Survival analyses of randomized clinical trials adjusted for patients who switch treatments.

Abstract:

:Patients who switch treatment groups in randomized clinical trials can cause problems in the interpretation of the results. Although the intention-to-treat method is recognized as being the most reliable analysis, it may result in an underestimate of the treatment effect if there have been patients who switch treatments. In this paper, an adjusted analysis, based on a Cox model, is proposed which estimates the treatment effect allowing for patients who switch treatments. Because this analysis requires much weaker assumptions than 'as treated' analyses, it is likely to be much more reliable. The method is illustrated by a trial comparing radical radiotherapy with radiotherapy followed by cystectomy in the treatment of invasive bladder cancer.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Law MG,Kaldor JM

doi

10.1002/(SICI)1097-0258(19961015)15:19<2069::AID-S

subject

Has Abstract

pub_date

1996-10-15 00:00:00

pages

2069-76

issue

19

eissn

0277-6715

issn

1097-0258

pii

10.1002/(SICI)1097-0258(19961015)15:19<2069::AID-S

journal_volume

15

pub_type

临床试验,杂志文章,随机对照试验
  • A random effects model for ordinal responses from a crossover trial.

    abstract::Crossover studies have been successfully conducted in the case of continuous responses. Existing procedures of analysis for ordinal responses, on the other hand, are rarely satisfactory unless strict, usually unrealistic, assumptions are made. In this paper we investigate a random effects model and show that the model...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780100611

    authors: Ezzet F,Whitehead J

    更新日期:1991-06-01 00:00:00

  • Prospective epidemiological studies involving paired organs.

    abstract::Standard methods for analysing survival data or case-control data normally concern factors affecting a subject as a whole. However, in a study of a condition that might develop in one or both of a pair of bodily organs information on response and on covariates may be available for each separately. This information can...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780070509

    authors: Whitehead J,Dorse C

    更新日期:1988-05-01 00:00:00

  • Methods for the statistical analysis of binary data in split-mouth designs with baseline measurements.

    abstract::Many split-mouth trials are characterized by the pairing of site-specific outcome and baseline data within each segment of a subject's mouth. However when the response variable of interest is binary, methods of statistical analysis for this design are not well developed. In this paper we present several analytic appro...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2782

    authors: Donner A,Zou GY

    更新日期:2007-08-15 00:00:00

  • Performance of weighted estimating equations for longitudinal binary data with drop-outs missing at random.

    abstract::The generalized estimating equations (GEE) approach is commonly used to model incomplete longitudinal binary data. When drop-outs are missing at random through dependence on observed responses (MAR), GEE may give biased parameter estimates in the model for the marginal means. A weighted estimating equations approach g...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1241

    authors: Preisser JS,Lohman KK,Rathouz PJ

    更新日期:2002-10-30 00:00:00

  • Comparison of population pharmacokinetic modeling methods using simulated data: results from the Population Modeling Workgroup.

    abstract::Statistical modeling methods have had increasing use in drug disposition studies, both to estimate pharmacokinetic parameters and to develop regression models that relate these parameter estimates to patient characteristics. These methods are particularly flexible as they allow non-linearity and sparse within-patient ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19970615)16:11<1241::aid-s

    authors: Roe DJ

    更新日期:1997-06-15 00:00:00

  • Two-stage sampling in pharmaceutical applications.

    abstract::In the pharmaceutical industry, some tests for quality assurance before, during and after the manufacture of a drug product involve a two-stage sampling technique. We propose statistical testing procedures in a two-stage sampling problem with large within-class sample sizes. We also derive a two-stage sampling plan by...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780122104

    authors: Shao J,Chow SC

    更新日期:1993-11-15 00:00:00

  • Measuring vaccine efficacy from epidemics of acute infectious agents.

    abstract::A good measure of field vaccine efficacy should evaluate the direct protective effect of vaccination on the person who receives the vaccine. The conventional estimator for vaccine efficacy depends on population level factors that are either unrelated or indirectly related to the direct biological action of the vaccine...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780120309

    authors: Longini IM Jr,Halloran ME,Haber M,Chen RT

    更新日期:1993-02-01 00:00:00

  • Modeling hospital length of stay by Coxian phase-type regression with heterogeneity.

    abstract::Hospital length of stay (LOS) is an important measure of healthcare utilization and is generally positively skewed and heterogeneous. We fit a Coxian phase-type distribution to LOS and identify the hidden states of the underlying latent homogeneous Markov model. We demonstrate that selecting an appropriate number of p...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4490

    authors: Tang X,Luo Z,Gardiner JC

    更新日期:2012-06-30 00:00:00

  • Simple models for repeated ordinal responses with an application to a seasonal rhinitis clinical trial.

    abstract::In contrast to other models for ordinal data, the continuation ratio model can be fitted with standard statistical software. This makes it particularly appropriate for large clinical trials with ordinal response variables. In addition, when the trials are longitudinal, this model can be applied to individual responses...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1002/(sici)1097-0258(19971230)16:24<2873::aid-s

    authors: Lindsey JK,Jones B,Ebbutt AF

    更新日期:1997-12-30 00:00:00

  • Flexible multistate models for interval-censored data: Specification, estimation, and an application to ageing research.

    abstract::Continuous-time multistate survival models can be used to describe health-related processes over time. In the presence of interval-censored times for transitions between the living states, the likelihood is constructed using transition probabilities. Models can be specified using parametric or semiparametric shapes fo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7604

    authors: Machado RJM,van den Hout A

    更新日期:2018-05-10 00:00:00

  • A nonparametric smoothing method for assessing GEE models with longitudinal binary data.

    abstract::Studies involving longitudinal binary responses are widely applied in the health and biomedical sciences research and frequently analyzed by generalized estimating equations (GEE) method. This article proposes an alternative goodness-of-fit test based on the nonparametric smoothing approach for assessing the adequacy ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3315

    authors: Lin KC,Chen YJ,Shyr Y

    更新日期:2008-09-30 00:00:00

  • Predicting analysis times in randomized clinical trials.

    abstract::Randomized clinical trial designs commonly include one or more planned interim analyses. At these times an external monitoring committee reviews the accumulated data and determines whether it is scientifically and ethically appropriate for the study to continue. With failure-time endpoints, it is common to schedule an...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.843

    authors: Bagiella E,Heitjan DF

    更新日期:2001-07-30 00:00:00

  • Estimation of the wild-type minimum inhibitory concentration value distribution.

    abstract::Antimicrobial resistance has become one of the main public health burdens of the last decades, and monitoring the development and spread of non-wild-type isolates has therefore gained increased interest. Monitoring is performed based on the minimum inhibitory concentration (MIC) values, which are collected through the...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5939

    authors: Jaspers S,Aerts M,Verbeke G,Beloeil PA

    更新日期:2014-01-30 00:00:00

  • Analyzing longitudinal data to characterize the accuracy of markers used to select treatment.

    abstract::With the increasing availability of detailed clinical information, there is optimism that treatment choices can be selectively directed to those individuals most likely to benefit. While standard clinical trials can establish whether a treatment appears to be effective on average, subsequent work is needed to determin...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6138

    authors: Sitlani CM,Heagerty PJ

    更新日期:2014-07-30 00:00:00

  • Human disease cost network analysis.

    abstract::Diseases can be interconnected. In the recent years, there has been a surge of multidisease studies. Among them, HDN (human disease network) analysis takes a system perspective, examines the interconnections among diseases along with their individual properties, and has demonstrated great potential. Most of the existi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8472

    authors: Ma C,Li Y,Shia B,Ma S

    更新日期:2020-04-30 00:00:00

  • Identifiability and estimation of causal mediation effects with missing data.

    abstract::Mediation analysis is a standard approach to understanding how and why an intervention works in social and medical sciences. However, the presence of missing data, especially missing not at random data, poses a great challenge for the applicability of this approach in practice. Current methods for handling such missin...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7413

    authors: Li W,Zhou XH

    更新日期:2017-11-10 00:00:00

  • Assessing goodness-of-fit of parametric regression models for lifetime data-graphical methods.

    abstract::Graphical methods are often used to check goodness-of-fit of models to data. It is common to plot residuals against a reference distribution so that when the model fits the data, the configuration should be close to a straight line. Since the resemblance to a straight line is often unclear, it has been suggested to ad...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780141607

    authors: Cohen A,Barnett O

    更新日期:1995-08-30 00:00:00

  • Policy developments in regulatory approval.

    abstract::Although radical changes in drug regulation are rare (e.g., the Federal Food, Drug and Cosmetic Act of 1938 and the 1962 amendment to the Act creating an effectiveness requirement), regulations and guidance do evolve significantly in the face of new problems and accumulating experience. Recent changes have been driven...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1298

    authors: Temple R

    更新日期:2002-10-15 00:00:00

  • Logistic regression with incompletely observed categorical covariates--investigating the sensitivity against violation of the missing at random assumption.

    abstract::Missing values in the covariates are a widespread complication in the statistical inference of regression models. The maximum likelihood principle requires specification of the distribution of the covariates, at least in part. For categorical covariates, log-linear models can be used. Additionally, the missing at rand...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780141205

    authors: Vach W,Blettner M

    更新日期:1995-06-30 00:00:00

  • Bounds on natural direct effects in the presence of confounded intermediate variables.

    abstract::In epidemiological studies we often want to learn about the direct effect of an exposure on an outcome, i.e. the effect that is not relayed by a specific intermediate variable. In the literature, there are two common definitions of direct effects; controlled and natural. When the intermediate variable and the outcome ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3493

    authors: Sjölander A

    更新日期:2009-02-15 00:00:00

  • Robust Bayesian sample size determination in clinical trials.

    abstract::This article deals with determination of a sample size that guarantees the success of a trial. We follow a Bayesian approach and we say an experiment is successful if it yields a large posterior probability that an unknown parameter of interest (an unknown treatment effect or an effects-difference) is greater than a c...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3175

    authors: Brutti P,De Santis F,Gubbiotti S

    更新日期:2008-06-15 00:00:00

  • Analysis of cluster randomized cross-over trial data: a comparison of methods.

    abstract::In a cluster randomized cross-over trial, all participating clusters receive both intervention and control treatments consecutively, in separate time periods. Patients recruited by each cluster within the same time period receive the same intervention, and randomization determines order of treatment within a cluster. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2537

    authors: Turner RM,White IR,Croudace T,PIP Study Group.

    更新日期:2007-01-30 00:00:00

  • Genetic association studies with bivariate mixed responses subject to measurement error and misclassification.

    abstract::In genetic association studies, mixed effects models have been widely used in detecting the pleiotropy effects which occur when one gene affects multiple phenotype traits. In particular, bivariate mixed effects models are useful for describing the association of a gene with a continuous trait and a binary trait. Howev...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8688

    authors: Zhang Q,Yi GY

    更新日期:2020-11-20 00:00:00

  • Circular-circular regression model with a spike at zero.

    abstract::With reference to a real data on cataract surgery, we discuss the problem of zero-inflated circular-circular regression when both covariate and response are circular random variables and a large proportion of the responses are zeros. The regression model is proposed, and the estimation procedure for the parameters is ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7496

    authors: Jha J,Biswas A

    更新日期:2018-01-15 00:00:00

  • Exact unconditional tables for significance testing in the 2 x 2 multinomial trial.

    abstract::This paper presents tables analogous to T-tables for use in the 2 x 2 multinomial trial, where the continuity corrected Z-statistic is used to make exact unconditional inference. This is the first solution of a discrete exact unconditional inference problem involving a multivariate nuisance parameter for which no anci...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780110708

    authors: Shuster JJ

    更新日期:1992-05-01 00:00:00

  • Pros and cons of permutation tests in clinical trials.

    abstract::Hypothesis testing, in which the null hypothesis specifies no difference between treatment groups, is an important tool in the assessment of new medical interventions. For randomized clinical trials, permutation tests that reflect the actual randomization are design-based analyses for such hypotheses. This means that ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(20000530)19:10<1319::aid-s

    authors: Berger VW

    更新日期:2000-05-30 00:00:00

  • A missing composite covariate in survival analysis: a case study of the Chinese Longitudinal Health and Longevity Survey.

    abstract::We estimate a Cox proportional hazards model where one of the covariates measures the level of a subject's cognitive functioning by grading the total score obtained by the subject on the items of a questionnaire. A case study is presented where the sample includes partial respondents, who did not answer some questionn...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3773

    authors: Lagona F,Zhang Z

    更新日期:2010-01-30 00:00:00

  • Construction of group sequential designs in clinical trials on the basis of detectable treatment differences.

    abstract::The treatment effect sizes that can be detected with sufficient power up to the different interim analyses constitute a clinically meaningful criterion for the selection of a group sequential test for a clinical trial. For any pre-specified sequence of effect sizes, it is possible to construct group sequential boundar...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1751

    authors: Schäfer H,Müller HH

    更新日期:2004-05-15 00:00:00

  • Sensitivity of Fisher's exact test to minor perturbations in 2 x 2 contingency tables.

    abstract::The two tailed Fisher's exact P value is extremely sensitive to small perturbations in 2 x 2 contingency tables. An example indicates that a 1 per cent increase in the denominator of one treatment group results in a 32 per cent drop in the exact P value, but a mere 0.1 per cent decrease in the treatment success rate. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780050610

    authors: Dupont WD

    更新日期:1986-11-01 00:00:00

  • On the propensity score weighting analysis with survival outcome: Estimands, estimation, and inference.

    abstract::Propensity score analysis is widely used in observational studies to adjust for confounding and estimate the causal effect of a treatment on the outcome. When the outcome is survival time, there are special considerations on the definition of the causal estimand, point, and variance estimation that have not been thoro...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7839

    authors: Mao H,Li L,Yang W,Shen Y

    更新日期:2018-11-20 00:00:00