Uncovering symptom progression history from disease registry data with application to young cystic fibrosis patients.
The growing availability of various disease registry data has brought precious opportunities to epidemiologists to understand the natural history of the registered diseases. It also presents challenges to the traditional data analysis techniques because of complicated censoring/truncation schemes and temporal dynamics of covariate influences. In a case study of the Cystic Fibrosis Foundation Patient Registry data, we propose analyses of progressive symptoms using temporal process regressions, as an alternative to the commonly employed proportional hazards models. Two endpoints are considered, the prevalence of ever positive and currently positive for Pseudomonas aeruginosa (PA) infection in the lungs, which capture different aspect of the disease process. The analysis of ever PA positive via a time-varying coefficient model demonstrates the lack of fit, as well as the potential loss of information, in the standard proportional hazards analysis. The analysis of currently PA positive yields results that are clinically meaningful and have not previously been reported in the cystic fibrosis literature. Our analyses demonstrate that prenatal/neonatal screening results in lower prevalence of PA infection compared to traditional diagnosis via signs and symptoms, but this benefit attenuates with age. Calendar years of diagnosis also affect the risk of PA infection; patients diagnosed in more recent cohort show higher prevalence of ever PA positive but lower prevalence of currently PA positive.
Yan
Jun
J
Department of Statistics and Institute for Public Health Research, University of Connecticut, Storrs, Connecticut 06269, USA. jun.yan@uconn.edu
Cheng
Yu
Y
Fine
Jason P
JP
Lai
Huichuan J
HJ
D003550:Cystic Fibrosis / Q000453:epidemiology*; D003627:Data Interpretation, Statistical; D018450:Disease Progression; D005315:Fetal Diseases / Q000175:diagnosis / Q000382:microbiology; D006801:Humans; D007231:Infant, Newborn; D007232:Infant, Newborn, Diseases / Q000175:diagnosis / Q000382:microbiology; D015233:Models, Statistical; D011296:Prenatal Diagnosis; D011552:Pseudomonas Infections / Q000453:epidemiology*; D011550:Pseudomonas aeruginosa; D012042:Registries; D013997:Time Factors