Causal inference in survival analysis using pseudo-observations.

abstract：:Patient non-compliance and drop-out can bias analyses of clinical trial data. I describe a parametric model for treatment cross-over and drop-out and demonstrate how the concept of ignorability, originally defined for incomplete-data problems, can elucidate sources of bias in clinical trials. I discuss some implicatio...

journal_title：Statistics in medicine

authors： Heitjan DF

更新日期：1999-09-15 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

authors： Berger U,Schäfer J,Ulm K

更新日期：2003-04-15 00:00:00

abstract：:Pearson's chi-squared, the likelihood-ratio, and Fisher-Freeman-Halton's test statistics are often used to test the association of unordered r x c tables. Asymptotical, exact conditional, or exact conditional with mid-p adjustment methods are commonly used to compute the p-value. We have compared test power and signif...

journal_title：Statistics in medicine

authors： Lydersen S,Pradhan V,Senchaudhuri P,Laake P

更新日期：2007-10-15 00:00:00

abstract：:One may encounter the application of the two independent samples t-test to ordinal scaled data (for example, data that assume only the values 0, 1, 2, 3) from small samples. This situation clearly violates the underlying normality assumption for the t-test and one cannot appeal to large sample theory for validity. In ...

journal_title：Statistics in medicine

authors： Heeren T,D'Agostino R

更新日期：1987-01-01 00:00:00

abstract：:In comparing two treatments under a typical sequential clinical trial setting, a 50-50 randomization design generates reliable data for making efficient inferences about the treatment difference for the benefit of patients in the general population. However, if the treatment difference is large and the endpoint of the...

journal_title：Statistics in medicine

authors： Yao Q,Wei LJ

更新日期：1996-11-15 00:00:00

abstract：:Frequently, multiple doses of an active treatment and multiple endpoints are simultaneously considered in the designs of clinical trials. For these trials, traditional multiplicity adjustment procedures such as Bonferroni, Hochberg and Hommel procedures can be applied when treating the comparisons of different doses t...

journal_title：Statistics in medicine

authors： Quan H,Luo X,Capizzi T

更新日期：2005-07-30 00:00:00

abstract：:We extend the method proposed in a recent work by the Authors for trial-level general surrogate evaluation to allow combinations of biomarkers and provide a procedure for finding the "best" combination of biomarkers based on the absolute prediction error summary of surrogate quality. We use a nonparametric Bayesian mo...

journal_title：Statistics in medicine

authors： Gabriel EE,Sachs MC,Daniels MJ,Halloran ME

更新日期：2019-03-30 00:00:00

abstract：:Random forest is a supervised learning method that combines many classification or regression trees for prediction. Here we describe an extension of the random forest method for building event risk prediction models in survival analysis with competing risks. In case of right-censored data, the event status at the pred...

journal_title：Statistics in medicine

authors： Mogensen UB,Gerds TA

更新日期：2013-08-15 00:00:00

abstract：:We simulated multinomial AIDS incidence counts from 27 'representative' AIDS epidemics that spanned a period corresponding to previous applications of backcalculation (1 January 1977 to 1 July 1987) and assessed mean square error for several back-calculated estimators of HIV prevalence and short-term AIDS projections....

journal_title：Statistics in medicine

authors： Rosenberg PS,Gail MH,Pee D

更新日期：1991-08-01 00:00:00

abstract：:Graphical methods based on the analysis of differences between log cumulative hazard functions are considered for a two-group semi-proportional hazard model which allows for interaction between treatments and covariates. Confidence procedures and test statistics that can be used to test for interaction and for main ef...

journal_title：Statistics in medicine

authors： Dabrowska DM,Doksum KA,Feduska NJ,Husing R,Neville P

更新日期：1992-08-01 00:00:00

abstract：:In this study, 401 cases of childhood cancer in four California counties in 1980-1988 were analysed with the innovative methodology of density equalizing map projections. The data were originally collected and analysed by the California State Department of Health Services (DHS). In addition to the new analytic techniq...

journal_title：Statistics in medicine

authors： Merrill DW

更新日期：2001-05-15 00:00:00

abstract：:The development of drugs and biologicals whose mechanisms of action may extend beyond their target indications has led to a need to identify unexpected potential toxicities promptly even while blinded clinical trials are under way. One component of recently issued FDA rules regarding safety reporting requirements rais...

journal_title：Statistics in medicine

authors： Gould AL,Wang WB

更新日期：2017-01-15 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：:Outcome-dependent sampling (ODS) scheme is a cost-effective sampling scheme where one observes the exposure with a probability that depends on the outcome. The well-known such design is the case-control design for binary response, the case-cohort design for the failure time data, and the general ODS design for a conti...

journal_title：Statistics in medicine

authors： Lu TS,Longnecker MP,Zhou H

更新日期：2017-03-15 00:00:00

abstract：:Intraclass correlation coefficient (ICC) estimates must be provided when reporting the results of a cluster randomized trial. This study demonstrates that estimating this parameter with one-way ANOVA and an underlying mixed-effects statistical model leads to biased estimates. The bias depends on the effect size of the...

journal_title：Statistics in medicine

authors： Giraudeau B

更新日期：2006-03-30 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

authors： Ukoumunne OC,Davison AC,Gulliford MC,Chinn S

更新日期：2003-12-30 00:00:00

abstract：:This paper presents tables analogous to T-tables for use in the 2 x 2 multinomial trial, where the continuity corrected Z-statistic is used to make exact unconditional inference. This is the first solution of a discrete exact unconditional inference problem involving a multivariate nuisance parameter for which no anci...

journal_title：Statistics in medicine

authors： Shuster JJ

更新日期：1992-05-01 00:00:00

abstract：:In natural history studies of chronic disease, it is of interest to understand the evolution of key variables that measure aspects of disease progression. This is particularly true for immunological variables among persons infected with the human immunodeficiency virus (HIV). The natural time scale for such studies is...

journal_title：Statistics in medicine

authors： Vittinghoff E,Malani HM,Jewell NP

更新日期：1994-06-15 00:00:00

abstract：:Among many other uses, positron emission tomography (PET) can be used in studies to estimate the density of a neuroreceptor at each location throughout the brain by measuring the concentration of a radiotracer over time and modeling its kinetics. There are a variety of kinetic models in common usage and these typicall...

journal_title：Statistics in medicine

authors： Chang C,Ogden RT

更新日期：2009-03-15 00:00:00

abstract：:For genome-wide association studies, we propose a new method for identifying significant biological pathways. In this approach, we aggregate data across single-nucleotide polymorphisms to obtain summary measures at the gene level. We then use a hierarchical Bayesian model, which takes the gene-level summary measures a...

journal_title：Statistics in medicine

authors： Shahbaba B,Shachaf CM,Yu Z

更新日期：2012-05-10 00:00:00

abstract：:In studies with survival or time-to-event outcomes, a competing risk is an event whose occurrence precludes the occurrence of the primary event of interest. Specialized statistical methods must be used to analyze survival data in the presence of competing risks. We conducted a review of randomized controlled trials wi...

journal_title：Statistics in medicine

authors： Austin PC,Fine JP

更新日期：2017-04-15 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

authors： Nagelkerke NJ,Borgdorff MW,Kim SJ

更新日期：2001-04-15 00:00:00

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

authors： Rosthøj S,Keiding N,Schmiegelow K

更新日期：2012-02-28 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

authors： Plummer M,Clayton D

更新日期：1993-05-30 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

authors： Horton BJ,Wages NA,Conaway MR

更新日期：2017-01-30 00:00:00

abstract：:For time-to-event outcomes, a rich literature exists on the bias introduced by covariate measurement error in regression models, such as the Cox model, and methods of analysis to address this bias. By comparison, less attention has been given to understanding the impact or addressing errors in the failure time outcome...

journal_title：Statistics in medicine

authors： Oh EJ,Shepherd BE,Lumley T,Shaw PA

更新日期：2018-04-15 00:00:00

abstract：:To study the spread of sexually transmitted diseases (STDs) using social/sexual mixing models, one must have quantitative information about sexual mixing. An unavoidable complication in gathering such information by survey is that members of the surveyed population will almost certainly have sexual contacts outside th...

journal_title：Statistics in medicine

authors： Rubin G,Umbach D,Shyu SF,Castillo-Chavez C

更新日期：1992-09-15 00:00:00

abstract：:The two tailed Fisher's exact P value is extremely sensitive to small perturbations in 2 x 2 contingency tables. An example indicates that a 1 per cent increase in the denominator of one treatment group results in a 32 per cent drop in the exact P value, but a mere 0.1 per cent decrease in the treatment success rate. ...

journal_title：Statistics in medicine

authors： Dupont WD

更新日期：1986-11-01 00:00:00

abstract：:The incremental life expectancy, defined as the difference in mean survival times between two treatment groups, is a crucial quantity of interest in cost-effectiveness analyses. Usually, this quantity is very difficult to estimate from censored survival data with a limited follow-up period. The paper develops estimati...

journal_title：Statistics in medicine

authors： Titman AC

更新日期：2016-09-10 00:00:00

abstract：:We describe the usual statistical concepts and consequent appropriate simulations of a prospective study for the simple case of a single risk variable and an assumed logistic model. We examine the simulations of Lilienfeld and Pyne, and show that they are seriously flawed. Contrary to those authors' claims, the estima...

journal_title：Statistics in medicine

authors： Halperin M,Abbott RD,Blackwelder WC,Jacobowitz R,Lan KK,Verter JI,Wedel H

更新日期：1985-04-01 00:00:00