Non-parametric Estimation of a Survival Function with Two-stage Design Studies

Abstract.  The two-stage design is popular in epidemiology studies and clinical trials due to its cost effectiveness. Typically, the first stage sample contains cheaper and possibly biased information, while the second stage validation sample consists of a subset of subjects with accurate and complete information. In this paper, we study estimation of a survival function with right-censored survival data from a two-stage design. A non-parametric estimator is derived by combining data from both stages. We also study its large sample properties and derive pointwise and simultaneous confidence intervals for the survival function. The proposed estimator effectively reduces the variance and finite-sample bias of the Kaplan–Meier estimator solely based on the second stage validation sample. Finally, we apply our method to a real data set from a medical device postmarketing surveillance study.     

On the mean squared error of nonparametric quantile estimators under random right-censorship

For randomly right-censored data, new asymptotic expressions for the mean squared errors of the product-limit quantile estimator and a kernel-type quantile estimator are presented in this paper. From these results a comparison of the two quantile estimators with respect to their mean squared errors is given.     

Maximum likelihood estimation of bivariate survival probabilities and its existence and uniqueness

Nonparametric maximum likelihood estimation of bivariate survival probabilities is developed for interval censored survival data. We restrict our attention to the situation where response times within pairs are not distinguishable, and the univariate survival distribution is the same for any individual within any pair. Campbell's (1981) model is modified to incorporate this restriction. Existence and uniqueness of maximum likelihood estimators are discussed. This methodology is illustrated with a bivariate life table analysis of an angioplasty study where each patient undergoes two procedures.     

Identity for the NPMLE in Censored Data Models

We derive an identity for nonparametric maximum likelihood estimators (NPMLE) and regularized MLEs in censored data models which expresses the standardized maximum likelihood estimator in terms of the standardized empirical process. This identity provides an effective starting point in proving both consistency and efficiency of NPMLE and regularized MLE. The identity and corresponding method for proving efficiency is illustrated for the NPMLE in the univariate right-censored data model, the regularized MLE in the current status data model and for an implicit NPMLE based on a mixture of right-censored and current status data. Furthermore, a general algorithm for estimation of the limiting variance of the NPMLE is provided. This revised version was published online in July 2006 with corrections to the Cover Date.     

Aalen's linear model for doubly censored data

Double censoring often occurs in registry studies when left censoring is present in addition to right censoring. In this work, we examine estimation of Aalen's nonparametric regression coefficients based on doubly censored data. We propose two estimation techniques. The first type of estimators, including ordinary least squared (OLS) estimator and weighted least squared (WLS) estimators, are obtained using martingale arguments. The second type of estimator, the maximum likelihood estimator (MLE), is obtained via expectation-maximization (EM) algorithms that treat the survival times of left censored observations as missing. Asymptotic properties, including the uniform consistency and weak convergence, are established for the MLE. Simulation results demonstrate that the MLE is more efficient than the OLS and WLS estimators.     

Variance estimation of the Buckley–James estimator under discrete assumptions

The Buckley–James estimator (BJE) is a widely recognized approach in dealing with right-censored linear regression models. There have been a lot of discussions in the literature on the estimation of the BJE as well as its asymptotic distribution. So far, no simulation has been done to directly estimate the asymptotic variance of the BJE. Kong and Yu [Asymptotic distributions of the Buckley–James estimator under nonstandard conditions, Statist. Sinica 17 (2007), pp. 341–360] studied the asymptotic distribution under discontinuous assumptions. Based on their methodology, we recalculate and correct some missing terms in the expression of the asymptotic variance in Theorem 2 of their work. We propose an estimator of the standard deviation of the BJE by using plug-in estimators. The estimator is shown to be consistent. The performance of the estimator is accessed through simulation studies under discrete underline distributions. We further extend our studies to several continuous underline distributions through simulation. The estimator is also applied to a real medical data set. The simulation results suggest that our estimation is a good approximation to the true standard deviation with reference to the empirical standard deviation.     

A weighted quantile regression for left-truncated and right-censored data

Quantile regression methods have been used to estimate upper and lower quantile reference curves as the function of several covariates. In this article, it is demonstrated that the estimating equation of Zhou [A weighted quantile regression for randomly truncated data, Comput. Stat. Data Anal. 55 (2011), pp. 554–566.] can be extended to analyse left-truncated and right-censored data. We evaluate the finite sample performance of the proposed estimators through simulation studies. The proposed estimator β?(q) is applied to the Veteran's Administration lung cancer data reported by Prentice [Exponential survival with censoring and explanatory variables, Biometrika 60 (1973), pp. 279–288].     

Semiparametric Proportional Mean Residual Life Model With Censoring Indicators Missing at Random

For right-censored survival data, the information that whether the observed time is survival or censoring time is frequently lost. This is the case for the competing risk data. In this article, we consider statistical inference for the right-censored survival data with censoring indicators missing at random under the proportional mean residual life model. Simple and augmented inverse probability weighted estimating equation approaches are developed, in which the nonmissingness probability and some unknown conditional expectations are estimated by the kernel smoothing technique. The asymptotic properties of all the proposed estimators are established, while extensive simulation studies demonstrate that our proposed methods perform well under the moderate sample size. At last, the proposed method is applied to a data set from a stage II breast cancer trial.     

Nonparametric Bayes estimation of gap-time distribution with recurrent event data

Nonparametric Bayes (NPB) estimation of the gap-time survivor function governing the time to occurrence of a recurrent event in the presence of censoring is considered. In our Bayesian approach, the gap-time distribution, denoted by F, has a Dirichlet process prior with parameter α. We derive NPB and nonparametric empirical Bayes (NPEB) estimators of the survivor function F?=1?F and construct point-wise credible intervals. The resulting Bayes estimator of F? extends that based on single-event right-censored data, and the PL-type estimator is a limiting case of this Bayes estimator. Through simulation studies, we demonstrate that the PL-type estimator has smaller biases but higher root-mean-squared errors (RMSEs) than those of the NPB and the NPEB estimators. Even in the case of a mis-specified prior measure parameter α, the NPB and the NPEB estimators have smaller RMSEs than the PL-type estimator, indicating robustness of the NPB and NPEB estimators. In addition, the NPB and NPEB estimators are smoother (in some sense) than the PL-type estimator.     

Second-order variance estimation in poststratified two-stage sampling

The linearization or Taylor series variance estimator and jackknife linearization variance estimator are popular for poststratified point estimators. In this note we propose a simple second-order linearization variance estimator for the poststratified estimator of the population total in two-stage sampling, using the second-order Taylor series expansion. We investigate the properties of the proposed variance estimator and its modified version and their empirical performance through some simulation studies in comparison to the standard and jackknife linearization variance estimators. Simulation studies are carried out on both artificially generated data and real data.     

Confidence regions based on inefficient estimators

We consider an unbiased estimator of a function of mean value parameters, which is not efficient. This inefficient estimator is correlated with a residual vector. Thus, if a unit dispersion is unknown, it is impossible to determine the correct confidence region for a function of mean value parameters via a standard estimator of an unknown dispersion with the exception of the case when the ordinary least squares (OLS) estimator is considered in a model with a special covariance structure such that the OLS and the generalized least squares (GLS) estimator are the same, that is the OLS estimator is efficient. Two different estimators of a unit dispersion independent of an inefficient estimator are derived in a singular linear statistical model. Their quality was verified by simulations for several types of experimental designs. Two new estimators of the unit dispersion were compared with the standard estimators based on the GLS and the OLS estimators of the function of the mean value parameters. The OLS estimator was considered in the incorrect model with a different covariance matrix such that the originally inefficient estimator became efficient. The numerical examples led to a slightly surprising result which seems to be due to data behaviour. An example from geodetic practice is presented in the paper.     

Logspline Density Estimation under Censoring and Truncation

ABSTRACT. In this paper we consider logspline density estimation for data that may be left-truncated or right-censored. For randomly left-truncated and right-censored data the product-limit estimator is known to be a consistent estimator of the survivor function, having a faster rate of convergence than many density estimators. The product-limit estimator and B-splines are used to construct the logspline density estimate for possibly censored or truncated data. Rates of convergence are established when the log-density function is assumed to be in a Besov space. An algorithm involving a procedure similar to maximum likelihood, stepwise knot addition, and stepwise knot deletion is proposed for the estimation of the density function based upon sample data. Numerical examples are used to show the finite-sample performance of inference based on the logspline density estimation.     

Likelihood-Based Inference for Semi-Competing Risks

Consider semi-competing risks data (two times to concurrent events are studied but only one of them is right-censored by the other one) where the link between the times Y and C to non-terminal and terminal events, respectively, is modeled by a family of Archimedean copulas. Moreover, both Y and C are submitted to an independent right censoring variable D. We propose to estimate the parameter of the copula and some resulting survival functions using a pseudo maximum likelihood approach. The main advantage of this procedure is that it extends to multidimensional parameters copulas. We perform simulations to study the behavior of our estimation procedure and its impact on other related estimators and we apply our method to real data coming from a study on the Hodgkin disease.     

A New Kernel Distribution Function Estimator Based on a Non-parametric Transformation of the Data

Abstract.  A new kernel distribution function (df) estimator based on a non-parametric transformation of the data is proposed. It is shown that the asymptotic bias and mean squared error of the estimator are considerably smaller than that of the standard kernel df estimator. For the practical implementation of the new estimator a data-based choice of the bandwidth is proposed. Two possible areas of application are the non-parametric smoothed bootstrap and survival analysis. In the latter case new estimators for the survival function and the mean residual life function are derived.     

Large sample properties of nonparametric copula estimators under bivariate censoring

A new nonparametric estimator is proposed for the copula function of a bivariate survival function for data subject to random right-censoring. We consider two censoring models: univariate and copula censoring. We show strong consistency and we obtain an i.i.d. representation for the copula estimator. In a simulation study we compare the new estimator to the one of Gribkova and Lopez [Nonparametric copula estimation under bivariate censoring; doi:10.1111/sjos.12144].     

Small sample study on estimators of life distributions and of mean survival times using randomly censored data

Whereas large-sample properties of the estimators of survival distributions using censored data have been studied by many authors, exact results for small samples have been difficult to obtain. In this paper we obtain the exact expression for the ath moment (a > 0) of the Bayes estimator of survival distribution using the censored data under proportional hazard model. Using the exact expression we compute the exact mean, variance and MSE of the Bayes estimator. Also two estimators ofthe mean survival time based on the Kaplan-Meier estimator and the Bayes estimator are compared for small samples under proportional hazards.     

Nonparametric inference on mean residual life function with length-biased right-censored data

Abstract

In survival or reliability studies, the mean residual life (MRL) function is an important characteristic in understanding the survival or ageing process. In this article, we consider the problem of nonparametric MRL function estimation with length-biased right-censored data. Two nonparametric estimators of the MRL are proposed and their weak convergence is presented. In order to evaluate the performance of these estimators, small Monte Carlo simulations are carried out. Results show that the proposed estimators work well especially when the sample size is small and their calculations are simple. Finally, a real data example is provided.     

A modified kernel quantile estimator under censoring

Based on right-censored data from a lifetime distribution F₀, a modification of the kernel quantile estimator is proposed. The advantage of this estimator is that the data play a role in the degree of smoothing of the estimator while retaining the desirable features of the kernel estimator. Convergence in probability and almost sure convergence of the estimator are discussed. Also, asymptotic normality and confidence bands are presented and some examples are given.     

Asymptotic Properties of Generalized Multivariate Rank Statistics

This article discusses generalization of the well-known multivariate rank statistics under right-censored data case. Empirical process representation used to get the generalization. The marginal distribution functions are estimated by Kaplan–Meier estimators. Sufficient conditions for asymptotic normality of the generalized multivariate rank statistics under independently right censored data are specified. Several auxiliary results on sup-norm convergence of Kaplan–Meier estimators in randomly exhausting regions are given too.     

The Robustness of Several Estimators of the Survivorship Function with Randomly Censored Data

The problem of estimating the survivorship function, R(t) = P(T > t), arises frequently in both engineering and biomedical sciences. In many applications the data one sees are censored due to the occurrence of some competing cause of failure such as withdrawal from the study, failure from some cause not under study, etc. In the biomedical sciences the distribution free estimator suggested by Kaplan and Meier (JASA 1958) is routinely used, while in the engineering sciences a parametric approach is more commonly used. In this report we study the efficiency of these two techniques when a particular parametric model such as the exponential, Weibull, normal, log normal, exponential power, Pareto, Gompertz, gamma, or bathtub shaped hazard distribution is assumed under a variety of censoring schemes and underlying failure models. We conclude that in most cases the parametric estimators outperform the distribution free estimator. The results are particularly striking if the Weibull forms of these estimators are used routinely.