Estimation of sex‐specific survival with uncertainty in sex assessment

In monomorphic species, determination of sex from behavior is prone to errors. The authors develop capture‐recapture survival models that account for uncertainty in the assessment of sex. They examine parameter redundancy for four basic models with constant or time‐dependent survival and encounter probabilities. They further develop a more refined and more appropriate model for an Audouin's gull data set where four distinct behavioral clues have been used. They examine how useful it is to incorporate the least reliable of the clues and the genetic determination of sex available for only a handful of individuals. They finally discuss the implications of their findings for the design of field studies.     

Flexible Latent‐State Modelling of Old Faithful's Eruption Inter‐Arrival Times in 2009

This paper is concerned with the analysis of a time series comprising the eruption inter‐arrival times of the Old Faithful geyser in 2009. The series is much longer than other well‐documented ones and thus gives a more comprehensive insight into the dynamics of the geyser. Basic hidden Markov models with gamma state‐dependent distributions and several extensions are implemented. In order to better capture the stochastic dynamics exhibited by Old Faithful, the different non‐standard models under consideration seek to increase the flexibility of the basic models in various ways: (i) by allowing non‐geometric distributions for the times spent in the different states; (ii) by increasing the memory of the underlying Markov chain, with or without assuming additional structure implied by mixture transition distribution models; and (iii) by incorporating feedback from the observation process on the latent process. In each case it is shown how the likelihood can be formulated as a matrix product which can be conveniently maximized numerically.     

Multistate recapture models: Modelling incomplete individual histories

Multistate capture-recapture models are a natural generalization of the usual one-site recapture models. Similarly, individuals are sampled on discrete occasions, at which they may be captured or not. However, contrary to the one-site case, the individuals can move within a finite set of states between occasions. The growing interest in spatial aspects of population dynamics presently contributes to making multistate models a very promising tool for population biology. We review first the interest and the potential of multistate models, in particular when they are used with individual states as well as geographical sites. Multistate models indeed constitute canonical capture-recapture models for individual categorical covariates changing over time, and can be linked to longitudinal studies with missing data and models such as hidden Markov chains. Multistate models also provide a promising tool for handling heterogeneity of capture, provided states related to capturability can be defined and used. Such an approach could be relevant for population size estimation in closed populations. Multistate models also constitute a natural framework for mixtures of information in individual history data. Presently, most models can be fit using program MARK. As an example, we present a canonical model for multisite accession to reproduction, which fully generalizes a classical one-site model. In the generalization proposed, one can estimate simultaneously age-dependent rates of accession to reproduction, natal and breeding dispersal. Finally, we discuss further generalizations - such as a multistate generalization of growth rate models and models for data where the state in which an individual is detected is known with uncertainty - and prospects for software development.     

Multistate recapture models: modelling incomplete individual histories

Multistate capture-recapture models are a natural generalization of the usual one-site recapture models. Similarly, individuals are sampled on discrete occasions, at which they may be captured or not. However, contrary to the one-site case, the individuals can move within a finite set of states between occasions. The growing interest in spatial aspects of population dynamics presently contributes to making multistate models a very promising tool for population biology. We review first the interest and the potential of multistate models, in particular when they are used with individual states as well as geographical sites. Multistate models indeed constitute canonical capture-recapture models for individual categorical covariates changing over time, and can be linked to longitudinal studies with missing data and models such as hidden Markov chains. Multistate models also provide a promising tool for handling heterogeneity of capture, provided states related to capturability can be defined and used. Such an approach could be relevant for population size estimation in closed populations. Multistate models also constitute a natural framework for mixtures of information in individual history data. Presently, most models can be fit using program MARK. As an example, we present a canonical model for multisite accession to reproduction, which fully generalizes a classical one-site model. In the generalization proposed, one can estimate simultaneously age-dependent rates of accession to reproduction, natal and breeding dispersal. Finally, we discuss further generalizations - such as a multistate generalization of growth rate models and models for data where the state in which an individual is detected is known with uncertainty - and prospects for software development.     

The time‐dependent “cure‐death” model investigating two equally important endpoints simultaneously in trials treating high‐risk patients with resistant pathogens

A variety of primary endpoints are used in clinical trials treating patients with severe infectious diseases, and existing guidelines do not provide a consistent recommendation. We propose to study simultaneously two primary endpoints, cure and death, in a comprehensive multistate cure‐death model as starting point for a treatment comparison. This technique enables us to study the temporal dynamic of the patient‐relevant probability to be cured and alive. We describe and compare traditional and innovative methods suitable for a treatment comparison based on this model. Traditional analyses using risk differences focus on one prespecified timepoint only. A restricted logrank‐based test of treatment effect is sensitive to ordered categories of responses and integrates information on duration of response. The pseudo‐value regression provides a direct regression model for examination of treatment effect via difference in transition probabilities. Applied to a topical real data example and simulation scenarios, we demonstrate advantages and limitations and provide an insight into how these methods can handle different kinds of treatment imbalances. The cure‐death model provides a suitable framework to gain a better understanding of how a new treatment influences the time‐dynamic cure and death process. This might help the future planning of randomised clinical trials, sample size calculations, and data analyses.     

Multistate Survival Models as Transient Electrical Networks

In multistate survival analysis, the sojourn of a patient through various clinical states is shown to correspond to the diffusion of 1 C of electrical charge through an electrical network. The essential comparison has differentials of probability for the patient to correspond to differentials of charge, and it equates clinical states to electrical nodes. Indeed, if the death state of the patient corresponds to the sink node of the circuit, then the transient current that would be seen on an oscilloscope as the sink output is a plot of the probability density for the survival time of the patient. This electrical circuit analogy is further explored by considering the simplest possible survival model with two clinical states, alive and dead (sink), that incorporates censoring and truncation. The sink output seen on an oscilloscope is a plot of the Kaplan–Meier mass function. Thus, the Kaplan–Meier estimator finds motivation from the dynamics of current flow, as a fundamental physical law, rather than as a nonparametric maximum likelihood estimate (MLE). Generalization to competing risks settings with multiple death states (sinks) leads to cause‐specific Kaplan–Meier submass functions as outputs at sink nodes. With covariates present, the electrical analogy provides for an intuitive understanding of partial likelihood and various baseline hazard estimates often used with the proportional hazards model.     

Estimation with right‐censored observations under a semi‐Markov model

The semi‐Markov process often provides a better framework than the classical Markov process for the analysis of events with multiple states. The purpose of this paper is twofold. First, we show that in the presence of right censoring, when the right end‐point of the support of the censoring time is strictly less than the right end‐point of the support of the semi‐Markov kernel, the transition probability of the semi‐Markov process is nonidentifiable, and the estimators proposed in the literature are inconsistent in general. We derive the set of all attainable values for the transition probability based on the censored data, and we propose a nonparametric inference procedure for the transition probability using this set. Second, the conventional approach to constructing confidence bands is not applicable for the semi‐Markov kernel and the sojourn time distribution. We propose new perturbation resampling methods to construct these confidence bands. Different weights and transformations are explored in the construction. We use simulation to examine our proposals and illustrate them with hospitalization data from a recent cancer survivor study. The Canadian Journal of Statistics 41: 237–256; 2013 © 2013 Statistical Society of Canada     

Non‐parametric Estimation of the Number of Zeros in Truncated Count Distributions

We present some lower bounds for the probability of zero for the class of count distributions having a log‐convex probability generating function, which includes compound and mixed‐Poisson distributions. These lower bounds allow the construction of new non‐parametric estimators of the number of unobserved zeros, which are useful for capture‐recapture models, or in areas like epidemiology and literary style analysis. Some of these bounds also lead to the well‐known Chao's and Turing's estimators. Several examples of application are analysed and discussed.     

Longitudinal data analysis for generalized linear models with follow‐up dependent on outcome‐related variables

In longitudinal studies, observation times are often irregular and subject‐specific. Frequently they are related to the outcome measure or other variables that are associated with the outcome measure but undesirable to condition upon in the model for outcome. Regression analyses that are unadjusted for outcome‐dependent follow‐up then yield biased estimates. The authors propose a class of inverse‐intensity rate‐ratio weighted estimators in generalized linear models that adjust for outcome‐dependent follow‐up. The estimators, based on estimating equations, are very simple and easily computed; they can be used under mixtures of continuous and discrete observation times. The predictors of observation times can be past observed outcomes, cumulative values of outcome‐model covariates and other factors associated with the outcome. The authors validate their approach through simulations and they illustrate it using data from a supported housing program from the US federal government.     

Semiparametric Time Series Models with Log‐concave Innovations: Maximum Likelihood Estimation and its Consistency

We study semiparametric time series models with innovations following a log‐concave distribution. We propose a general maximum likelihood framework that allows us to estimate simultaneously the parameters of the model and the density of the innovations. This framework can be easily adapted to many well‐known models, including autoregressive moving average (ARMA), generalized autoregressive conditionally heteroscedastic (GARCH), and ARMA‐GARCH models. Furthermore, we show that the estimator under our new framework is consistent in both ARMA and ARMA‐GARCH settings. We demonstrate its finite sample performance via a thorough simulation study and apply it to model the daily log‐return of the FTSE 100 index.     

Estimating the variance for heterogeneity in arm‐based network meta‐analysis

Network meta‐analysis can be implemented by using arm‐based or contrast‐based models. Here we focus on arm‐based models and fit them using generalized linear mixed model procedures. Full maximum likelihood (ML) estimation leads to biased trial‐by‐treatment interaction variance estimates for heterogeneity. Thus, our objective is to investigate alternative approaches to variance estimation that reduce bias compared with full ML. Specifically, we use penalized quasi‐likelihood/pseudo‐likelihood and hierarchical (h) likelihood approaches. In addition, we consider a novel model modification that yields estimators akin to the residual maximum likelihood estimator for linear mixed models. The proposed methods are compared by simulation, and 2 real datasets are used for illustration. Simulations show that penalized quasi‐likelihood/pseudo‐likelihood and h‐likelihood reduce bias and yield satisfactory coverage rates. Sum‐to‐zero restriction and baseline contrasts for random trial‐by‐treatment interaction effects, as well as a residual ML‐like adjustment, also reduce bias compared with an unconstrained model when ML is used, but coverage rates are not quite as good. Penalized quasi‐likelihood/pseudo‐likelihood and h‐likelihood are therefore recommended.     

Bayesian analysis of mark‐recapture data with travel time‐dependent survival probabilities

The authors extend the classical Cormack‐Jolly‐Seber mark‐recapture model to account for both temporal and spatial movement through a series of markers (e.g., dams). Survival rates are modeled as a function of (possibly) unobserved travel times. Because of the complex nature of the likelihood, they use a Bayesian approach based on the complete data likelihood, and integrate the posterior through Markov chain Monte Carlo methods. They test the model through simulations and apply it also to actual salmon data arising from the Columbia river system. The methodology was developed for use by the Pacific Ocean Shelf Tracking (POST) project.     

ROBUST ESTIMATION OF SMALL‐AREA MEANS AND QUANTILES

Small‐area estimation techniques have typically relied on plug‐in estimation based on models containing random area effects. More recently, regression M‐quantiles have been suggested for this purpose, thus avoiding conventional Gaussian assumptions, as well as problems associated with the specification of random effects. However, the plug‐in M‐quantile estimator for the small‐area mean can be shown to be the expected value of this mean with respect to a generally biased estimator of the small‐area cumulative distribution function of the characteristic of interest. To correct this problem, we propose a general framework for robust small‐area estimation, based on representing a small‐area estimator as a functional of a predictor of this small‐area cumulative distribution function. Key advantages of this framework are that it naturally leads to integrated estimation of small‐area means and quantiles and is not restricted to M‐quantile models. We also discuss mean squared error estimation for the resulting estimators, and demonstrate the advantages of our approach through model‐based and design‐based simulations, with the latter using economic data collected in an Australian farm survey.     

Building and Fitting Non‐Gaussian Latent Variable Models via the Moment‐Generating Function

Abstract. For certain classes of hierarchical models, it is easy to derive an expression for the joint moment‐generating function (MGF) of data, whereas the joint probability density has an intractable form which typically involves an integral. The most important example is the class of linear models with non‐Gaussian latent variables. Parameters in the model can be estimated by approximate maximum likelihood, using a saddlepoint‐type approximation to invert the MGF. We focus on modelling heavy‐tailed latent variables, and suggest a family of mixture distributions that behaves well under the saddlepoint approximation (SPA). It is shown that the well‐known normalization issue renders the ordinary SPA useless in the present context. As a solution we extend the non‐Gaussian leading term SPA to a multivariate setting, and introduce a general rule for choosing the leading term density. The approach is applied to mixed‐effects regression, time‐series models and stochastic networks and it is shown that the modified SPA is very accurate.     

DYNAMICAL AND STATISTICAL MODELS FOR EXPLOITED POPULATIONS†

Human activities have indirectly modified the dynamics of many populations, accelerating considerably the natural rate of species extinction and raising strong concerns about biodiversity. In many such cases, the underlying ‘natural’ dynamics of the population has been modified by human‐induced increases in mortality, even if the populations are not exploited or harvested in the strict sense. Both dynamical and statistical models are needed to investigate the consequences of human‐induced mortality on the overall dynamics of a population. This paper reviews existing approaches and the potential of recent developments to help form a conceptual and practical framework for analysing the dynamics of exploited populations. It examines both the simple case of an extra source of mortality instantaneously in time, and the theory involved when both risks compete over a continuous time scale. This basic theory is expanded to structured populations, using matrix population models, with applications to the conservation biology of long‐lived vertebrates. The type and degree of compensation expected and approaches to detect it are reviewed, and ways of handling uncertainty are discussed.     

Regression Analysis for Multistate Models Based on a Pseudo-value Approach, with Applications to Bone Marrow Transplantation Studies

Abstract.  Typically, regression analysis for multistate models has been based on regression models for the transition intensities. These models lead to highly nonlinear and very complex models for the effects of covariates on state occupation probabilities. We present a technique that models the state occupation or transition probabilities in a multistate model directly. The method is based on the pseudo-values from a jackknife statistic constructed from non-parametric estimators for the probability in question. These pseudo-values are used as outcome variables in a generalized estimating equation to obtain estimates of model parameters. We examine this approach and its properties in detail for two special multistate model probabilities, the cumulative incidence function in competing risks and the current leukaemia-free survival used in bone marrow transplants. The latter is the probability a patient is alive and in either a first or second post-transplant remission. The techniques are illustrated on a dataset of leukaemia patients given a marrow transplant. We also discuss extensions of the model that are of current research interest.     

Efficient estimation for case‐cohort studies

The author considers time‐to‐event data from case‐cohort designs. As existing methods are either inefficient or based on restrictive assumptions concerning the censoring mechanism, he proposes a semi‐parametrically efficient estimator under the usual assumptions for Cox regression models. The estimator in question is obtained by a one‐step Newton‐Raphson approximation that solves the efficient score equations with initial value obtained from an existing method. The author proves that the estimator is consistent, asymptotically efficient and normally distributed in the limit. He also resorts to simulations to show that the proposed estimator performs well in finite samples and that it considerably improves the efficiency of existing pseudo‐likelihood estimators when a correlate of the missing covariate is available. Although he focuses on the situation where covariates are discrete, the author also explores how the method can be applied to models with continuous covariates.     

Empirical Likelihood Inference for the Rao‐Hartley‐Cochran Sampling Design

The Hartley‐Rao‐Cochran sampling design is an unequal probability sampling design which can be used to select samples from finite populations. We propose to adjust the empirical likelihood approach for the Hartley‐Rao‐Cochran sampling design. The approach proposed intrinsically incorporates sampling weights, auxiliary information and allows for large sampling fractions. It can be used to construct confidence intervals. In a simulation study, we show that the coverage may be better for the empirical likelihood confidence interval than for standard confidence intervals based on variance estimates. The approach proposed is simple to implement and less computer intensive than bootstrap. The confidence interval proposed does not rely on re‐sampling, linearization, variance estimation, design‐effects or joint inclusion probabilities.     

A Class of Convolution‐Based Models for Spatio‐Temporal Processes with Non‐Separable Covariance Structure

Abstract. In this article, we propose a new parametric family of models for real‐valued spatio‐temporal stochastic processes S ( x , t ) and show how low‐rank approximations can be used to overcome the computational problems that arise in fitting the proposed class of models to large datasets. Separable covariance models, in which the spatio‐temporal covariance function of S ( x , t ) factorizes into a product of purely spatial and purely temporal functions, are often used as a convenient working assumption but are too inflexible to cover the range of covariance structures encountered in applications. We define positive and negative non‐separability and show that in our proposed family we can capture positive, zero and negative non‐separability by varying the value of a single parameter.