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Scott Zuo

Publications and source records attributed to Scott Zuo.

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Clustering Informed Inverse Probability Weighting Strategies for Causal Effect Estimation in Observational Studies

Inverse probability weighting (IPW) is widely used to estimate causal effects in observational studies but depends on adequate propensity-score specification. We compare three strategies for addressing treatment assignment heterogeneity: standard IPW, clustering augmented IPW with cluster specific propensity score models, and a global propensity score model including estimated cluster membership as a covariate. Through simulations with and without latent cluster structure and under correctly specified and omitted covariate propensity score models, we evaluate bias, mean squared error (MSE), and confidence interval coverage across sample sizes of 100 to 500. Both cluster informed strategies reduced bias and MSE from omitted covariate misspecification relative to standard IPW, but neither uniformly dominated: clustering augmented IPW achieved lower MSE when latent cluster structure was present, whereas the global model generally provided lower bias and better coverage at smaller sample sizes. We also apply the methods to 966 breast cancer patients treated with carboplatin, using generalized propensity scores to estimate the dose response relationship between treatment cycles and hypersensitivity reaction risk. Standard and clustered analyses produced similar pooled estimates, while clustering additionally provided subgroup specific estimates and diagnostic profiles. Overall, cluster informed strategies may improve robustness to propensity score misspecification, with relative performance depending on subgroup structure, sample size, and inferential priorities.

stat.ME

Covariate-adjusted win statistics in randomized clinical trials with ordinal outcomes

Ordinal outcomes are common in clinical settings where they often represent increasing levels of disease progression or different levels of functional impairment. In this article, we focus on representing the average treatment effect for ordinal outcomes via intrinsic pairwise outcome comparisons captured through win estimands, such as the win ratio and win difference. Recognizing the value of baseline covariate adjustment toward enhanced precision, we first develop propensity score weighting estimators, including both inverse probability weighting (IPW) and overlap weighting (OW), tailored to estimating win estimands. Furthermore, we develop augmented weighting estimators that leverage an additional ordinal outcome regression to potentially improve efficiency over weighting alone. Leveraging the theory of U-statistics, we establish the asymptotic theory for all estimators, and derive closed-form variance estimators to support statistical inference. We also prove that all of the covariate-adjusted estimators do not compromise consistency for the target estimand even when the associated working models are incorrectly specified; hence these covariate-adjusted estimators are model-robust. Through simulations we demonstrate the enhanced efficiency of the weighted estimators over the unadjusted estimator, with the augmented weighting estimators showing a further improvement in efficiency except for extreme cases. Finally, we illustrate our proposed methods with the ORCHID trial, and implement our covariate adjustment methods in an R package winPSW.

stat.ME