Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy
Identifieur interne : 001361 ( PascalFrancis/Corpus ); précédent : 001360; suivant : 001362Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy
Auteurs : Susanne May ; Sid Gilman ; B. Brooke Sowell ; Ronald G. Thomas ; Matthew B. Stem ; 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 [ 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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Format Inist (serveur)
| NO : | PASCAL 08-0147084 INIST |
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| ET : | Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy |
| AU : | MAY (Susanne); GILMAN (Sid); SOWELL (B. Brooke); THOMAS (Ronald G.); STEM (Matthew B.); COLCHER (Amy); TANNER (Caroline M.); NENG HUANG; NOVAK (Peter); REICH (Stephen G.); JANKOVIC (Joseph); ONDO (William G.); LOW (Phillip A.); SANDRONI (Paola); LIPP (Axel); MARSHALL (Frederick J.); WOOTEN (Frederick); SHULTS (Clifford W.) |
| AF : | Department of Family and Preventive Medicine, University of California, San Diego/La Jolla, California/Etats-Unis (1 aut., 3 aut., 4 aut.); Department of Neurosciences, University of California, San Diego/La Jolla, California/Etats-Unis (1 aut., 4 aut., 18 aut.); Department of Neurology, University of Michigan/Ann Arbor, Michigan/Etats-Unis (2 aut.); Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital/Philadelphia, Pennsylvania/Etats-Unis (5 aut., 6 aut.); Parkinson's Institute/Sunnyvale, California/Etats-Unis (7 aut., 8 aut.); Department of Neurology, Boston University/Boston, Massachusetts/Etats-Unis (9 aut.); Department of Neurology, University of Maryland, School of Medicine/Baltimore, Maryland/Etats-Unis (10 aut.); Department of Neurology, Baylor College of Medicine/Houston, Texas/Etats-Unis (11 aut., 12 aut.); Department of Neurology, Mayo Clinic/Rochester, Minnesota/Etats-Unis (13 aut., 14 aut., 15 aut.); Department of Neurology, University of Rochester/Rochester, New York/Etats-Unis (16 aut.); Department of Neurology, University of Virginia Health System/Charlottesville, Virginia/Etats-Unis (17 aut.); Veterans Affairs San Diego Healthcare System/San Diego, California/Etats-Unis (18 aut.) |
| DT : | Publication en série; Niveau analytique |
| SO : | Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2007; Vol. 22; No. 16; Pp. 2371-2377; Bibl. 20 ref. |
| LA : | Anglais |
| EA : | 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. |
| CC : | 002B17 |
| FD : | Atrophie multisystématisée; Parkinsonisme; Pathologie du système nerveux; Pronostic |
| ED : | Multiple system atrophy; Parkinsonism; Nervous system diseases; Prognosis |
| SD : | Atrofia multisistematizada; Parkinson síndrome; Sistema nervioso patología; Pronóstico |
| LO : | INIST-20953.354000162715700110 |
| ID : | 08-0147084 |
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Marshall</name><affiliation><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></affiliation></author><author><name sortKey="Wooten, Frederick" sort="Wooten, Frederick" uniqKey="Wooten F" first="Frederick" last="Wooten">Frederick Wooten</name><affiliation><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></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><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></affiliation><affiliation><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></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></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><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></affiliation><affiliation><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></affiliation></author><author><name sortKey="Gilman, Sid" sort="Gilman, Sid" uniqKey="Gilman S" first="Sid" last="Gilman">Sid Gilman</name><affiliation><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></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><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></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><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></affiliation><affiliation><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></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><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></affiliation></author><author><name sortKey="Colcher, Amy" sort="Colcher, Amy" uniqKey="Colcher A" first="Amy" last="Colcher">Amy Colcher</name><affiliation><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></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><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></affiliation></author><author><name sortKey="Neng Huang" sort="Neng Huang" uniqKey="Neng Huang" last="Neng Huang">NENG HUANG</name><affiliation><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></affiliation></author><author><name sortKey="Novak, Peter" sort="Novak, Peter" uniqKey="Novak P" first="Peter" last="Novak">Peter Novak</name><affiliation><inist:fA14 i1="06"><s1>Department of Neurology, Boston University</s1><s2>Boston, Massachusetts</s2><s3>USA</s3><sZ>9 aut.</sZ></inist:fA14></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><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></affiliation></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation><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></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><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></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><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></affiliation></author><author><name sortKey="Sandroni, Paola" sort="Sandroni, Paola" uniqKey="Sandroni P" first="Paola" last="Sandroni">Paola Sandroni</name><affiliation><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></affiliation></author><author><name sortKey="Lipp, Axel" sort="Lipp, Axel" uniqKey="Lipp A" first="Axel" last="Lipp">Axel Lipp</name><affiliation><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></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><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></affiliation></author><author><name sortKey="Wooten, Frederick" sort="Wooten, Frederick" uniqKey="Wooten F" first="Frederick" last="Wooten">Frederick Wooten</name><affiliation><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></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><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></affiliation><affiliation><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></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><inist><standard h6="B"><pA><fA01 i1="01" i2="1"><s0>0885-3185</s0></fA01><fA03 i2="1"><s0>Mov. disord.</s0></fA03><fA05><s2>22</s2></fA05><fA06><s2>16</s2></fA06><fA08 i1="01" i2="1" l="ENG"><s1>Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy</s1></fA08><fA11 i1="01" i2="1"><s1>MAY (Susanne)</s1></fA11><fA11 i1="02" i2="1"><s1>GILMAN (Sid)</s1></fA11><fA11 i1="03" i2="1"><s1>SOWELL (B. Brooke)</s1></fA11><fA11 i1="04" i2="1"><s1>THOMAS (Ronald G.)</s1></fA11><fA11 i1="05" i2="1"><s1>STEM (Matthew B.)</s1></fA11><fA11 i1="06" i2="1"><s1>COLCHER (Amy)</s1></fA11><fA11 i1="07" i2="1"><s1>TANNER (Caroline M.)</s1></fA11><fA11 i1="08" i2="1"><s1>NENG HUANG</s1></fA11><fA11 i1="09" i2="1"><s1>NOVAK (Peter)</s1></fA11><fA11 i1="10" i2="1"><s1>REICH (Stephen G.)</s1></fA11><fA11 i1="11" i2="1"><s1>JANKOVIC (Joseph)</s1></fA11><fA11 i1="12" i2="1"><s1>ONDO (William G.)</s1></fA11><fA11 i1="13" i2="1"><s1>LOW (Phillip A.)</s1></fA11><fA11 i1="14" i2="1"><s1>SANDRONI (Paola)</s1></fA11><fA11 i1="15" i2="1"><s1>LIPP (Axel)</s1></fA11><fA11 i1="16" i2="1"><s1>MARSHALL (Frederick J.)</s1></fA11><fA11 i1="17" i2="1"><s1>WOOTEN (Frederick)</s1></fA11><fA11 i1="18" i2="1"><s1>SHULTS (Clifford W.)</s1></fA11><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></fA14><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></fA14><fA14 i1="03"><s1>Department of Neurology, University of Michigan</s1><s2>Ann Arbor, Michigan</s2><s3>USA</s3><sZ>2 aut.</sZ></fA14><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></fA14><fA14 i1="05"><s1>Parkinson's Institute</s1><s2>Sunnyvale, California</s2><s3>USA</s3><sZ>7 aut.</sZ><sZ>8 aut.</sZ></fA14><fA14 i1="06"><s1>Department of Neurology, Boston University</s1><s2>Boston, Massachusetts</s2><s3>USA</s3><sZ>9 aut.</sZ></fA14><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></fA14><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></fA14><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></fA14><fA14 i1="10"><s1>Department of Neurology, University of Rochester</s1><s2>Rochester, New York</s2><s3>USA</s3><sZ>16 aut.</sZ></fA14><fA14 i1="11"><s1>Department of Neurology, University of Virginia Health System</s1><s2>Charlottesville, Virginia</s2><s3>USA</s3><sZ>17 aut.</sZ></fA14><fA14 i1="12"><s1>Veterans Affairs San Diego Healthcare System</s1><s2>San Diego, California</s2><s3>USA</s3><sZ>18 aut.</sZ></fA14><fA17 i1="01" i2="1"><s1>North American Multiple System Atrophy Study Group</s1><s3>USA</s3></fA17><fA20><s1>2371-2377</s1></fA20><fA21><s1>2007</s1></fA21><fA23 i1="01"><s0>ENG</s0></fA23><fA43 i1="01"><s1>INIST</s1><s2>20953</s2><s5>354000162715700110</s5></fA43><fA44><s0>0000</s0><s1>© 2008 INIST-CNRS. All rights reserved.</s1></fA44><fA45><s0>20 ref.</s0></fA45><fA47 i1="01" i2="1"><s0>08-0147084</s0></fA47><fA60><s1>P</s1></fA60><fA61><s0>A</s0></fA61><fA64 i1="01" i2="1"><s0>Movement disorders</s0></fA64><fA66 i1="01"><s0>USA</s0></fA66><fC01 i1="01" l="ENG"><s0>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.</s0></fC01><fC02 i1="01" i2="X"><s0>002B17</s0></fC02><fC03 i1="01" i2="X" l="FRE"><s0>Atrophie multisystématisée</s0><s2>NM</s2><s5>01</s5></fC03><fC03 i1="01" i2="X" l="ENG"><s0>Multiple system atrophy</s0><s2>NM</s2><s5>01</s5></fC03><fC03 i1="01" i2="X" l="SPA"><s0>Atrofia multisistematizada</s0><s2>NM</s2><s5>01</s5></fC03><fC03 i1="02" i2="X" l="FRE"><s0>Parkinsonisme</s0><s2>NM</s2><s5>02</s5></fC03><fC03 i1="02" i2="X" l="ENG"><s0>Parkinsonism</s0><s2>NM</s2><s5>02</s5></fC03><fC03 i1="02" i2="X" l="SPA"><s0>Parkinson síndrome</s0><s2>NM</s2><s5>02</s5></fC03><fC03 i1="03" i2="X" l="FRE"><s0>Pathologie du système nerveux</s0><s5>03</s5></fC03><fC03 i1="03" i2="X" l="ENG"><s0>Nervous system diseases</s0><s5>03</s5></fC03><fC03 i1="03" i2="X" l="SPA"><s0>Sistema nervioso patología</s0><s5>03</s5></fC03><fC03 i1="04" i2="X" l="FRE"><s0>Pronostic</s0><s5>09</s5></fC03><fC03 i1="04" i2="X" l="ENG"><s0>Prognosis</s0><s5>09</s5></fC03><fC03 i1="04" i2="X" l="SPA"><s0>Pronóstico</s0><s5>09</s5></fC03><fN21><s1>091</s1></fN21><fN44 i1="01"><s1>OTO</s1></fN44><fN82><s1>OTO</s1></fN82></pA></standard><server><NO>PASCAL 08-0147084 INIST</NO><ET>Potential Outcome Measures and Trial Design Issues for Multiple System Atrophy</ET><AU>MAY (Susanne); GILMAN (Sid); SOWELL (B. Brooke); THOMAS (Ronald G.); STEM (Matthew B.); COLCHER (Amy); TANNER (Caroline M.); NENG HUANG; NOVAK (Peter); REICH (Stephen G.); JANKOVIC (Joseph); ONDO (William G.); LOW (Phillip A.); SANDRONI (Paola); LIPP (Axel); MARSHALL (Frederick J.); WOOTEN (Frederick); SHULTS (Clifford W.)</AU><AF>Department of Family and Preventive Medicine, University of California, San Diego/La Jolla, California/Etats-Unis (1 aut., 3 aut., 4 aut.); Department of Neurosciences, University of California, San Diego/La Jolla, California/Etats-Unis (1 aut., 4 aut., 18 aut.); Department of Neurology, University of Michigan/Ann Arbor, Michigan/Etats-Unis (2 aut.); Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital/Philadelphia, Pennsylvania/Etats-Unis (5 aut., 6 aut.); Parkinson's Institute/Sunnyvale, California/Etats-Unis (7 aut., 8 aut.); Department of Neurology, Boston University/Boston, Massachusetts/Etats-Unis (9 aut.); Department of Neurology, University of Maryland, School of Medicine/Baltimore, Maryland/Etats-Unis (10 aut.); Department of Neurology, Baylor College of Medicine/Houston, Texas/Etats-Unis (11 aut., 12 aut.); Department of Neurology, Mayo Clinic/Rochester, Minnesota/Etats-Unis (13 aut., 14 aut., 15 aut.); Department of Neurology, University of Rochester/Rochester, New York/Etats-Unis (16 aut.); Department of Neurology, University of Virginia Health System/Charlottesville, Virginia/Etats-Unis (17 aut.); Veterans Affairs San Diego Healthcare System/San Diego, California/Etats-Unis (18 aut.)</AF><DT>Publication en série; Niveau analytique</DT><SO>Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2007; Vol. 22; No. 16; Pp. 2371-2377; Bibl. 20 ref.</SO><LA>Anglais</LA><EA>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.</EA><CC>002B17</CC><FD>Atrophie multisystématisée; Parkinsonisme; Pathologie du système nerveux; Pronostic</FD><ED>Multiple system atrophy; Parkinsonism; Nervous system diseases; Prognosis</ED><SD>Atrofia multisistematizada; Parkinson síndrome; Sistema nervioso patología; Pronóstico</SD><LO>INIST-20953.354000162715700110</LO><ID>08-0147084</ID></server></inist></record>Pour manipuler ce document sous Unix (Dilib)
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