Abstract:
:Many extensions of survival models based on the Cox proportional hazards approach have been proposed to handle clustered or multiple event data. Of particular note are five Cox-based models for recurrent event data: Andersen and Gill (AG); Wei, Lin and Weissfeld (WLW); Prentice, Williams and Peterson, total time (PWP-CP) and gap time (PWP-GT); and Lee, Wei and Amato (LWA). Some authors have compared these models by observing differences that arise from fitting the models to real and simulated data. However, no attempt has been made to systematically identify the components of the models that are appropriate for recurrent event data. We propose a systematic way of characterizing such Cox-based models using four key components: risk intervals; baseline hazard; risk set, and correlation adjustment. From the definitions of risk interval and risk set there are conceptually seven such Cox-based models that are permissible, five of which are those previously identified. The two new variant models are termed the 'total time - restricted' (TT-R) and 'gap time - unrestricted' (GT-UR) models. The aim of the paper is to determine which models are appropriate for recurrent event data using the key components. The models are fitted to simulated data sets and to a data set of childhood recurrent infectious diseases. The LWA model is not appropriate for recurrent event data because it allows a subject to be at risk several times for the same event. The WLW model overestimates treatment effect and is not recommended. We conclude that PWP-GT and TT-R are useful models for analysing recurrent event data, providing answers to slightly different research questions. Further, applying a robust variance to any of these models does not adequately account for within-subject correlation.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Kelly PJ,Lim LLdoi
10.1002/(sici)1097-0258(20000115)19:1<13::aid-sim2subject
Has Abstractpub_date
2000-01-15 00:00:00pages
13-33issue
1eissn
0277-6715issn
1097-0258pii
10.1002/(SICI)1097-0258(20000115)19:1<13::AID-SIM2journal_volume
19pub_type
临床试验,杂志文章,随机对照试验abstract::Childhood acute lymphoblastic leukaemia is treated with long-term intensive chemotherapy. During the latter part of the treatment, the maintenance therapy, the patients receive oral doses of two cytostatics. The doses are tailored to blood counts measured on a weekly basis, and the treatment is therefore highly dynami...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4393
更新日期:2012-02-28 00:00:00
abstract::The identification of changes in the recent trend is an important issue in the analysis of cancer mortality and incidence data. We apply a joinpoint regression model to describe such continuous changes and use the grid-search method to fit the regression function with unknown joinpoints assuming constant variance and ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(20000215)19:3<335::aid-sim
更新日期:2000-02-15 00:00:00
abstract::This paper concerns using modified weighted Schoenfeld residuals to test the proportionality of subdistribution hazards for the Fine-Gray model, similar to the tests proposed by Grambsch and Therneau for independently censored data. We develop a score test for the time-varying coefficients based on the modified Schoen...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5815
更新日期:2013-09-30 00:00:00
abstract::Using both simulated and real datasets, we compared two approaches for estimating absolute risk from nested case-control (NCC) data and demonstrated the feasibility of using the NCC design for estimating absolute risk. In contrast to previously published results, we successfully demonstrated not only that data from a ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7143
更新日期:2017-02-10 00:00:00
abstract::The authors measure the efficacy of three methods for predicting the time to infection for susceptible individuals in a population undergoing an HIV epidemic. The methods differ in whether they require detailed information of the contact network and whether they require knowledge of the initial source of infection. Ef...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780130107
更新日期:1994-01-15 00:00:00
abstract::We explore the 'reassessment' design in a logistic regression setting, where a second wave of sampling is applied to recover a portion of the missing data on a binary exposure and/or outcome variable. We construct a joint likelihood function based on the original model of interest and a model for the missing data mech...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6456
更新日期:2015-05-20 00:00:00
abstract::Standard approaches to analysis of randomized controlled trials (RCTs) using Markov models make it difficult to generalize treatment effects to new patient groups and synthesize evidence across trials. This paper demonstrates how pair-wise and mixed treatment comparison meta-analysis can be applied to event history da...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4059
更新日期:2011-01-30 00:00:00
abstract::Clinical trials often assess therapeutic benefit on the basis of an event such as death or the diagnosis of disease. Usually, there are several additional longitudinal measures of clinical status which are collected to be used in the treatment comparison. This paper proposes a simple non-parametric test which combines...
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(19990615)18:11<1341::aid-s
更新日期:1999-06-15 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 the analysis of serial biomarkers to screen and monitor individuals in a given population for onset of a specific disease of interest. The biomarker readings are subject to error. We survey some of the existing literature and concentrate on two recently proposed models. The first is a fully Bayesian hierar...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(20000229)19:4<617::aid-sim
更新日期:2000-02-29 00:00:00
abstract::Phase II studies in oncology are frequently conducted as two-stage single-arm trials with a binary endpoint indicating tumor response. As a common feature of these designs, the sample sizes of the two stages and the decision rules for the interim and the final analysis have to be pre-specified and adhered to strictly ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6479
更新日期:2015-06-15 00:00:00
abstract::In Part I we presented a covariance structure model for analysing measurement error in the assessment of nitrogen intake. In this paper we include data on urine nitrogen excretion which allows a critical assessment of the model proposed. Inclusion of urine nitrogen data produces more pessimistic estimates of the quali...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780121005
更新日期:1993-05-30 00:00:00
abstract::In modern observational studies using electronic health records or other routinely collected data, both the outcome and covariates of interest can be error-prone and their errors often correlated. A cost-effective solution is the two-phase design, under which the error-prone outcome and covariates are observed for all...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8799
更新日期:2021-02-10 00:00:00
abstract::Meta-analysis has generally been accepted as a fundamental tool for combining effect estimates from several studies. For binary studies with rare events, the Peto odds ratio (POR) method has become the relative effect estimator of choice. However, the POR leads to biased estimates for the OR when treatment effects are...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6301
更新日期:2014-12-10 00:00:00
abstract::We analyse the effect of using prevalence rates based on populations with different sizes in the power of spatial independence tests. We compare the well known spatial correlation Moran's index to three indexes obtained after adjusting for population density, one proposed by Oden, another proposed by Waldhör, and a th...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990830)18:16<2147::aid-s
更新日期:1999-08-30 00:00:00
abstract::Ewell and Ibrahim derived the large sample distribution of the logrank statistic under general local alternatives. Their asymptotic results enable us to extend several group sequential designs which allow for early stopping in favour of the null hypothesis to the setting in which the cure rate model is appropriate. In...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/1097-0258(20001130)19:22<3023::aid-sim638>
更新日期:2000-11-30 00:00:00
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
更新日期:1994-03-15 00:00:00
abstract::In the recent two decades, data mining methods for signal detection have been developed for drug safety surveillance, using large post-market safety data. Several of these methods assume that the number of reports for each drug-adverse event combination is a Poisson random variable with mean proportional to the unknow...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6510
更新日期:2015-08-30 00:00:00
abstract::The focus of this paper is the development of a range of cluster detection diagnostics that can be used to assess the degree to which a clustering method recovers the true clustering behaviour of small area data. The diagnostics proposed range from individual region specific diagnostics to neighbourhood diagnostics, a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2401
更新日期:2006-03-15 00:00:00
abstract::In this paper we examine the efficiency of a generalization of the traditional normal linear (LDA) or quadratic (QDA) discriminant analysis. This procedure (the generalized discriminant analysis, GDA) replaces each normal density used in the traditional classification rule by a Fourier series density estimator which '...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780100319
更新日期:1991-03-01 00:00:00
abstract::Comparative studies of the accuracy of diagnostic procedures often use a paired design to gain in efficiency. Standard methods for analysing data from paired designs require complete observations. In many studies, however, one of the test results may be missing for some patients. In this paper, we propose a simple cor...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章
doi:10.1002/(SICI)1097-0258(19960815)15:15<1687::AID-S
更新日期:1996-08-15 00:00:00
abstract::The most popular model used for survival analysis is the proportional hazards regression model proposed by Cox. This is mainly due to its exceptional simplicity. Nevertheless the fundamental assumption of the Cox model is the proportionality of the hazards. For many applications, however, this assumption is doubtful. ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1411
更新日期:2003-04-15 00:00:00
abstract::A popular method for analysing repeated-measures data is generalized estimating equations (GEE). When response data are missing at random (MAR), two modifications of GEE use inverse-probability weighting and imputation. The weighted GEE (WGEE) method involves weighting observations by their inverse probability of bein...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3520
更新日期:2009-03-15 00:00:00
abstract::Longitudinal designs are important in medical research and in many other disciplines. Complete longitudinal studies, in which each subject is evaluated at each measurement occasion, are often very expensive and motivate a search for more efficient designs. Recently developed statistical methods foster the use of inten...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780111411
更新日期:1992-10-01 00:00:00
abstract::The log-rank test is the most powerful non-parametric test for detecting a proportional hazards alternative and thus is the most commonly used testing procedure for comparing time-to-event distributions between different treatments in clinical trials. When the log-rank test is used for the primary data analysis, the s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3501
更新日期:2009-02-28 00:00:00
abstract::The World Health Organization and collaborating institutions in four developing countries have conducted a multi-centre randomized controlled trial, in which clinics were allocated at random to two antenatal care (ANC) models. These were the standard 'Western' ANC model and a 'new' ANC model consisting of tests, clini...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1002/1097-0258(20010215)20:3<401::aid-sim801>3.
更新日期:2001-02-15 00:00:00
abstract::It is naive and incorrect to use the proportions of successful operations to compare the performance of surgeons because the patients' risk profiles are different. In this paper, we explore the use of risk-adjusted procedures to compare the performance of surgeons. One such risk-adjusted statistic is the standardized ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7310
更新日期:2017-07-20 00:00:00
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
更新日期:2018-05-10 00:00:00
abstract::The objective of a phase II cancer clinical trial is to screen a treatment that can produce a similar or better response rate compared to the current treatment results. This screening is usually carried out in two stages as proposed by Simon. For ineffective treatment, the trial should terminate at the first stage. En...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19971215)16:23<2701::aid-s
更新日期:1997-12-15 00:00:00
abstract::Assessing and comparing the performance of correlated predictive scores are of current interest in precision medicine. Given the limitations of available theoretical approaches for assessing and comparing the predictive accuracy, numerical methods are highly desired which, however, have not been systematically develop...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8566
更新日期:2020-09-10 00:00:00