Sample size estimates for determining treatment effects in high-risk patients with early relapsing-remitting multiple sclerosis.
BACKGROUND
Risk factors for short-term progression in early relapsing remitting MS have been identified recently. Previously we determined potential risk factors for rapid progression of early relapsing remitting MS and identified three groups of high-risk patients. These non-mutually exclusive groups of patients were drawn from a consecutively studied sample of 98 patients with newly diagnosed MS. High-risk patients had a history of either poor recovery from initial attacks, more than two attacks in the first two years of disease, or a combination of at least four other risk factors.


OBJECTIVE
To determine differences in sample sizes required to show a meaningful treatment effect when using a high-risk sample versus a random sample of patients.


METHODS
Power analyses were used to calculate the different sample sizes needed for hypothetical treatment trials.


RESULTS
We found that substantially smaller numbers of patients should be needed to show a significant treatment effect by employing these high-risk groups of patients as compared to a random population of MS patients (e.g., 58% reduction in sample size in one model).


CONCLUSIONS
The use of patients at higher risk of progression to perform drug treatment trials can be considered as a means to reduce the number of patients needed to show a significant treatment effect for patients with very early MS.
Scott
Thomas F
TF
Drexel University College of Medicine, Department of Neurology, Allegheny General Hospital, Pittsburgh, Pennsylvania, USA. tscott@wpahs.org
Schramke
Carol J
CJ
Cutter
Gary
G
D000368:Aged; D018450:Disease Progression; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000188:drug therapy* / Q000503:physiopathology*; D011379:Prognosis; D012307:Risk Factors; D018401:Sample Size; D016896:Treatment Outcome