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Amanda Ricciuto

Publications and source records attributed to Amanda Ricciuto.

3 recordsLinked to original sources

Bayesian hierarchical bootstrap framework for causal subgroup estimation with a time-to-event outcome

Causal estimation of treatment effects within prespecified subgroups, such as biomarker-defined strata, disease phenotypes, or demographic groups are often of clinical interest. Bayesian approaches are attractive for subgroup effect estimation because flexible priors can represent complex treatment heterogeneity and propagate posterior uncertainty. Frequentist methods for prespecified causal subgroup analysis with time-to-event outcomes are also available, including propensity-score weighting approaches that emphasize subgroup-level covariate balance. In the posterior g-formula, subgroup survival estimands also depend on the subgroup-specific distribution of baseline covariates, which may be unstable when some subgroups are small or unevenly represented. Right censoring separately reduces information for the outcome-model component of the estimand, increasing overall uncertainty in subgroup causal survival contrasts. We extend the hierarchical Bayesian bootstrap (HBB) to subgroup causal inference with right-censored time-to-event outcomes. The HBB places a nonparametric hierarchical prior on subgroup-specific baseline covariate distributions, enabling principled borrowing of information across related subgroups while preserving subgroup-specific structure. We combine this distributional regularization with a Bayesian accelerated failure time model and right censoring to perform a posterior g-formula that propagates uncertainty from both the survival model and the subgroup covariate distribution. The resulting framework stabilizes subgroup causal survival estimands in sparse strata without imposing parametric assumptions on the covariate distribution. Simulation studies examine performance across varying degrees of subgroup sparsity and censoring and compare the proposed approach to the popular Bayesian additive regression tree for heterogeneous survival effects.

stat.ME

Machine learning models for estimating counterfactuals in a single-arm inflammatory bowel disease study

Single-arm trials accelerate study timelines by reducing the number of patients that must be recruited for a concurrent control group. However, these designs require an alternative comparator to estimate treatment effects. One approach is to construct a virtual control arm using a machine learning (ML) model trained on external control data to predict the counterfactual outcomes of the treatment arm. Our aim in this study was to leverage virtual controls by developing and evaluating ML-based counterfactual outcome models trained on IFX-treated patients to predict 1-year steroid-free clinical remission (SFCR ) and a composite of C-reactive protein remission plus steroid-free clinical remission (CRP-SFCR) for ADA-treated pediatric Crohn's disease patients, and to compare the resulting IFX-versus-ADA treatment effect estimates with those obtained using propensity score matching to external controls. Five ML models were used to train counterfactual models on the observed IFX cohort data. The resulting models were used to predict the counterfactual outcomes for the ADA arm patients. LGBM yields the best OR closest to the propensity score matched reference, and all 95% CI results align with the conclusion from the reference study that no statistical difference in the primary and secondary outcomes has been observed between the patients treated with ADA or IFX. Our study supports virtual controls as a viable and effective substitute for expensive, lengthy or unethical patient recruitment in an inflammatory bowel disease (IBD) trial. The developed gradient boosted prediction model can be used as a pretrained model to generate IFX counterfactual predictions in future studies, pending external validation and assessment of transportability.

cs.LG

Bayesian Sensitivity Analysis for Causal Estimation with Time-varying Unmeasured Confounding

Causal inference relies on the untestable assumption of no unmeasured confounding. Sensitivity analysis can be used to quantify the impact of unmeasured confounding on causal estimates. Among sensitivity analysis methods proposed in the literature for unmeasured confounding, the latent confounder approach is favoured for its intuitive interpretation via the use of bias parameters to specify the relationship between the observed and unobserved variables and the sensitivity function approach directly characterizes the net causal effect of the unmeasured confounding without explicitly introducing latent variables to the causal models. In this paper, we developed and extended two sensitivity analysis approaches, namely the Bayesian sensitivity analysis with latent confounding variables and the Bayesian sensitivity function approach for the estimation of time-varying treatment effects with longitudinal observational data subjected to time-varying unmeasured confounding. We investigated the performance of these methods in a series of simulation studies and applied them to a multi-center pediatric disease registry data to provide practical guidance on their implementation.

stat.ME