Optimal seamless phase 2/3 oncology trial designs based on Probability of Success (PoS).

abstract：:Conditional power and predictive power provide estimates of the probability of success at the end of the trial based on the information from the interim analysis. The observed value of the time to event endpoint at the interim analysis could be biased for the true treatment effect due to early censoring, leading to a ...

journal_title：Statistics in medicine

authors： Sun L,Wan Y

更新日期：2018-08-15 00:00:00

abstract：:Conventional seamless phase 2/3 design with fixed sample size determination (SSD) has gained its popularity in oncology drug development due to attractive features such as significantly shortening the development timeline, minimizing sample size, as well as early decision making. However, this design is not immune to ...

journal_title：Statistics in medicine

authors： Teng Z,Tian Y,Liu Y,Liu G

更新日期：2020-08-15 00:00:00

abstract：:It is well established that neural activity is stochastically modulated over time. Therefore, direct comparisons across experimental conditions and determination of change points or maximum firing rates are not straightforward. This study sought to compare temporal firing probability curves that may vary across groups...

journal_title：Statistics in medicine

authors： Roca-Pardiñas J,Cadarso-Suárez C,Pardo-Vazquez JL,Leboran V,Molenberghs G,Faes C,Acuña C

更新日期：2011-06-30 00:00:00

abstract：:Prevalent sampling is frequently a convenient and economical sampling technique for the collection of time-to-event data and thus is commonly used in studies of the natural history of a disease. However, it is biased by design because it tends to recruit individuals with longer survival times. This paper considers est...

journal_title：Statistics in medicine

authors： Li S

更新日期：2017-04-15 00:00:00

abstract：:In medical research, risk difference (RD) and number needed to treat (NNT) measures for survival times have been mainly proposed without consideration of covariates. In this paper, we develop adjusted RD and NNT measures for use in observational studies with survival time outcomes within the framework of the Cox propo...

journal_title：Statistics in medicine

authors： Laubender RP,Bender R

更新日期：2010-03-30 00:00:00

abstract：:To study the effect of a mega hydropower dam in southwest Ethiopia on malaria incidence, we have set up a longitudinal study. To gain insight in temporal and spatial aspects, that is, in time (period  =  year-season combination) and location (village), we need models that account for these effects. The frailty model w...

journal_title：Statistics in medicine

authors： Getachew Y,Janssen P,Yewhalaw D,Speybroeck N,Duchateau L

更新日期：2013-08-15 00:00:00

abstract：:In vaccine safety studies, subjects are considered at increased risk for adverse events for a period of time after vaccination known as risk window. To our knowledge, risk windows for vaccine safety studies have tended to be pre-defined and not to use information from the current study. Inaccurate specification of the...

journal_title：Statistics in medicine

authors： Xu S,Zhang L,Nelson JC,Zeng C,Mullooly J,McClure D,Glanz J

更新日期：2011-03-30 00:00:00

abstract：:Subjective judgements of complex variables are commonly recorded as ordered categorical data. The rank-invariant properties of such data are well known, and there are various statistical approaches to the analysis and modelling of ordinal data. This paper focuses on the non-additive property of ordered categorical dat...

journal_title：Statistics in medicine

authors： Svensson E

更新日期：1998-12-30 00:00:00

abstract：:Appropriate quantification of added usefulness offered by new markers included in risk prediction algorithms is a problem of active research and debate. Standard methods, including statistical significance and c statistic are useful but not sufficient. Net reclassification improvement (NRI) offers a simple intuitive w...

journal_title：Statistics in medicine

authors： Pencina MJ,D'Agostino RB Sr,Steyerberg EW

更新日期：2011-01-15 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：:New combination regimens evaluated in phase II cancer clinical trials often show promising results compared to the standard therapy for a disease system. Selection of patients with a better prognosis can be a prominent factor for this optimism. For most disease systems, prognostic variables that are related to the out...

journal_title：Statistics in medicine

authors： Mazumdar M,Fazzari M,Panageas KS

更新日期：2001-03-30 00:00:00

abstract：:Sensible plans for health-care needs and determination of priorities for expenditure require regular assessment of trends in HIV incidences. In particular, trends in the relative HIV incidences of different risk categories are useful when assessing whether current control strategies are working equally well for all ri...

journal_title：Statistics in medicine

authors： Becker NG,Cui JS

更新日期：1996-08-30 00:00:00

abstract：:A technique is presented which provides guidance on the spacing of follow-up waves in a multiwave study. Only information from the baseline wave is needed, as well as rough parameter estimates for the survival distribution. The computations use the expected Fisher information; a new method for its calculation is given...

journal_title：Statistics in medicine

authors： Chappell R

更新日期：1991-12-01 00:00:00

abstract：:Service on the Data Monitoring Committee of the CPEP (Calcium for Pre-eclampsia Prevention) has led us to four conclusions about clinical trials which we should like to present to this gathering of biostatisticians for their reactions: (i) meta-analyses of the pertinent published trials of the same therapy should alwa...

journal_title：Statistics in medicine

authors： Chalmers TC,Lau J

更新日期：1996-06-30 00:00:00

abstract：:Lung function tests are used both clinically, in assessing disease, and epidemiologically, in identifying those factors which influence the growth and aging process of the lungs. The user must beware of several common pitfalls in the use of these tests, however. First, the commonly used tests of lung function can only...

journal_title：Statistics in medicine

authors： Buist AS,Vollmer WM

更新日期：1988-01-01 00:00:00

abstract：:In this paper, we define a modified version τ(b) of Kendall's tau to measure the association in a pair (X,Y) of random variables subject to fixed left censoring due to known lower detection limits. We provide a nonparametric estimator of τ(b) and investigate its asymptotic properties. We then assume an Archimedean cop...

journal_title：Statistics in medicine

authors： Romdhani H,Lakhal-Chaieb L

更新日期：2011-11-20 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

authors： Rosner B,Glynn RJ

更新日期：2007-02-10 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

authors： Zhou XH,Katz BP,Holleman E,Melfi CA,Dittus R

更新日期：1996-09-15 00:00:00

abstract：:Evaluating treatment effects within different subsets of patients is a common practice in the analysis of individual randomized clinical trials. Such analyses are limited, however, by the number of patients available. Overviews, by providing evidence based on large numbers of patients, can be useful for overcoming the...

journal_title：Statistics in medicine

authors： Gelber RD,Goldhirsch A

更新日期：1987-04-01 00:00:00

abstract：:Propensity score analysis is widely used in observational studies to adjust for confounding and estimate the causal effect of a treatment on the outcome. When the outcome is survival time, there are special considerations on the definition of the causal estimand, point, and variance estimation that have not been thoro...

journal_title：Statistics in medicine

authors： Mao H,Li L,Yang W,Shen Y

更新日期：2018-11-20 00:00:00

abstract：:Proportional hazards (or Cox) regression is a popular method for modelling the effects of prognostic factors on survival. Use of cubic spline functions to model time-by-covariate interactions in Cox regression allows investigation of the shape of a possible covariate-time dependence without having to specify a specifi...

journal_title：Statistics in medicine

authors： Hess KR

更新日期：1994-05-30 00:00:00

abstract：:The need for resource-intensive laboratory assays to assess exposures in many epidemiologic studies provides ample motivation to consider study designs that incorporate pooled samples. In this paper, we consider the case in which specimens are combined for the purpose of determining the presence or absence of a pool-w...

journal_title：Statistics in medicine

authors： Lyles RH,Tang L,Lin J,Zhang Z,Mukherjee B

更新日期：2012-09-28 00:00:00

abstract：:We discuss some of the fundamental concepts underlying the development of frailty and random effects models in survival. One of these fundamental concepts was the idea of a frailty model where each subject has his or her own disposition to failure, their so-called frailty, additional to any effects we wish to quantify...

journal_title：Statistics in medicine

authors： O'Quigley J,Stare J

更新日期：2002-11-15 00:00:00

abstract：:When estimating the average effect of a binary treatment (or exposure) on an outcome, methods that incorporate propensity scores, the G-formula, or targeted maximum likelihood estimation (TMLE) are preferred over naïve regression approaches, which are biased under misspecification of a parametric outcome model. In con...

journal_title：Statistics in medicine

authors： Luque-Fernandez MA,Schomaker M,Rachet B,Schnitzer ME

更新日期：2018-07-20 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：: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

authors： Eichner M,Diebner HH,Schubert C,Kreth HW,Dietz K

更新日期：2002-08-30 00:00:00

abstract：:We study prevalence-dependent diagnostic accuracy measures, specifically, positive and negative predictive values. These measures permit an assessment of the clinical utility of diagnostic tests across populations with different disease prevalences. In many cases, prevalence may not be known with certainty and the eva...

journal_title：Statistics in medicine

authors： Li J,Fine JP,Safdar N

更新日期：2007-07-30 00:00:00

abstract：:A common problem encountered in many medical applications is the comparison of survival curves. Often, rather than comparison of the entire survival curves, interest is focused on the comparison at a fixed point in time. In most cases, the naive test based on a difference in the estimates of survival is used for this ...

journal_title：Statistics in medicine

authors： Klein JP,Logan B,Harhoff M,Andersen PK

更新日期：2007-10-30 00:00:00

abstract：:We applied a two-stage random effects model to pulmonary function data from 31 sarcoidosis patients to illustrate its usefulness in analysing unbalanced longitudinal data. For the first stage, repeated measurements of percentage of predicted forced vital capacity (FVC%) from an individual were modelled as a function o...

journal_title：Statistics in medicine

authors： Vacek PM,Mickey RM,Bell DY

更新日期：1989-02-01 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

authors： Dai T,Guo Y,Peng L,Manatunga A

更新日期：2020-06-30 00:00:00