Movement Disorders (revue)

Attention, ce site est en cours de développement !
Attention, site généré par des moyens informatiques à partir de corpus bruts.
Les informations ne sont donc pas validées.

Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias

Identifieur interne : 000155 ( France/Analysis ); précédent : 000154; suivant : 000156

Quantitative 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 :

RBID : ISTEX:F5E1EA37EAE8A09F4FE0D467DA5ECD11BB4E90A6

Descripteurs français

English descriptors

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:


Links toward previous steps (curation, corpus...)


Links to Exploration step

ISTEX:F5E1EA37EAE8A09F4FE0D467DA5ECD11BB4E90A6

Le document en format XML

<record>
<TEI wicri:istexFullTextTei="biblStruct">
<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>
</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>
</author>
<author>
<name sortKey="Freeman, Leorah" sort="Freeman, Leorah" uniqKey="Freeman L" first="Leorah" last="Freeman">Leorah Freeman</name>
</author>
<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>
</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>
<publicationStmt>
<idno type="wicri:source">ISTEX</idno>
<idno type="RBID">ISTEX:F5E1EA37EAE8A09F4FE0D467DA5ECD11BB4E90A6</idno>
<date when="2011" year="2011">2011</date>
<idno type="doi">10.1002/mds.23531</idno>
<idno type="url">https://api.istex.fr/document/F5E1EA37EAE8A09F4FE0D467DA5ECD11BB4E90A6/fulltext/pdf</idno>
<idno type="wicri:Area/Istex/Corpus">001498</idno>
<idno type="wicri:Area/Istex/Curation">001498</idno>
<idno type="wicri:Area/Istex/Checkpoint">000173</idno>
<idno type="wicri:doubleKey">0885-3185:2011:Chan E:quantitative:assessment:of</idno>
<idno type="wicri:source">PubMed</idno>
<idno type="RBID">pubmed:21287600</idno>
<idno type="wicri:Area/PubMed/Corpus">001464</idno>
<idno type="wicri:Area/PubMed/Curation">001464</idno>
<idno type="wicri:Area/PubMed/Checkpoint">001134</idno>
<idno type="wicri:Area/Ncbi/Merge">002F56</idno>
<idno type="wicri:Area/Ncbi/Curation">002F56</idno>
<idno type="wicri:Area/Ncbi/Checkpoint">002F56</idno>
<idno type="wicri:Area/Main/Merge">001494</idno>
<idno type="wicri:source">INIST</idno>
<idno type="RBID">Pascal:11-0211284</idno>
<idno type="wicri:Area/PascalFrancis/Corpus">000682</idno>
<idno type="wicri:Area/PascalFrancis/Curation">002636</idno>
<idno type="wicri:Area/PascalFrancis/Checkpoint">000430</idno>
<idno type="wicri:doubleKey">0885-3185:2011:Chan E:quantitative:assessment:of</idno>
<idno type="wicri:Area/Main/Merge">001A20</idno>
<idno type="wicri:Area/Main/Curation">001441</idno>
<idno type="wicri:Area/Main/Exploration">001441</idno>
<idno type="wicri:Area/France/Extraction">000155</idno>
</publicationStmt>
<sourceDesc>
<biblStruct>
<analytic>
<title level="a" type="main" 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">
<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>
<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="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>
<settlement type="city">Paris</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Ribai, Pascale" sort="Ribai, Pascale" uniqKey="Ribai P" first="Pascale" last="Ribai">Pascale Ribai</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>
<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="Goizet, Cyril" sort="Goizet, Cyril" uniqKey="Goizet C" first="Cyril" last="Goizet">Cyril Goizet</name>
<affiliation wicri:level="1">
<country xml:lang="fr">France</country>
<wicri:regionArea>Department of Medical Genetics and University Victor Segalen Bordeaux 2, Bordeaux CHU Hospitals, Pellegrin Hospital, Laboratory of Human Genetics, Bordeaux</wicri:regionArea>
<placeName>
<settlement type="city">Bordeaux</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Marelli, Cecilia" sort="Marelli, Cecilia" uniqKey="Marelli C" first="Cecilia" last="Marelli">Cecilia Marelli</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>
<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="Vincitorio, Carlo Aria" sort="Vincitorio, Carlo Aria" uniqKey="Vincitorio C" first="Carlo-Maria" last="Vincitorio">Carlo-Maria Vincitorio</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>
</author>
<author>
<name sortKey="Bayon, Alice Le" sort="Bayon, Alice Le" uniqKey="Bayon A" first="Alice Le" last="Bayon">Alice Le Bayon</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>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Guyant Arechal, Lucie" sort="Guyant Arechal, Lucie" uniqKey="Guyant Arechal L" first="Lucie" last="Guyant-Maréchal">Lucie Guyant-Maréchal</name>
<affiliation wicri:level="3">
<country xml:lang="fr">France</country>
<wicri:regionArea>Departement of Neurology, Departement of Genetics and Inserm Unit 614, Rouen University Hospital 76031, Rouen Cedex</wicri:regionArea>
<placeName>
<region type="region" nuts="2">Région Normandie</region>
<region type="old region" nuts="2">Haute-Normandie</region>
<settlement type="city">Rouen</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Vandenberghe, Nadia" sort="Vandenberghe, Nadia" uniqKey="Vandenberghe N" first="Nadia" last="Vandenberghe">Nadia Vandenberghe</name>
<affiliation wicri:level="1">
<country xml:lang="fr">France</country>
<wicri:regionArea>Lyon Hospitals, Pierre Wertheimer Neurological Hospital, Service of Neurology C, Lyon</wicri:regionArea>
<placeName>
<settlement type="city">Lyon</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Anheim, Mathieu" sort="Anheim, Mathieu" uniqKey="Anheim M" first="Mathieu" last="Anheim">Mathieu Anheim</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>
</author>
<author>
<name sortKey="Devos, David" sort="Devos, David" uniqKey="Devos D" first="David" last="Devos">David Devos</name>
<affiliation wicri:level="1">
<country xml:lang="fr">France</country>
<wicri:regionArea>Department of Neurology, CNRS FRE3291, IFR114 IMPRT, Université Lille Nord de France, Faculté Lille 2, CHU Lille</wicri:regionArea>
<wicri:noRegion>CHU Lille</wicri:noRegion>
<wicri:noRegion>CHU Lille</wicri:noRegion>
</affiliation>
</author>
<author>
<name sortKey="Freeman, Leorah" sort="Freeman, Leorah" uniqKey="Freeman L" first="Leorah" last="Freeman">Leorah Freeman</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>
</author>
<author>
<name sortKey="Ber, Isabelle Le" sort="Ber, Isabelle Le" uniqKey="Ber I" first="Isabelle Le" last="Ber">Isabelle Le Ber</name>
<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>
<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>
<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>
<affiliation wicri:level="1">
<country xml:lang="fr">France</country>
<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>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Hannequin, Didier" sort="Hannequin, Didier" uniqKey="Hannequin D" first="Didier" last="Hannequin">Didier Hannequin</name>
<affiliation wicri:level="3">
<country xml:lang="fr">France</country>
<wicri:regionArea>Departement of Neurology, Departement of Genetics and Inserm Unit 614, Rouen University Hospital 76031, Rouen Cedex</wicri:regionArea>
<placeName>
<region type="region" nuts="2">Région Normandie</region>
<region type="old region" nuts="2">Haute-Normandie</region>
<settlement type="city">Rouen</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Brice, Alexis" sort="Brice, Alexis" uniqKey="Brice A" first="Alexis" last="Brice">Alexis Brice</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="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>
<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>
<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="Durr, Alexandra" sort="Durr, Alexandra" uniqKey="Durr A" first="Alexandra" last="Durr">Alexandra Durr</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>
<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="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>

Pour manipuler ce document sous Unix (Dilib)

EXPLOR_STEP=$WICRI_ROOT/Wicri/Santé/explor/MovDisordV3/Data/France/Analysis
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 000155 | SxmlIndent | more

Ou

HfdSelect -h $EXPLOR_AREA/Data/France/Analysis/biblio.hfd -nk 000155 | SxmlIndent | more

Pour mettre un lien sur cette page dans le réseau Wicri

{{Explor lien
   |wiki=    Wicri/Santé
   |area=    MovDisordV3
   |flux=    France
   |étape=   Analysis
   |type=    RBID
   |clé=     ISTEX:F5E1EA37EAE8A09F4FE0D467DA5ECD11BB4E90A6
   |texte=   Quantitative assessment of the evolution of cerebellar signs in spinocerebellar ataxias
}}

Wicri

This area was generated with Dilib version V0.6.23.
Data generation: Sun Jul 3 12:29:32 2016. Site generation: Wed Feb 14 10:52:30 2024