Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Identifieur interne : 002024 ( Main/Merge ); précédent : 002023; suivant : 002025Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Auteurs : Lisa S. Friedman [États-Unis] ; Jennifer M. Farmer [États-Unis] ; Susan Perlman [États-Unis] ; George Wilmot [États-Unis] ; Christopher M. Gomez [États-Unis] ; Khalaf O. Bushara [États-Unis] ; Katherine D. Mathews [États-Unis] ; S. H. Subramony [États-Unis] ; Tetsuo Ashizawa [États-Unis] ; Laura J. Balcer [États-Unis] ; Robert B. Wilson [États-Unis] ; David R. Lynch [États-Unis]Source :
- Movement Disorders [ 0885-3185 ] ; 2010-03-15.
English descriptors
- KwdEn :
- Activities of Daily Living, Adult, Aged, Clinical Trials as Topic, DNA, Mitochondrial (genetics), Disease Progression, Double-Blind Method, Female, Friedreich Ataxia (diagnosis), Friedreich Ataxia (genetics), Friedreich Ataxia (physiopathology), Humans, Iron-Binding Proteins (genetics), Male, Middle Aged, Neurologic Examination, Point Mutation (genetics), Severity of Illness Index, Speech Disorders (diagnosis), Trinucleotide Repeats (genetics), Walking, Young Adult, ataxia, clinical neurology examination, mitochondrial disorder, natural history study, trinucleotide repeat disease.
- MESH :
- chemical , genetics : DNA, Mitochondrial, Iron-Binding Proteins.
- diagnosis : Friedreich Ataxia, Speech Disorders.
- genetics : Friedreich Ataxia, Point Mutation, Trinucleotide Repeats.
- physiopathology : Friedreich Ataxia.
- Activities of Daily Living, Adult, Aged, Clinical Trials as Topic, Disease Progression, Double-Blind Method, Female, Humans, Male, Middle Aged, Neurologic Examination, Severity of Illness Index, Walking, Young Adult.
Abstract
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner‐rated functional disability scales, self‐reported activities of daily living and performance measures such as the timed 25‐foot walk, 9‐hole pegboard test, PATA speech test, and low‐contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance‐measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long‐term success of therapeutic agents and defining sample‐size calculations for double‐blind clinical trials. © 2010 Movement Disorder Society
Url:
- https://api.istex.fr/document/A06CB388546EB8D4B4B45FDF7F94DCE8AC0A1CCF/fulltext/pdf
- http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2954653
DOI: 10.1002/mds.22912
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Bushara</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Minnesota, Minneapolis, Minnesota</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D." last="Mathews">Katherine D. Mathews</name><affiliation wicri:level="4"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Departments of Neurology and Pediatrics, University of Iowa, Iowa City, Iowa</wicri:regionArea><placeName><region type="state">Iowa</region><settlement type="city">Iowa City</settlement></placeName><orgName type="university">Université de l'Iowa</orgName></affiliation></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Texas Medical Branch, Galveston, Texas</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Mississippi, Jackson, Mississippi</wicri:regionArea><placeName><region type="state">État du Mississippi</region></placeName></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Texas Medical Branch, Galveston, Texas</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Florida, Gainesville, Florida</wicri:regionArea><placeName><region type="state">Floride</region></placeName></affiliation></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J." last="Balcer">Laura J. Balcer</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B." last="Wilson">Robert B. Wilson</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children's Hospital of Philadelphia, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></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="2010-03-15">2010-03-15</date><biblScope unit="vol">25</biblScope><biblScope unit="issue">4</biblScope><biblScope unit="page" from="426">426</biblScope><biblScope unit="page" to="432">432</biblScope></imprint><idno type="ISSN">0885-3185</idno></series><idno type="istex">A06CB388546EB8D4B4B45FDF7F94DCE8AC0A1CCF</idno><idno type="DOI">10.1002/mds.22912</idno><idno type="ArticleID">MDS22912</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0885-3185</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Activities of Daily Living</term><term>Adult</term><term>Aged</term><term>Clinical Trials as Topic</term><term>DNA, Mitochondrial (genetics)</term><term>Disease Progression</term><term>Double-Blind Method</term><term>Female</term><term>Friedreich Ataxia (diagnosis)</term><term>Friedreich Ataxia (genetics)</term><term>Friedreich Ataxia (physiopathology)</term><term>Humans</term><term>Iron-Binding Proteins (genetics)</term><term>Male</term><term>Middle Aged</term><term>Neurologic Examination</term><term>Point Mutation (genetics)</term><term>Severity of Illness Index</term><term>Speech Disorders (diagnosis)</term><term>Trinucleotide Repeats (genetics)</term><term>Walking</term><term>Young Adult</term><term>ataxia</term><term>clinical neurology examination</term><term>mitochondrial disorder</term><term>natural history study</term><term>trinucleotide repeat disease</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>DNA, Mitochondrial</term><term>Iron-Binding Proteins</term></keywords><keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Friedreich Ataxia</term><term>Speech Disorders</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Friedreich Ataxia</term><term>Point Mutation</term><term>Trinucleotide Repeats</term></keywords><keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Activities of Daily Living</term><term>Adult</term><term>Aged</term><term>Clinical Trials as Topic</term><term>Disease Progression</term><term>Double-Blind Method</term><term>Female</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Neurologic Examination</term><term>Severity of Illness Index</term><term>Walking</term><term>Young Adult</term></keywords></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner‐rated functional disability scales, self‐reported activities of daily living and performance measures such as the timed 25‐foot walk, 9‐hole pegboard test, PATA speech test, and low‐contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance‐measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long‐term success of therapeutic agents and defining sample‐size calculations for double‐blind clinical trials. © 2010 Movement Disorder Society</div></front></TEI><double doi="10.1002/mds.22912"><ISTEX><TEI wicri:istexFullTextTei="biblStruct"><teiHeader><fileDesc><titleStmt><title xml:lang="en">Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design</title><author><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name></author><author><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name></author><author><name sortKey="Gomez, Christopher M" sort="Gomez, Christopher M" uniqKey="Gomez C" first="Christopher M." last="Gomez">Christopher M. Gomez</name></author><author><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O." last="Bushara">Khalaf O. Bushara</name></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D." last="Mathews">Katherine D. Mathews</name></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J." last="Balcer">Laura J. Balcer</name></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B." last="Wilson">Robert B. Wilson</name></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name></author></titleStmt><publicationStmt><idno type="wicri:source">ISTEX</idno><idno type="RBID">ISTEX:A06CB388546EB8D4B4B45FDF7F94DCE8AC0A1CCF</idno><date when="2010" year="2010">2010</date><idno type="doi">10.1002/mds.22912</idno><idno type="url">https://api.istex.fr/document/A06CB388546EB8D4B4B45FDF7F94DCE8AC0A1CCF/fulltext/pdf</idno><idno type="wicri:Area/Istex/Corpus">001341</idno><idno type="wicri:Area/Istex/Curation">001341</idno><idno type="wicri:Area/Istex/Checkpoint">000867</idno><idno type="wicri:doubleKey">0885-3185:2010:Friedman L:measuring:the:rate</idno></publicationStmt><sourceDesc><biblStruct><analytic><title level="a" type="main" xml:lang="en">Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design</title><author><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children's Hospital of Philadelphia, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></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 wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children's Hospital of Philadelphia, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles, Los Angeles, California</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, Emory University, Atlanta, Georgia</wicri:regionArea><placeName><region type="state">Géorgie (États-Unis)</region></placeName></affiliation></author><author><name sortKey="Gomez, Christopher M" sort="Gomez, Christopher M" uniqKey="Gomez C" first="Christopher M." last="Gomez">Christopher M. Gomez</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Minnesota, Minneapolis, Minnesota</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Chicago, Chicago, Illinois</wicri:regionArea><placeName><region type="state">Illinois</region></placeName></affiliation></author><author><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O." last="Bushara">Khalaf O. Bushara</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Minnesota, Minneapolis, Minnesota</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D." last="Mathews">Katherine D. Mathews</name><affiliation wicri:level="4"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Departments of Neurology and Pediatrics, University of Iowa, Iowa City, Iowa</wicri:regionArea><placeName><region type="state">Iowa</region><settlement type="city">Iowa City</settlement></placeName><orgName type="university">Université de l'Iowa</orgName></affiliation></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Texas Medical Branch, Galveston, Texas</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Mississippi, Jackson, Mississippi</wicri:regionArea><placeName><region type="state">État du Mississippi</region></placeName></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Texas Medical Branch, Galveston, Texas</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Florida, Gainesville, Florida</wicri:regionArea><placeName><region type="state">Floride</region></placeName></affiliation></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J." last="Balcer">Laura J. Balcer</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B." last="Wilson">Robert B. Wilson</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children's Hospital of Philadelphia, Philadelphia, Pennsylvania</wicri:regionArea><placeName><region type="state">Pennsylvanie</region></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="2010-03-15">2010-03-15</date><biblScope unit="vol">25</biblScope><biblScope unit="issue">4</biblScope><biblScope unit="page" from="426">426</biblScope><biblScope unit="page" to="432">432</biblScope></imprint><idno type="ISSN">0885-3185</idno></series><idno type="istex">A06CB388546EB8D4B4B45FDF7F94DCE8AC0A1CCF</idno><idno type="DOI">10.1002/mds.22912</idno><idno type="ArticleID">MDS22912</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0885-3185</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>ataxia</term><term>clinical neurology examination</term><term>mitochondrial disorder</term><term>natural history study</term><term>trinucleotide repeat disease</term></keywords></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner‐rated functional disability scales, self‐reported activities of daily living and performance measures such as the timed 25‐foot walk, 9‐hole pegboard test, PATA speech test, and low‐contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance‐measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long‐term success of therapeutic agents and defining sample‐size calculations for double‐blind clinical trials. © 2010 Movement Disorder Society</div></front></TEI></ISTEX><PMC><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design</title><author><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S" last="Friedman">Lisa S. Friedman</name><affiliation wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pediatrics, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A3">Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104</wicri:regionArea><wicri:noRegion>19104</wicri:noRegion></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 wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pediatrics, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A3">Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104</wicri:regionArea><wicri:noRegion>19104</wicri:noRegion></affiliation></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><affiliation wicri:level="2"><nlm:aff id="A4">University of California Los Angeles, Los Angeles, CA, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of California Los Angeles, Los Angeles, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><affiliation wicri:level="2"><nlm:aff id="A5">Emory University, Atlanta, GA, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Emory University, Atlanta, GA</wicri:regionArea><placeName><region type="state">Géorgie (États-Unis)</region></placeName></affiliation></author><author><name sortKey="Gomez, Christopher" sort="Gomez, Christopher" uniqKey="Gomez C" first="Christopher" last="Gomez">Christopher Gomez</name><affiliation wicri:level="2"><nlm:aff id="A6">University of Minnesota, Minneapolis, MN, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Minnesota, Minneapolis, MN</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation><affiliation wicri:level="2"><nlm:aff id="A7">University of Chicago, Chicago, IL, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Chicago, Chicago, IL</wicri:regionArea><placeName><region type="state">Illinois</region></placeName></affiliation></author><author><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O" last="Bushara">Khalaf O. Bushara</name><affiliation wicri:level="2"><nlm:aff id="A6">University of Minnesota, Minneapolis, MN, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Minnesota, Minneapolis, MN</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D" last="Mathews">Katherine D. Mathews</name><affiliation wicri:level="4"><nlm:aff id="A8">University of Iowa, Iowa City, IA, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Iowa, Iowa City, IA</wicri:regionArea><placeName><region type="state">Iowa</region><settlement type="city">Iowa City</settlement></placeName><orgName type="university">Université de l'Iowa</orgName></affiliation></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H" last="Subramony">S. H. Subramony</name><affiliation wicri:level="2"><nlm:aff id="A9">University of Texas Medical Branch, Galveston, TX, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Texas Medical Branch, Galveston, TX</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><nlm:aff id="A10">University of Mississippi, Jackson, MS, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Mississippi, Jackson, MS</wicri:regionArea><placeName><region type="state">État du Mississippi</region></placeName></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="2"><nlm:aff id="A9">University of Texas Medical Branch, Galveston, TX, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Texas Medical Branch, Galveston, TX</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><nlm:aff id="A11">University of Florida, Gainesville, FL, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Florida, Gainesville, FL</wicri:regionArea><placeName><region type="state">Floride</region></placeName></affiliation></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J" last="Balcer">Laura J. Balcer</name><affiliation wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B" last="Wilson">Robert B. Wilson</name><affiliation wicri:level="1"><nlm:aff id="A12">Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R" last="Lynch">David R. Lynch</name><affiliation wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pediatrics, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A3">Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104</wicri:regionArea><wicri:noRegion>19104</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">20063431</idno><idno type="pmc">2954653</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2954653</idno><idno type="RBID">PMC:2954653</idno><idno type="doi">10.1002/mds.22912</idno><date when="2010">2010</date><idno type="wicri:Area/Pmc/Corpus">000075</idno><idno type="wicri:Area/Pmc/Curation">000075</idno><idno type="wicri:Area/Pmc/Checkpoint">000329</idno><idno type="wicri:source">PubMed</idno><idno type="wicri:Area/PubMed/Corpus">001A34</idno><idno type="wicri:Area/PubMed/Curation">001A34</idno><idno type="wicri:Area/PubMed/Checkpoint">001785</idno><idno type="wicri:Area/Ncbi/Merge">002968</idno><idno type="wicri:Area/Ncbi/Curation">002968</idno><idno type="wicri:Area/Ncbi/Checkpoint">002968</idno><idno type="wicri:doubleKey">0885-3185:2010:Friedman L:measuring:the:rate</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design</title><author><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S" last="Friedman">Lisa S. Friedman</name><affiliation wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pediatrics, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A3">Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104</wicri:regionArea><wicri:noRegion>19104</wicri:noRegion></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 wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pediatrics, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A3">Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104</wicri:regionArea><wicri:noRegion>19104</wicri:noRegion></affiliation></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><affiliation wicri:level="2"><nlm:aff id="A4">University of California Los Angeles, Los Angeles, CA, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of California Los Angeles, Los Angeles, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><affiliation wicri:level="2"><nlm:aff id="A5">Emory University, Atlanta, GA, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Emory University, Atlanta, GA</wicri:regionArea><placeName><region type="state">Géorgie (États-Unis)</region></placeName></affiliation></author><author><name sortKey="Gomez, Christopher" sort="Gomez, Christopher" uniqKey="Gomez C" first="Christopher" last="Gomez">Christopher Gomez</name><affiliation wicri:level="2"><nlm:aff id="A6">University of Minnesota, Minneapolis, MN, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Minnesota, Minneapolis, MN</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation><affiliation wicri:level="2"><nlm:aff id="A7">University of Chicago, Chicago, IL, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Chicago, Chicago, IL</wicri:regionArea><placeName><region type="state">Illinois</region></placeName></affiliation></author><author><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O" last="Bushara">Khalaf O. Bushara</name><affiliation wicri:level="2"><nlm:aff id="A6">University of Minnesota, Minneapolis, MN, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Minnesota, Minneapolis, MN</wicri:regionArea><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D" last="Mathews">Katherine D. Mathews</name><affiliation wicri:level="4"><nlm:aff id="A8">University of Iowa, Iowa City, IA, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Iowa, Iowa City, IA</wicri:regionArea><placeName><region type="state">Iowa</region><settlement type="city">Iowa City</settlement></placeName><orgName type="university">Université de l'Iowa</orgName></affiliation></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H" last="Subramony">S. H. Subramony</name><affiliation wicri:level="2"><nlm:aff id="A9">University of Texas Medical Branch, Galveston, TX, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Texas Medical Branch, Galveston, TX</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><nlm:aff id="A10">University of Mississippi, Jackson, MS, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Mississippi, Jackson, MS</wicri:regionArea><placeName><region type="state">État du Mississippi</region></placeName></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="2"><nlm:aff id="A9">University of Texas Medical Branch, Galveston, TX, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Texas Medical Branch, Galveston, TX</wicri:regionArea><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><nlm:aff id="A11">University of Florida, Gainesville, FL, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>University of Florida, Gainesville, FL</wicri:regionArea><placeName><region type="state">Floride</region></placeName></affiliation></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J" last="Balcer">Laura J. Balcer</name><affiliation wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B" last="Wilson">Robert B. Wilson</name><affiliation wicri:level="1"><nlm:aff id="A12">Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R" last="Lynch">David R. Lynch</name><affiliation wicri:level="1"><nlm:aff id="A1">Department of Neurology, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A2">Department of Pediatrics, University of Pennsylvania School of Medicine, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Pediatrics, University of Pennsylvania School of Medicine</wicri:regionArea><wicri:noRegion>University of Pennsylvania School of Medicine</wicri:noRegion></affiliation><affiliation wicri:level="1"><nlm:aff id="A3">Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104</wicri:regionArea><wicri:noRegion>19104</wicri:noRegion></affiliation></author></analytic><series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title><idno type="ISSN">0885-3185</idno><idno type="eISSN">1531-8257</idno><imprint><date when="2010">2010</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Activities of Daily Living</term><term>Adult</term><term>Aged</term><term>Clinical Trials as Topic</term><term>DNA, Mitochondrial (genetics)</term><term>Disease Progression</term><term>Double-Blind Method</term><term>Female</term><term>Friedreich Ataxia (diagnosis)</term><term>Friedreich Ataxia (genetics)</term><term>Friedreich Ataxia (physiopathology)</term><term>Humans</term><term>Iron-Binding Proteins (genetics)</term><term>Male</term><term>Middle Aged</term><term>Neurologic Examination</term><term>Point Mutation (genetics)</term><term>Severity of Illness Index</term><term>Speech Disorders (diagnosis)</term><term>Trinucleotide Repeats (genetics)</term><term>Walking</term><term>Young Adult</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>DNA, Mitochondrial</term><term>Iron-Binding Proteins</term></keywords><keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Friedreich Ataxia</term><term>Speech Disorders</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Friedreich Ataxia</term><term>Point Mutation</term><term>Trinucleotide Repeats</term></keywords><keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Activities of Daily Living</term><term>Adult</term><term>Aged</term><term>Clinical Trials as Topic</term><term>Disease Progression</term><term>Double-Blind Method</term><term>Female</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Neurologic Examination</term><term>Severity of Illness Index</term><term>Walking</term><term>Young Adult</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p id="P1">Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner-rated functional disability scales, self-reported activities of daily living and performance measures such as the timed 25-foot walk, 9-hole pegboard test, PATA speech test, and low-contrast letter acuity vision charts. The present study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance-measure composites captured disease progression, with a greater sensitivity to change over two years than over one year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long-term success of therapeutic agents and defining sample-size calculations for double-blind clinical trials.</p></div></front></TEI></PMC></double></record>Pour manipuler ce document sous Unix (Dilib)
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