Potential outcome measures and trial design issues for multiple system atrophy.
Identifieur interne : 002498 ( PubMed/Curation ); précédent : 002497; suivant : 002499Potential outcome measures and trial design issues for multiple system atrophy.
Auteurs : Susanne May [États-Unis] ; Sid Gilman ; B Brooke Sowell ; Ronald G. Thomas ; Matthew B. Stern ; Amy Colcher ; Caroline M. Tanner ; Neng Huang ; Peter Novak ; Stephen G. Reich ; Joseph Jankovic ; William G. Ondo ; Phillip A. Low ; Paola Sandroni ; Axel Lipp ; Frederick J. Marshall ; Frederick Wooten ; Clifford W. ShultsSource :
- Movement disorders : official journal of the Movement Disorder Society [ 0885-3185 ] ; 2007.
English descriptors
- KwdEn :
- Aged, Antiparkinson Agents (therapeutic use), Cerebellar Diseases (physiopathology), Cerebellar Diseases (therapy), Clinical Trials as Topic, Disability Evaluation, Disease Progression, Female, Humans, Levodopa (therapeutic use), Male, Middle Aged, Multiple System Atrophy (epidemiology), Multiple System Atrophy (physiopathology), Multiple System Atrophy (therapy), Research Design, Risk Factors, Sample Size, Socioeconomic Factors, Treatment Outcome.
- MESH :
- chemical , therapeutic use : Antiparkinson Agents, Levodopa.
- epidemiology : Multiple System Atrophy.
- physiopathology : Cerebellar Diseases, Multiple System Atrophy.
- therapy : Cerebellar Diseases, Multiple System Atrophy.
- Aged, Clinical Trials as Topic, Disability Evaluation, Disease Progression, Female, Humans, Male, Middle Aged, Research Design, Risk Factors, Sample Size, Socioeconomic Factors, Treatment Outcome.
Abstract
Multiple system atrophy (MSA) is a neurodegenerative disorder exhibiting a combination of parkinsonism, cerebellar ataxia, and autonomic failure. A disease-specific scale, the Unified Multiple System Atrophy Rating Scale (UMSARS), has been developed and validated to measure progression of MSA, but its use as an outcome measure for therapeutic trials has not been evaluated. On the basis of twelve months of follow-up from an observational study of 67 patients with probable MSA, we evaluated three disease-specific scores: Activities of Daily Living, Motor Examination, and a combined score from the UMSARS and two general health scores, the Physical Health and Mental Health scores of the SF-36 health survey, for their use as outcome measures in a therapeutic trial. We discuss related design issues and provide sample size estimates. Scores based on the disease-specific UMSARS seemed to be equal or superior to scores based on the SF-36 health survey. They appeared to capture disease progression, were well correlated and required the smallest sample size. The UMSARS Motor Examination score exhibited the most favorable characteristics as an outcome measure for a therapeutic trial in MSA with 1 year of follow-up.
DOI: 10.1002/mds.21734
PubMed: 17914727
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Reich</name></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name></author><author><name sortKey="Ondo, William G" sort="Ondo, William G" uniqKey="Ondo W" first="William G" last="Ondo">William G. Ondo</name></author><author><name sortKey="Low, Phillip A" sort="Low, Phillip A" uniqKey="Low P" first="Phillip A" last="Low">Phillip A. Low</name></author><author><name sortKey="Sandroni, Paola" sort="Sandroni, Paola" uniqKey="Sandroni P" first="Paola" last="Sandroni">Paola Sandroni</name></author><author><name sortKey="Lipp, Axel" sort="Lipp, Axel" uniqKey="Lipp A" first="Axel" last="Lipp">Axel Lipp</name></author><author><name sortKey="Marshall, Frederick J" sort="Marshall, Frederick J" uniqKey="Marshall F" first="Frederick J" last="Marshall">Frederick J. 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Thomas</name></author><author><name sortKey="Stern, Matthew B" sort="Stern, Matthew B" uniqKey="Stern M" first="Matthew B" last="Stern">Matthew B. Stern</name></author><author><name sortKey="Colcher, Amy" sort="Colcher, Amy" uniqKey="Colcher A" first="Amy" last="Colcher">Amy Colcher</name></author><author><name sortKey="Tanner, Caroline M" sort="Tanner, Caroline M" uniqKey="Tanner C" first="Caroline M" last="Tanner">Caroline M. Tanner</name></author><author><name sortKey="Huang, Neng" sort="Huang, Neng" uniqKey="Huang N" first="Neng" last="Huang">Neng Huang</name></author><author><name sortKey="Novak, Peter" sort="Novak, Peter" uniqKey="Novak P" first="Peter" last="Novak">Peter Novak</name></author><author><name sortKey="Reich, Stephen G" sort="Reich, Stephen G" uniqKey="Reich S" first="Stephen G" last="Reich">Stephen G. Reich</name></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name></author><author><name sortKey="Ondo, William G" sort="Ondo, William G" uniqKey="Ondo W" first="William G" last="Ondo">William G. Ondo</name></author><author><name sortKey="Low, Phillip A" sort="Low, Phillip A" uniqKey="Low P" first="Phillip A" last="Low">Phillip A. Low</name></author><author><name sortKey="Sandroni, Paola" sort="Sandroni, Paola" uniqKey="Sandroni P" first="Paola" last="Sandroni">Paola Sandroni</name></author><author><name sortKey="Lipp, Axel" sort="Lipp, Axel" uniqKey="Lipp A" first="Axel" last="Lipp">Axel Lipp</name></author><author><name sortKey="Marshall, Frederick J" sort="Marshall, Frederick J" uniqKey="Marshall F" first="Frederick J" last="Marshall">Frederick J. Marshall</name></author><author><name sortKey="Wooten, Frederick" sort="Wooten, Frederick" uniqKey="Wooten F" first="Frederick" last="Wooten">Frederick Wooten</name></author><author><name sortKey="Shults, Clifford W" sort="Shults, Clifford W" uniqKey="Shults C" first="Clifford W" last="Shults">Clifford W. Shults</name></author></analytic><series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title><idno type="ISSN">0885-3185</idno><imprint><date when="2007" type="published">2007</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Aged</term><term>Antiparkinson Agents (therapeutic use)</term><term>Cerebellar Diseases (physiopathology)</term><term>Cerebellar Diseases (therapy)</term><term>Clinical Trials as Topic</term><term>Disability Evaluation</term><term>Disease Progression</term><term>Female</term><term>Humans</term><term>Levodopa (therapeutic use)</term><term>Male</term><term>Middle Aged</term><term>Multiple System Atrophy (epidemiology)</term><term>Multiple System Atrophy (physiopathology)</term><term>Multiple System Atrophy (therapy)</term><term>Research Design</term><term>Risk Factors</term><term>Sample Size</term><term>Socioeconomic Factors</term><term>Treatment Outcome</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Antiparkinson Agents</term><term>Levodopa</term></keywords><keywords scheme="MESH" qualifier="epidemiology" xml:lang="en"><term>Multiple System Atrophy</term></keywords><keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Cerebellar Diseases</term><term>Multiple System Atrophy</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Cerebellar Diseases</term><term>Multiple System Atrophy</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Aged</term><term>Clinical Trials as Topic</term><term>Disability Evaluation</term><term>Disease Progression</term><term>Female</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Research Design</term><term>Risk Factors</term><term>Sample Size</term><term>Socioeconomic Factors</term><term>Treatment Outcome</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Multiple system atrophy (MSA) is a neurodegenerative disorder exhibiting a combination of parkinsonism, cerebellar ataxia, and autonomic failure. A disease-specific scale, the Unified Multiple System Atrophy Rating Scale (UMSARS), has been developed and validated to measure progression of MSA, but its use as an outcome measure for therapeutic trials has not been evaluated. On the basis of twelve months of follow-up from an observational study of 67 patients with probable MSA, we evaluated three disease-specific scores: Activities of Daily Living, Motor Examination, and a combined score from the UMSARS and two general health scores, the Physical Health and Mental Health scores of the SF-36 health survey, for their use as outcome measures in a therapeutic trial. We discuss related design issues and provide sample size estimates. Scores based on the disease-specific UMSARS seemed to be equal or superior to scores based on the SF-36 health survey. They appeared to capture disease progression, were well correlated and required the smallest sample size. The UMSARS Motor Examination score exhibited the most favorable characteristics as an outcome measure for a therapeutic trial in MSA with 1 year of follow-up.</div></front></TEI><pubmed><MedlineCitation Owner="NLM" Status="MEDLINE"><PMID Version="1">17914727</PMID><DateCreated><Year>2007</Year><Month>12</Month><Day>27</Day></DateCreated><DateCompleted><Year>2008</Year><Month>02</Month><Day>20</Day></DateCompleted><DateRevised><Year>2013</Year><Month>11</Month><Day>21</Day></DateRevised><Article PubModel="Print"><Journal><ISSN IssnType="Print">0885-3185</ISSN><JournalIssue CitedMedium="Print"><Volume>22</Volume><Issue>16</Issue><PubDate><Year>2007</Year><Month>Dec</Month></PubDate></JournalIssue><Title>Movement disorders : official journal of the Movement Disorder Society</Title><ISOAbbreviation>Mov. Disord.</ISOAbbreviation></Journal><ArticleTitle>Potential outcome measures and trial design issues for multiple system atrophy.</ArticleTitle><Pagination><MedlinePgn>2371-7</MedlinePgn></Pagination><Abstract><AbstractText>Multiple system atrophy (MSA) is a neurodegenerative disorder exhibiting a combination of parkinsonism, cerebellar ataxia, and autonomic failure. A disease-specific scale, the Unified Multiple System Atrophy Rating Scale (UMSARS), has been developed and validated to measure progression of MSA, but its use as an outcome measure for therapeutic trials has not been evaluated. On the basis of twelve months of follow-up from an observational study of 67 patients with probable MSA, we evaluated three disease-specific scores: Activities of Daily Living, Motor Examination, and a combined score from the UMSARS and two general health scores, the Physical Health and Mental Health scores of the SF-36 health survey, for their use as outcome measures in a therapeutic trial. We discuss related design issues and provide sample size estimates. Scores based on the disease-specific UMSARS seemed to be equal or superior to scores based on the SF-36 health survey. They appeared to capture disease progression, were well correlated and required the smallest sample size. The UMSARS Motor Examination score exhibited the most favorable characteristics as an outcome measure for a therapeutic trial in MSA with 1 year of follow-up.</AbstractText><CopyrightInformation>2007 Movement Disorder Society</CopyrightInformation></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>May</LastName><ForeName>Susanne</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>Department of Family and Preventive Medicine, University of California, San Diego, La Jolla, California 92093-0717, USA. smay@ucsd.edu</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Gilman</LastName><ForeName>Sid</ForeName><Initials>S</Initials></Author><Author ValidYN="Y"><LastName>Sowell</LastName><ForeName>B Brooke</ForeName><Initials>BB</Initials></Author><Author ValidYN="Y"><LastName>Thomas</LastName><ForeName>Ronald G</ForeName><Initials>RG</Initials></Author><Author ValidYN="Y"><LastName>Stern</LastName><ForeName>Matthew B</ForeName><Initials>MB</Initials></Author><Author ValidYN="Y"><LastName>Colcher</LastName><ForeName>Amy</ForeName><Initials>A</Initials></Author><Author ValidYN="Y"><LastName>Tanner</LastName><ForeName>Caroline M</ForeName><Initials>CM</Initials></Author><Author ValidYN="Y"><LastName>Huang</LastName><ForeName>Neng</ForeName><Initials>N</Initials></Author><Author ValidYN="Y"><LastName>Novak</LastName><ForeName>Peter</ForeName><Initials>P</Initials></Author><Author ValidYN="Y"><LastName>Reich</LastName><ForeName>Stephen G</ForeName><Initials>SG</Initials></Author><Author ValidYN="Y"><LastName>Jankovic</LastName><ForeName>Joseph</ForeName><Initials>J</Initials></Author><Author ValidYN="Y"><LastName>Ondo</LastName><ForeName>William G</ForeName><Initials>WG</Initials></Author><Author ValidYN="Y"><LastName>Low</LastName><ForeName>Phillip A</ForeName><Initials>PA</Initials></Author><Author ValidYN="Y"><LastName>Sandroni</LastName><ForeName>Paola</ForeName><Initials>P</Initials></Author><Author ValidYN="Y"><LastName>Lipp</LastName><ForeName>Axel</ForeName><Initials>A</Initials></Author><Author ValidYN="Y"><LastName>Marshall</LastName><ForeName>Frederick J</ForeName><Initials>FJ</Initials></Author><Author ValidYN="Y"><LastName>Wooten</LastName><ForeName>Frederick</ForeName><Initials>F</Initials></Author><Author ValidYN="Y"><LastName>Shults</LastName><ForeName>Clifford W</ForeName><Initials>CW</Initials></Author><Author ValidYN="Y"><CollectiveName>North American Multiple System Atrophy Study Group</CollectiveName></Author></AuthorList><Language>eng</Language><GrantList CompleteYN="Y"><Grant><GrantID>P01 NS044233</GrantID><Acronym>NS</Acronym><Agency>NINDS NIH HHS</Agency><Country>United States</Country></Grant></GrantList><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D016448">Multicenter Study</PublicationType><PublicationType UI="D052061">Research Support, N.I.H., Extramural</PublicationType><PublicationType UI="D013485">Research Support, Non-U.S. Gov't</PublicationType></PublicationTypeList></Article><MedlineJournalInfo><Country>United States</Country><MedlineTA>Mov Disord</MedlineTA><NlmUniqueID>8610688</NlmUniqueID><ISSNLinking>0885-3185</ISSNLinking></MedlineJournalInfo><ChemicalList><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="D000978">Antiparkinson Agents</NameOfSubstance></Chemical><Chemical><RegistryNumber>46627O600J</RegistryNumber><NameOfSubstance UI="D007980">Levodopa</NameOfSubstance></Chemical></ChemicalList><CitationSubset>IM</CitationSubset><MeshHeadingList><MeshHeading><DescriptorName MajorTopicYN="N" UI="D000368">Aged</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D000978">Antiparkinson Agents</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D002526">Cerebellar Diseases</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000503">physiopathology</QualifierName><QualifierName MajorTopicYN="N" UI="Q000628">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="Y" UI="D002986">Clinical Trials as Topic</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D004185">Disability Evaluation</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D018450">Disease Progression</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D005260">Female</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D006801">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D007980">Levodopa</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D008297">Male</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D008875">Middle Aged</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D019578">Multiple System Atrophy</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000453">epidemiology</QualifierName><QualifierName MajorTopicYN="N" UI="Q000503">physiopathology</QualifierName><QualifierName MajorTopicYN="Y" UI="Q000628">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="Y" UI="D012107">Research Design</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D012307">Risk Factors</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D018401">Sample Size</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D012959">Socioeconomic Factors</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="Y" UI="D016896">Treatment Outcome</DescriptorName></MeshHeading></MeshHeadingList></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="pubmed"><Year>2007</Year><Month>10</Month><Day>5</Day><Hour>9</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="medline"><Year>2008</Year><Month>2</Month><Day>21</Day><Hour>9</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="entrez"><Year>2007</Year><Month>10</Month><Day>5</Day><Hour>9</Hour><Minute>0</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="doi">10.1002/mds.21734</ArticleId><ArticleId IdType="pubmed">17914727</ArticleId></ArticleIdList></PubmedData></pubmed></record>Pour manipuler ce document sous Unix (Dilib)
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