Estimating individualized treatment rules by optimizing the adjusted probability of a longer survival.
Qijia HeShixiao ZhangMichael L LeBlancYing-Qi ZhaoPublished in: Statistical methods in medical research (2024)
Individualized treatment rules inform tailored treatment decisions based on the patient's information, where the goal is to optimize clinical benefit for the population. When the clinical outcome of interest is survival time, most of current approaches typically aim to maximize the expected time of survival. We propose a new criterion for constructing Individualized treatment rules that optimize the clinical benefit with survival outcomes, termed as the adjusted probability of a longer survival. This objective captures the likelihood of living longer with being on treatment, compared to the alternative, which provides an alternative and often straightforward interpretation to communicate with clinicians and patients. We view it as an alternative to the survival analysis standard of the hazard ratio and the increasingly used restricted mean survival time. We develop a new method to construct the optimal Individualized treatment rule by maximizing a nonparametric estimator of the adjusted probability of a longer survival for a decision rule. Simulation studies demonstrate the reliability of the proposed method across a range of different scenarios. We further perform data analysis using data collected from a randomized Phase III clinical trial (SWOG S0819).
Keyphrases
- clinical trial
- phase iii
- chronic kidney disease
- randomized controlled trial
- end stage renal disease
- machine learning
- palliative care
- climate change
- free survival
- open label
- newly diagnosed
- artificial intelligence
- deep learning
- replacement therapy
- health information
- electronic health record
- smoking cessation
- virtual reality