AI Article Synopsis

  • - Competing risks data in clinical studies can lead to biased results if not properly analyzed, especially when estimating treatment effects from observational data; the R package causalCmprsk was developed to address this issue.
  • - causalCmprsk utilizes an inverse probability weighting approach to correct for treatment selection bias and employs both non-parametric and semi-parametric methods from survival analysis for causal estimation.
  • - The package features two main functions for analyzing treatment effects: fit.cox for Cox proportional hazards models, and fit.nonpar for flexible modeling; it provides estimators for absolute risks and relative treatment effects that help quantify the impact of treatments in terms of time.

Article Abstract

Background And Objective: Competing risks data arise in both observational and experimental clinical studies with time-to-event outcomes, when each patient might follow one of the multiple mutually exclusive competing paths. Ignoring competing risks in the analysis can result in biased conclusions. In addition, possible confounding bias of the treatment-outcome relationship has to be addressed, when estimating treatment effects from observational data. In order to provide tools for estimation of average treatment effects on time-to-event outcomes in the presence of competing risks, we developed the R package causalCmprsk. We illustrate the package functionality in the estimation of effects of a right heart catheterization procedure on discharge and in-hospital death from observational data.

Methods: The causalCmprsk package implements an inverse probability weighting estimation approach, aiming to emulate baseline randomization and alleviate possible treatment selection bias. The package allows for different types of weights, representing different target populations. causalCmprsk builds on existing methods from survival analysis and adapts them to the causal analysis in non-parametric and semi-parametric frameworks.

Results: The causalCmprsk package has two main functions: fit.cox assumes a semiparametric structural Cox proportional hazards model for the counterfactual cause-specific hazards, while fit.nonpar does not impose any structural assumptions. In both frameworks, causalCmprsk implements estimators of (i) absolute risks for each treatment arm, e.g., cumulative hazards or cumulative incidence functions, and (ii) relative treatment effects, e.g., hazard ratios, or restricted mean time differences. The latter treatment effect measure translates the treatment effect from probability into more intuitive time domain and allows the user to quantify, for example, by how many days or months the treatment accelerates the recovery or postpones illness or death.

Conclusions: The causalCmprsk package provides a convenient and useful tool for causal analysis of competing risks data. It allows the user to distinguish between different causes of the end of follow-up and provides several time-varying measures of treatment effects. The package is accompanied by a vignette that contains more details, examples and code, making the package accessible even for non-expert users.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10841064PMC
http://dx.doi.org/10.1016/j.cmpb.2023.107819DOI Listing

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