apreprint-march30
Targeted learning of heterogeneous treatment effect curves for right censored or left truncated time-to-event data
Pryce, Matthew, Diaz-Ordaz, Karla, Keogh, Ruth H., Vansteelandt, Stijn
In recent years, there has been growing interest in causal machine learning estimators for quantifying subject-specific effects of a binary treatment on time-to-event outcomes. Estimation approaches have been proposed which attenuate the inherent regularisation bias in machine learning predictions, with each of these estimators addressing measured confounding, right censoring, and in some cases, left truncation. However, the existing approaches are found to exhibit suboptimal finite-sample performance, with none of the existing estimators fully leveraging the temporal structure of the data, yielding non-smooth treatment effects over time. We address these limitations by introducing surv-iTMLE, a targeted learning procedure for estimating the difference in the conditional survival probabilities under two treatments. Unlike existing estimators, surv-iTMLE accommodates both left truncation and right censoring while enforcing smoothness and boundedness of the estimated treatment effect curve over time. Through extensive simulation studies under both right censoring and left truncation scenarios, we demonstrate that surv-iTMLE outperforms existing methods in terms of bias and smoothness of time-varying effect estimates in finite samples. We then illustrate surv-iTMLE's practical utility by exploring heterogeneity in the effects of immunotherapy on survival among non-small cell lung cancer (NSCLC) patients, revealing clinically meaningful temporal patterns that existing estimators may obscure.
- Research Report > Experimental Study (1.00)
- Research Report > New Finding (0.67)
- Health & Medicine > Therapeutic Area > Pulmonary/Respiratory Diseases (1.00)
- Health & Medicine > Therapeutic Area > Oncology > Lung Cancer (0.88)
A Power-Weighted Noncentral Complex Gaussian Distribution
The complex Gaussian distribution has been widely used as a fundamental spectral and noise model in signal processing and communication. However, its Gaussian structure often limits its ability to represent the diverse amplitude characteristics observed in individual source signals. On the other hand, many existing non-Gaussian amplitude distributions derived from hyperspherical models achieve good empirical fit due to their power-law structures, while they do not explicitly account for the complex-plane geometry inherent in complex-valued observations. In this paper, we propose a new probabilistic model for complex-valued random variables, which can be interpreted as a power-weighted noncentral complex Gaussian distribution. Unlike conventional hyperspherical amplitude models, the proposed model is formulated directly on the complex plane and preserves the geometric structure of complex-valued observations while retaining a higher-dimensional interpretation. The model introduces a nonlinear phase diffusion through a single shape parameter, enabling continuous control of the distributional geometry from arc-shaped diffusion along the phase direction to concentration of probability mass toward the origin. We formulate the proposed distribution and analyze the statistical properties of the induced amplitude distribution. The derived amplitude and power distributions provide a unified framework encompassing several widely used distributions in signal modeling, including the Rice, Nakagami, and gamma distributions. Experimental results on speech power spectra demonstrate that the proposed model consistently outperforms conventional distributions in terms of log-likelihood.