Estimating heterogeneous treatment effects for latent subgroups in observational studies.

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

authors： Mandrekar SJ,Qin R,Sargent DJ

更新日期：2010-05-10 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

authors： Piaggio G,Carroli G,Villar J,Pinol A,Bakketeig L,Lumbiganon P,Bergsjø P,Al-Mazrou Y,Ba'aqeel H,Belizán JM,Farnot U,Berendes H,WHO Antenatal Care Trial Research Group.

更新日期：2001-02-15 00:00:00

abstract：:We consider using observational data to estimate the effect of a treatment on disease recurrence, when the decision to initiate treatment is based on longitudinal factors associated with the risk of recurrence. The effect of salvage androgen deprivation therapy (SADT) on the risk of recurrence of prostate cancer is in...

journal_title：Statistics in medicine

authors： Kennedy EH,Taylor JM,Schaubel DE,Williams S

更新日期：2010-11-10 00:00:00

abstract：:The goal of screening programmes for cancer is early detection and treatment with a consequent reduction in mortality from the disease. Screening programmes need to assess the true benefit of screening, that is, the length of time of extension of survival beyond the time of advancement of diagnosis (lead-time). This p...

journal_title：Statistics in medicine

authors： Xu JL,Prorok PC

更新日期：1995-12-30 00:00:00

abstract：:The design of clinical trials often requires knowledge of quantities such as between- and within subject variances about which only imprecise information exists. To provide assurance that the study has the desired power to detect a minimum clinically meaningful difference between treatment groups. Gould, Gould and Shi...

journal_title：Statistics in medicine

authors： Shih WJ,Gould AL

更新日期：1995-10-30 00:00:00

abstract：:We consider the design of both cohort and case-control studies in which an initial ('stage 1') sample of complete data on an error-free disease indicator (D), a correct ('gold standard') dichotomous exposure measurement (X) and an error-prone exposure measurement (Z) are available. We calculate the amount of additiona...

journal_title：Statistics in medicine

authors： Dahm PF,Gail MH,Rosenberg PS,Pee D

更新日期：1995-12-15 00:00:00

abstract：:Before introducing a new measurement tool it is necessary to evaluate its performance. Several statistical methods have been developed, or used, to evaluate the reliability and validity of a new assessment method in such circumstances. In this paper we review some commonly used methods. Data from a study that was cond...

journal_title：Statistics in medicine

authors： White SA,van den Broek NR

更新日期：2004-05-30 00:00:00

abstract：:Laboratory determinations on children aged 6 to 10 years obtained over a 5-year period are analysed by a method described in detail for differentiating between children from exposed and control areas of Seveso, Italy. In the analysis, stratification is employed to distinguish the separate days of laboratory measuremen...

journal_title：Statistics in medicine

authors： Mantel N,Mocarelli P,Marocchi A,Brambilla P,Baretta R

更新日期：1983-04-01 00:00:00

abstract：:I reflect on the statistical methods of the Christakis-Fowler studies on network-based contagion of traits by checking the sensitivity of these kinds of results to various alternate specifications and generative mechanisms. Despite the honest efforts of all involved, I remain pessimistic about establishing whether bin...

journal_title：Statistics in medicine

authors： Thomas AC

更新日期：2013-02-20 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

authors： Jung I,Kulldorff M,Klassen AC

更新日期：2007-03-30 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

authors： Assunção RM,Reis EA

更新日期：1999-08-30 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

authors： Donald A,Donner A

更新日期：1987-06-01 00:00:00

abstract：:Box-Cox transformation is investigated for regression models for left-censored data. Examples are provided using coronary calcification data from the Multi-Ethnic Study of Atherosclerosis and pharmacokinetic data of a nicotine nasal spray. ...

journal_title：Statistics in medicine

authors： Han C,Kronmal R

更新日期：2004-12-15 00:00:00

abstract：:In order for national statistical offices to maintain the trust of the public to collect data and publish statistics of importance to society and decision-making, it is imperative that respondents (persons or establishments) be guaranteed privacy and confidentiality in return for providing requested confidential data....

journal_title：Statistics in medicine

authors： Gonzalez JF Jr,Cox LH

更新日期：2005-02-28 00:00:00

abstract：:This paper considers the effect of non-differential outcome misclassification on the population attributable fraction and the population prevented fraction. I examine the bias in the attributable and the prevented fraction derived from a risk ratio estimate as a function of the sensitivity and specificity of the outco...

journal_title：Statistics in medicine

authors： Hsieh CC

更新日期：1991-03-01 00:00:00

abstract：:Screening and diagnostic tests are important in disease prevention or control. The predictive values of positive and negative (PPV and NPV) test results are two of four operational characteristics of a screening test. We review an existing method based on the generalized estimating equation (GEE) methodology for compa...

journal_title：Statistics in medicine

authors： Wang W,Davis CS,Soong SJ

更新日期：2006-07-15 00:00:00

abstract：:This paper presents a general approach for simultaneously assessing, from serial data, diagnostic consistency, interrater reliability and incidence of a strictly progressive disease. Observed data are viewed as incomplete: diagnostic errors are not distinguished from true diagnoses. We introduce a broad class of model...

journal_title：Statistics in medicine

authors： Espeland MA,Murphy WC,Leverett DH

更新日期：1988-03-01 00:00:00

abstract：:Appropriate methods for meta-regression applied to a set of clinical trials, and the limitations and pitfalls in interpretation, are insufficiently recognized. Here we summarize recent research focusing on these issues, and consider three published examples of meta-regression in the light of this work. One principal m...

journal_title：Statistics in medicine

authors： Thompson SG,Higgins JP

更新日期：2002-06-15 00:00:00

abstract：:In the presence of confounding, the consistency assumption required for identification of causal effects may be violated due to misclassification of the outcome variable. We introduce an inverse probability weighted approach to rebalance covariates across treatment groups while mitigating the influence of differential...

journal_title：Statistics in medicine

authors： Gravel CA,Platt RW

更新日期：2018-02-10 00:00:00

abstract：:There is considerable public concern about health disparities among different cultural/racial/ethnic groups. Important process measures that might reflect inequities are treated prevalence and the service utilization rate in a defined period of time. We have previously described a method for estimating N, the distinct...

journal_title：Statistics in medicine

authors： Laska EM,Meisner M,Wanderling J,Siegel C

更新日期：2010-07-20 00:00:00

abstract：:We consider evaluation and comparison of the diagnostic accuracy of biomarkers with continuous test outcomes, possibly correlated due to repeated measurements. We develop nonparametric group sequential testing procedures to evaluate and compare the area of biomarkers under their receiver operating characteristic curve...

journal_title：Statistics in medicine

authors： Liu A,Wu C,Schisterman EF

更新日期：2008-05-10 00:00:00

abstract：:Longitudinal biomarker data are often collected in studies, providing important information regarding the probability of an outcome of interest occurring at a future time. With many new and evolving technologies for biomarker discovery, the number of biomarker measurements available for analysis of disease progression...

journal_title：Statistics in medicine

authors： Zhao L,Murray S,Mariani LH,Ju W

更新日期：2020-11-20 00:00:00

abstract：:In this article, we investigate procedures for comparing two independent Poisson variates that are observed over unequal sampling frames (i.e. time intervals, populations, areas or any combination thereof). We consider two statistics (with and without the logarithmic transformation) for testing the equality of two Poi...

journal_title：Statistics in medicine

authors： Ng HK,Tang ML

更新日期：2005-03-30 00:00:00

abstract：:In this paper, we investigate the impact of time-invariant covariates when fitting transition mixed models. This is carried out by emphasizing on the role of baseline responses on the estimation process. Transition models are allowed for two cases of exogenous and endogenous baseline responses. We illustrate these con...

journal_title：Statistics in medicine

authors： Rikhtehgaran R,Kazemi I,Verbeke G

更新日期：2014-11-30 00:00:00

abstract：:A method is proposed to infer the randomized treatment effect on survival after an adjustment for a post-randomization variable. The post-randomization variable is made independent of the treatment assignment and is considered a surrogate for baseline prognostic factors. The relationship between the post-randomization...

journal_title：Statistics in medicine

authors： Heller G

更新日期：2001-11-30 00:00:00

abstract：:There is a single-minded focus on events in survival analysis, and we often ignore longitudinal data that are collected together with the event data. This is due to a lack of methodology but also a result of the artificial distinction between survival and longitudinal data analyses. Understanding the dynamics of such ...

journal_title：Statistics in medicine

authors： Aalen OO

更新日期：2012-08-15 00:00:00

abstract：:We consider a latent variable hazard model for clustered survival data where clusters are a random sample from an underlying population. We allow interactions between the random cluster effect and covariates. We use a maximum pseudo-likelihood estimator to estimate the mean hazard ratio parameters. We propose a bootst...

journal_title：Statistics in medicine

authors： Cai J,Zhou H,Davis CE

更新日期：1997-09-15 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

authors： Remontet L,Bossard N,Belot A,Estève J,French network of cancer registries FRANCIM.

更新日期：2007-05-10 00:00:00

abstract：:Recurrent event data occur in many clinical and observational studies, and in these situations, there may exist a terminal event such as death that is related to the recurrent event of interest. In addition, sometimes more than one type of recurrent events may occur, that is, one may encounter multivariate recurrent e...

journal_title：Statistics in medicine

authors： Zhu L,Sun J,Srivastava DK,Tong X,Leisenring W,Zhang H,Robison LL

更新日期：2011-11-10 00:00:00

abstract：:The treatment effect sizes that can be detected with sufficient power up to the different interim analyses constitute a clinically meaningful criterion for the selection of a group sequential test for a clinical trial. For any pre-specified sequence of effect sizes, it is possible to construct group sequential boundar...

journal_title：Statistics in medicine

authors： Schäfer H,Müller HH

更新日期：2004-05-15 00:00:00