Defining the endpoints and the formulas for sensitivity and false detection rates, as well as the statistical analysis using binomial and repeated measures analysis for sensitivity, and using the Poisson model for false detection rates. Power analysis to determine sample size, as well as the number of subjects and trial duration as a function of accrual rate.
Based on visit histories of some 100 Fabry patients in a major European medical center, and using a range of measure modalities, the aim is to describe and model the typical disease progression through disease states until device implanting or until death. Descriptive as well as Markov models are being used.
