudinally in 223 SCA3 patients with a follow-up assessment after 1 year. Results: An increase in SARA score from 10 to 20 points was mainly driven by axial and speech items, with a markedly smaller contribution of appendicular items. Finger chase and nose-finger test scores not only showed the lowest variability at baseline, but also the least deterioration at follow-up. Compared with the full set of SARA items, omission of both tests would result in lower sample size requirements for therapeutic trials. Sex was associated with change in SARA sum score and appendicular, but not axial, subscore, with a significantly faster progression in men. Despite considerable interindividual variability, the average annual progression rate of SARA score was approximately three times higher in subjects with a disease duration over 10 years than in those within 10 years from onset. Conclusion: Our findings provide evidence for a difference in temporal dynamics between axial and appendicular ataxia in SCA3 patients, which will help inform the design of clinical trials and development of new (etiology-specific) outcome measures. © 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
KW  - Science & Technology
KW  -  Life Sciences & Biomedicine
KW  -  Clinical Neurology
KW  -  Neurosciences & Neurology
KW  -  spinocerebellar ataxia type 3
KW  -  natural history
KW  -  Scale for the Assessment and Rating of Ataxia
KW  -  disease progression
KW  -  DISEASE PROGRESSION
KW  -  CEREBELLAR-ATAXIA
KW  -  NATURAL-HISTORY
KW  -  SPINOCEREBELLAR
TI  - Differential Temporal Dynamics of Axial and Appendicular Ataxia in SCA3
UR  - https://doi.org/10.1002/mds.29135
ER  -