Abstract:
:Risk prediction procedures can be quite useful for the patient's treatment selection, prevention strategy, or disease management in evidence-based medicine. Often, potentially important new predictors are available in addition to the conventional markers. The question is how to quantify the improvement from the new markers for prediction of the patient's risk in order to aid cost-benefit decisions. The standard method, using the area under the receiver operating characteristic curve, to measure the added value may not be sensitive enough to capture incremental improvements from the new markers. Recently, some novel alternatives to area under the receiver operating characteristic curve, such as integrated discrimination improvement and net reclassification improvement, were proposed. In this paper, we consider a class of measures for evaluating the incremental values of new markers, which includes the preceding two as special cases. We present a unified procedure for making inferences about measures in the class with censored event time data. The large sample properties of our procedures are theoretically justified. We illustrate the new proposal with data from a cancer study to evaluate a new gene score for prediction of the patient's survival.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Uno H,Tian L,Cai T,Kohane IS,Wei LJdoi
10.1002/sim.5647subject
Has Abstractpub_date
2013-06-30 00:00:00pages
2430-42issue
14eissn
0277-6715issn
1097-0258journal_volume
32pub_type
杂志文章abstract::To compare the survival functions based on right-truncated data, Lagakos et al. proposed a weighted logrank test based on a reverse time scale. This is in contrast to Bilker and Wang, who suggested a semi-parametric version of the Mann-Whitney test by assuming that the distribution of truncation times is known or can ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2556
更新日期:2007-02-20 00:00:00
abstract::In conventional survival analysis there is an underlying assumption that all study subjects are susceptible to the event. In general, this assumption does not adequately hold when investigating the time to an event other than death. Owing to genetic and/or environmental etiology, study subjects may not be susceptible ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5845
更新日期:2013-10-30 00:00:00
abstract::The primary goal of a phase I trial is to find the maximally tolerated dose (MTD) of a treatment. The MTD is usually defined in terms of a tolerable probability, q(*), of toxicity. Our objective is to find the highest dose with toxicity risk that does not exceed q(*), a criterion that is often desired in designing pha...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.970
更新日期:2002-01-15 00:00:00
abstract::In observational cohort studies we may wish to examine the associations between fixed patient characteristics and the longitudinal changes from baseline in a repeated outcome measure. Many biological and other outcome measures are known to be subject to measurement error and biological variation. In an initial analysi...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3725
更新日期:2009-11-20 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::Relative survival is used to estimate patient survival excluding causes of death not related to the disease of interest. Rather than using cause of death information from death certificates, which is often poorly recorded, relative survival compares the observed survival to that expected in a matched group from the ge...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2399
更新日期:2005-12-30 00:00:00
abstract::The "some invalid, some valid instrumental variable estimator" (sisVIVE) is a lasso-based method for instrumental variables (IVs) regression of outcome on an exposure. In principle, sisVIVE is robust to some of the IVs in the analysis being invalid, in the sense of being related to the outcome variable through pathway...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8066
更新日期:2019-04-30 00:00:00
abstract::This paper investigates the Bayesian procedures for comparing proportions. These procedures are especially suitable for accepting (or rejecting) the equivalence of two population proportions. Furthermore the Bayesian predictive probabilities provide a natural and flexible tool in monitoring trials, especially for choo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780140924
更新日期:1995-05-15 00:00:00
abstract::Exploring the possible reasons for heterogeneity between studies is an important aspect of conducting a meta-analysis. This paper compares a number of methods which can be used to investigate whether a particular covariate, with a value defined for each study in the meta-analysis, explains any heterogeneity. The main ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19991030)18:20<2693::aid-s
更新日期:1999-10-30 00:00:00
abstract::The relative concentration index (RCI) and the absolute concentration index (ACI) have been widely used for monitoring health disparities with ranked health determinants. The RCI has been extended to allow value judgments about inequality aversion by Pereira in 1998 and by Wagstaff in 2002. Previous studies of the ext...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7952
更新日期:2019-01-15 00:00:00
abstract::A statistical analysis is presented of the intensities of admissions to psychiatric hospitals among women in Denmark who either gave birth to a child in the year 1975 or had an induced abortion in the same year. The models used are time-continuous Markov and semi-Markov processes, and the methods employed include non-...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780050305
更新日期:1986-05-01 00:00:00
abstract::To construct a confidence interval for the mean of a log-normal distribution in small samples, we propose likelihood-based approaches - the signed log-likelihood ratio and modified signed log-likelihood ratio methods. Extensive Monte Carlo simulation results show the advantages of the modified signed log-likelihood ra...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1381
更新日期:2003-06-15 00:00:00
abstract::The weighted average treatment effect is a causal measure for the comparison of interventions in a specific target population, which may be different from the population where data are sampled from. For instance, when the goal is to introduce a new treatment to a target population, the question is what efficacy (or ef...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7980
更新日期:2019-02-10 00:00:00
abstract::The original article to which this Correction refers was published in Statistics in Medicine 2000 19(14): 1901-1914. ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/1097-0258(20001115)19:21<3017::aid-sim785>
更新日期:2000-11-15 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
abstract::Variation in heart disease (HD) mortality rates across census tracts is greater than expected given binomial error and available explanatory variables. We extended an extra-binomial variation model for rates standardized by the direct method. The overdispersion parameter accounted for 36 per cent of the observed varia...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780091009
更新日期:1990-10-01 00:00:00
abstract::Paired data from matched case-control studies are commonly used to estimate the association between the exposure to a risk factor and the occurrence of a disease. The odds ratio is typically used to quantify this association. Difficulties in estimating the true odds ratio with matched pairs arise, however, when the ex...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5355
更新日期:2012-11-30 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::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::Although transportation safety has greatly improved over the past 2 decades, motor vehicle crash injuries remain a leading cause of morbidity and mortality, particularly among young drivers. Driver errors and behaviors such as speeding and distraction contribute disproportionately to crashes among inexperienced novice...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7404
更新日期:2017-10-30 00:00:00
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::Comparative calibration is the broad statistical methodology used to assess the calibration of a set of p instruments, each designed to measure the same characteristic, on a common group of individuals. Different from the usual calibration problem, the true underlying quantity measured is unobservable. Many authors ha...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970830)16:16<1889::aid-s
更新日期:1997-08-30 00:00:00
abstract::A new, intuitive method has recently been proposed to explore treatment-covariate interactions in survival data arising from two treatment arms of a clinical trial. The method is based on constructing overlapping subpopulations of patients with respect to one (or more) covariates of interest and in observing the patte...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3524
更新日期:2009-04-15 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::Thomas et al. presented the application of empirical-Bayes methods to the problem of multiple inference in epidemiologic studies. One limitation of their approach, which they noted, was the need to assume exchangeable log relative-risk parameters, and independent relative-risk estimates. Numerical integration was also...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780110208
更新日期:1992-01-30 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::Biomarkers, increasingly used in biomedical studies for the diagnosis and prognosis of acute and chronic diseases, provide insight into the effectiveness of treatments and potential pathways that can be used to guide future treatment targets. The measurement of these markers is often limited by the sensitivity of the ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4503
更新日期:2012-07-30 00:00:00
abstract::This paper presents a statistical analysis of treatment effects in 24-hour ambulatory blood pressure recordings. The statistical models account for circadian rhythms, subject effects, and the effects of treatment with drugs or relaxation therapy. In view of the heterogeneity of the subjects, we fit a separate linear m...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780070608
更新日期:1988-06-01 00:00:00
abstract::The statistical analysis of panel count data has recently attracted a great deal of attention, and a number of approaches have been developed. However, most of these approaches are for situations where the observation and follow-up processes are independent of the underlying recurrent event process unconditional or co...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5724
更新日期:2013-07-30 00:00:00
abstract::In this paper, we consider pooling schemes in which samples are to be tested in two-stages. We show that when batch size is limited as well as pool size, selection schemes tend to be more efficient and flexible. Formulae for the efficiencies of square arrays in all dimensions and for all selection schemes are given in...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3965
更新日期:2010-09-20 00:00:00