Estimating treatment effect in a proportional hazards model in randomized clinical trials with all-or-nothing compliance.

Abstract:

:We consider methods for estimating the treatment effect and/or the covariate by treatment interaction effect in a randomized clinical trial under noncompliance with time-to-event outcome. As in Cuzick et al. (2007), assuming that the patient population consists of three (possibly latent) subgroups based on treatment preference: the ambivalent group, the insisters, and the refusers, we estimate the effects among the ambivalent group. The parameters have causal interpretations under standard assumptions. The article contains two main contributions. First, we propose a weighted per-protocol (Wtd PP) estimator through incorporating time-varying weights in a proportional hazards model. In the second part of the article, under the model considered in Cuzick et al. (2007), we propose an EM algorithm to maximize a full likelihood (FL) as well as the pseudo likelihood (PL) considered in Cuzick et al. (2007). The E step of the algorithm involves computing the conditional expectation of a linear function of the latent membership, and the main advantage of the EM algorithm is that the risk parameters can be updated by fitting a weighted Cox model using standard software and the baseline hazard can be updated using closed-form solutions. Simulations show that the EM algorithm is computationally much more efficient than directly maximizing the observed likelihood. The main advantage of the Wtd PP approach is that it is more robust to model misspecifications among the insisters and refusers since the outcome model does not impose distributional assumptions among these two groups.

journal_name

Biometrics

journal_title

Biometrics

authors

Li S,Gray RJ

doi

10.1111/biom.12472

subject

Has Abstract

pub_date

2016-09-01 00:00:00

pages

742-50

issue

3

eissn

0006-341X

issn

1541-0420

journal_volume

72

pub_type

杂志文章
  • Models for circular-linear and circular-circular data constructed from circular distributions based on nonnegative trigonometric sums.

    abstract::Johnson and Wehrly (1978, Journal of the American Statistical Association 73, 602-606) and Wehrly and Johnson (1980, Biometrika 67, 255-256) show one way to construct the joint distribution of a circular and a linear random variable, or the joint distribution of a pair of circular random variables from their marginal ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00716.x

    authors: Fernández-Durán JJ

    更新日期:2007-06-01 00:00:00

  • Aberrant crypt foci and semiparametric modeling of correlated binary data.

    abstract::Motivated by the spatial modeling of aberrant crypt foci (ACF) in colon carcinogenesis, we consider binary data with probabilities modeled as the sum of a nonparametric mean plus a latent Gaussian spatial process that accounts for short-range dependencies. The mean is modeled in a general way using regression splines....

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2007.00892.x

    authors: Apanasovich TV,Ruppert D,Lupton JR,Popovic N,Turner ND,Chapkin RS,Carroll RJ

    更新日期:2008-06-01 00:00:00

  • The estimation of maternal genetic variances.

    abstract::The estimation of maternal genetic variances by a multivariate maximum likelihood method is discussed. As an illustration the method is applied to data on Tribolium using a model based on partitioning the maternal genetic effect into additive and dominance components. An alternative model due to Falconer (1965) is als...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Thompson R

    更新日期:1976-12-01 00:00:00

  • A spatial Bayesian latent factor model for image-on-image regression.

    abstract::Image-on-image regression analysis, using images to predict images, is a challenging task, due to (1) the high dimensionality and (2) the complex spatial dependence structures in image predictors and image outcomes. In this work, we propose a novel image-on-image regression model, by extending a spatial Bayesian laten...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13420

    authors: Guo C,Kang J,Johnson TD

    更新日期:2020-12-27 00:00:00

  • On logit confidence intervals for the odds ratio with small samples.

    abstract::Unless the true association is very strong, simple large-sample confidence intervals for the odds ratio based on the delta method perform well even for small samples. Such intervals include the Woolf logit interval and the related Gart interval based on adding .5 before computing the log odds ratio estimate and its st...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.1999.00597.x

    authors: Agresti A

    更新日期:1999-06-01 00:00:00

  • Quantifying the predictive performance of prognostic models for censored survival data with time-dependent covariates.

    abstract::Prognostic models in survival analysis typically aim to describe the association between patient covariates and future outcomes. More recently, efforts have been made to include covariate information that is updated over time. However, there exists as yet no standard approach to assess the predictive accuracy of such ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2007.00889.x

    authors: Schoop R,Graf E,Schumacher M

    更新日期:2008-06-01 00:00:00

  • Ultra high-dimensional semiparametric longitudinal data analysis.

    abstract::As ultra high-dimensional longitudinal data are becoming ever more apparent in fields such as public health and bioinformatics, developing flexible methods with a sparse model is of high interest. In this setting, the dimension of the covariates can potentially grow exponentially as ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13348

    authors: Green B,Lian H,Yu Y,Zu T

    更新日期:2020-08-04 00:00:00

  • The gamma-frailty Poisson model for the nonparametric estimation of panel count data.

    abstract::In this article, we study nonparametric estimation of the mean function of a counting process with panel observations. We introduce the gamma frailty variable to account for the intracorrelation between the panel counts of the counting process and construct a maximum pseudo-likelihood estimate with the frailty variabl...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2003.00126.x

    authors: Zhang Y,Jamshidian M

    更新日期:2003-12-01 00:00:00

  • Maximum likelihood estimation for incomplete repeated-measures experiments under an ARMA covariance structure.

    abstract::A stochastic model is presented for the analysis of incomplete repeated-measures experiments. The general linear model is used to relate the response measures to other variables which are thought to account for inherent variation; an autoregressive moving average (ARMA) time series representation is used to model dist...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Rochon J,Helms RW

    更新日期:1989-03-01 00:00:00

  • Semiparametric regression of multidimensional genetic pathway data: least-squares kernel machines and linear mixed models.

    abstract::We consider a semiparametric regression model that relates a normal outcome to covariates and a genetic pathway, where the covariate effects are modeled parametrically and the pathway effect of multiple gene expressions is modeled parametrically or nonparametrically using least-squares kernel machines (LSKMs). This un...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2007.00799.x

    authors: Liu D,Lin X,Ghosh D

    更新日期:2007-12-01 00:00:00

  • Bayesian model selection for incomplete data using the posterior predictive distribution.

    abstract::We explore the use of a posterior predictive loss criterion for model selection for incomplete longitudinal data. We begin by identifying a property that most model selection criteria for incomplete data should consider. We then show that a straightforward extension of the Gelfand and Ghosh (1998, Biometrika, 85, 1-11...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2012.01766.x

    authors: Daniels MJ,Chatterjee AS,Wang C

    更新日期:2012-12-01 00:00:00

  • Confidence intervals for the generalized ROC criterion.

    abstract::Receiver operating characteristic (ROC) curves are frequently used to assess the usefulness of diagnostic markers. When several diagnostic markers are available, they can be combined by a best linear combination: that is, when the area under the ROC curve of this combination is maximized among all possible linear comb...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Reiser B,Faraggi D

    更新日期:1997-06-01 00:00:00

  • Nonparametric discrete survival function estimation with uncertain endpoints using an internal validation subsample.

    abstract::When a true survival endpoint cannot be assessed for some subjects, an alternative endpoint that measures the true endpoint with error may be collected, which often occurs when obtaining the true endpoint is too invasive or costly. We develop an estimated likelihood function for the situation where we have both uncert...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12316

    authors: Zee J,Xie SX

    更新日期:2015-09-01 00:00:00

  • Heterogeneity models of disease susceptibility, with application to diabetic nephropathy.

    abstract::It is not, in general, possible to include all relevant risk factors in a model of survival or disease incidence. This heterogeneity must be accounted for in the interpretation, as it can imply otherwise unexpected results. This is illustrated by diabetic nephropathy, a serious complication experienced by some diabeti...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Hougaard P,Myglegaard P,Borch-Johnsen K

    更新日期:1994-12-01 00:00:00

  • Marginal analysis of correlated failure time data with informative cluster sizes.

    abstract::We consider modeling correlated survival data when cluster sizes may be informative to the outcome of interest based on a within-cluster resampling (WCR) approach and a weighted score function (WSF) method. We derive the large sample properties for the WCR estimators under the Cox proportional hazards model. We establ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00730.x

    authors: Cong XJ,Yin G,Shen Y

    更新日期:2007-09-01 00:00:00

  • Likelihood ratio tests for a dose-response effect using multiple nonlinear regression models.

    abstract::We consider the problem of testing for a dose-related effect based on a candidate set of (typically nonlinear) dose-response models using likelihood-ratio tests. For the considered models this reduces to assessing whether the slope parameter in these nonlinear regression models is zero or not. A technical problem is t...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12563

    authors: Gutjahr G,Bornkamp B

    更新日期:2017-03-01 00:00:00

  • Confidence interval estimation for the ratio of simple and standardized rates in cohort studies.

    abstract::Computer simulation has been used to compare four methods for calculating confidence intervals for simple rate ratios estimated from cohort studies. The method proposed by Cornfield (1956. In Proceedings of the Third Berkeley Symposium on Mathematical Statistics and Probability. Vol. IV, 135-148) for interval estimati...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Howe GR

    更新日期:1983-06-01 00:00:00

  • On modelling microbial infections.

    abstract::Several models for the course of microbial infections during the incubation period are examined. Each model fits sore throat incubation data very well. Together with the lack of precision of the resulting parameter estimates, this suggests that incubation data alone are insufficient for elucidating the finer details o...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Morgan BJ,Watts SA

    更新日期:1980-06-01 00:00:00

  • Bayesian inferences in the Cox model for order-restricted hypotheses.

    abstract::In studying the relationship between an ordered categorical predictor and an event time, it is standard practice to include dichotomous indicators of the different levels of the predictor in a Cox model. One can then use a multiple degree-of-freedom score or partial likelihood ratio test for hypothesis testing. Often,...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2003.00106.x

    authors: Dunson DB,Herring AH

    更新日期:2003-12-01 00:00:00

  • Randomization inference with general interference and censoring.

    abstract::Interference occurs between individuals when the treatment (or exposure) of one individual affects the outcome of another individual. Previous work on causal inference methods in the presence of interference has focused on the setting where it is a priori assumed that there is "partial interference," in the sense that...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13125

    authors: Loh WW,Hudgens MG,Clemens JD,Ali M,Emch ME

    更新日期:2020-03-01 00:00:00

  • Sample size determination for establishing equivalence/noninferiority via ratio of two proportions in matched-pair design.

    abstract::In this article, we propose approximate sample size formulas for establishing equivalence or noninferiority of two treatments in match-pairs design. Using the ratio of two proportions as the equivalence measure, we derive sample size formulas based on a score statistic for two types of analyses: hypothesis testing and...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2002.00957.x

    authors: Tang ML,Tang NS,Chan IS,Chan BP

    更新日期:2002-12-01 00:00:00

  • Semiparametric frailty models for zero-inflated event count data in the presence of informative dropout.

    abstract::Recurrent events data are commonly encountered in medical studies. In many applications, only the number of events during the follow-up period rather than the recurrent event times is available. Two important challenges arise in such studies: (a) a substantial portion of subjects may not experience the event, and (b) ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13085

    authors: Diao G,Zeng D,Hu K,Ibrahim JG

    更新日期:2019-12-01 00:00:00

  • Comparing the performances of Diggle's tests of spatial randomness for small samples with and without edge-effect correction: application to ecological data.

    abstract::Diggle's tests of spatial randomness based on empirical distributions of interpoint distances can be performed with and without edge-effect correction. We present here numerical results illustrating that tests without the edge-effect correction proposed by Diggle (1979, Biometrics 35, 87-101) have a higher power for s...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.1999.00156.x

    authors: Gignoux J,Duby C,Barot S

    更新日期:1999-03-01 00:00:00

  • Calculating sample size for studies with expected all-or-none nonadherence and selection bias.

    abstract:SUMMARY:We develop sample size formulas for studies aiming to test mean differences between a treatment and control group when all-or-none nonadherence (noncompliance) and selection bias are expected. Recent work by Fay, Halloran, and Follmann (2007, Biometrics 63, 465-474) addressed the increased variances within grou...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2008.01114.x

    authors: Shardell MD,El-Kamary SS

    更新日期:2009-06-01 00:00:00

  • A general noninteractive multiple toxicity model including probit, logit, and Weibull transformations.

    abstract::A multiple toxicity model for the quantal response of organisms is constructed based on an existing bivariate theory. The main assumption is that the tolerances follow a multivariate normal distribution function. However, any monotone tolerance distribution can be applied by mapping the integration region in the n-dim...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Christensen ER,Chen CY

    更新日期:1985-09-01 00:00:00

  • A proposal for a goodness-of-fit test to the Arnason-Schwarz multisite capture-recapture model.

    abstract::In an analysis of capture-recapture data, the identification of a model that fits is a critical step. For the multisite (also called multistate) models used to analyze data gathered at several sites, no reliable test for assessing fit is currently available. We propose a test for the JMV model, a simple generalization...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/1541-0420.00006

    authors: Pradel R,Wintrebert CM,Gimenez O

    更新日期:2003-03-01 00:00:00

  • Nonparametric functional mapping of quantitative trait loci.

    abstract::Functional mapping is a useful tool for mapping quantitative trait loci (QTL) that control dynamic traits. It incorporates mathematical aspects of biological processes into the mixture model-based likelihood setting for QTL mapping, thus increasing the power of QTL detection and the precision of parameter estimation. ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2008.01063.x

    authors: Yang J,Wu R,Casella G

    更新日期:2009-03-01 00:00:00

  • Evaluating multiple diagnostic tests with partial verification.

    abstract::To evaluate diagnostic tests, one would ideally like to verify, for example, with a biopsy, the disease state of all subjects in a study. Often, however, no all subjects are verified. Previous methods for evaluation assume that the decision to verify depends only on recorded variables. Sometimes, particularly if the d...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Baker SG

    更新日期:1995-03-01 00:00:00

  • Criteria for the validation of surrogate endpoints in randomized experiments.

    abstract::The validation of surrogate endpoints has been studied by Prentice (1989, Statistics in Medicine 8, 431-440) and Freedman, Graubard, and Schatzkin (1992, Statistics in Medicine 11, 167-178). We extended their proposals in the cases where the surrogate and the final endpoints are both binary or normally distributed. Le...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Buyse M,Molenberghs G

    更新日期:1998-09-01 00:00:00

  • Fitting nonlinear and constrained generalized estimating equations with optimization software.

    abstract::In this article, we present an estimation approach for solving nonlinear constrained generalized estimating equations that can be implemented using object-oriented software for nonlinear programming, such as nlminb in Splus or fmincon and lsqnonlin in Matlab. We show how standard estimating equation theory includes th...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2000.01268.x

    authors: Contreras M,Ryan LM

    更新日期:2000-12-01 00:00:00