scholarly journals Regression models for interval censored data using parametric pseudo-observations

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Martin Nygård Johansen ◽  
Søren Lundbye-Christensen ◽  
Jacob Moesgaard Larsen ◽  
Erik Thorlund Parner

Abstract Background Time-to-event data that is subject to interval censoring is common in the practice of medical research and versatile statistical methods for estimating associations in such settings have been limited. For right censored data, non-parametric pseudo-observations have been proposed as a basis for regression modeling with the possibility to use different association measures. In this article, we propose a method for calculating pseudo-observations for interval censored data. Methods We develop an extension of a recently developed set of parametric pseudo-observations based on a spline-based flexible parametric estimator. The inherent competing risk issue with an interval censored event of interest necessitates the use of an illness-death model, and we formulate our method within this framework. To evaluate the empirical properties of the proposed method, we perform a simulation study and calculate pseudo-observations based on our method as well as alternative approaches. We also present an analysis of a real dataset on patients with implantable cardioverter-defibrillators who are monitored for the occurrence of a particular type of device failures by routine follow-up examinations. In this dataset, we have information on exact event times as well as the interval censored data, so we can compare analyses of pseudo-observations based on the interval censored data to those obtained using the non-parametric pseudo-observations for right censored data. Results Our simulations show that the proposed method for calculating pseudo-observations provides unbiased estimates of the cumulative incidence function as well as associations with exposure variables with appropriate coverage probabilities. The analysis of the real dataset also suggests that our method provides estimates which are in agreement with estimates obtained from the right censored data. Conclusions The proposed method for calculating pseudo-observations based on the flexible parametric approach provides a versatile solution to the specific challenges that arise with interval censored data. This solution allows regression modeling using a range of different association measures.

2020 ◽  
Author(s):  
Martin Nygård Johansen ◽  
Søren Lundbye-Christensen ◽  
Jacob Moesgaard Larsen ◽  
Erik Thorlund Parner

Abstract Background: Time-to-event data that is subject to interval censoring is common in the practice of medical research and versatile statistical methods for estimating associations in such settings have been limited. For right censored data, non-parametric pseudo-observations have been proposed as a basis for regression modeling with the possibility to use different association measures. In this article, we propose a method for calculating pseudo-observations for interval censored data. Methods: We develop an extension of a recently developed set of parametric pseudo-observations based on a spline-based flexible parametric estimator. The inherent competing risk issue with an interval censored event of interest necessitates the use of an illness-death model, and we formulate our method within this framework. To evaluate the empirical properties of the proposed method, we perform a simulation study and calculate pseudo-observations based on our method as well as alternative approaches. We also present an analysis of a real dataset on patients with implantable cardioverter-defibrillators who are monitored for the occurrence of a particular type of device failures by routine follow-up examinations. In this dataset, we have information on exact event times as well as the interval censored data, so we can compare analyses of pseudo-observations based on the interval censored data to those obtained using the non-parametric pseudo-observations for right censored data. Results: Our simulations show that the proposed method for calculating pseudo-observations provides unbiased estimates of the cumulative incidence function as well as associations with exposure variables with appropriate coverage probabilities. The analysis of the real dataset also suggests that our method provides estimates which are in agreement with estimates obtained from the right censored data. Conclusions: The proposed method for calculating pseudo-observations based on the flexible parametric approach provides a versatile solution to the specific challenges that arise with interval censored data. This solution allows regression modeling using a range of different association measures.


2009 ◽  
Vol 9 (4) ◽  
pp. 259-297 ◽  
Author(s):  
Guadalupe Gómez ◽  
M Luz Calle ◽  
Ramon Oller ◽  
Klaus Langohr

Interval censoring is encountered in many practical situations when the event of interest cannot be observed and it is only known to have occurred within a time window. The theory for the analysis of interval-censored data has been developed over the past three decades and several reviews have been written. However, it is still a common practice in medical and reliability studies to simplify the interval censoring structure of the data into a more standard right censoring situation by, for instance, imputing the midpoint of the censoring interval. The availability of software for right censoring might well be the main reason for this simplifying practice. In contrast, several methods have been developed to deal with interval-censored data and the corresponding algorithms to make the procedures feasible are scattered across the statistical software or remain behind the personal computers of many researchers. The purpose of this tutorial is to present, in a pedagogical and unified manner, the methodology and the available software for analyzing interval-censored data. The paper covers frequentist non-parametric, parametric and semiparametric estimating approaches, non-parametric tests for comparing survival curves and a section on simulation of interval-censored data. The methods and the software are described using the data from a dental study.


2019 ◽  
Vol 29 (3) ◽  
pp. 752-764 ◽  
Author(s):  
Camille Sabathé ◽  
Per K Andersen ◽  
Catherine Helmer ◽  
Thomas A Gerds ◽  
Hélène Jacqmin-Gadda ◽  
...  

Pseudo-values provide a method to perform regression analysis for complex quantities with right-censored data. A further complication, interval-censored data, appears when events such as dementia are studied in an epidemiological cohort. We propose an extension of the pseudo-value approach for interval-censored data based on a semi-parametric estimator computed using penalised likelihood and splines. This estimator takes interval-censoring and competing risks into account in an illness-death model. We apply the pseudo-value approach to three mean value parameters of interest in studies of dementia: the probability of staying alive and non-demented, the restricted mean survival time without dementia and the absolute risk of dementia. Simulation studies are conducted to examine properties of pseudo-values based on this semi-parametric estimator. The method is applied to the French cohort PAQUID, which included more than 3,000 non-demented subjects, followed for dementia for more than 25 years.


2018 ◽  
Vol 88 (16) ◽  
pp. 3132-3150
Author(s):  
Yuh-Jenn Wu ◽  
Wei-Quan Fang ◽  
Li-Hsueh Cheng ◽  
Kai-Chi Chu ◽  
Yin-Tzer Shih ◽  
...  

2021 ◽  
pp. 096228022110239
Author(s):  
Liuquan Sun ◽  
Shuwei Li ◽  
Lianming Wang ◽  
Xinyuan Song

Failure time data with a cured subgroup are frequently confronted in various scientific fields and many methods have been proposed for their analysis under right or interval censoring. However, a cure model approach does not seem to exist in the analysis of partly interval-censored data, which consist of both exactly observed and interval-censored observations on the failure time of interest. In this article, we propose a two-component mixture cure model approach for analyzing such type of data. We employ a logistic model to describe the cured probability and a proportional hazards model to model the latent failure time distribution for uncured subjects. We consider maximum likelihood estimation and develop a new expectation-maximization algorithm for its implementation. The asymptotic properties of the resulting estimators are established and the finite sample performance of the proposed method is examined through simulation studies. An application to a set of real data on childhood mortality in Nigeria is provided.


2019 ◽  
Vol 29 (8) ◽  
pp. 2151-2166 ◽  
Author(s):  
Shuwei Li ◽  
Qiwei Wu ◽  
Jianguo Sun

Variable selection or feature extraction is fundamental to identify important risk factors from a large number of covariates and has applications in many fields. In particular, its applications in failure time data analysis have been recognized and many methods have been proposed for right-censored data. However, developing relevant methods for variable selection becomes more challenging when one confronts interval censoring that often occurs in practice. In this article, motivated by an Alzheimer’s disease study, we develop a variable selection method for interval-censored data with a general class of semiparametric transformation models. Specifically, a novel penalized expectation–maximization algorithm is developed to maximize the complex penalized likelihood function, which is shown to perform well in the finite-sample situation through a simulation study. The proposed methodology is then applied to the interval-censored data arising from the Alzheimer’s disease study mentioned above.


2016 ◽  
Author(s):  
◽  
Yeqian Liu

[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Failure time data arise in many fields including biomedical studies and industrial life testing. Right-censored failure time data are often observed from a cohort of prevalent cases that are subject to length-biased sampling, which are termed as length-biased and right-censored data. Interval-censored failure time data arise when the failure time of interest in a survival study is not exactly observed but known only to fall within some interval or window. One area that often produces such data is medical studies with periodic follow-ups, in which the medical condition of interest such as the onset of a disease is only known to occur between two adjacent examination times. An important special case of interval-censored data is current status data which arise when each study subject is observed only once and the only information available is whether the failure event of interest has occurred or not by the observation time. Sometimes we also refer current status data as case I interval-censored data and the general case as case II interval-censored data. Semiparametric regression analysis of both right-censored and interval-censored failure time data has recently attracted a great deal of attention. Many procedures have been proposed for their regression analysis under various models. However, in many settings, the population include a cured (nonsusceptible) subpopulation, where only individuals in the susceptible subpopulation will go on to experience the event. Since classical survival models implicitly assume that all individuals will eventually experience the event of interest, they cannot be used in such contexts. They would in fact lead to incorrect results such as, among others, an overestimation of the survival of the non-cured subjects. The research in this dissertation focuses on the statistical analysis for right-censored data with length-biased sampling, interval-censored data with a cured subgroup in the presence of potential dependent censoring and measurement errors. Chapter 1 describes specific examples of right-censored and interval-censored failure time data and reviews the literature on some important topics, including nonparametric and semiparametric estimation, regression analysis in the presence of length-biased sampling and a cured subgroup respectively. Chapter 2 discusses regression analysis of length-biased and right-censored data with with partially linear varying effects. For this problem, we consider quantile regression analysis of right-censored and length-biased data and present a semiparametric varying coefficient partially linear model. For estimation of regression parameters, a three-stage procedure that makes use of the inverse probability weighted technique is developed, and the asymptotic properties of the resulting estimators are established. In addition, the approach allows the dependence of the censoring variable on covariates, while most of the existing methods assume the independence between censoring variables and covariates. A simulation study is conducted and suggests that the proposed approach works well in practical situations. Also an illustrative example is provided. Chapter 3 considers regression analysis of current status data in the presence of a cured subgroup and dependent censoring. For the problem, we develop a sieve maximum likelihood estimation approach with the use of latent variables and Bernstein polynomials. For the determination of the proposed estimators, an EM algorithm and the asymptotic properties of the estimators are established. An extensive simulation study conducted to asses the finite sample performance of the method indicates that it performs well for practical situations. An illustrative example using a data set from a tumor toxicological study is provided. Chapter 4 considers regression analysis of interval-censored data in the presence of a cured subgroup and the case where one or more explanatory variables in the model are subject to measurement errors. These errors should be taken into account in the estimation of the model, to avoid biased estimations. A general approach that exists in the literature is the SIMEX algorithm, a method based on simulations which allows one to estimate the effect of measurement error on the bias of the estimators and to reduce this bias. We extend the SIMEX approach to the mixture cure model with interval-censored data. Comprehensive simulations study as well as a real data application are provided. Several directions for future research are discussed in Chapter 5.


Sign in / Sign up

Export Citation Format

Share Document