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A mixed-effects two-part model for twin-data and an application on identifying important factors associated with
Baiming Zou1, Hudson P Santos2, James G Xenakis3
1Department of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States of America.
Insights
This study introduces a new statistical model for analyzing child health index data, improving the detection of health disorder factors in preterm infants. The novel approach offers enhanced power and robustness compared to existing methods.
Area of Science:
- Biostatistics
- Pediatric Health Research
- Statistical Modeling
Background:
- Analyzing the Positive Child Health Index (PCHI) in preterm infants presents challenges due to distinct data processes for healthy versus affected children.
- Correlations within multiple births (twins, triplets) require specialized statistical approaches.
- Existing methods dichotomize health disorder data, potentially reducing statistical power.
Purpose of the Study:
- To develop and evaluate a novel mixed-effects two-part model for analyzing PCHI data.
- To jointly model the two distinct data processes (healthy vs. disordered children) within a unified framework.
- To account for correlations in health outcomes among multiple births.
Main Methods:
- Development of a mixed-effects two-part model to analyze correlated semi-continuous PCHI data.
- Joint modeling of two data processes: one for children with no disorders and another for those with disorders.
- Simulation studies to compare the proposed method with existing logistic regression approaches.
Main Results:
- The proposed joint-test procedure demonstrates increased power in detecting factors associated with health disorders.
- The new method consistently outperforms existing approaches in simulations when controlling for Type I error.
- The model is robust to misspecifications and applicable to correlated semi-continuous data.
Conclusions:
- The proposed mixed-effects two-part model provides a more powerful and robust framework for analyzing PCHI data.
- This approach enhances the ability to identify factors influencing child health outcomes in preterm populations.
- The method is suitable for complex health index data, particularly in the presence of multiple births and varying health statuses.
Abstract:
Our recent studies identifying factors significantly associated with the positive child health index (PCHI) in a mixed cohort of preterm-born singletons, twins, and triplets posed some analytic and modeling challenges. The PCHI transforms the total number of health disorders experienced (of the eleven ascertained) to a scale from 0 to 100%. While some of the children had none of the eleven health disorders (i.e., PCHI = 1), others experienced a subset or all (i.e., 0 ≤PCHI< 1). This indicates the existence of two distinct data processes-one for the healthy children, and another for those with at least one health disorder, necessitating a two-part model to accommodate both. Further, the scores for twins and triplets are potentially correlated since these children share similar genetics and early environments. The existing approach for analyzing PCHI data dichotomizes the data (i.e., number of health disorders) and uses a mixed-effects logistic or multiple logistic regression to model the binary feature of the PCHI (1 vs. < 1). To provide an alternate analytic framework, in this study we jointly model the two data processes under a mixed-effects two-part model framework that accounts for the sample correlations between and within the two data processes. The proposed method increases power to detect factors associated with disorders. Extensive numerical studies demonstrate that the proposed joint-test procedure consistently outperforms the existing method when the type I error is controlled at the same level. Our numerical studies also show that the proposed method is robust to model misspecifications and it is applicable to a set of correlated semi-continuous data.
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