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 of the parameter estimates for statistical inference. Our extensive simulation studies indicate that the variance estimators by our proposed methods can not only correct the bias of the sandwich estimator but also improve the confidence interval coverage. We applied the proposed method to a data set from a clinical trial of antibiotics for leprosy.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Li Y,Wang YGdoi
10.1002/sim.3027subject
Has Abstractpub_date
2008-03-30 00:00:00pages
937-53issue
7eissn
0277-6715issn
1097-0258journal_volume
27pub_type
杂志文章abstract::Cancer immunotherapy trials have two special features: a delayed treatment effect and a cure rate. Both features violate the proportional hazard model assumption and ignoring either one of the two features in an immunotherapy trial design will result in substantial loss of statistical power. To properly design immunot...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8440
更新日期:2020-03-15 00:00:00
abstract::The setting of a quarantine time for an emerging infectious disease will depend on current knowledge concerning incubation times. Methods for the analysis of information on incubation times are investigated with a particular focus on inference regarding a possible maximum incubation time, after which an exposed indivi...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2206
更新日期:2005-11-30 00:00:00
abstract::To update the British growth reference, anthropometric data for weight, height, body mass index (weight/height2) and head circumference from 17 distinct surveys representative of England, Scotland and Wales (37,700 children, age range 23 weeks gestation to 23 years) were analysed by maximum penalized likelihood using ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:
更新日期:1998-02-28 00:00:00
abstract::Current methods for statistical analysis of twin studies focus on continuous and dichotomous data, while only limited methodology exists for analysing multinomial data. As a consequence, investigators are often tempted to collapse multinomial data into two categories simply to facilitate the analysis. We address this ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/1097-0258(20010130)20:2<249::aid-sim641>3.
更新日期:2001-01-30 00:00:00
abstract::To benefit Alzheimer's disease research, a central data co-ordinating centre (CDCC) is planned that will systematically collect data from 27 Alzheimer's disease centres (ADCs) located nationwide. This CDCC will combine, analyse and disseminate epidemiologic, demographic, clinical and neuropathological data to research...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(20000615/30)19:11/12<1453:
更新日期:2000-06-15 00:00:00
abstract::We present a method to estimate the time-dependent vaccine efficacy from the cohort-specific vaccination coverage and from data on the vaccination status of cases and apply it to a measles epidemic in Germany which involved 529 cases, 88 of whom were vaccinated and 370 unvaccinated (for the remaining 71 cases the vacc...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1043
更新日期:2002-08-30 00:00:00
abstract::The intraclass correlation coefficient rho plays a key role in the design of cluster randomized trials. Estimates of rho obtained from previous cluster trials and used to inform sample size calculation in planned trials may be imprecise due to the typically small numbers of clusters in such studies. It may be useful t...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1643
更新日期:2003-12-30 00:00:00
abstract::Toxicity probability interval designs have received increasing attention as a dose-finding method in recent years. In this study, we compared the two-stage, likelihood-based continual reassessment method (CRM), modified toxicity probability interval (mTPI), and the Bayesian optimal interval design (BOIN) in order to e...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7043
更新日期:2017-01-30 00:00:00
abstract::Pattern-mixture models (PMM) and selection models (SM) are alternative approaches for statistical analysis when faced with incomplete data and a nonignorable missing-data mechanism. Both models make empirically unverifiable assumptions and need additional constraints to identify the parameters. Here, we first introduc...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6302
更新日期:2014-11-30 00:00:00
abstract::Multi-type recurrent event data arise when two or more different kinds of events may occur repeatedly over a period of observation. The scientific objectives in such settings are often to describe features of the marginal processes and to study the association between the different types of events. Interval-censored m...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1936
更新日期:2005-03-15 00:00:00
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
更新日期:2012-06-30 00:00:00
abstract::This paper is a compendium of exact and asymptotic formulae and tables for estimating the sample size in a clinical trial with two treatment groups and a dichotomous outcome. The paper provides separate formulae for equal and unequal treatment group sizes, formulae for the calculation of power given the sample size, a...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/(SICI)1097-0258(19960115)15:1<1::AID-SIM13
更新日期:1996-01-15 00:00:00
abstract::Estimates of relative efficacy between alternative treatments are crucial for decision making in health care. Bayesian mixed treatment comparison models provide a powerful methodology to obtain such estimates when head-to-head evidence is not available or insufficient. In recent years, this methodology has become wide...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5764
更新日期:2013-07-30 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
abstract::We consider bivariate survival times for heterogeneous populations, where heterogeneity induces deviations in an individual's risk of an event as well as associations between survival times. The heterogeneity is characterized by a bivariate frailty model. We measure the heterogeneity effects through deviations associa...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990430)18:8<907::aid-sim
更新日期:1999-04-30 00:00:00
abstract::This article concerns construction of a confidence surface for tangential slopes of the dose-response surface of a combination therapy to identify where response increases as a function of drug dosage. This approach extends to the assessment of the effectiveness of the combination therapy. ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780110513
更新日期:1992-03-01 00:00:00
abstract::The cost and efficiency of training clinical centre staff and of duplicate data entry in clinical trials is reviewed. Training is an essential component of quality assurance programmes and it is usually carried out at regular intervals in long-term clinical trials. Initial training of staff and regular retraining is i...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780090118
更新日期:1990-01-01 00:00:00
abstract::Austin Bradford Hill was once a friend to The Lancet, but, as occasionally happens, friends fall out. The great legacy of his association with the journal, however, was Principles of Medical Statistics. As each edition was succeeded by another--the first in 1937, the last in 1991--he seemed to shift his view about the...
journal_title:Statistics in medicine
pub_type: 传
doi:10.1002/1097-0258(20001215)19:23<3149::aid-sim617>
更新日期:2000-12-15 00:00:00
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
更新日期:1983-04-01 00:00:00
abstract::Juvenile dermatomyositis (JDM) is a rare autoimmune disease that may lead to serious complications, even to death. We develop a 2-state Markov regression model in a Bayesian framework to characterise disease progression in JDM over time and gain a better understanding of the factors influencing disease risk. The trans...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7613
更新日期:2018-05-10 00:00:00
abstract::The back-calculation method has been used to estimate the number of HIV infections from AIDS incidence data in a particular population. We present an extension of back calculation that provides estimates of the numbers of HIV infectives in different stages of infection. We model the staging process with a time-depende...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780110612
更新日期:1992-04-01 00:00:00
abstract::Case-control studies are prone to low power for testing gene-environment interactions (GXE) given the need for a sufficient number of individuals on each strata of disease, gene, and environment. We propose a new study design to increase power by strategically pooling biospecimens. Pooling biospecimens allows us to in...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5357
更新日期:2012-11-20 00:00:00
abstract::The introduction of potent antiretroviral therapies for treatment of HIV infection typically results in a dramatic reduction in plasma HIV RNA concentration, often to levels undetectable by current measurement practices. However, although a high proportion of patients achieve 'undetectability', many then experience a ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1325
更新日期:2003-02-15 00:00:00
abstract::The concept of broad sense agreement (BSA) has recently been proposed for studying the relationship between a continuous measurement and an ordinal measurement. They developed a nonparametric procedure for estimating the BSA index, which is only applicable to completely observed data. In this work, we consider the pro...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8523
更新日期:2020-06-30 00:00:00
abstract::In this paper we give an informal introduction to a robust method for survival analysis which is based on a modification of the usual partial likelihood estimator (PLE). Large sample results lead us to expect reduced bias for this robust estimator compared with the PLE whenever there are even slight violations of the ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(SICI)1097-0258(19960530)15:10<1033::AID-S
更新日期:1996-05-30 00:00:00
abstract::Biological drug products are therapeutic moieties manufactured by a living system or organisms. These are important life-saving drug products for patients with unmet medical needs. Because of expensive cost, only a few patients have access to life-saving biological products. Most of the early biological products will ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5565
更新日期:2013-02-10 00:00:00
abstract::An observed confidence distribution is proposed as a measure of strength of evidence for practically equivalent efficacies of two treatments. The concept is independent of prior opinions about relevant sizes of a difference in efficacy. It also avoids retrospective power calculations for trials with missed recruitment...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780071207
更新日期:1988-12-01 00:00:00
abstract::Joint models initially dedicated to a single longitudinal marker and a single time-to-event need to be extended to account for the rich longitudinal data of cohort studies. Multiple causes of clinical progression are indeed usually observed, and multiple longitudinal markers are collected when the true latent trait of...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6731
更新日期:2016-02-10 00:00:00
abstract::When the event time of interest depends on the censoring time, conventional two-sample test methods, such as the log-rank and Wilcoxon tests, can produce an invalid test result. We extend our previous work on estimation using auxiliary variables to adjust for dependent censoring via multiple imputation, to the compari...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3480
更新日期:2009-02-01 00:00:00
abstract::We discuss a sample size calculation for a pair-matched case-control study to test for interaction between a specific exposure and a second risk factor. The second risk factor could be either binary or continuous. An algorithm for the calculation of sample size is suggested which is based on a logistic regression mode...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(20000415)19:7<923::aid-sim
更新日期:2000-04-15 00:00:00