title: Design and analysis of multiple events case-control studies.
abstract: In case-control research where there are multiple case groups, standard analyses fail to make use of all available information. Multiple events case-control (MECC) studies provide a new approach to sampling from a cohort and are useful when it is desired to study multiple types of events in the cohort. In this design, subjects in the cohort who develop any event of interest are sampled, as well as a fraction of the remaining subjects. We show that a simple case-control analysis of data arising from MECC studies is biased and develop three general estimating-equation-based approaches to analyzing data from these studies. We conduct simulation studies to compare the efficiency of the various MECC analyses with each other and with the corresponding conventional analyses. It is shown that the gain in efficiency by using the new design is substantial in many situations. We demonstrate the application of our approach to a nested case-control study of the effect of oral sodium phosphate use on chronic kidney injury with multiple case definitions.
authors.affiliation: Department of Statistics, North Carolina State University, Raleigh, North Carolina 27695, USA. sun@stat.ncsu.edu
authors.forename: Wenguang
authors.initials: W
authors.lastname: Sun
authors.forename: Marshall M
authors.initials: MM
authors.lastname: Joffe
authors.forename: Jinbo
authors.initials: J
authors.lastname: Chen
authors.forename: Steven M
authors.initials: SM
authors.lastname: Brunelli
mesh_terms: D000284:Administration, Oral; D001699:Biometry / Q000379:methods; D016022:Case-Control Studies; D006801:Humans; D010710:Phosphates / Q000008:administration & dosage / Q000009:adverse effects; D051436:Renal Insufficiency, Chronic / Q000139:chemically induced; D012107:Research Design / Q000706:statistics & numerical data*
chemical_list: D010710:Phosphates; C018279:sodium phosphate