Quantitative Assessment of the Evolution of Cerebellar Signs in Spinocerebellar Ataxias

Ellis Chan, Perrine Charles, Pascale Ribai, Cyril Goizet, Cecilia Marelli, Carlo-Maria Vincitorio, Alice Le Bayon, Lucie Guyant-Maréchal, Nadia Vandenberghe, Mathieu Anheim, David Devos, Leorah Freeman, Isabelle Le Ber, Karine N'Guyen, Maya Tchikviladzé, Pierre Labauge, Didier Hannequin, Alexis Brice, Alexandra Durr, Sophie Tezenas du Montcel
Mov. Disord.. 2011-02-01; 26(3): 534-538
DOI: 10.1002/MDS.23531

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1. Mov Disord. 2011 Feb 15;26(3):534-8. doi: 10.1002/mds.23531. Epub 2011 Feb 1.

Quantitative assessment of the evolution of cerebellar signs in spinocerebellar

Chan E(1), Charles P, Ribai P, Goizet C, Marelli C, Vincitorio CM, Le Bayon A,
Guyant-Maréchal L, Vandenberghe N, Anheim M, Devos D, Freeman L, Le Ber I,
N’Guyen K, Tchikviladzé M, Labauge P, Hannequin D, Brice A, Durr A, du Montcel

Author information:
(1)Department of Biostatistics and Medical Informatics and Pitié-Salpêtrière
Charles-Foix Clinical Research Unit, AP-HP, Paris, France.

BACKGROUND: Responsive ataxia rating scales are essential for determining outcome
measures in clinical trials.
METHODS: We evaluated the responsiveness over time of the composite cerebellar
functional severity score, a quantitative score measuring cerebellar ataxia in
133 patients with autosomal dominant cerebellar ataxias (ADCA), which were
prospectively evaluated at inclusion and after one-year of follow-up. A more
responsive tool was developed, the Cerebellar Functional Severity score writing,
incorporating the writing test at dominant hand to the Cerebellar Functional
Severity score.
RESULTS: Within the one-year follow-up period, the Cerebellar Functional Severity
score and its writing version increased significantly and the Scale for the
Assessment and Rating of Ataxia decreased significantly reflecting increased
severity of the cerebellar symptoms. The Cerebellar Functional Severity score
writing responsiveness was best in genotypes SCA1, 2, and 3 compared with the
other genotypes (effect size = 0.196, standardized response mean (SRM) = 0.624
versus effect size = -0.051, SRM = -0.150). The Cerebellar Functional Severity
score writing used as an outcome measure would require only 163 SCA1, 2, or 3
patients per group in a two-arm interventional trial for a 50% reduction in
progression and 80% of power.
DISCUSSION: Our study demonstrates that the Cerebellar Functional Severity score
and Cerebellar Functional Severity score writing are responsive quantitative
scores for evaluating sensitivity to change in ADCA patients and can be used as
outcome measures in clinical trials, especially when targeting genotypes SCA1, 2
and 3.

Copyright © 2011 Movement Disorder Society.

DOI: 10.1002/mds.23531
PMID: 21287600 [Indexed for MEDLINE]

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