Biostatistics 5:31-43 (2004)
© 2004 Oxford University Press
Estimating treatment effects in studies of perinatal transmission of HIV
Department of Biostatistics, University of North Carolina, 137E Franklin Street, Suite 400, Chapel Hill, NC 27599-8030, USA heejung_bang{at}unc.edu
Departments of Biostatistics and Epidemiology, Harvard School of Public Health, 677 Huntington Avenue, Boston, MA 02115, USA
Fetal loss often precludes the ascertainment of infection status in studies of perinatal transmission of HIV. The standard analysis based on liveborn babies can result in biased estimation and invalid inference in the presence of fetal death. This paper focuses on the problem of estimating treatment effects for mother-to-child transmission when infection status is unknown for some babies. Minimal data structures for identifiability of parameters are given. Methods using full likelihood and the inverse probability of selection-weighted estimators are suggested. Simulation studies are used to show that these estimators perform well in finite samples. Methods are applied to the data from a clinical trial in Dar es Salaam, Tanzania. To validly estimate the treatment effect using likelihood methods, investigators should make sure that the design includes a mini-study among uninfected mothers and that efforts are made to ascertain the infection status of as many babies lost as possible. The inverse probability weighting methods need precise estimation of the probability of observing infection status. We can further apply our methodology to the study of other vertically transmissible infections which are potentially fatal pre- and perinatally.
Keywords: AIDS; HIV; Logistic regression; Missing data; Perinatal transmission; Semiparametric efficiency; Selection bias; Vertical transmission