Potential outcome measures and trial design issues for multiple system atrophy

Susanne May; 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. Shults; Eliezer Masliah; Ron Thomas; Caroline Tanner; Walter Kukull; Virginia Lee; John Trojanowski; Phillip Low; Ira Shoulson; Laurie Ozelius; Tatiana Foroud

doi:10.1002/mds.21734

Potential outcome measures and trial design issues for multiple system atrophy

Susanne May, 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. Shults, Eliezer Masliah, Ron ThomasCaroline Tanner, Walter Kukull, Virginia Lee, John Trojanowski, Phillip Low, Ira Shoulson, Laurie Ozelius, Tatiana Foroud

Neurology

Research output: Contribution to journal › Article › peer-review

54 Scopus citations

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.

Original language	English (US)
Pages (from-to)	2371-2377
Number of pages	7
Journal	Movement Disorders
Volume	22
Issue number	16
DOIs	https://doi.org/10.1002/mds.21734
State	Published - Dec 2007

Keywords

Parkinsonism
Power, study design
SF-36
UMSARS

ASJC Scopus subject areas

Neurology
Clinical Neurology

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10.1002/mds.21734

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May, S., Gilman, S., Sowell, B. B., Thomas, R. G., Stern, M. B., Colcher, A., Tanner, C. M., Huang, N., Novak, P., Reich, S. G., Jankovic, J., Ondo, W. G., Low, P. A., Sandroni, P., Lipp, A., Marshall, F. J., Wooten, F., Shults, C. W., Masliah, E., ... Foroud, T. (2007). Potential outcome measures and trial design issues for multiple system atrophy. Movement Disorders, 22(16), 2371-2377. https://doi.org/10.1002/mds.21734

May, S, Gilman, S, Sowell, BB, Thomas, RG, Stern, MB, Colcher, A, Tanner, CM, Huang, N, Novak, P, Reich, SG, Jankovic, J, Ondo, WG, Low, PA, Sandroni, P, Lipp, A, Marshall, FJ, Wooten, F, Shults, CW, Masliah, E, Thomas, R, Tanner, C, Kukull, W, Lee, V, Trojanowski, J, Low, P, Shoulson, I, Ozelius, L & Foroud, T 2007, 'Potential outcome measures and trial design issues for multiple system atrophy', Movement Disorders, vol. 22, no. 16, pp. 2371-2377. https://doi.org/10.1002/mds.21734

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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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AU - May, Susanne

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AU - Sowell, B. Brooke

AU - Thomas, Ronald G.

AU - Stern, Matthew B.

AU - Colcher, Amy

AU - Tanner, Caroline M.

AU - Huang, Neng

AU - Novak, Peter

AU - Reich, Stephen G.

AU - Jankovic, Joseph

AU - Ondo, William G.

AU - Low, Phillip A.

AU - Sandroni, Paola

AU - Lipp, Axel

AU - Marshall, Frederick J.

AU - Wooten, Frederick

AU - Shults, Clifford W.

AU - Masliah, Eliezer

AU - Thomas, Ron

AU - Tanner, Caroline

AU - Kukull, Walter

AU - Lee, Virginia

AU - Trojanowski, John

AU - Low, Phillip

AU - Shoulson, Ira

AU - Ozelius, Laurie

AU - Foroud, Tatiana

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AB - 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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Potential outcome measures and trial design issues for multiple system atrophy

Abstract

Keywords

ASJC Scopus subject areas

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