Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Identifieur interne : 000075 ( Pmc/Curation ); précédent : 000074; suivant : 000076Measuring 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 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 : official journal of the Movement Disorder Society [ 0885-3185 ] ; 2010.
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. 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.
Url:
DOI: 10.1002/mds.22912
PubMed: 20063431
PubMed Central: 2954653
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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></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></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></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></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></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></affiliation></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><affiliation wicri:level="1"><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></affiliation></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><affiliation wicri:level="1"><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></affiliation></author><author><name sortKey="Gomez, Christopher" sort="Gomez, Christopher" uniqKey="Gomez C" first="Christopher" last="Gomez">Christopher Gomez</name><affiliation wicri:level="1"><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></affiliation><affiliation wicri:level="1"><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></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="1"><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></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="1"><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></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="1"><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></affiliation><affiliation wicri:level="1"><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></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="1"><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></affiliation><affiliation wicri:level="1"><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></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></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></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></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></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></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></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 article-type="research-article" xml:lang="EN"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<pmc-dir>properties manuscript</pmc-dir>
<front><journal-meta><journal-id journal-id-type="nlm-journal-id">8610688</journal-id><journal-id journal-id-type="pubmed-jr-id">5937</journal-id><journal-id journal-id-type="nlm-ta">Mov Disord</journal-id><journal-title>Movement disorders : official journal of the Movement Disorder Society</journal-title><issn pub-type="ppub">0885-3185</issn><issn pub-type="epub">1531-8257</issn></journal-meta><article-meta><article-id pub-id-type="pmid">20063431</article-id><article-id pub-id-type="pmc">2954653</article-id><article-id pub-id-type="doi">10.1002/mds.22912</article-id><article-id pub-id-type="manuscript">NIHMS217747</article-id><article-categories><subj-group subj-group-type="heading"><subject>Article</subject></subj-group></article-categories><title-group><article-title>Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Friedman</surname><given-names>Lisa S</given-names></name><xref ref-type="aff" rid="A1">a</xref><xref ref-type="aff" rid="A2">b</xref><xref ref-type="aff" rid="A3">c</xref></contrib><contrib contrib-type="author"><name><surname>Farmer</surname><given-names>Jennifer M</given-names></name><xref ref-type="aff" rid="A1">a</xref><xref ref-type="aff" rid="A2">b</xref><xref ref-type="aff" rid="A3">c</xref></contrib><contrib contrib-type="author"><name><surname>Perlman</surname><given-names>Susan</given-names></name><xref ref-type="aff" rid="A4">d</xref></contrib><contrib contrib-type="author"><name><surname>Wilmot</surname><given-names>George</given-names></name><xref ref-type="aff" rid="A5">e</xref></contrib><contrib contrib-type="author"><name><surname>Gomez</surname><given-names>Christopher</given-names></name><xref ref-type="aff" rid="A6">f</xref><xref ref-type="aff" rid="A7">g</xref></contrib><contrib contrib-type="author"><name><surname>Bushara</surname><given-names>Khalaf O</given-names></name><xref ref-type="aff" rid="A6">f</xref></contrib><contrib contrib-type="author"><name><surname>Mathews</surname><given-names>Katherine D</given-names></name><xref ref-type="aff" rid="A8">h</xref></contrib><contrib contrib-type="author"><name><surname>Subramony</surname><given-names>S. H</given-names></name><xref ref-type="aff" rid="A9">i</xref><xref ref-type="aff" rid="A10">j</xref></contrib><contrib contrib-type="author"><name><surname>Ashizawa</surname><given-names>Tetsuo</given-names></name><xref ref-type="aff" rid="A9">i</xref><xref ref-type="aff" rid="A11">k</xref></contrib><contrib contrib-type="author"><name><surname>Balcer</surname><given-names>Laura J</given-names></name><xref ref-type="aff" rid="A1">a</xref></contrib><contrib contrib-type="author"><name><surname>Wilson</surname><given-names>Robert B</given-names></name><xref ref-type="aff" rid="A12">l</xref></contrib><contrib contrib-type="author"><name><surname>Lynch</surname><given-names>David R</given-names></name><xref ref-type="aff" rid="A1">a</xref><xref ref-type="aff" rid="A2">b</xref><xref ref-type="aff" rid="A3">c</xref><xref ref-type="corresp" rid="cor1">+</xref></contrib></contrib-group><aff id="A1"><label>a</label>Department of Neurology, University of Pennsylvania School of Medicine, United States</aff><aff id="A2"><label>b</label>Department of Pediatrics, University of Pennsylvania School of Medicine, United States</aff><aff id="A3"><label>c</label>Children’s Hospital of Philadelphia, Philadelphia, PA, 19104, United States</aff><aff id="A4"><label>d</label>University of California Los Angeles, Los Angeles, CA, United States</aff><aff id="A5"><label>e</label>Emory University, Atlanta, GA, United States</aff><aff id="A6"><label>f</label>University of Minnesota, Minneapolis, MN, United States</aff><aff id="A7"><label>g</label>University of Chicago, Chicago, IL, United States</aff><aff id="A8"><label>h</label>University of Iowa, Iowa City, IA, United States</aff><aff id="A9"><label>i</label>University of Texas Medical Branch, Galveston, TX, United States</aff><aff id="A10"><label>j</label>University of Mississippi, Jackson, MS, United States</aff><aff id="A11"><label>k</label>University of Florida, Gainesville, FL, United States</aff><aff id="A12"><label>l</label>Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, United States</aff><author-notes><corresp id="cor1"><label>+</label>Address correspondence to: David R. Lynch, MD, PhD, Children’s Hospital of Philadelphia, Department of Neurology, Abramson Research Center, Room 502, 3615 Civic Center Blvd, Philadelphia, PA 19104, Tel: 1 215 590 2242, Fax: 1 215 590 3779, <email>lynchd@mail.med.upenn.edu</email></corresp></author-notes><pub-date pub-type="nihms-submitted"><day>28</day><month>9</month><year>2010</year></pub-date><pub-date pub-type="ppub"><day>15</day><month>3</month><year>2010</year></pub-date><pub-date pub-type="pmc-release"><day>14</day><month>10</month><year>2010</year></pub-date><volume>25</volume><issue>4</issue><fpage>426</fpage><lpage>432</lpage><abstract><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></abstract><kwd-group><kwd>ataxia</kwd><kwd>natural history study</kwd><kwd>clinical neurology examination</kwd><kwd>mitochondrial disorder</kwd><kwd>trinucleotide repeat disease</kwd></kwd-group><contract-num rid="NS1">RC1 NS068897-01
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