| Title: |
Potential outcome measures and trial design issues for multiple system atrophy Members of ???The North American Multiple System Atrophy Study Group??? are listed as an Appendix . |
| Authors: |
Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA; Department of Family and Preventive Medicine, University of California, San Diego, La Jolla, California, USA ; Department of Neurosciences, University of California, San Diego, La Jolla, California, USA ; University of California San Diego, Division of Biostatistics and Bioinformatics, 9500 Gilman Dr.-M/C 0717, La Jolla, CA 92093-0717; Department of Family and Preventive Medicine, University of California, San Diego, La Jolla, California, USA; Department of Family and Preventive Medicine, University of California, San Diego, La Jolla, California, USA ; Department of Neurosciences, University of California, San Diego, La Jolla, California, USA; Parkinson's Disease and Movement Disorders Center, Pennsylvania Hospital, Philadelphia, Pennsylvania, USA; Parkinson's Institute, Sunnyvale, California, USA; Department of Neurology, Boston University, Boston, Massachusetts, USA; Department of Neurology, University of Maryland, School of Medicine, Baltimore, Maryland, USA; Department of Neurology, Baylor College of Medicine, Houston, Texas, USA; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA; Department of Neurology, University of Rochester, Rochester, New York, USA; Department of Neurology, University of Virginia Health System, Charlottesville, Virginia, USA; Department of Neurosciences, University of California, San Diego, La Jolla, California, USA ; Veterans Affairs San Diego Healthcare System, San Diego, California, USA ; This manuscript is dedicated to the memory of Clifford W. Shults, MD.; May, Susanne; Gilman, Sid; Sowell, B. Brooke; Thomas, Ronald G.; Stern, Matthew B.; Colcher, Amy; Tanner, Caroline M.; Huang, Neng; 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. |
| Publisher Information: |
Wiley Subscription Services, Inc., A Wiley Company 2008-01-04T20:09:23Z WITHHELD_ONE_YEAR 2008-01-04T20:09:23Z 2007-12 |
| Document Type: |
Electronic Resource |
| 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. ?? 2007 Movement Disorder Society |
| URL: |
http://hdl.handle.net/2027.42/57524; http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=retrieve&db=pubmed&list_uids=17914727&dopt=citation |
| Availability: |
Open access content. Open access content; IndexNoFollow |
| Other Numbers: |
EYM oai:deepblue.lib.umich.edu:2027.42/57524; 0885-3185; 1531-8257; 17914727; Movement Disorders; 894385237 |
| Contributing Source: |
UNIV OF MICHIGAN LIBR; From OAIster®, provided by the OCLC Cooperative. |
| Accession Number: |
edsoai.ocn894385237 |
| Database: |
OAIster |