Multi-state models for colon cancer recurrence and death with a cured fraction.

Abstract:

:In cancer clinical trials, patients often experience a recurrence of disease prior to the outcome of interest, overall survival. Additionally, for many cancers, there is a cured fraction of the population who will never experience a recurrence. There is often interest in how different covariates affect the probability of being cured of disease and the time to recurrence, time to death, and time to death after recurrence. We propose a multi-state Markov model with an incorporated cured fraction to jointly model recurrence and death in colon cancer. A Bayesian estimation strategy is used to obtain parameter estimates. The model can be used to assess how individual covariates affect the probability of being cured and each of the transition rates. Checks for the adequacy of the model fit and for the functional forms of covariates are explored. The methods are applied to data from 12 randomized trials in colon cancer, where we show common effects of specific covariates across the trials.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Conlon AS,Taylor JM,Sargent DJ

doi

10.1002/sim.6056

subject

Has Abstract

pub_date

2014-05-10 00:00:00

pages

1750-66

issue

10

eissn

0277-6715

issn

1097-0258

journal_volume

33

pub_type

杂志文章
  • Deficiencies in clinical reports for registration of drugs.

    abstract::A considerable number of the clinical reports which are presented to the Dutch Board for the Evaluation of Drugs, have deficiencies and/or shortcomings. A number of these, including loose description of the target population and sampling method, methodological flaws, incorrect treatment of withdrawals, confounding of ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780020209

    authors: De Jonge H

    更新日期:1983-04-01 00:00:00

  • Play the winner for phase II/III clinical trials.

    abstract::In comparing two treatments under a typical sequential clinical trial setting, a 50-50 randomization design generates reliable data for making efficient inferences about the treatment difference for the benefit of patients in the general population. However, if the treatment difference is large and the endpoint of the...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19961130)15:22<2413::aid-s

    authors: Yao Q,Wei LJ

    更新日期:1996-11-15 00:00:00

  • R2: a useful measure of model performance when predicting a dichotomous outcome.

    abstract::R2 has been criticized as a measure of model performance when predicting a dichotomous outcome, both because its value is often low and because it is sensitive to the prevalence of the event of interest. The C statistic is more widely used to measure model performance in a 0/1 setting. We use a simple parametric famil...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19990228)18:4<375::aid-sim

    authors: Ash A,Shwartz M

    更新日期:1999-02-28 00:00:00

  • Modelling heterogeneity in clustered count data with extra zeros using compound Poisson random effect.

    abstract::In medical and health studies, heterogeneities in clustered count data have been traditionally modeled by positive random effects in Poisson mixed models; however, excessive zeros often occur in clustered medical and health count data. In this paper, we consider a three-level random effects zero-inflated Poisson model...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3619

    authors: Ma R,Hasan MT,Sneddon G

    更新日期:2009-08-15 00:00:00

  • Estimating the stenosis probabilities in arteriosclerosis obliterans using generalized estimating equations.

    abstract::For each of 211 arteriosclerosis obliterans patients, the degree of stenosis of arteries at four sites were examined at Hiroshima University Hospital to analyse the relationship between the degree of stenosis and age, sex and site. The generalized estimating equations using a proportional odds model for the stenosis p...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1363

    authors: Nakashima E,Tsuji S,Fukuoka H,Ohtaki M,Ito K

    更新日期:2003-07-15 00:00:00

  • Multilevel latent variable models for global health-related quality of life assessment.

    abstract::Quality of life (QOL) assessment is a key component of many clinical studies and frequently requires the use of single global summary measures that capture the overall balance of findings from a potentially wide-ranging assessment of QOL issues. We propose and evaluate an irregular multilevel latent variable model sui...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4455

    authors: Kifley A,Heller GZ,Beath KJ,Bulger D,Ma J,Gebski V

    更新日期:2012-05-20 00:00:00

  • Variance estimators for attributable fraction estimates consistent in both large strata and sparse data.

    abstract::A number of variance formulae for the attributable fraction have been presented, but none is consistent in sparse data, such as found in individually matched case-control studies. This paper employs Mantel-Haenszel estimation to derive variance estimators for attributable fractions that are dually consistent, that is,...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780060607

    authors: Greenland S

    更新日期:1987-09-01 00:00:00

  • Monitoring clinical trials: issues and controversies regarding confidentiality.

    abstract::During phase III clinical trials in life-threatening disease settings, it is important to ensure that the Data Monitoring Committee (DMC) has exclusive access to the interim efficacy and safety data generated by the data analysis centre, in order to minimize the risk of widespread prejudgement of unreliable trial resu...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1288

    authors: Fleming TR,Ellenberg S,DeMets DL

    更新日期:2002-10-15 00:00:00

  • Covariate adjusted weighted normal spatial scan statistics with applications to study geographic clustering of obesity and lung cancer mortality in the United States.

    abstract::In the field of cluster detection, a weighted normal model-based scan statistic was recently developed to analyze regional continuous data and to evaluate the clustering pattern of pre-defined cells (such as state, county, tract, school, hospital) that include many individuals. The continuous measures of interest are,...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3990

    authors: Huang L,Tiwari RC,Pickle LW,Zou Z

    更新日期:2010-10-15 00:00:00

  • Interval estimation for rank correlation coefficients based on the probit transformation with extension to measurement error correction of correlated ranked data.

    abstract::The Spearman (rho(s)) and Kendall (tau) rank correlation coefficient are routinely used as measures of association between non-normally distributed random variables. However, confidence limits for rho(s) are only available under the assumption of bivariate normality and for tau under the assumption of asymptotic norma...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2547

    authors: Rosner B,Glynn RJ

    更新日期:2007-02-10 00:00:00

  • Analyzing survival curves at a fixed point in time.

    abstract::A common problem encountered in many medical applications is the comparison of survival curves. Often, rather than comparison of the entire survival curves, interest is focused on the comparison at a fixed point in time. In most cases, the naive test based on a difference in the estimates of survival is used for this ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2864

    authors: Klein JP,Logan B,Harhoff M,Andersen PK

    更新日期:2007-10-30 00:00:00

  • Two-stage residual inclusion for survival data and competing risks-An instrumental variable approach with application to SEER-Medicare linked data.

    abstract::Instrumental variable is an essential tool for addressing unmeasured confounding in observational studies. Two-stage predictor substitution (2SPS) estimator and two-stage residual inclusion (2SRI) are two commonly used approaches in applying instrumental variables. Recently, 2SPS was studied under the additive hazards...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8071

    authors: Ying A,Xu R,Murphy J

    更新日期:2019-05-10 00:00:00

  • Testing whether genetic variation explains correlation of quantitative measures of gene expression, and application to genetic network analysis.

    abstract::Genetic networks for gene expression data are often built by graphical models, which in turn are built from pair-wise correlations of gene expression levels. A key feature of building graphical models is the evaluation of conditional independence of two traits, given other traits. When conditional independence can be ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3274

    authors: Yu Z,Wang L,Hildebrandt MA,Schaid DJ

    更新日期:2008-08-30 00:00:00

  • Constructing intervals for the intracluster correlation coefficient using Bayesian modelling, and application in cluster randomized trials.

    abstract::Studies in health research are commonly carried out in clustered settings, where the individual response data are correlated within clusters. Estimation and modelling of the extent of between-cluster variation contributes to understanding of the current study and to design of future studies. It is common to express be...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2304

    authors: Turner RM,Omar RZ,Thompson SG

    更新日期:2006-05-15 00:00:00

  • Comparison of methods for estimating the effect of salvage therapy in prostate cancer when treatment is given by indication.

    abstract::For patients who were previously treated for prostate cancer, salvage hormone therapy is frequently given when the longitudinal marker prostate-specific antigen begins to rise during follow-up. Because the treatment is given by indication, estimating the effect of the hormone therapy is challenging. In a previous pape...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5890

    authors: Taylor JM,Shen J,Kennedy EH,Wang L,Schaubel DE

    更新日期:2014-01-30 00:00:00

  • Infant growth modelling using a shape invariant model with random effects.

    abstract::Models for infant growth have usually been based on parametric forms, commonly an exponential or similar model, which have been shown to fit poorly especially during the first year of life. An alternative approach is to use a non-parametric model, based on a shape invariant model (SIM), where a single function is tran...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2718

    authors: Beath KJ

    更新日期:2007-05-30 00:00:00

  • Use of max and min scores for trend tests for association when the genetic model is unknown.

    abstract::In case-control studies, the Cochran-Armitage (CA) trend test is powerful for detection of an association between a risk allele and a marker. To apply this test, a score should be assigned to the genotypes based on the genetic model. When the underlying genetic model is unknown, the trend test statistic is a function ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1474

    authors: Zheng G

    更新日期:2003-08-30 00:00:00

  • A large class of models derived from generalized linear models.

    abstract::Generalized linear models may be extended in several ways. This paper describes five such extensions: (i) generalized additive models; (ii) the use of quasi-likelihood; (iii) joint modelling of mean and dispersion; (iv) introduction of extra random components to give hierarchical generalized linear models; (v) modelli...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19981215)17:23<2747::aid-s

    authors: Nelder JA

    更新日期:1998-12-15 00:00:00

  • Multilevel time series models with applications to repeated measures data.

    abstract::The analysis of repeated measures data can be conducted efficiently using a two-level random coefficients model. A standard assumption is that the within-individual (level 1) residuals are uncorrelated. In some cases, especially where measurements are made close together in time, this may not be reasonable and this ad...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780131605

    authors: Goldstein H,Healy MJ,Rasbash J

    更新日期:1994-08-30 00:00:00

  • Random models for margins of a 2 x 2 contingency table and application to pharmacovigilance.

    abstract::The identification of new adverse drug reactions is often tricky. For a given case, the relationship between drug exposure and symptom occurrence is usually questionable. It could be investigated statistically from a series of drug-event association cases with an independence test between the two variables. Analysing ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780100621

    authors: Tubert P,Begaud B

    更新日期:1991-06-01 00:00:00

  • Effects of time-invariant covariates on the estimation of longitudinal trends for transition mixed models.

    abstract::In this paper, we investigate the impact of time-invariant covariates when fitting transition mixed models. This is carried out by emphasizing on the role of baseline responses on the estimation process. Transition models are allowed for two cases of exogenous and endogenous baseline responses. We illustrate these con...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6270

    authors: Rikhtehgaran R,Kazemi I,Verbeke G

    更新日期:2014-11-30 00:00:00

  • The SAEM algorithm for group comparison tests in longitudinal data analysis based on non-linear mixed-effects model.

    abstract::Non-linear mixed-effects models (NLMEMs) are used to improve information gathering from longitudinal studies and are applied to treatment evaluation in disease-evolution studies, such as human immunodeficiency virus (HIV) infection. The estimation of parameters and the statistical tests are critical issues in NLMEMs s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2950

    authors: Samson A,Lavielle M,Mentré F

    更新日期:2007-11-30 00:00:00

  • Evaluation of surrogate endpoints in randomized experiments with mixed discrete and continuous outcomes.

    abstract::A statistical definition of surrogate endpoints as well as validation criteria was first presented by Prentice. Freedman et al. supplemented these criteria with the so-called proportion explained. Buyse and Molenberghs pointed to inadequacies of these criteria and suggested a new definition of surrogacy based on (i) t...

    journal_title:Statistics in medicine

    pub_type: 评论,杂志文章

    doi:10.1002/sim.923

    authors: Molenberghs G,Geys H,Buyse M

    更新日期:2001-10-30 00:00:00

  • Bayesian clinical trials in action.

    abstract::Although the frequentist paradigm has been the predominant approach to clinical trial design since the 1940s, it has several notable limitations. Advancements in computational algorithms and computer hardware have greatly enhanced the alternative Bayesian paradigm. Compared with its frequentist counterpart, the Bayesi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/sim.5404

    authors: Lee JJ,Chu CT

    更新日期:2012-11-10 00:00:00

  • A prediction-based test for multiple endpoints.

    abstract::This article introduces a global hypothesis test intended for studies with multiple endpoints. Our test makes use of a priori predictions about the direction of the result of each endpoint and we weight these predictions using the sample correlation matrix. The global alternative hypothesis concerns a parameter, ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8724

    authors: Montgomery RN,Mahnken JD

    更新日期:2020-12-10 00:00:00

  • A comparison of likelihood-based and marginal estimating equation methods for analysing repeated ordered categorical responses with missing data: application to an intervention trial of vitamin prophylaxis for oesophageal dysplasia.

    abstract::The purpose of this research was to develop appropriate methods for analysing repeated ordinal categorical data that arose in an intervention trial to prevent oesophageal cancer. The measured response was the degree of oesophageal dysplasia at 2.5 and 6 years after randomization. An important feature was that some res...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780130511

    authors: Mark SD,Gail MH

    更新日期:1994-03-15 00:00:00

  • Violations of the independent increment assumption when using generalized estimating equation in longitudinal group sequential trials.

    abstract::In phase 3 clinical trials, ethical and financial concerns motivate sequential analyses in which the data are analyzed prior to completion of the entire planned study. Existing group sequential software accounts for the effects of these interim analyses on the sampling density by assuming that the contribution of subs...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6306

    authors: Shoben AB,Emerson SS

    更新日期:2014-12-20 00:00:00

  • A pathway analysis method for genome-wide association studies.

    abstract::For genome-wide association studies, we propose a new method for identifying significant biological pathways. In this approach, we aggregate data across single-nucleotide polymorphisms to obtain summary measures at the gene level. We then use a hierarchical Bayesian model, which takes the gene-level summary measures a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4477

    authors: Shahbaba B,Shachaf CM,Yu Z

    更新日期:2012-05-10 00:00:00

  • A simulation study of finite-sample properties of marginal structural Cox proportional hazards models.

    abstract::Motivated by a previously published study of HIV treatment, we simulated data subject to time-varying confounding affected by prior treatment to examine some finite-sample properties of marginal structural Cox proportional hazards models. We compared (a) unadjusted, (b) regression-adjusted, (c) unstabilized, and (d) s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5317

    authors: Westreich D,Cole SR,Schisterman EF,Platt RW

    更新日期:2012-08-30 00:00:00

  • Assessing local influence in principal component analysis with application to haematology study data.

    abstract::In many medical and health studies, high-dimensional data are often encountered. Principal component analysis (PCA) is a commonly used technique to reduce such data to a few components that includes most of the information provided by the original data. However, PCA is known to be very sensitive to some abnormal obser...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2747

    authors: Fung WK,Gu H,Xiang L,Yau KK

    更新日期:2007-06-15 00:00:00