On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes

In randomized clinical trials, methods of pairwise comparisons such as the ‘Net Benefit’ or the ‘win ratio’ have recently gained much attention when interests lies in assessing the effect of a treatment as compared to a standard of care. Among other advantages, these methods are usually praised for delivering a treatment measure that can easily handle multiple outcomes of different nature, while keeping a meaningful interpretation for patients and clinicians. For time-to-event outcomes, a recent suggestion emerged in the literature for estimating these treatment measures by providing a natural handling of censored outcomes. However, this estimation procedure may lead to biased estimates when tails of survival functions cannot be reliably estimated using Kaplan–Meier estimators. The problem then extrapolates to the other outcomes incorporated in the pairwise comparison construction. In this work, we suggest to extend the procedure by the consideration of a hybrid survival function estimator that relies on an extreme value tail model through the Generalized Pareto distribution. We provide an estimator of treatment effect measures that notably improves on bias and remains easily apprehended for practical implementation. This is illustrated in an extensive simulation study as well as in an actual trial of a new cancer immunotherapy.     

Power and sample size calculation for paired right-censored data based on survival copula models

Sample size determination is essential during the planning phases of clinical trials. To calculate the required sample size for paired right-censored data, the structure of the within-paired correlations needs to be pre-specified. In this article, we consider using popular parametric copula models, including the Clayton, Gumbel, or Frank families, to model the distribution of joint survival times. Under each copula model, we derive a sample size formula based on the testing framework for rank-based tests and non-rank-based tests (i.e., logrank test and Kaplan–Meier statistic, respectively). We also investigate how the power or the sample size was affected by the choice of testing methods and copula model under different alternative hypotheses. In addition to this, we examine the impacts of paired-correlations, accrual times, follow-up times, and the loss to follow-up rates on sample size estimation. Finally, two real-world studies are used to illustrate our method and R code is available to the user.     

An unbiased estimator for the survival function of censored data

A monotonic. pointwise unbiased and uniformly consistent estimator for the survival function of failure time under the random censorship model is proposed. This estimator is closely related to the Kaplan-Meier. the Nelson-Aalen. and the reduced sample estimator. Large sample properties of the new estimator are discussed.     

Asymptotic Behaviors of the Lorenz Curve for Censored Data Under Strong Mixing

In this article, we consider a nonparametric estimator of the Lorenz curve under censored dependent model. We show that this estimator is uniformly strongly consistent for the associated Lorenz curve. Also, a strong Gaussian approximation for the associated Lorenz process are established under appropriate assumptions. A law of the iterated logarithm for the Lorenz process is also derived.     

Confidence Intervals for Survival Probabilities: A Comparison Study

The confidence interval of the Kaplan–Meier estimate of the survival probability at a fixed time point is often constructed by the Greenwood formula. This normal approximation-based method can be looked as a Wald type confidence interval for a binomial proportion, the survival probability, using the “effective” sample size defined by Cutler and Ederer. Wald-type binomial confidence interval has been shown to perform poorly comparing to other methods. We choose three methods of binomial confidence intervals for the construction of confidence interval for survival probability: Wilson's method, Agresti–Coull's method, and higher-order asymptotic likelihood method. The methods of “effective” sample size proposed by Peto et al. and Dorey and Korn are also considered. The Greenwood formula is far from satisfactory, while confidence intervals based on the three methods of binomial proportion using Cutler and Ederer's “effective” sample size have much better performance.     

Imputation methods for doubly censored HIV data

In medical research, it is common to have doubly censored survival data: origin time and event time are both subject to censoring. In this paper, we review simple and probability-based methods that are used to impute interval censored origin time and compare the performance of these methods through extensive simulations in the one-sample problem, two-sample problem and Cox regression model problem. The use of a bootstrap procedure for inference is demonstrated.     

On the Kaplan–Meier estimator based on ranked set samples

When quantification of all sampling units is expensive but a set of units can be ranked, without formal measurement, ranked set sampling (RSS) is a cost-efficient alternate to simple random sampling (SRS). In this paper, we study the Kaplan–Meier estimator of survival probability based on RSS under random censoring time setup, and propose nonparametric estimators of the population mean. We present a simulation study to compare the performance of the suggested estimators. It turns out that RSS design can yield a substantial improvement in efficiency over the SRS design. Additionally, we apply the proposed methods to a real data set from an environmental study.     

A jackknife-based versatile test for two-sample problems with right-censored data

For testing the equality of two survival functions, the weighted logrank test and the weighted Kaplan–Meier test are the two most widely used methods. Actually, each of these tests has advantages and defects against various alternatives, while we cannot specify in advance the possible types of the survival differences. Hence, how to choose a single test or combine a number of competitive tests for indicating the diversities of two survival functions without suffering a substantial loss in power is an important issue. Instead of directly using a particular test which generally performs well in some situations and poorly in others, we further consider a class of tests indexed by a weighted parameter for testing the equality of two survival functions in this paper. A delete-1 jackknife method is implemented for selecting weights such that the variance of the test is minimized. Some numerical experiments are performed under various alternatives for illustrating the superiority of the proposed method. Finally, the proposed testing procedure is applied to two real-data examples as well.     

Estimation of reliability with cumulative stress and strength degradation

In many real-life scenarios, system reliability depends on dynamic stress–strength interference where strength degrades and stress accumulates concurrently over time. In this paper, we consider the problem of estimating reliability of a system under deterministic strength degradation and cumulative damage due to shocks arriving according to a point process. Maximum likelihood estimation under two different sampling plans has been considered. Large sample properties in general are discussed. The method is illustrated through simulation and real-life data analysis.