Abstract:
:We consider counting process methods for analysing time-to-event data with multiple or recurrent outcomes, using the models developed by Anderson and Gill, Wei, Lin and Weissfeld and Prentice, Williams and Peterson. We compare the methods, and show how to implement them using popular statistical software programs. By analysing three data sets, we illustrate the strengths and pitfalls of each method. The first example is simulated and involves the effect of a hidden covariate. The second is based on a trial of gamma interferon, and behaves remarkably like the first. The third and most interesting example involves both multiple events and discontinuous intervals at risk, and the three approaches give dissimilar answers. We recommend the AG and marginal models for the analysis of this type of data.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Therneau TM,Hamilton SAdoi
10.1002/(sici)1097-0258(19970930)16:18<2029::aid-ssubject
Has Abstractpub_date
1997-09-30 00:00:00pages
2029-47issue
18eissn
0277-6715issn
1097-0258pii
10.1002/(SICI)1097-0258(19970930)16:18<2029::AID-Sjournal_volume
16pub_type
杂志文章abstract::The use of outcome-dependent sampling with longitudinal data analysis has previously been shown to improve efficiency in the estimation of regression parameters. The motivating scenario is when outcome data exist for all cohort members but key exposure variables will be gathered only on a subset. Inference with outcom...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7633
更新日期:2018-06-15 00:00:00
abstract::Binary matched-pairs data occur commonly in longitudinal studies, such as in cross-over experiments. Many analyses for comparing the matched probabilities of a particular outcome do not utilize pairs having the same outcome for each observation. An example is McNemar's test. Some methodologists find this to be counter...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.1589
更新日期:2004-01-15 00:00:00
abstract::Technologic advances give rise to new tests for detecting disease in many fields, including cancer and sexually transmitted disease. Before a new disease screening test is approved for public use, its accuracy should be shown to be better than or at least not inferior to an existing test. Standards do not yet exist fo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1058
更新日期:2002-03-30 00:00:00
abstract::Biomedical studies often measure variables with error. Examples in the literature include investigation of the association between the change in some outcome variable (blood pressure, cholesterol level etc.) and a set of explanatory variables (age, smoking status etc.). Typically, one fits linear regression models to ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19981130)17:22<2597::aid-s
更新日期:1998-11-30 00:00:00
abstract::We extend the shared frailty model of recurrent events and a dependent terminal event to allow for a nonparametric covariate function. We include a Gaussian random effect (frailty) in the intensity functions of both the recurrent and terminal events to capture correlation between the two processes. We employ the penal...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4297
更新日期:2011-09-30 00:00:00
abstract::One Sentence Summary: A Bayesian repeated measures model based on quantitative muscle strength data from a prospective Natural History Study was developed to determine disease progression and design clinical trials for GNE myopathy, a rare and slowly progressive muscle disease. GNE myopathy is a rare muscle disease ch...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8050
更新日期:2019-04-15 00:00:00
abstract::Dependence between observations on a dichotomous variable renders invalid the usual chi-square tests of independence and inflates the variances of parameter estimates. Such a situation occurs, for example, when subjects consist of members of the same family or with repeated observations on the same person. In this pap...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780060408
更新日期:1987-06-01 00:00:00
abstract::One is often interested in the ratio of two variables, for example in genetics, assessing drug effectiveness, and in health economics. In this paper, we derive an explicit geometric solution to the general problem of identifying the two tangents from an arbitrary external point to an ellipse. This solution permits num...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3398
更新日期:2008-12-10 00:00:00
abstract::Knee replacement is the most commonly used surgical treatment for knee arthritis. It has been reported that knee replacement rates vary across both regions and counties. This paper used data from Medicare patients to develop explanations for the variation. One problem with our data is that we do not have patient level...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(SICI)1097-0258(19960915)15:17<1875::AID-S
更新日期:1996-09-15 00:00:00
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journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/1097-0258(20001215)19:23<3291::aid-sim627>
更新日期:2000-12-15 00:00:00
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journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4157
更新日期:2011-04-15 00:00:00
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journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780090114
更新日期:1990-01-01 00:00:00
abstract::In a phase I clinical trial in cancer patients, the drug involved had one known main adverse effect, which also occurs spontaneously in cancer patients with a fairly high frequency. Experiments in rats have shown marked effects of the drug on tumour growth in high doses, but also dose-dependent toxicity. Consequently,...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780140909
更新日期:1995-05-15 00:00:00
abstract::We propose a new design for dose finding for cytotoxic agents in two ordered groups of patients. By ordered groups, we mean that prior to the study there is clinical information that would indicate that for a given dose one group would be more susceptible to toxicities than patients in the other group. The designs are...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7133
更新日期:2017-01-30 00:00:00
abstract::We discuss some of the statistical approaches to the design and analysis of phase I clinical trials in cancer. An attempt is made to identify the issues, particular to this type of trial, that should be addressed by an appropriate methodology. A brief review of schemes currently in use is provided together with our vi...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.4780101104
更新日期:1991-11-01 00:00:00
abstract::In analysis of longitudinal data, the variance matrix of the parameter estimates is usually estimated by the 'sandwich' method, in which the variance for each subject is estimated by its residual products. We propose smooth bootstrap methods by perturbing the estimating functions to obtain 'bootstrapped' realizations ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3027
更新日期:2008-03-30 00:00:00
abstract::Meta-analyses of clinical trials are increasingly seeking to go beyond estimating the effect of a treatment and may also aim to investigate the effect of other covariates and how they alter treatment effectiveness. This requires the estimation of treatment-covariate interactions. Meta-regression can be used to estimat...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2768
更新日期:2007-07-10 00:00:00
abstract::A review is given of different ways of estimating the error rate of a prediction rule based on a statistical model. A distinction is drawn between apparent, optimum and actual error rates. Moreover it is shown how cross-validation can be used to obtain an adjusted predictor with smaller error rate. A detailed discussi...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780091109
更新日期:1990-11-01 00:00:00
abstract::Causal inference for non-censored response variables, such as binary or quantitative outcomes, is often based on either (1) direct standardization ('G-formula') or (2) inverse probability of treatment assignment weights ('propensity score'). To do causal inference in survival analysis, one needs to address right-censo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7297
更新日期:2017-07-30 00:00:00
abstract::Relative survival provides a measure of the proportion of patients dying from the disease under study without requiring the knowledge of the cause of death. We propose an overall strategy based on regression models to estimate the relative survival and model the effects of potential prognostic factors. The baseline ha...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2656
更新日期:2007-05-10 00:00:00
abstract::We consider Cox proportional hazards regression when the covariate vector includes error-prone discrete covariates along with error-free covariates, which may be discrete or continuous. The misclassification in the discrete error-prone covariates is allowed to be of any specified form. Building on the work of Nakamura...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3159
更新日期:2008-05-20 00:00:00
abstract::Spatial scan statistics are widely used for count data to detect geographical disease clusters of high or low incidence, mortality or prevalence and to evaluate their statistical significance. Some data are ordinal or continuous in nature, however, so that it is necessary to dichotomize the data to use a traditional s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2607
更新日期:2007-03-30 00:00:00
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
更新日期:2008-09-30 00:00:00
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
更新日期:1995-06-30 00:00:00
abstract::Comparative effectiveness research represents the kind of research that arguably more directly affects clinical practice and policy. It includes observational studies, clinical trials, and systematic syntheses of existing literature. In this commentary, I argue for the ongoing and critical role of randomization in com...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5400
更新日期:2012-11-10 00:00:00
abstract::We consider several sources of heterogeneity in a clinical trial with patients' survival time as the main response criterion: differences in prognosis which can be attributed to a latent or ignored prognostic factor; differences in treatment efficacy in subgroups of patients, and differences in treatment combinations ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780060708
更新日期:1987-10-01 00:00:00
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
更新日期:1998-12-15 00:00:00
abstract::Both traditional phase I designs and the increasingly popular continual reassessment method (CRM) designs select an estimate of maximum tolerable dose (MTD) from among a set of prespecified dose levels. Although CRM designs use an implied dose-response model to select the next dose level, in general it is neither assu...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.903
更新日期:2001-08-30 00:00:00
abstract::Having a surrogate for a definitive endpoint in a clinical trial can sometimes be useful when it is impractical, invasive or very time consuming to obtain the definitive endpoint. This paper discusses methods for assessing whether the surrogate-endpoint results of a trial can be used in place of definitive-endpoint re...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1779
更新日期:2005-01-30 00:00:00
abstract::Misclassification introduces errors in categorical variables. This paper presents a review of methods for misclassified categorical data in epidemiology. Different sampling schemes for a 2 x 2 x 2 table and methods of analyses will be discussed first. A misclassification matrix is defined, and the usual misclassificat...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.4780080908
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