Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy
Identifieur interne : 004120 ( Main/Merge ); précédent : 004119; suivant : 004121Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy
Auteurs : Susanne May [États-Unis] ; Sid Gilman [États-Unis] ; B. Brooke Sowell [États-Unis] ; Ronald G. Thomas [États-Unis] ; Matthew B. Stem [États-Unis] ; Amy Colcher [États-Unis] ; Caroline M. Tanner [États-Unis] ; NENG HUANG [États-Unis] ; Peter Novak [États-Unis] ; Stephen G. Reich [États-Unis] ; Joseph Jankovic [États-Unis] ; William G. Ondo [États-Unis] ; Phillip A. Low [États-Unis] ; Paola Sandroni [États-Unis] ; Axel Lipp [États-Unis] ; Frederick J. Marshall [États-Unis] ; Frederick Wooten [États-Unis] ; Clifford W. Shults [États-Unis]Source :
- Movement disorders [ 0885-3185 ] ; 2007.
Descripteurs français
- Pascal (Inist)
English descriptors
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.
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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en" level="a">Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy</title><author><name sortKey="May, Susanne" sort="May, Susanne" uniqKey="May S" first="Susanne" last="May">Susanne May</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Family and Preventive Medicine, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Neurosciences, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>4 aut.</sZ><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Gilman, Sid" sort="Gilman, Sid" uniqKey="Gilman S" first="Sid" last="Gilman">Sid Gilman</name><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Department of Neurology, University of Michigan</s1><s2>Ann Arbor, Michigan</s2><s3>USA</s3><sZ>2 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Michigan</region></placeName></affiliation></author><author><name sortKey="Sowell, B Brooke" sort="Sowell, B Brooke" uniqKey="Sowell B" first="B. Brooke" last="Sowell">B. Brooke Sowell</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Family and Preventive Medicine, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Thomas, Ronald G" sort="Thomas, Ronald G" uniqKey="Thomas R" first="Ronald G." last="Thomas">Ronald G. Thomas</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Family and Preventive Medicine, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Neurosciences, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>4 aut.</sZ><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Stem, Matthew B" sort="Stem, Matthew B" uniqKey="Stem M" first="Matthew B." last="Stem">Matthew B. Stem</name><affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Colcher, Amy" sort="Colcher, Amy" uniqKey="Colcher A" first="Amy" last="Colcher">Amy Colcher</name><affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Tanner, Caroline M" sort="Tanner, Caroline M" uniqKey="Tanner C" first="Caroline M." last="Tanner">Caroline M. Tanner</name><affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Parkinson's Institute</s1><s2>Sunnyvale, California</s2><s3>USA</s3><sZ>7 aut.</sZ><sZ>8 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Neng Huang" sort="Neng Huang" uniqKey="Neng Huang" last="Neng Huang">NENG HUANG</name><affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Parkinson's Institute</s1><s2>Sunnyvale, California</s2><s3>USA</s3><sZ>7 aut.</sZ><sZ>8 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Novak, Peter" sort="Novak, Peter" uniqKey="Novak P" first="Peter" last="Novak">Peter Novak</name><affiliation wicri:level="2"><inist:fA14 i1="06"><s1>Department of Neurology, Boston University</s1><s2>Boston, Massachusetts</s2><s3>USA</s3><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Massachusetts</region></placeName></affiliation></author><author><name sortKey="Reich, Stephen G" sort="Reich, Stephen G" uniqKey="Reich S" first="Stephen G." last="Reich">Stephen G. Reich</name><affiliation wicri:level="2"><inist:fA14 i1="07"><s1>Department of Neurology, University of Maryland, School of Medicine</s1><s2>Baltimore, Maryland</s2><s3>USA</s3><sZ>10 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Maryland</region></placeName></affiliation></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation wicri:level="2"><inist:fA14 i1="08"><s1>Department of Neurology, Baylor College of Medicine</s1><s2>Houston, Texas</s2><s3>USA</s3><sZ>11 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName><placeName><settlement type="city">Houston</settlement><region type="state">Texas</region></placeName><orgName type="university" n="3">Baylor College of Medicine</orgName></affiliation></author><author><name sortKey="Ondo, William G" sort="Ondo, William G" uniqKey="Ondo W" first="William G." last="Ondo">William G. Ondo</name><affiliation wicri:level="2"><inist:fA14 i1="08"><s1>Department of Neurology, Baylor College of Medicine</s1><s2>Houston, Texas</s2><s3>USA</s3><sZ>11 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName></affiliation></author><author><name sortKey="Low, Phillip A" sort="Low, Phillip A" uniqKey="Low P" first="Phillip A." last="Low">Phillip A. Low</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, Mayo Clinic</s1><s2>Rochester, Minnesota</s2><s3>USA</s3><sZ>13 aut.</sZ><sZ>14 aut.</sZ><sZ>15 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Sandroni, Paola" sort="Sandroni, Paola" uniqKey="Sandroni P" first="Paola" last="Sandroni">Paola Sandroni</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, Mayo Clinic</s1><s2>Rochester, Minnesota</s2><s3>USA</s3><sZ>13 aut.</sZ><sZ>14 aut.</sZ><sZ>15 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Lipp, Axel" sort="Lipp, Axel" uniqKey="Lipp A" first="Axel" last="Lipp">Axel Lipp</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, Mayo Clinic</s1><s2>Rochester, Minnesota</s2><s3>USA</s3><sZ>13 aut.</sZ><sZ>14 aut.</sZ><sZ>15 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Marshall, Frederick J" sort="Marshall, Frederick J" uniqKey="Marshall F" first="Frederick J." last="Marshall">Frederick J. Marshall</name><affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Department of Neurology, University of Rochester</s1><s2>Rochester, New York</s2><s3>USA</s3><sZ>16 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État de New York</region></placeName></affiliation></author><author><name sortKey="Wooten, Frederick" sort="Wooten, Frederick" uniqKey="Wooten F" first="Frederick" last="Wooten">Frederick Wooten</name><affiliation wicri:level="2"><inist:fA14 i1="11"><s1>Department of Neurology, University of Virginia Health System</s1><s2>Charlottesville, Virginia</s2><s3>USA</s3><sZ>17 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Virginie</region></placeName></affiliation></author><author><name sortKey="Shults, Clifford W" sort="Shults, Clifford W" uniqKey="Shults C" first="Clifford W." last="Shults">Clifford W. Shults</name><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Neurosciences, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>4 aut.</sZ><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="12"><s1>Veterans Affairs San Diego Healthcare System</s1><s2>San Diego, California</s2><s3>USA</s3><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">INIST</idno><idno type="inist">08-0147084</idno><date when="2007">2007</date><idno type="stanalyst">PASCAL 08-0147084 INIST</idno><idno type="RBID">Pascal:08-0147084</idno><idno type="wicri:Area/PascalFrancis/Corpus">001361</idno><idno type="wicri:Area/PascalFrancis/Curation">001958</idno><idno type="wicri:Area/PascalFrancis/Checkpoint">001530</idno><idno type="wicri:doubleKey">0885-3185:2007:May S:potential:outcome:measures</idno><idno type="wicri:Area/Main/Merge">004120</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy</title><author><name sortKey="May, Susanne" sort="May, Susanne" uniqKey="May S" first="Susanne" last="May">Susanne May</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Family and Preventive Medicine, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Neurosciences, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>4 aut.</sZ><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Gilman, Sid" sort="Gilman, Sid" uniqKey="Gilman S" first="Sid" last="Gilman">Sid Gilman</name><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Department of Neurology, University of Michigan</s1><s2>Ann Arbor, Michigan</s2><s3>USA</s3><sZ>2 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Michigan</region></placeName></affiliation></author><author><name sortKey="Sowell, B Brooke" sort="Sowell, B Brooke" uniqKey="Sowell B" first="B. Brooke" last="Sowell">B. Brooke Sowell</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Family and Preventive Medicine, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Thomas, Ronald G" sort="Thomas, Ronald G" uniqKey="Thomas R" first="Ronald G." last="Thomas">Ronald G. Thomas</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Family and Preventive Medicine, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Neurosciences, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>4 aut.</sZ><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Stem, Matthew B" sort="Stem, Matthew B" uniqKey="Stem M" first="Matthew B." last="Stem">Matthew B. Stem</name><affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Colcher, Amy" sort="Colcher, Amy" uniqKey="Colcher A" first="Amy" last="Colcher">Amy Colcher</name><affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Tanner, Caroline M" sort="Tanner, Caroline M" uniqKey="Tanner C" first="Caroline M." last="Tanner">Caroline M. Tanner</name><affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Parkinson's Institute</s1><s2>Sunnyvale, California</s2><s3>USA</s3><sZ>7 aut.</sZ><sZ>8 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Neng Huang" sort="Neng Huang" uniqKey="Neng Huang" last="Neng Huang">NENG HUANG</name><affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Parkinson's Institute</s1><s2>Sunnyvale, California</s2><s3>USA</s3><sZ>7 aut.</sZ><sZ>8 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Novak, Peter" sort="Novak, Peter" uniqKey="Novak P" first="Peter" last="Novak">Peter Novak</name><affiliation wicri:level="2"><inist:fA14 i1="06"><s1>Department of Neurology, Boston University</s1><s2>Boston, Massachusetts</s2><s3>USA</s3><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Massachusetts</region></placeName></affiliation></author><author><name sortKey="Reich, Stephen G" sort="Reich, Stephen G" uniqKey="Reich S" first="Stephen G." last="Reich">Stephen G. Reich</name><affiliation wicri:level="2"><inist:fA14 i1="07"><s1>Department of Neurology, University of Maryland, School of Medicine</s1><s2>Baltimore, Maryland</s2><s3>USA</s3><sZ>10 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Maryland</region></placeName></affiliation></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation wicri:level="2"><inist:fA14 i1="08"><s1>Department of Neurology, Baylor College of Medicine</s1><s2>Houston, Texas</s2><s3>USA</s3><sZ>11 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName><placeName><settlement type="city">Houston</settlement><region type="state">Texas</region></placeName><orgName type="university" n="3">Baylor College of Medicine</orgName></affiliation></author><author><name sortKey="Ondo, William G" sort="Ondo, William G" uniqKey="Ondo W" first="William G." last="Ondo">William G. Ondo</name><affiliation wicri:level="2"><inist:fA14 i1="08"><s1>Department of Neurology, Baylor College of Medicine</s1><s2>Houston, Texas</s2><s3>USA</s3><sZ>11 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName></affiliation></author><author><name sortKey="Low, Phillip A" sort="Low, Phillip A" uniqKey="Low P" first="Phillip A." last="Low">Phillip A. Low</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, Mayo Clinic</s1><s2>Rochester, Minnesota</s2><s3>USA</s3><sZ>13 aut.</sZ><sZ>14 aut.</sZ><sZ>15 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Sandroni, Paola" sort="Sandroni, Paola" uniqKey="Sandroni P" first="Paola" last="Sandroni">Paola Sandroni</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, Mayo Clinic</s1><s2>Rochester, Minnesota</s2><s3>USA</s3><sZ>13 aut.</sZ><sZ>14 aut.</sZ><sZ>15 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Lipp, Axel" sort="Lipp, Axel" uniqKey="Lipp A" first="Axel" last="Lipp">Axel Lipp</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, Mayo Clinic</s1><s2>Rochester, Minnesota</s2><s3>USA</s3><sZ>13 aut.</sZ><sZ>14 aut.</sZ><sZ>15 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Marshall, Frederick J" sort="Marshall, Frederick J" uniqKey="Marshall F" first="Frederick J." last="Marshall">Frederick J. Marshall</name><affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Department of Neurology, University of Rochester</s1><s2>Rochester, New York</s2><s3>USA</s3><sZ>16 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État de New York</region></placeName></affiliation></author><author><name sortKey="Wooten, Frederick" sort="Wooten, Frederick" uniqKey="Wooten F" first="Frederick" last="Wooten">Frederick Wooten</name><affiliation wicri:level="2"><inist:fA14 i1="11"><s1>Department of Neurology, University of Virginia Health System</s1><s2>Charlottesville, Virginia</s2><s3>USA</s3><sZ>17 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Virginie</region></placeName></affiliation></author><author><name sortKey="Shults, Clifford W" sort="Shults, Clifford W" uniqKey="Shults C" first="Clifford W." last="Shults">Clifford W. Shults</name><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Neurosciences, University of California, San Diego</s1><s2>La Jolla, California</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>4 aut.</sZ><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="12"><s1>Veterans Affairs San Diego Healthcare System</s1><s2>San Diego, California</s2><s3>USA</s3><sZ>18 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</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="2007">2007</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>Multiple system atrophy</term><term>Nervous system diseases</term><term>Parkinsonism</term><term>Prognosis</term></keywords><keywords scheme="Pascal" xml:lang="fr"><term>Atrophie multisystématisée</term><term>Parkinsonisme</term><term>Pathologie du système nerveux</term><term>Pronostic</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><affiliations><list><country><li>États-Unis</li></country><region><li>Californie</li><li>Maryland</li><li>Massachusetts</li><li>Michigan</li><li>Minnesota</li><li>Pennsylvanie</li><li>Texas</li><li>Virginie</li><li>État de New York</li></region><settlement><li>Houston</li></settlement><orgName><li>Baylor College of Medicine</li></orgName></list><tree><country name="États-Unis"><region name="Californie"><name sortKey="May, Susanne" sort="May, Susanne" uniqKey="May S" first="Susanne" last="May">Susanne May</name></region><name sortKey="Colcher, Amy" sort="Colcher, Amy" uniqKey="Colcher A" first="Amy" last="Colcher">Amy Colcher</name><name sortKey="Gilman, Sid" sort="Gilman, Sid" uniqKey="Gilman S" first="Sid" last="Gilman">Sid Gilman</name><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><name sortKey="Lipp, Axel" sort="Lipp, Axel" uniqKey="Lipp A" first="Axel" last="Lipp">Axel Lipp</name><name sortKey="Low, Phillip A" sort="Low, Phillip A" uniqKey="Low P" first="Phillip A." last="Low">Phillip A. Low</name><name sortKey="Marshall, Frederick J" sort="Marshall, Frederick J" uniqKey="Marshall F" first="Frederick J." last="Marshall">Frederick J. Marshall</name><name sortKey="May, Susanne" sort="May, Susanne" uniqKey="May S" first="Susanne" last="May">Susanne May</name><name sortKey="Neng Huang" sort="Neng Huang" uniqKey="Neng Huang" last="Neng Huang">NENG HUANG</name><name sortKey="Novak, Peter" sort="Novak, Peter" uniqKey="Novak P" first="Peter" last="Novak">Peter Novak</name><name sortKey="Ondo, William G" sort="Ondo, William G" uniqKey="Ondo W" first="William G." last="Ondo">William G. Ondo</name><name sortKey="Reich, Stephen G" sort="Reich, Stephen G" uniqKey="Reich S" first="Stephen G." last="Reich">Stephen G. Reich</name><name sortKey="Sandroni, Paola" sort="Sandroni, Paola" uniqKey="Sandroni P" first="Paola" last="Sandroni">Paola Sandroni</name><name sortKey="Shults, Clifford W" sort="Shults, Clifford W" uniqKey="Shults C" first="Clifford W." last="Shults">Clifford W. Shults</name><name sortKey="Shults, Clifford W" sort="Shults, Clifford W" uniqKey="Shults C" first="Clifford W." last="Shults">Clifford W. Shults</name><name sortKey="Sowell, B Brooke" sort="Sowell, B Brooke" uniqKey="Sowell B" first="B. Brooke" last="Sowell">B. Brooke Sowell</name><name sortKey="Stem, Matthew B" sort="Stem, Matthew B" uniqKey="Stem M" first="Matthew B." last="Stem">Matthew B. Stem</name><name sortKey="Tanner, Caroline M" sort="Tanner, Caroline M" uniqKey="Tanner C" first="Caroline M." last="Tanner">Caroline M. Tanner</name><name sortKey="Thomas, Ronald G" sort="Thomas, Ronald G" uniqKey="Thomas R" first="Ronald G." last="Thomas">Ronald G. Thomas</name><name sortKey="Thomas, Ronald G" sort="Thomas, Ronald G" uniqKey="Thomas R" first="Ronald G." last="Thomas">Ronald G. Thomas</name><name sortKey="Wooten, Frederick" sort="Wooten, Frederick" uniqKey="Wooten F" first="Frederick" last="Wooten">Frederick Wooten</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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