Lifetime Risk Estimators in Epidemiological Studies of Krabbe Disease: Review and Monte Carlo Comparison

• Activity Number: 550
• Type: Contributed
• Date/Time: Wednesday, August 7, 2013 : 10:30 AM to 12:20 PM
• Sponsor: Section on Statistics in Epidemiology
• Author(s): Alexander Foss*+ and Randy Carter
• Companies: Univ At Buffalo, Dept. of Biostatistics and University At Buffalo
• Keywords: Monte Carlo ; Prevalence ; Incidence ; Krabbe disease ; Inborn errors of metabolism ; Epidemiology

Abstract

Krabbe disease is a lysosomal storage disorder that affects both the central and peripheral nervous system. Although it is known to be rare, previous epidemiological studies of the disease are difficult to reconcile, because they have used different study populations and different methods of calculation. Furthermore, confusion exists in the literature regarding which epidemiological parameters have been estimated. In the current poster, it is shown that most existing estimates can be properly interpreted as lifetime risk at birth. Of the two most common estimation methods, it is shown herein using a Monte Carlo simulation that one method is biased, while the other is approximately unbiased. A third estimation method, that has been previously neglected by studies of Krabbe disease, is shown to outperform the other two estimation methods. Based on the results and conclusions of the current paper, specific recommendations are made that are expected to improve the quality of future studies of Krabbe disease. It is anticipated that these recommendations will be broadly applicable to epidemiological studies of many rare genetic diseases.