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A Unified Causal Inference Framework for the Desirability of Outcome Ranking Paradigm in Benefit-Risk Evaluation

获益—风险评价中结局期望排序概率的统一因果推断框架

We developed a unified covariate-adjusted causal inference framework for estimating the desirability of outcome ranking (DOOR) probability for benefit-risk evaluation in randomized trials and observational studies. The framework expresses the DOOR probability as a bilinear functional of the marginal ordinal outcome distributions under the two treatment strategies, estimates conditional ordinal distributions through sequential risk-set hazards, and derives the efficient…

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On Cluster Randomized Trials with the Desirability of Outcome Ranking (DOOR) Endpoints

采用结局优劣排序端点的整群随机试验

Cluster randomized trials are widely used when individual randomization is logistically infeasible or when correlations between observations cannot be ignored, especially in fields such as ophthalmology, infectious disease, vaccine research, and sociology. The desirability of outcome ranking (DOOR) framework evaluates patient-centric benefit-risk using an ordinal outcome and a Wilcoxon-Mann-Whitney statistic-based approach to compare outcome distributions between…

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Doubly Robust Estimation of Desirability of Outcome Ranking (DOOR) Probability with Application to MDRO Studies

结局优劣排序概率的双重稳健估计及其耐多药菌研究应用

In observational studies, adjusting for confounders is required if a treatment comparison is planned. A crude comparison of the primary endpoint without covariate adjustment will suffer from biases, and the addition of regression models could improve precision by incorporating imbalanced covariates and thus help make correct inference. Desirability of outcome ranking (DOOR) is a patient-centric benefit-risk evaluation methodology designed for randomized clinical trials.…

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