A responsive outcome for Parkinson's disease neuroprotection futility studies
Identifieur interne : 001541 ( Main/Exploration ); précédent : 001540; suivant : 001542A responsive outcome for Parkinson's disease neuroprotection futility studies
Auteurs : Jordan J. Elm [États-Unis] ; Christopher G. Goetz [États-Unis] ; Bernard Ravina [États-Unis] ; Kathleen Shannon [États-Unis] ; George Fredrick Wooten [États-Unis] ; Caroline M. Tanner [États-Unis] ; Yuko Y. Palesch [États-Unis] ; Peng Huang [États-Unis] ; Paulo Guimaraes [États-Unis] ; Cornelia Kamp [États-Unis] ; Barbara C. Tilley [États-Unis] ; Karl Kieburtz [États-Unis]Source :
- Annals of Neurology [ 0364-5134 ] ; 2005-02.
Abstract
Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. Ann Neurol 2005;57:197–203
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DOI: 10.1002/ana.20361
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Tanner</name><affiliation wicri:level="2"><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>The Parkinson's Institute, Sunnyvale</wicri:cityArea></affiliation></author><author><name sortKey="Palesch, Yuko Y" sort="Palesch, Yuko Y" uniqKey="Palesch Y" first="Yuko Y." last="Palesch">Yuko Y. 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An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short‐term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies. Ann Neurol 2005;57:197–203</div></front></TEI><affiliations><list><country><li>États-Unis</li></country><region><li>Californie</li><li>Caroline du Sud</li><li>Illinois</li><li>Maryland</li><li>Virginie</li><li>État de New York</li></region></list><tree><country name="États-Unis"><region name="Caroline du Sud"><name sortKey="Elm, Jordan J" sort="Elm, Jordan J" uniqKey="Elm J" first="Jordan J." last="Elm">Jordan J. Elm</name></region><name sortKey="Goetz, Christopher G" sort="Goetz, Christopher G" uniqKey="Goetz C" first="Christopher G." last="Goetz">Christopher G. Goetz</name><name sortKey="Guimaraes, Paulo" sort="Guimaraes, Paulo" uniqKey="Guimaraes P" first="Paulo" last="Guimaraes">Paulo Guimaraes</name><name sortKey="Huang, Peng" sort="Huang, Peng" uniqKey="Huang P" first="Peng" last="Huang">Peng Huang</name><name sortKey="Kamp, Cornelia" sort="Kamp, Cornelia" uniqKey="Kamp C" first="Cornelia" last="Kamp">Cornelia Kamp</name><name sortKey="Kieburtz, Karl" sort="Kieburtz, Karl" uniqKey="Kieburtz K" first="Karl" last="Kieburtz">Karl Kieburtz</name><name sortKey="Palesch, Yuko Y" sort="Palesch, Yuko Y" uniqKey="Palesch Y" first="Yuko Y." last="Palesch">Yuko Y. Palesch</name><name sortKey="Ravina, Bernard" sort="Ravina, Bernard" uniqKey="Ravina B" first="Bernard" last="Ravina">Bernard Ravina</name><name sortKey="Shannon, Kathleen" sort="Shannon, Kathleen" uniqKey="Shannon K" first="Kathleen" last="Shannon">Kathleen Shannon</name><name sortKey="Tanner, Caroline M" sort="Tanner, Caroline M" uniqKey="Tanner C" first="Caroline M." last="Tanner">Caroline M. Tanner</name><name sortKey="Tilley, Barbara C" sort="Tilley, Barbara C" uniqKey="Tilley B" first="Barbara C." last="Tilley">Barbara C. Tilley</name><name sortKey="Wooten, George Fredrick" sort="Wooten, George Fredrick" uniqKey="Wooten G" first="George Fredrick" last="Wooten">George Fredrick Wooten</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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