Comparison of relative change with effect size metrics in Alzheimer's disease clinical trials.

ABSTRACT:

Background

Per cent slowing of decline is frequently used as a metric of outcome in Alzheimer's disease (AD) clinical trials, but it may be misleading. Our objective was to determine whether per cent slowing of decline or Cohen's d is the more valid and informative measure of efficacy.

Methods

Outcome measures of interest were per cent slowing of decline; Cohen's d effect size and number-needed-to-treat (NNT). Data from a graphic were used to model the inter-relationships among Cohen's d, placebo decline in raw score units and per cent slowing of decline with active treatment. NNTs were computed based on different magnitudes of d. Last, we tabulated recent AD anti-amyloid clinical trials that reported per cent slowing and for which we computed their respective d's and NNTs.<

SUBMITTER: Goldberg TE 

PROVIDER: S-EPMC11299058 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature