Abstract:
:If the sample size for a t-test is calculated on the basis of a prior estimate of the variance then the power of the test at the treatment difference of interest is not robust to misspecification of the variance. We propose a t-test for a two-treatment comparison based on Stein's two-stage test which involves the use of an internal pilot to estimate variance and thus the final sample size required. We evaluate our procedure's performance and show that it controls the type I and II error rates more closely than existing methods for the same problem. We also propose a rule for choosing the size of the internal pilot, and show that this is reasonable in terms of the efficiency of the procedure.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Denne JS,Jennison Cdoi
10.1002/(sici)1097-0258(19990715)18:13<1575::aid-ssubject
Has Abstractpub_date
1999-07-15 00:00:00pages
1575-85issue
13eissn
0277-6715issn
1097-0258pii
10.1002/(SICI)1097-0258(19990715)18:13<1575::AID-Sjournal_volume
18pub_type
杂志文章abstract::Assessment of quality of life is becoming standard in clinical trials. A popular method for measuring quality of life is with instruments which utilize multiple-item subscales, in which each item is scored on a Likert scale. Most statistical methods for the analysis of quality of life data in clinical trials do not ex...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19991115)18:21<2917::aid-s
更新日期:1999-11-15 00:00:00
abstract::A problem occurring in chronic disease modeling is the estimation of transition probabilities of moving from one state of a categorical risk factor to another. Transitions could be obtained from a cohort study, but often such data may not be available. However, under the assumption that transitions remain stable over ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4423
更新日期:2012-03-15 00:00:00
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
更新日期:2007-02-10 00:00:00
abstract::Generalized linear models are often assumed to fit propensity scores, which are used to compute inverse probability weighted (IPW) estimators. To derive the asymptotic properties of IPW estimators, the propensity score is supposed to be bounded away from zero. This condition is known in the literature as strict positi...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7827
更新日期:2018-10-30 00:00:00
abstract::This paper describes compliance with the completion of a quality of life questionnaire in the Breast Cancer Prevention Trial, a large multi-centre randomized trial that is studying the efficacy of Tamoxifen in preventing breast cancer. In the first 4875 women enrolled in the control arm of the study, there was a very ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19980315/15)17:5/7<613::ai
更新日期:1998-03-15 00:00:00
abstract::Standard randomization-based tests of sharp null hypotheses in randomized clinical trials, that is, intent-to-treat analyses, are valid without extraneous assumptions, but generally can be appropriately powerful only with alternative hypotheses that involve treatment assignment having an effect on outcome. In the cont...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19980215)17:3<371::aid-sim
更新日期:1998-02-15 00:00:00
abstract::In recent years randomized trials designed to establish non-inferiority of a new treatment as compared to a standard one have been more widely used. Two-sample statistics have been proposed for this equivalence testing problem. However, they are not suited to situations where a long-term survivor fraction is expected....
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1453
更新日期:2003-03-30 00:00:00
abstract::The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8506
更新日期:2020-05-30 00:00:00
abstract::Many quantitative assay measurements of metabolites of environmental toxicants in clinical investigations are subject to left censoring due to values falling below assay detection limits. Moreover, when observations occur in both unexposed individuals and exposed individuals who reflect a mixture of two distributions ...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1002/sim.2079
更新日期:2005-07-15 00:00:00
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 treatmen...
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(19961015)15:19<2069::AID-S
更新日期:1996-10-15 00:00:00
abstract::There is a rich literature that considers whether an observed relation between treatment and response is due to an unobserved covariate. In order to quantify this unmeasured bias, an assumption is made about the distribution of this unobserved covariate; typically that it is either binary or at least confined to the u...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2344
更新日期:2006-07-15 00:00:00
abstract::Novel therapies are challenging the standards of drug development. Agents with specific biologic targets, unknown dose-efficacy curves, and limited toxicity mandate novel designs to identify biologically optimal doses. We review two model-based designs that utilize either a proportional odds model or a continuation ra...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3706
更新日期:2010-05-10 00:00:00
abstract::It is often assumed that randomisation will prevent bias in estimation of treatment effects from clinical trials, but this is not true of the semiparametric Proportional Hazards model for survival data when there is underlying risk heterogeneity. Here, a new formula is proposed for estimation of this bias, improving o...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7343
更新日期:2017-09-20 00:00:00
abstract::Interpretation of the Mantoux test for tuberculous infection can be complicated by cross-reactions caused by infection with non-specific mycobacteria. Thus, the distribution of positive indurations is a mixture of two distributions. To estimate tuberculous infection prevalence, the marginal distribution of indurations...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.745
更新日期:2001-04-15 00:00:00
abstract::We have formulated the problem of determining whether there has been an upturn in HIV-1 seroconversion incidence over the first five years of follow-up in the Multicenter AIDS Cohort Study (MACS) as that of locating the minimum of a quadratic regression or examination of two-knot piecewise spline models. Under a quadr...
journal_title:Statistics in medicine
pub_type: 杂志文章,多中心研究
doi:10.1002/sim.4780120207
更新日期:1993-01-30 00:00:00
abstract::This paper presents combinatorial (exact) methods that are useful in the analysis of disease cluster data obtained from small environments, such as buildings and neighbourhoods. Maxwell-Boltzmann and Fermi-Dirac occupancy models are compared in terms of appropriateness of representation of disease incidence patterns (...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780121906
更新日期:1993-10-01 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
abstract::Statisticians have long argued that randomized controlled trials should be sufficiently large to achieve their purpose, and for common diseases with major public health implications this has brought many benefits. However, there are many instances where it is unrealistic to expect clinicians to provide the information...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.4780140204
更新日期:1995-01-30 00:00:00
abstract::The purpose of this paper is to show that the sensitivity and specificity estimates obtained by 'discrepant analysis' are biased. Discrepant analysis is a widely used technique that attempts to provide estimates of sensitivity and specificity in the presence of an imperfect gold standard. Many researchers have applied...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970630)16:12<1391::aid-s
更新日期:1997-06-30 00:00:00
abstract::The Box-Cox power exponential (BCPE) distribution, developed in this paper, provides a model for a dependent variable Y exhibiting both skewness and kurtosis (leptokurtosis or platykurtosis). The distribution is defined by a power transformation Y(nu) having a shifted and scaled (truncated) standard power exponential ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1861
更新日期:2004-10-15 00:00:00
abstract::When asking 'what is known' about a drug or therapy or program at any time, both researchers and practitioners often confront more than a single study. Facing a variety of findings, where conflicts may outweigh agreement, how can a reviewer constructively approach the task? In this discussion, I will outline some ques...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780060304
更新日期:1987-04-01 00:00:00
abstract::Assessment of equivalence or non-inferiority in accuracy between two diagnostic procedures often involves comparisons of paired areas under the receiver operating characteristic (ROC) curves. With some pre-specified clinically meaningful limits, the current approach to evaluating equivalence is to perform the two one-...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2358
更新日期:2006-04-15 00:00:00
abstract::This paper reviews methods for mapping geographical variation in disease incidence and mortality. Recent results in Bayesian hierarchical modelling of relative risk are discussed. Two approaches to relative risk estimation, along with the related computational procedures, are described and compared. The first is an em...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780110802
更新日期:1992-06-15 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::Correlation is inherent in longitudinal studies due to the repeated measurements on subjects, as well as due to time-dependent covariates in the study. In the National Longitudinal Study of Adolescent to Adult Health (Add Health), data were repeatedly collected on children in grades 7-12 across four waves. Thus, obser...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8099
更新日期:2019-05-30 00:00:00
abstract::Correlation analysis is widely used in biomedical and psychosocial research for assessing rater reliability, precision of diagnosis and accuracy of proxy outcomes. The popularity of longitudinal study designs has propelled the proliferation in recent years of new methods for longitudinal and other multi-level clustere...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2857
更新日期:2007-09-30 00:00:00
abstract::In biomedical studies and clinical trials, repeated measures are often subject to some upper and/or lower limits of detection. Hence, the responses are either left or right censored. A complication arises when more than one series of responses is repeatedly collected on each subject at irregular intervals over a perio...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8017
更新日期:2019-03-15 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::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 the study of multiple failure times for the same subjects, for example, recurrent infections for patients with a given disease, there are often subject effects, that is, subjects have different risks that cannot be explained by known covariates. Standard methods, which ignore subject effects, lead to overestimation...
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(19980615)17:11<1201::aid-s
更新日期:1998-06-15 00:00:00