Title: Internal pilots for a class of linear mixed models with Gaussian and compound symmetric data.

Authors: Gurka, Matthew J; Coffey, Christopher S; Muller, Keith E

Published In Stat Med, (2007 Sep 30)

Abstract: An internal pilot design uses interim sample size analysis, without interim data analysis, to adjust the final number of observations. The approach helps to choose a sample size sufficiently large (to achieve the statistical power desired), but not too large (which would waste money and time). We report on recent research in cerebral vascular tortuosity (curvature in three dimensions) which would benefit greatly from internal pilots due to uncertainty in the parameters of the covariance matrix used for study planning. Unfortunately, observations correlated across the four regions of the brain and small sample sizes preclude using existing methods. However, as in a wide range of medical imaging studies, tortuosity data have no missing or mistimed data, a factorial within-subject design, the same between-subject design for all responses, and a Gaussian distribution with compound symmetry. For such restricted models, we extend exact, small sample univariate methods for internal pilots to linear mixed models with any between-subject design (not just two groups). Planning a new tortuosity study illustrates how the new methods help to avoid sample sizes that are too small or too large while still controlling the type I error rate.

PubMed ID: 17318914

MeSH Terms: Cerebral Veins/physiology; Clinical Trials as Topic/standards; Clinical Trials as Topic/statistics & numerical data; Humans; Magnetic Resonance Imaging; Models, Statistical*; Normal Distribution*; Research Design/statistics & numerical data; Sample Size*; United States