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Accommodating Time-Varying Heterogeneity in Risk Estimation under the Cox Model: A Transfer Learning Approach

容纳风险估计中的时变异质性:Cox 模型下的迁移学习

Transfer learning has attracted increasing attention in recent years for adaptively borrowing information across different data cohorts in various settings. Cancer registries have been widely used in clinical research because of their easy accessibility and large sample size. Our method is motivated by the question of how to utilize cancer registry data as a complement to improve the estimation precision of individual risks of death for inflammatory breast cancer (IBC)…

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Semiparametric Model for Bivariate Survival Data Subject to Biased Sampling

偏倚抽样下二元生存数据的半参数模型

Summary To understand better the relationship between patient characteristics and their residual survival after an intermediate event such as the local recurrence of cancer, it is of interest to identify patients with the intermediate event and then to analyse their residual survival data. One challenge in analysing such data is that the observed residual survival times tend to be longer than those in the target population, since patients who die before experiencing the…

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Semiparametric Estimation of Longitudinal Medical Cost Trajectory

纵向医疗费用轨迹的半参数估计

Estimating the average monthly medical costs from disease diagnosis to a terminal event such as death for an incident cohort of patients is a topic of immense interest to researchers in health policy and health economics because patterns of average monthly costs over time reveal how medical costs vary across phases of care. The statistical challenges to estimating monthly medical costs longitudinally are multifold; the longitudinal cost trajectory (formed by plotting the…

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Score Estimating Equations from Embedded Likelihood Functions Under Accelerated Failure Time Model

加速失效时间模型中基于嵌入似然的得分估计方程

The semiparametric accelerated failure time (AFT) model is one of the most popular models for analyzing time-to-event outcomes. One appealing feature of the AFT model is that the observed failure time data can be transformed to identically independent distributed random variables without covariate effects. We describe a class of estimating equations based on the score functions for the transformed data, which are derived from the full likelihood function under commonly used…

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Maximum Likelihood Estimations and EM Algorithms With Length-Biased Data

长度偏倚数据的最大似然估计与 EM 算法

Length-biased sampling has been well recognized in economics, industrial reliability, etiology applications, epidemiological, genetic and cancer screening studies. Length-biased right-censored data have a unique data structure different from traditional survival data. The nonparametric and semiparametric estimations and inference methods for traditional survival data are not directly applicable for length-biased right-censored data. We propose new expectation-maximization…

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Non-Parametric Tests for Right-Censored Data with Biased Sampling

有偏抽样下右删失数据的非参数检验

Summary Testing the equality of two survival distributions can be difficult in a prevalent cohort study when non-random sampling of subjects is involved. Owing to the biased sampling scheme, the independent censoring assumption is often violated. Although the issues about biased inference caused by length-biased sampling have been widely recognized in the statistical, epidemiological and economical literature, there is no satisfactory solution for efficient two-sample…

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Analyzing Length-Biased Data With Semiparametric Transformation and Accelerated Failure Time Models

利用半参数变换与加速失效时间模型分析长度偏倚数据

Right-censored time-to-event data are often observed from a cohort of prevalent cases that are subject to length-biased sampling. Informative right censoring of data from the prevalent cohort within the population often makes it difficult to model risk factors on the unbiased failure times for the general population, because the observed failure times are length biased. In this paper, we consider two classes of flexible semiparametric models: the transformation models and…

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A Semiparametric Regression Cure Model for Interval-Censored Data

区间删失数据的半参数回归治愈模型

Motivated by medical studies in which patients could be cured of disease but the disease event time may be subject to interval censoring, we presents a semiparametric non-mixture cure model for the regression analysis of interval-censored time-to-event datxa. We develop semiparametric maximum likelihood estimation for the model using the expectation-maximization method for interval-censored data. The maximization step for the baseline function is nonparametric and…

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Inference of Tamoxifen's Effects on Prevention of Breast Cancer From a Randomized Controlled Trial

随机对照试验中他莫昔芬预防乳腺癌效应的推断

The largest randomized, double-blind, placebo-controlled chemoprevention trial, the National Surgical Adjuvant Breast and Bowel Project's Breast Cancer Prevention Trial (NSABP-BCPT), evaluated the efficacy of tamoxifen in the prevention of breast cancer among women at high risk of developing the disease. The trial has reported a reduction of breast cancer incidence for the tamoxifen group. However, the effect of tamoxifen on the time to diagnosis of the disease over the…

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Bayesian adaptive designs for clinical trials

临床试验的贝叶斯自适应设计

A Bayesian adaptive design is proposed for a comparative two-armed clinical trial using decision-theoretic approaches. A loss function is specified, based on the cost for each patient and the costs of making incorrect decisions at the end of a trial. At each interim analysis, the decision to terminate or to continue the trial is based on the expected loss function while concurrently incorporating efficacy, futility and cost. The maximum number of interim analyses is…

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Sample Size Reestimation for Clinical Trials With Censored Survival Data

含删失生存数据的临床试验样本量再估计

A flexible design with updating of sample size in clinical trials is proposed for censored survival data. Patients enter trials serially and are subject to random loss to follow-up. The statistical inference is based on a weighted average of the linear rank statistics, where the weight function at each look depends on prior observed data. A stopping rule is devised to allow early termination and acceptance of the null hypothesis when the experimental treatment offers no…

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Weighted Mean Survival Test Statistics: A Class of Distance Tests for Censored Survival Data

加权平均生存检验统计量:删失生存数据的一类距离检验

Summary A class of test statistics is introduced which is sensitive against the alternative of stochastic ordering in the two-sample censored data problem. The test statistics for evaluating a cumulative weighted difference in survival distributions are developed while taking into account the imbalances in base-line covariates between two groups. This procedure can be used to test the null hypothesis of no treatment effect, especially when base-line hazards cross and…

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