TITLE

Sample size requirements for treatment effects using gray matter, white matter and whole brain volume in relapsing–remitting multiple sclerosis

AUTHOR(S)
B Healy
PUB. DATE
November 2009
SOURCE
Journal of Neurology, Neurosurgery & Psychiatry;Nov2009, Vol. 80 Issue 11, p1218
SOURCE TYPE
Academic Journal
DOC. TYPE
Article
ABSTRACT
OBJECTIVE: To compare the sample size requirements for a neuroprotection trial with change in cerebral gray matter volume (GMV), white matter volume (WMV) or whole brain parenchymal volume (BPV) as outcome measures in patients with relapsing–remitting multiple sclerosis (RRMS). METHODS: Two datasets with longitudinal MRI measures of untreated patients with RRMS (n = 116 and n = 26) and one dataset of treated patients with RRMS (n = 109) were investigated. In each dataset, normalised GMV, normalised WMV and normalised BPV were analysed using a random intercepts and slopes model to estimate the variance components and per cent change. The required sample size to observe a 33%, 50% and 90% reduction in the per cent change was calculated for each dataset using both a constant per cent change for each measurement and the estimated per cent change for each dataset. RESULTS: The per cent change was greatest in GMV but all variance components were smallest in BPV. Using the estimated per cent change, the sample size required in the untreated cohorts was similar for GMV and BPV, and both were lower than WMV. In the treated cohort, the sample size for GMV was the smallest of all measures. Including additional scans reduced the sample size but increasing the length of the trial and clustering scans led to greater reductions. CONCLUSIONS: Cerebral GMV may be a viable outcome measure for clinical trials investigating neuroprotection in RRMS patients, especially considering that the treatment effect may be larger on GMV compared with BPV. However, GMV was somewhat limited by increased variability versus BPV.
ACCESSION #
45076712

 

Related Articles

  • Rasch analysis of the Fatigue Severity Scale in multiple sclerosis. Mills, R. J.; Young, C. A.; Nicholas, R. S.; Pallant, J. F.; Tennant, A. // Multiple Sclerosis (13524585);Jan2009, Vol. 15 Issue 1, p81 

    Background The 9-item, Fatigue Severity Scale (FSS-9) has been widely used as an outcome measure in multiple sclerosis (MS). Modern psychometric theory, in the form of the Rasch measurement model, has set new quality standards for outcome measures by appraising a broad range of measurement...

  • Relationship of the Multiple Sclerosis Neuropsychological Questionnaire (MSNQ) to functional, emotional, and neuropsychological outcomes O’Brien, Amanda; Gaudino-Goering, Elizabeth; Shawaryn, Marla; Komaroff, Eugene; Moore, Nancy B.; DeLuca, John // Archives of Clinical Neuropsychology;Nov2007, Vol. 22 Issue 8, p933 

    Abstract: Objective: To explore the relationship of the Multiple Sclerosis Neuropsychology Questionnaire (MSNQ; [Benedict, R. H. B., Cox, D., Thompson, L. L., Foley, F., Weinstock-Guttman, B., & Munschauer, F. (2004). Reliable screening for neuropsychological impairment in multiple sclerosis....

  • Development and validation of a patient self-assessed questionnaire on satisfaction with communication of the multiple sclerosis diagnosis. Solari, A.; Mattarozzi, K.; Vignatelli, L.; Giordano, A.; Russo, P. M.; Uccelli, M. Messmer.; D'Alessandro, R. // Multiple Sclerosis (13524585);Oct2010, Vol. 16 Issue 10, p1237 

    Background: We describe the development and clinical validation of a patient self-administered tool assessing the quality of multiple sclerosis diagnosis disclosure. Method: A multiple sclerosis expert panel generated questionnaire items from the Doctor's Interpersonal Skills Questionnaire,...

  • Clinical scales in progressive MS: predicting long-term disability. Bosma, Libertje VAE; Kragt, Jolijn J; Knol, Dirk L; Polman, Chris H; Uitdehaag, Bernard MJ // Multiple Sclerosis Journal;Mar2012, Vol. 18 Issue 3, p345 

    Background: To determine which short-term changes on clinical scales including the Expanded Disability Status Scale (EDSS), Timed 25-Foot Walk (T25FW), 9-Hole Peg test (9HPT) and Guy�s Neurological Disability Scale (GNDS) are most predictive of long-term outcome of disability as rated by...

  • Assessment of Definitions of Sustained Disease Progression in Relapsing-Remitting Multiple Sclerosis. Healy, Brian C.; Engler, David; Glanz, Bonnie; Musallam, Alexander; Chitnis, Tanuja // Multiple Sclerosis International;2013, p1 

    Sustained progression on the expanded disability status scale (EDSS) is a common outcome measure of disease progression in clinical studies ofMS. Unfortunately, this outcome may not accurately measure long-term and irreversible disease progression. To assess the performance of definitions of...

  • Future MS care: a consensus statement of the MS in the 21st Century Steering Group. Rieckmann, Peter; Boyko, Alexey; Centonze, Diego; Coles, Alasdair; Elovaara, Irina; Havrdová, Eva; Hommes, Otto; LeLorier, Jacques; Morrow, Sarah; Oreja-Guevara, Celia; Rijke, Nick; Schippling, Sven // Journal of Neurology;Feb2013, Vol. 260 Issue 2, p462 

    The 'MS in the 21st Century' initiative was established with the purpose of (1) defining how multiple sclerosis (MS) treatment and standards of care should look in the 21st century; (2) developing a minimum standard of care across the world; and (3) motivating the broad MS community to align...

  • Grasping multiple sclerosis: do quantitative motor assessments provide a link between structure and function? Reilmann, R.; Holtbernd, F.; Bachmann, R.; Mohammadi, S.; Ringelstein, E.; Deppe, M. // Journal of Neurology;Feb2013, Vol. 260 Issue 2, p407 

    Motor disability in MS is commonly assessed by the Expanded Disability Status Scale (EDSS). Categorical rating scales are limited by subjective error and inter-rater variability. Therefore, objective and quantitative measures of motor disability may be useful to supplement the EDSS in the...

  • Robot-assisted gait training in multiple sclerosis patients: a randomized trial. Schwartz, Isabella; Sajin, Anna; Moreh, Elior; Fisher, Iris; Neeb, Martin; Forest, Adina; Vaknin-Dembinsky, Adi; Karusis, Dimitrios; Meiner, Zeev // Multiple Sclerosis Journal;Jun2012, Vol. 18 Issue 6, p881 

    Background: Preservation of locomotor activity in multiple sclerosis (MS) patients is of utmost importance. Robotic-assisted body weight-supported treadmill training is a promising method to improve gait functions in neurologically impaired patients, although its effectiveness in MS patients is...

  • Modelling the natural history of primary progressive multiple sclerosis. Harding, Katharine E.; Wardle, Mark; Moore, Perry; Tomassini, Valentina; Pickersgill, Trevor; Ben-Shlomo, Yoav; Robertson, Neil P. // Journal of Neurology, Neurosurgery & Psychiatry;Jan2015, Vol. 86 Issue 1, p13 

    Background A minority of patients with multiple sclerosis (MS) have primary progressive disease (PPMS). Treatment options are currently limited, but as prospects for interventional studies become more realistic, understanding contemporary outcome data will be key to successful trial design....

Share

Read the Article

Courtesy of VIRGINIA BEACH PUBLIC LIBRARY AND SYSTEM

Sign out of this library

Other Topics