Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias
Identifieur interne : 000155 ( France/Analysis ); précédent : 000154; suivant : 000156Quantitative 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.
Descripteurs français
- Pascal (Inist)
- Wicri :
- topic : Neurologie.
English descriptors
- KwdEn :
- Adult, Analysis of Variance, Cerebellar ataxia, Cerebellum (pathology), Cerebellum (physiopathology), Disability Evaluation, Disease Progression, Evolution, Female, Follow-Up Studies, Gait disorder, Humans, Male, Middle Aged, Nervous system diseases, Neurology, Prognosis, Sample Size, Severity of Illness Index, Sign, Spinocerebellar Ataxias (diagnosis), Spinocerebellar Ataxias (physiopathology), Spinocerebellar ataxia, 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
Affiliations:
- France
- Haute-Normandie, Languedoc-Roussillon, Région Normandie, Île-de-France
- Bordeaux, Lyon, Marseille, Nîmes, Paris, Rouen
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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="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>
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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>
</placeName>
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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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<affiliation wicri:level="1"><country xml:lang="fr">France</country>
<wicri:regionArea>Inserm, U975, 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>Cnrs, UMR 7225, Paris</wicri:regionArea>
<placeName><settlement type="city">Paris</settlement>
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</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>
<affiliation wicri:level="1"><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>
<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>
</placeName>
</affiliation>
</author>
</analytic>
<monogr></monogr>
<series><title level="j">Movement Disorders</title>
<title level="j" type="abbrev">Mov. Disord.</title>
<idno type="ISSN">0885-3185</idno>
<idno type="eISSN">1531-8257</idno>
<imprint><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher>
<pubPlace>Hoboken</pubPlace>
<date type="published" when="2011-02-15">2011-02-15</date>
<biblScope unit="vol">26</biblScope>
<biblScope unit="issue">3</biblScope>
<biblScope unit="page" from="534">534</biblScope>
<biblScope unit="page" to="538">538</biblScope>
</imprint>
<idno type="ISSN">0885-3185</idno>
</series>
<idno type="istex">F5E1EA37EAE8A09F4FE0D467DA5ECD11BB4E90A6</idno>
<idno type="DOI">10.1002/mds.23531</idno>
<idno type="ArticleID">MDS23531</idno>
</biblStruct>
</sourceDesc>
<seriesStmt><idno type="ISSN">0885-3185</idno>
</seriesStmt>
</fileDesc>
<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term>
<term>Analysis of Variance</term>
<term>Cerebellar ataxia</term>
<term>Cerebellum (pathology)</term>
<term>Cerebellum (physiopathology)</term>
<term>Disability Evaluation</term>
<term>Disease Progression</term>
<term>Evolution</term>
<term>Female</term>
<term>Follow-Up Studies</term>
<term>Gait disorder</term>
<term>Humans</term>
<term>Male</term>
<term>Middle Aged</term>
<term>Nervous system diseases</term>
<term>Neurology</term>
<term>Prognosis</term>
<term>Sample Size</term>
<term>Severity of Illness Index</term>
<term>Sign</term>
<term>Spinocerebellar Ataxias (diagnosis)</term>
<term>Spinocerebellar Ataxias (physiopathology)</term>
<term>Spinocerebellar ataxia</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>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Ataxie cérébelleuse</term>
<term>Ataxie spinocérébelleuse</term>
<term>Evolution</term>
<term>Neurologie</term>
<term>Pathologie du système nerveux</term>
<term>Pronostic</term>
<term>Signe</term>
<term>Trouble de la marche</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Neurologie</term>
</keywords>
</textClass>
<langUsage><language ident="en">en</language>
</langUsage>
</profileDesc>
</teiHeader>
<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>
<affiliations><list><country><li>France</li>
</country>
<region><li>Haute-Normandie</li>
<li>Languedoc-Roussillon</li>
<li>Région Normandie</li>
<li>Île-de-France</li>
</region>
<settlement><li>Bordeaux</li>
<li>Lyon</li>
<li>Marseille</li>
<li>Nîmes</li>
<li>Paris</li>
<li>Rouen</li>
</settlement>
</list>
<tree><country name="France"><noRegion><name sortKey="Chan, Ellis" sort="Chan, Ellis" uniqKey="Chan E" first="Ellis" last="Chan">Ellis Chan</name>
</noRegion>
<name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</name>
<name sortKey="Bayon, Alice Le" sort="Bayon, Alice Le" uniqKey="Bayon A" first="Alice Le" last="Bayon">Alice Le Bayon</name>
<name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
<name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
<name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
<name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
<name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
<name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
<name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
<name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
<name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</name>
<name sortKey="Charles, Perrine" sort="Charles, Perrine" uniqKey="Charles P" first="Perrine" last="Charles">Perrine Charles</name>
<name sortKey="Charles, Perrine" sort="Charles, Perrine" uniqKey="Charles P" first="Perrine" last="Charles">Perrine Charles</name>
<name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
<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>
<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>
<name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
<name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
<name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
<name sortKey="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</name>
<name sortKey="Freeman, Leorah" sort="Freeman, Leorah" uniqKey="Freeman L" first="Leorah" last="Freeman">Leorah Freeman</name>
<name sortKey="Goizet, Cyril" sort="Goizet, Cyril" uniqKey="Goizet C" first="Cyril" last="Goizet">Cyril Goizet</name>
<name sortKey="Guyant Arechal, Lucie" sort="Guyant Arechal, Lucie" uniqKey="Guyant Arechal L" first="Lucie" last="Guyant-Maréchal">Lucie Guyant-Maréchal</name>
<name sortKey="Hannequin, Didier" sort="Hannequin, Didier" uniqKey="Hannequin D" first="Didier" last="Hannequin">Didier Hannequin</name>
<name sortKey="Labauge, Pierre" sort="Labauge, Pierre" uniqKey="Labauge P" first="Pierre" last="Labauge">Pierre Labauge</name>
<name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
<name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
<name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
<name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</name>
<name sortKey="N Guyen, Karine" sort="N Guyen, Karine" uniqKey="N Guyen K" first="Karine" last="N'Guyen">Karine N'Guyen</name>
<name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
<name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
<name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
<name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</name>
<name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
<name sortKey="Tchikviladze, Maya" sort="Tchikviladze, Maya" uniqKey="Tchikviladze M" first="Maya" last="Tchikviladzé">Maya Tchikviladzé</name>
<name sortKey="Vandenberghe, Nadia" sort="Vandenberghe, Nadia" uniqKey="Vandenberghe N" first="Nadia" last="Vandenberghe">Nadia Vandenberghe</name>
<name sortKey="Vincitorio, Carlo Aria" sort="Vincitorio, Carlo Aria" uniqKey="Vincitorio C" first="Carlo-Maria" last="Vincitorio">Carlo-Maria Vincitorio</name>
</country>
</tree>
</affiliations>
</record>
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