Performance measures in Friedreich ataxia: Potential utility as clinical outcome tools
Identifieur interne : 001708 ( Istex/Corpus ); précédent : 001707; suivant : 001709Performance measures in Friedreich ataxia: Potential utility as clinical outcome tools
Auteurs : David R. Lynch ; Jennifer M. Farmer ; Robert L. Wilson ; Laura J. BalcerSource :
- Movement Disorders [ 0885-3185 ] ; 2005-07.
English descriptors
Abstract
Although several neuroprotective agents have been proposed as potential therapies in Friedreich ataxia (FA), clinical trials of their efficacy are limited by a lack of sensitive outcome measures. We assessed whether performance measures (nine‐hole peg test, the timed 25‐foot walk, and low‐contrast letter acuity) provide valid measures of disease status in FA. Scores for each measure correlated significantly with neurologic disability and disease duration. Rank correlations between scores for performance measures were moderate in magnitude, suggesting that the each test captures separate yet related dimensions of neurological function in FA. Linear regression models demonstrated that scores from the nine‐hole peg test and the timed 25‐foot walk (after reciprocal transformation) were predicted by age and triplet repeat length in patients with FA. In addition, comparison of the temporal courses of change for each performance measure demonstrated that scores from the timed 25‐foot walk change early in the course of FA, nine‐hole peg test scores change slowly over the full course of the disorder, and low‐contrast letter acuity scores change in the later stages of the disease. Thus, a composite scale derived from these performance measures may provide the best overall measure for assessing disease progression throughout the illness. © 2005 Movement Disorder Society
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DOI: 10.1002/mds.20449
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ISTEX:56C9C7F682A11ADFD80C1BD7B809FC29973612E2Le document en format XML
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Lynch</name><affiliation><mods:affiliation>Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</mods:affiliation></affiliation><affiliation><mods:affiliation>Department of Pediatrics, University of Pennsylvania School of Medicine, and Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA</mods:affiliation></affiliation></author><author><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name><affiliation><mods:affiliation>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</mods:affiliation></affiliation></author><author><name sortKey="Wilson, Robert L" sort="Wilson, Robert L" uniqKey="Wilson R" first="Robert L." last="Wilson">Robert L. 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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="2005-07">2005-07</date><biblScope unit="vol">20</biblScope><biblScope unit="issue">7</biblScope><biblScope unit="page" from="777">777</biblScope><biblScope unit="page" to="782">782</biblScope></imprint><idno type="ISSN">0885-3185</idno></series><idno type="istex">56C9C7F682A11ADFD80C1BD7B809FC29973612E2</idno><idno type="DOI">10.1002/mds.20449</idno><idno type="ArticleID">MDS20449</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0885-3185</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>antioxidant</term><term>cardiomyopathy</term><term>composite measure</term><term>diabetes</term><term>mitochondrial disease</term></keywords></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Although several neuroprotective agents have been proposed as potential therapies in Friedreich ataxia (FA), clinical trials of their efficacy are limited by a lack of sensitive outcome measures. We assessed whether performance measures (nine‐hole peg test, the timed 25‐foot walk, and low‐contrast letter acuity) provide valid measures of disease status in FA. Scores for each measure correlated significantly with neurologic disability and disease duration. Rank correlations between scores for performance measures were moderate in magnitude, suggesting that the each test captures separate yet related dimensions of neurological function in FA. Linear regression models demonstrated that scores from the nine‐hole peg test and the timed 25‐foot walk (after reciprocal transformation) were predicted by age and triplet repeat length in patients with FA. In addition, comparison of the temporal courses of change for each performance measure demonstrated that scores from the timed 25‐foot walk change early in the course of FA, nine‐hole peg test scores change slowly over the full course of the disorder, and low‐contrast letter acuity scores change in the later stages of the disease. Thus, a composite scale derived from these performance measures may provide the best overall measure for assessing disease progression throughout the illness. © 2005 Movement Disorder Society</div></front></TEI><istex><corpusName>wiley</corpusName><author><json:item><name>David R. Lynch MD, PhD</name><affiliations><json:string>Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</json:string><json:string>Department of Pediatrics, University of Pennsylvania School of Medicine, and Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA</json:string></affiliations></json:item><json:item><name>Jennifer M. Farmer MSGC</name><affiliations><json:string>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</json:string></affiliations></json:item><json:item><name>Robert L. Wilson MD, PhD</name><affiliations><json:string>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</json:string></affiliations></json:item><json:item><name>Laura J. Balcer MD, MSCE</name><affiliations><json:string>Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</json:string></affiliations></json:item></author><subject><json:item><lang><json:string>eng</json:string></lang><value>composite measure</value></json:item><json:item><lang><json:string>eng</json:string></lang><value>mitochondrial disease</value></json:item><json:item><lang><json:string>eng</json:string></lang><value>antioxidant</value></json:item><json:item><lang><json:string>eng</json:string></lang><value>cardiomyopathy</value></json:item><json:item><lang><json:string>eng</json:string></lang><value>diabetes</value></json:item></subject><language><json:string>eng</json:string></language><abstract>Although several neuroprotective agents have been proposed as potential therapies in Friedreich ataxia (FA), clinical trials of their efficacy are limited by a lack of sensitive outcome measures. We assessed whether performance measures (nine‐hole peg test, the timed 25‐foot walk, and low‐contrast letter acuity) provide valid measures of disease status in FA. Scores for each measure correlated significantly with neurologic disability and disease duration. Rank correlations between scores for performance measures were moderate in magnitude, suggesting that the each test captures separate yet related dimensions of neurological function in FA. Linear regression models demonstrated that scores from the nine‐hole peg test and the timed 25‐foot walk (after reciprocal transformation) were predicted by age and triplet repeat length in patients with FA. In addition, comparison of the temporal courses of change for each performance measure demonstrated that scores from the timed 25‐foot walk change early in the course of FA, nine‐hole peg test scores change slowly over the full course of the disorder, and low‐contrast letter acuity scores change in the later stages of the disease. 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We assessed whether performance measures (nine‐hole peg test, the timed 25‐foot walk, and low‐contrast letter acuity) provide valid measures of disease status in FA. Scores for each measure correlated significantly with neurologic disability and disease duration. Rank correlations between scores for performance measures were moderate in magnitude, suggesting that the each test captures separate yet related dimensions of neurological function in FA. Linear regression models demonstrated that scores from the nine‐hole peg test and the timed 25‐foot walk (after reciprocal transformation) were predicted by age and triplet repeat length in patients with FA. In addition, comparison of the temporal courses of change for each performance measure demonstrated that scores from the timed 25‐foot walk change early in the course of FA, nine‐hole peg test scores change slowly over the full course of the disorder, and low‐contrast letter acuity scores change in the later stages of the disease. Thus, a composite scale derived from these performance measures may provide the best overall measure for assessing disease progression throughout the illness. © 2005 Movement Disorder Society</p></abstract></abstractGroup></contentMeta></header></component></istex:document></istex:metadataXml><!--Version 0.6 générée le 4-12-2015--><mods version="3.6"><titleInfo lang="en"><title>Performance measures in Friedreich ataxia: Potential utility as clinical outcome tools</title></titleInfo><titleInfo type="abbreviated" lang="en"><title>Clinical Measures in Friedreich Ataxia</title></titleInfo><titleInfo type="alternative" contentType="CDATA" lang="en"><title>Performance measures in Friedreich ataxia: Potential utility as clinical outcome tools</title></titleInfo><name type="personal"><namePart type="given">David R.</namePart><namePart type="family">Lynch</namePart><namePart type="termsOfAddress">MD, PhD</namePart><affiliation>Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</affiliation><affiliation>Department of Pediatrics, University of Pennsylvania School of Medicine, and Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA</affiliation><description>Correspondence: Division of Neurology, Children's Hospital of Philadelphia, 502 Abramson Building, Philadelphia, PA 19104‐4318</description><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Jennifer M.</namePart><namePart type="family">Farmer</namePart><namePart type="termsOfAddress">MSGC</namePart><affiliation>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Robert L.</namePart><namePart type="family">Wilson</namePart><namePart type="termsOfAddress">MD, PhD</namePart><affiliation>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Laura J.</namePart><namePart type="family">Balcer</namePart><namePart type="termsOfAddress">MD, MSCE</namePart><affiliation>Department of Neurology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA</affiliation><role><roleTerm type="text">author</roleTerm></role></name><typeOfResource>text</typeOfResource><genre authority="originalCategForm">article</genre><originInfo><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><place><placeTerm type="text">Hoboken</placeTerm></place><dateIssued encoding="w3cdtf">2005-07</dateIssued><dateCaptured encoding="w3cdtf">2004-07-14</dateCaptured><dateValid encoding="w3cdtf">2004-11-12</dateValid><copyrightDate encoding="w3cdtf">2005</copyrightDate></originInfo><language><languageTerm type="code" authority="rfc3066">en</languageTerm><languageTerm type="code" authority="iso639-2b">eng</languageTerm></language><physicalDescription><internetMediaType>text/html</internetMediaType><extent unit="figures">2</extent><extent unit="tables">4</extent><extent unit="references">18</extent><extent unit="words">4237</extent></physicalDescription><abstract lang="en">Although several neuroprotective agents have been proposed as potential therapies in Friedreich ataxia (FA), clinical trials of their efficacy are limited by a lack of sensitive outcome measures. We assessed whether performance measures (nine‐hole peg test, the timed 25‐foot walk, and low‐contrast letter acuity) provide valid measures of disease status in FA. Scores for each measure correlated significantly with neurologic disability and disease duration. Rank correlations between scores for performance measures were moderate in magnitude, suggesting that the each test captures separate yet related dimensions of neurological function in FA. Linear regression models demonstrated that scores from the nine‐hole peg test and the timed 25‐foot walk (after reciprocal transformation) were predicted by age and triplet repeat length in patients with FA. In addition, comparison of the temporal courses of change for each performance measure demonstrated that scores from the timed 25‐foot walk change early in the course of FA, nine‐hole peg test scores change slowly over the full course of the disorder, and low‐contrast letter acuity scores change in the later stages of the disease. Thus, a composite scale derived from these performance measures may provide the best overall measure for assessing disease progression throughout the illness. © 2005 Movement Disorder Society</abstract><note type="funding">Friedreich Ataxia Research Alliancem</note><note type="funding">Beeson Scholar Award from AFAR</note><note type="funding">National Institutes of Health - No. EY 00351; </note><subject lang="en"><genre>Keywords</genre><topic>composite measure</topic><topic>mitochondrial disease</topic><topic>antioxidant</topic><topic>cardiomyopathy</topic><topic>diabetes</topic></subject><relatedItem type="host"><titleInfo><title>Movement Disorders</title><subTitle>Official Journal of the Movement Disorder Society</subTitle></titleInfo><titleInfo type="abbreviated"><title>Mov. Disord.</title></titleInfo><subject><genre>article category</genre><topic>Research Article</topic></subject><identifier type="ISSN">0885-3185</identifier><identifier type="eISSN">1531-8257</identifier><identifier type="DOI">10.1002/(ISSN)1531-8257</identifier><identifier type="PublisherID">MDS</identifier><part><date>2005</date><detail type="volume"><caption>vol.</caption><number>20</number></detail><detail type="issue"><caption>no.</caption><number>7</number></detail><extent unit="pages"><start>777</start><end>782</end><total>6</total></extent></part></relatedItem><identifier type="istex">56C9C7F682A11ADFD80C1BD7B809FC29973612E2</identifier><identifier type="DOI">10.1002/mds.20449</identifier><identifier type="ArticleID">MDS20449</identifier><accessCondition type="use and reproduction" contentType="copyright">Copyright © 2005 Movement Disorder Society</accessCondition><recordInfo><recordOrigin>Wiley Subscription Services, Inc., A Wiley Company</recordOrigin><recordContentSource>WILEY</recordContentSource></recordInfo></mods></metadata><serie></serie></istex></record>Pour manipuler ce document sous Unix (Dilib)
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