Modeling MR imaging enhancing-lesion volumes in multiple sclerosis: application in clinical trials
Abstract
Background and purpose: Although the number of enhancing lesions is the typical outcome measure of choice in clinical trials in MS, a potentially more sensitive and statistically more powerful outcome measure is the volume of enhancing lesions. In this study, we assessed the distribution and statistical power of the volume of enhancing brain lesions as an outcome measure by means of their required sample size, and we compared the results with the number of enhancing lesions.
Material and methods: First, a literature search was performed to compare the effects of treatment on the number and volume of enhancing lesions. Then, a statistical model was proposed to describe the distribution of the volume of enhancing lesions in 2 datasets of patients with RRMS, and sample sizes for enhancing-lesion volume as primary outcome measure were calculated.
Results: A mixture of the binomial and Weibull distribution was determined to model enhancing-lesion volumes in patients. Sample size calculations for enhancing-lesion volumes showed that approximately 94 patients per arm would be required to detect a combination of 20% decrease in lesion volume and 20% increase in patients without enhancing lesions, whereas calculations for enhancing-lesion counts showed that approximately 129 patients would be required to detect a 50% decrease.
Conclusions: The mixture of the binomial and Weibull distribution is a suitable approach in modeling new enhancing-lesion volumes in MS and yielded feasible…
How strong is the evidence?
Evidence Hub reads this as a meta-analysis, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 129 participants or records.
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