Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias
Identifieur interne : 001494 ( Main/Merge ); précédent : 001493; suivant : 001495Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias
Auteurs : Ellis Chan [France] ; Perrine Charles [France] ; Pascale Ribai [France] ; Cyril Goizet [France] ; Cecilia Marelli [France] ; Carlo-Maria Vincitorio [France] ; Alice Le Bayon [France] ; Lucie Guyant-Maréchal [France] ; Nadia Vandenberghe [France] ; Mathieu Anheim [France] ; David Devos [France] ; Leorah Freeman [France] ; Isabelle Le Ber [France] ; Karine N'Guyen [France] ; Maya Tchikviladzé [France] ; Pierre Labauge [France] ; Didier Hannequin [France] ; Alexis Brice [France] ; Alexandra Durr [France] ; Sophie Tezenas Du Montcel [France]Source :
- Movement Disorders [ 0885-3185 ] ; 2011-02-15.
English descriptors
- KwdEn :
- Adult, Analysis of Variance, Cerebellum (pathology), Cerebellum (physiopathology), Disability Evaluation, Disease Progression, Female, Follow-Up Studies, Humans, Male, Middle Aged, Sample Size, Severity of Illness Index, Spinocerebellar Ataxias (diagnosis), Spinocerebellar Ataxias (physiopathology), Statistics as Topic, Young Adult, clinical neurology examination, gait disorder/ataxia, outcome research, responsiveness, spinocerebellar ataxia.
- MESH :
- diagnosis : Spinocerebellar Ataxias.
- pathology : Cerebellum.
- physiopathology : Cerebellum, Spinocerebellar Ataxias.
- Adult, Analysis of Variance, Disability Evaluation, Disease Progression, Female, Follow-Up Studies, Humans, Male, Middle Aged, Sample Size, Severity of Illness Index, Statistics as Topic, Young Adult.
Abstract
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. © 2011 Movement Disorder Society
Url:
DOI: 10.1002/mds.23531
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<author><name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</name>
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<author><name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
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<author><name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
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<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
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<wicri:regionArea>Inserm, U975, Paris</wicri:regionArea>
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<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Cnrs, UMR 7225, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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<author><name sortKey="N Guyen, Karine" sort="N Guyen, Karine" uniqKey="N Guyen K" first="Karine" last="N'Guyen">Karine N'Guyen</name>
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<wicri:regionArea>Department of Genetics, Marseille Hospitals, La Timone Hospital, Marseille</wicri:regionArea>
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<author><name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
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<author><name sortKey="Labauge, Pierre" sort="Labauge, Pierre" uniqKey="Labauge P" first="Pierre" last="Labauge">Pierre Labauge</name>
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<wicri:regionArea>Departement of Neurology, Departement of Genetics and Inserm Unit 614, Rouen University Hospital 76031, Rouen Cedex</wicri:regionArea>
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<author><name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
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<affiliation wicri:level="3"><country xml:lang="fr">France</country>
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<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
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<author><name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
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<author><name sortKey="Du Montcel, Sophie Tezenas" sort="Du Montcel, Sophie Tezenas" uniqKey="Du Montcel S" first="Sophie Tezenas" last="Du Montcel">Sophie Tezenas Du Montcel</name>
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<wicri:regionArea>Department of Biostatistics and Medical Informatics and Pitié‐Salpêtrière Charles‐Foix Clinical Research Unit, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>ER4, Modelling in Clinical Research, University Pierre et Marie Curie, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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<series><title level="j">Movement Disorders</title>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term>
<term>Analysis of Variance</term>
<term>Cerebellum (pathology)</term>
<term>Cerebellum (physiopathology)</term>
<term>Disability Evaluation</term>
<term>Disease Progression</term>
<term>Female</term>
<term>Follow-Up Studies</term>
<term>Humans</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Sample Size</term>
<term>Severity of Illness Index</term>
<term>Spinocerebellar Ataxias (diagnosis)</term>
<term>Spinocerebellar Ataxias (physiopathology)</term>
<term>Statistics as Topic</term>
<term>Young Adult</term>
<term>clinical neurology examination</term>
<term>gait disorder/ataxia</term>
<term>outcome research</term>
<term>responsiveness</term>
<term>spinocerebellar ataxia</term>
</keywords>
<keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Spinocerebellar Ataxias</term>
</keywords>
<keywords scheme="MESH" qualifier="pathology" xml:lang="en"><term>Cerebellum</term>
</keywords>
<keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Cerebellum</term>
<term>Spinocerebellar Ataxias</term>
</keywords>
<keywords scheme="MESH" xml:lang="en"><term>Adult</term>
<term>Analysis of Variance</term>
<term>Disability Evaluation</term>
<term>Disease Progression</term>
<term>Female</term>
<term>Follow-Up Studies</term>
<term>Humans</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Sample Size</term>
<term>Severity of Illness Index</term>
<term>Statistics as Topic</term>
<term>Young Adult</term>
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<front><div type="abstract" xml:lang="en">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. © 2011 Movement Disorder Society</div>
</front>
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<author><name sortKey="Chan, Ellis" sort="Chan, Ellis" uniqKey="Chan E" first="Ellis" last="Chan">Ellis Chan</name>
</author>
<author><name sortKey="Charles, Perrine" sort="Charles, Perrine" uniqKey="Charles P" first="Perrine" last="Charles">Perrine Charles</name>
</author>
<author><name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
</author>
<author><name sortKey="Goizet, Cyril" sort="Goizet, Cyril" uniqKey="Goizet C" first="Cyril" last="Goizet">Cyril Goizet</name>
</author>
<author><name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
</author>
<author><name sortKey="Vincitorio, Carlo Aria" sort="Vincitorio, Carlo Aria" uniqKey="Vincitorio C" first="Carlo-Maria" last="Vincitorio">Carlo-Maria Vincitorio</name>
</author>
<author><name sortKey="Bayon, Alice Le" sort="Bayon, Alice Le" uniqKey="Bayon A" first="Alice Le" last="Bayon">Alice Le Bayon</name>
</author>
<author><name sortKey="Guyant Arechal, Lucie" sort="Guyant Arechal, Lucie" uniqKey="Guyant Arechal L" first="Lucie" last="Guyant-Maréchal">Lucie Guyant-Maréchal</name>
</author>
<author><name sortKey="Vandenberghe, Nadia" sort="Vandenberghe, Nadia" uniqKey="Vandenberghe N" first="Nadia" last="Vandenberghe">Nadia Vandenberghe</name>
</author>
<author><name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</name>
</author>
<author><name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
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<author><name sortKey="Freeman, Leorah" sort="Freeman, Leorah" uniqKey="Freeman L" first="Leorah" last="Freeman">Leorah Freeman</name>
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<author><name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
</author>
<author><name sortKey="N Guyen, Karine" sort="N Guyen, Karine" uniqKey="N Guyen K" first="Karine" last="N'Guyen">Karine N'Guyen</name>
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<author><name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
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<author><name sortKey="Labauge, Pierre" sort="Labauge, Pierre" uniqKey="Labauge P" first="Pierre" last="Labauge">Pierre Labauge</name>
</author>
<author><name sortKey="Hannequin, Didier" sort="Hannequin, Didier" uniqKey="Hannequin D" first="Didier" last="Hannequin">Didier Hannequin</name>
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<author><name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
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<author><name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
</author>
<author><name sortKey="Du Montcel, Sophie Tezenas" sort="Du Montcel, Sophie Tezenas" uniqKey="Du Montcel S" first="Sophie Tezenas" last="Du Montcel">Sophie Tezenas Du Montcel</name>
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<wicri:regionArea>Department of Biostatistics and Medical Informatics and Pitié‐Salpêtrière Charles‐Foix Clinical Research Unit, AP‐HP, Paris</wicri:regionArea>
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<author><name sortKey="Charles, Perrine" sort="Charles, Perrine" uniqKey="Charles P" first="Perrine" last="Charles">Perrine Charles</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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<affiliation wicri:level="3"><country xml:lang="fr">France</country>
<wicri:regionArea>Federation of Neurology, Pitié‐Salpêtrière Hospital, AP‐HP, F‐75013 Paris</wicri:regionArea>
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<author><name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
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<placeName><settlement type="city">Paris</settlement>
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</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
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<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Cnrs, UMR 7225, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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<author><name sortKey="Vincitorio, Carlo Aria" sort="Vincitorio, Carlo Aria" uniqKey="Vincitorio C" first="Carlo-Maria" last="Vincitorio">Carlo-Maria Vincitorio</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Bayon, Alice Le" sort="Bayon, Alice Le" uniqKey="Bayon A" first="Alice Le" last="Bayon">Alice Le Bayon</name>
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<wicri:regionArea>Service of Neurology, CHU Nimes, Carémeau Hospital, Nimes</wicri:regionArea>
<placeName><region type="région">Languedoc-Roussillon</region>
<settlement type="city">Nîmes</settlement>
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<author><name sortKey="Guyant Arechal, Lucie" sort="Guyant Arechal, Lucie" uniqKey="Guyant Arechal L" first="Lucie" last="Guyant-Maréchal">Lucie Guyant-Maréchal</name>
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<wicri:regionArea>Departement of Neurology, Departement of Genetics and Inserm Unit 614, Rouen University Hospital 76031, Rouen Cedex</wicri:regionArea>
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<settlement type="city">Rouen</settlement>
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<author><name sortKey="Vandenberghe, Nadia" sort="Vandenberghe, Nadia" uniqKey="Vandenberghe N" first="Nadia" last="Vandenberghe">Nadia Vandenberghe</name>
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<wicri:regionArea>Lyon Hospitals, Pierre Wertheimer Neurological Hospital, Service of Neurology C, Lyon</wicri:regionArea>
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<author><name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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</affiliation>
</author>
<author><name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
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<wicri:regionArea>Department of Neurology, CNRS FRE3291, IFR114 IMPRT, Université Lille Nord de France, Faculté Lille 2, CHU Lille</wicri:regionArea>
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</affiliation>
</author>
<author><name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
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<wicri:regionArea>Federation of Neurology, Pitié‐Salpêtrière Hospital, AP‐HP, F‐75013 Paris</wicri:regionArea>
<placeName><region type="region" nuts="2">Île-de-France</region>
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</placeName>
</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Inserm, U975, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Cnrs, UMR 7225, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="N Guyen, Karine" sort="N Guyen, Karine" uniqKey="N Guyen K" first="Karine" last="N'Guyen">Karine N'Guyen</name>
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<wicri:regionArea>Department of Genetics, Marseille Hospitals, La Timone Hospital, Marseille</wicri:regionArea>
<placeName><settlement type="city">Marseille</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Labauge, Pierre" sort="Labauge, Pierre" uniqKey="Labauge P" first="Pierre" last="Labauge">Pierre Labauge</name>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Service of Neurology, CHU Nimes, Carémeau Hospital, Nimes</wicri:regionArea>
<placeName><region type="région">Languedoc-Roussillon</region>
<settlement type="city">Nîmes</settlement>
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<author><name sortKey="Hannequin, Didier" sort="Hannequin, Didier" uniqKey="Hannequin D" first="Didier" last="Hannequin">Didier Hannequin</name>
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<wicri:regionArea>Departement of Neurology, Departement of Genetics and Inserm Unit 614, Rouen University Hospital 76031, Rouen Cedex</wicri:regionArea>
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<author><name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
<affiliation wicri:level="3"><country xml:lang="fr">France</country>
<wicri:regionArea>Federation of Neurology, Pitié‐Salpêtrière Hospital, AP‐HP, F‐75013 Paris</wicri:regionArea>
<placeName><region type="region" nuts="2">Île-de-France</region>
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</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Université Pierre et Marie Curie‐Paris6, Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière, UMR‐S975, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Inserm, U975, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Cnrs, UMR 7225, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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<author><name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
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<wicri:regionArea>Department of Genetics and Cytogenetics, Pitié‐Salpêtrière Hospital, AP‐HP, Paris</wicri:regionArea>
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</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
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<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Inserm, U975, Paris</wicri:regionArea>
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<affiliation wicri:level="1"><country xml:lang="fr">France</country>
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<placeName><settlement type="city">Paris</settlement>
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<author><name sortKey="Du Montcel, Sophie Tezenas" sort="Du Montcel, Sophie Tezenas" uniqKey="Du Montcel S" first="Sophie Tezenas" last="Du Montcel">Sophie Tezenas Du Montcel</name>
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<wicri:regionArea>Department of Biostatistics and Medical Informatics and Pitié‐Salpêtrière Charles‐Foix Clinical Research Unit, AP‐HP, Paris</wicri:regionArea>
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</placeName>
</affiliation>
<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>ER4, Modelling in Clinical Research, University Pierre et Marie Curie, Paris</wicri:regionArea>
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<series><title level="j">Movement Disorders</title>
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<front><div type="abstract" xml:lang="en">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. © 2011 Movement Disorder Society</div>
</front>
</TEI>
</ISTEX>
<PubMed><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias.</title>
<author><name sortKey="Chan, Ellis" sort="Chan, Ellis" uniqKey="Chan E" first="Ellis" last="Chan">Ellis Chan</name>
<affiliation wicri:level="1"><nlm:affiliation>Department of Biostatistics and Medical Informatics and Pitié-Salpêtrière Charles-Foix Clinical Research Unit, AP-HP, Paris, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>Department of Biostatistics and Medical Informatics and Pitié-Salpêtrière Charles-Foix Clinical Research Unit, AP-HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Charles, Perrine" sort="Charles, Perrine" uniqKey="Charles P" first="Perrine" last="Charles">Perrine Charles</name>
</author>
<author><name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
</author>
<author><name sortKey="Goizet, Cyril" sort="Goizet, Cyril" uniqKey="Goizet C" first="Cyril" last="Goizet">Cyril Goizet</name>
</author>
<author><name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
</author>
<author><name sortKey="Vincitorio, Carlo Maria" sort="Vincitorio, Carlo Maria" uniqKey="Vincitorio C" first="Carlo-Maria" last="Vincitorio">Carlo-Maria Vincitorio</name>
</author>
<author><name sortKey="Le Bayon, Alice" sort="Le Bayon, Alice" uniqKey="Le Bayon A" first="Alice" last="Le Bayon">Alice Le Bayon</name>
</author>
<author><name sortKey="Guyant Marechal, Lucie" sort="Guyant Marechal, Lucie" uniqKey="Guyant Marechal L" first="Lucie" last="Guyant-Maréchal">Lucie Guyant-Maréchal</name>
</author>
<author><name sortKey="Vandenberghe, Nadia" sort="Vandenberghe, Nadia" uniqKey="Vandenberghe N" first="Nadia" last="Vandenberghe">Nadia Vandenberghe</name>
</author>
<author><name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</name>
</author>
<author><name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
</author>
<author><name sortKey="Freeman, Leorah" sort="Freeman, Leorah" uniqKey="Freeman L" first="Leorah" last="Freeman">Leorah Freeman</name>
</author>
<author><name sortKey="Le Ber, Isabelle" sort="Le Ber, Isabelle" uniqKey="Le Ber I" first="Isabelle" last="Le Ber">Isabelle Le Ber</name>
</author>
<author><name sortKey="N Guyen, Karine" sort="N Guyen, Karine" uniqKey="N Guyen K" first="Karine" last="N'Guyen">Karine N'Guyen</name>
</author>
<author><name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
</author>
<author><name sortKey="Labauge, Pierre" sort="Labauge, Pierre" uniqKey="Labauge P" first="Pierre" last="Labauge">Pierre Labauge</name>
</author>
<author><name sortKey="Hannequin, Didier" sort="Hannequin, Didier" uniqKey="Hannequin D" first="Didier" last="Hannequin">Didier Hannequin</name>
</author>
<author><name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
</author>
<author><name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
</author>
<author><name sortKey="Du Montcel, Sophie Tezenas" sort="Du Montcel, Sophie Tezenas" uniqKey="Du Montcel S" first="Sophie Tezenas" last="Du Montcel">Sophie Tezenas Du Montcel</name>
</author>
</titleStmt>
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<date when="2011">2011</date>
<idno type="doi">10.1002/mds.23531</idno>
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<sourceDesc><biblStruct><analytic><title xml:lang="en">Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias.</title>
<author><name sortKey="Chan, Ellis" sort="Chan, Ellis" uniqKey="Chan E" first="Ellis" last="Chan">Ellis Chan</name>
<affiliation wicri:level="1"><nlm:affiliation>Department of Biostatistics and Medical Informatics and Pitié-Salpêtrière Charles-Foix Clinical Research Unit, AP-HP, Paris, France.</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>Department of Biostatistics and Medical Informatics and Pitié-Salpêtrière Charles-Foix Clinical Research Unit, AP-HP, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Charles, Perrine" sort="Charles, Perrine" uniqKey="Charles P" first="Perrine" last="Charles">Perrine Charles</name>
</author>
<author><name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
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<author><name sortKey="Goizet, Cyril" sort="Goizet, Cyril" uniqKey="Goizet C" first="Cyril" last="Goizet">Cyril Goizet</name>
</author>
<author><name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
</author>
<author><name sortKey="Vincitorio, Carlo Maria" sort="Vincitorio, Carlo Maria" uniqKey="Vincitorio C" first="Carlo-Maria" last="Vincitorio">Carlo-Maria Vincitorio</name>
</author>
<author><name sortKey="Le Bayon, Alice" sort="Le Bayon, Alice" uniqKey="Le Bayon A" first="Alice" last="Le Bayon">Alice Le Bayon</name>
</author>
<author><name sortKey="Guyant Marechal, Lucie" sort="Guyant Marechal, Lucie" uniqKey="Guyant Marechal L" first="Lucie" last="Guyant-Maréchal">Lucie Guyant-Maréchal</name>
</author>
<author><name sortKey="Vandenberghe, Nadia" sort="Vandenberghe, Nadia" uniqKey="Vandenberghe N" first="Nadia" last="Vandenberghe">Nadia Vandenberghe</name>
</author>
<author><name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</name>
</author>
<author><name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
</author>
<author><name sortKey="Freeman, Leorah" sort="Freeman, Leorah" uniqKey="Freeman L" first="Leorah" last="Freeman">Leorah Freeman</name>
</author>
<author><name sortKey="Le Ber, Isabelle" sort="Le Ber, Isabelle" uniqKey="Le Ber I" first="Isabelle" last="Le Ber">Isabelle Le Ber</name>
</author>
<author><name sortKey="N Guyen, Karine" sort="N Guyen, Karine" uniqKey="N Guyen K" first="Karine" last="N'Guyen">Karine N'Guyen</name>
</author>
<author><name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
</author>
<author><name sortKey="Labauge, Pierre" sort="Labauge, Pierre" uniqKey="Labauge P" first="Pierre" last="Labauge">Pierre Labauge</name>
</author>
<author><name sortKey="Hannequin, Didier" sort="Hannequin, Didier" uniqKey="Hannequin D" first="Didier" last="Hannequin">Didier Hannequin</name>
</author>
<author><name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
</author>
<author><name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
</author>
<author><name sortKey="Du Montcel, Sophie Tezenas" sort="Du Montcel, Sophie Tezenas" uniqKey="Du Montcel S" first="Sophie Tezenas" last="Du Montcel">Sophie Tezenas Du Montcel</name>
</author>
</analytic>
<series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title>
<idno type="eISSN">1531-8257</idno>
<imprint><date when="2011" type="published">2011</date>
</imprint>
</series>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term>
<term>Analysis of Variance</term>
<term>Cerebellum (pathology)</term>
<term>Cerebellum (physiopathology)</term>
<term>Disability Evaluation</term>
<term>Disease Progression</term>
<term>Female</term>
<term>Follow-Up Studies</term>
<term>Humans</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Sample Size</term>
<term>Severity of Illness Index</term>
<term>Spinocerebellar Ataxias (diagnosis)</term>
<term>Spinocerebellar Ataxias (physiopathology)</term>
<term>Statistics as Topic</term>
<term>Young Adult</term>
</keywords>
<keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Spinocerebellar Ataxias</term>
</keywords>
<keywords scheme="MESH" qualifier="pathology" xml:lang="en"><term>Cerebellum</term>
</keywords>
<keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Cerebellum</term>
<term>Spinocerebellar Ataxias</term>
</keywords>
<keywords scheme="MESH" xml:lang="en"><term>Adult</term>
<term>Analysis of Variance</term>
<term>Disability Evaluation</term>
<term>Disease Progression</term>
<term>Female</term>
<term>Follow-Up Studies</term>
<term>Humans</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Sample Size</term>
<term>Severity of Illness Index</term>
<term>Statistics as Topic</term>
<term>Young Adult</term>
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<front><div type="abstract" xml:lang="en">Responsive ataxia rating scales are essential for determining outcome measures in clinical trials.</div>
</front>
</TEI>
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