Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Identifieur interne : 001B88 ( Main/Curation ); précédent : 001B87; suivant : 001B89Measuring 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.
Descripteurs français
- Pascal (Inist)
- Wicri :
- topic : Neurologie.
English descriptors
- KwdEn :
- Activities of Daily Living, Adult, Aged, Ataxia, Clinical Trials as Topic, Clinical trial, DNA, Mitochondrial (genetics), Disease Progression, Double-Blind Method, Female, Friedreich Ataxia (diagnosis), Friedreich Ataxia (genetics), Friedreich Ataxia (physiopathology), Friedreich ataxia, Humans, Iron-Binding Proteins (genetics), Male, Middle Aged, Mitochondrial disorder, Nervous system diseases, Neurologic Examination, Neurology, Point Mutation (genetics), Severity of Illness Index, Speech Disorders (diagnosis), Trinucleotide, 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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<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>
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<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
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<affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1>
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<author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name>
<affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles</s1>
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<sZ>3 aut.</sZ>
</inist:fA14>
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<author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name>
<affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Department of Neurology, Emory University</s1>
<s2>Atlanta, Georgia</s2>
<s3>USA</s3>
<sZ>4 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1>
<s2>Minneapolis, Minnesota</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
<sZ>6 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Minnesota</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="07"><s1>Department of Neurology, University of Chicago</s1>
<s2>Chicago, Illinois</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Illinois</region>
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</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"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1>
<s2>Minneapolis, Minnesota</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
<sZ>6 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="08"><s1>Departments of Neurology and Pediatrics, University of Iowa</s1>
<s2>Iowa City, Iowa</s2>
<s3>USA</s3>
<sZ>7 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1>
<s2>Galveston, Texas</s2>
<s3>USA</s3>
<sZ>8 aut.</sZ>
<sZ>9 aut.</sZ>
</inist:fA14>
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<placeName><region type="state">Texas</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Department of Neurology, University of Mississippi</s1>
<s2>Jackson, Mississippi</s2>
<s3>USA</s3>
<sZ>8 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1>
<s2>Galveston, Texas</s2>
<s3>USA</s3>
<sZ>8 aut.</sZ>
<sZ>9 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Texas</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="11"><s1>Department of Neurology, University of Florida</s1>
<s2>Gainesville, Florida</s2>
<s3>USA</s3>
<sZ>9 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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</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"><inist:fA14 i1="12"><s1>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>11 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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</inist:fA14>
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<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
<sZ>12 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
</author>
</titleStmt>
<publicationStmt><idno type="wicri:source">INIST</idno>
<idno type="inist">10-0178204</idno>
<date when="2010">2010</date>
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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">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>
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<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
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</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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<country>États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
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</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
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</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="04"><s1>Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles</s1>
<s2>Los Angeles, California</s2>
<s3>USA</s3>
<sZ>3 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="05"><s1>Department of Neurology, Emory University</s1>
<s2>Atlanta, Georgia</s2>
<s3>USA</s3>
<sZ>4 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1>
<s2>Minneapolis, Minnesota</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
<sZ>6 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Minnesota</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="07"><s1>Department of Neurology, University of Chicago</s1>
<s2>Chicago, Illinois</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1>
<s2>Minneapolis, Minnesota</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
<sZ>6 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="08"><s1>Departments of Neurology and Pediatrics, University of Iowa</s1>
<s2>Iowa City, Iowa</s2>
<s3>USA</s3>
<sZ>7 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1>
<s2>Galveston, Texas</s2>
<s3>USA</s3>
<sZ>8 aut.</sZ>
<sZ>9 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Texas</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Department of Neurology, University of Mississippi</s1>
<s2>Jackson, Mississippi</s2>
<s3>USA</s3>
<sZ>8 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1>
<s2>Galveston, Texas</s2>
<s3>USA</s3>
<sZ>8 aut.</sZ>
<sZ>9 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Texas</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="11"><s1>Department of Neurology, University of Florida</s1>
<s2>Gainesville, Florida</s2>
<s3>USA</s3>
<sZ>9 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
<sZ>10 aut.</sZ>
<sZ>12 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="12"><s1>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>11 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<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"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
<sZ>10 aut.</sZ>
<sZ>12 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
<sZ>12 aut.</sZ>
</inist:fA14>
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<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
<affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1>
<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
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</inist:fA14>
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<placeName><region type="state">Pennsylvanie</region>
</placeName>
</affiliation>
</author>
</analytic>
<series><title level="j" type="main">Movement disorders</title>
<title level="j" type="abbreviated">Mov. disord.</title>
<idno type="ISSN">0885-3185</idno>
<imprint><date when="2010">2010</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
<seriesStmt><title level="j" type="main">Movement disorders</title>
<title level="j" type="abbreviated">Mov. disord.</title>
<idno type="ISSN">0885-3185</idno>
</seriesStmt>
</fileDesc>
<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Ataxia</term>
<term>Clinical trial</term>
<term>Friedreich ataxia</term>
<term>Mitochondrial disorder</term>
<term>Nervous system diseases</term>
<term>Neurology</term>
<term>Trinucleotide</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Hérédodégénérescence spinocérébelleuse de Friedreich</term>
<term>Ataxie</term>
<term>Cytopathie mitochondriale</term>
<term>Pathologie du système nerveux</term>
<term>Essai clinique</term>
<term>Neurologie</term>
<term>Trinucléotide</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Neurologie</term>
</keywords>
</textClass>
</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.</div>
</front>
</TEI>
</INIST>
<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>
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<wicri:regionArea>Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea>
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</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>
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</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>
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</placeName>
</affiliation>
<affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country>
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<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>
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<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>
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<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>
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<wicri:regionArea>Department of Neurology, University of Mississippi, Jackson, Mississippi</wicri:regionArea>
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</placeName>
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<author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name>
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</affiliation>
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</placeName>
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<wicri:regionArea>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea>
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</placeName>
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</placeName>
</affiliation>
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<wicri:regionArea>Department of Pediatrics, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania</wicri:regionArea>
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</placeName>
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<series><title level="j">Movement Disorders</title>
<title level="j" type="abbrev">Mov. Disord.</title>
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<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>
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<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>
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<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>
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<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>
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