Cancer immunotherapy trial design with cure rate and delayed treatment effect.

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：:Longitudinal studies with repeated measures are often subject to non-response. Methods currently employed to alleviate the difficulties caused by missing data are typically unsatisfactory, especially when the cause of the missingness is related to the outcomes. We present an approach for incomplete categorical data in...

journal_title：Statistics in medicine

authors： West CP,Dawson JD

更新日期：2002-01-30 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

authors： Lachos VH,A Matos L,Castro LM,Chen MH

更新日期：2019-03-15 00:00:00

abstract：:It has been increasingly common to analyze simultaneously repeated measures and time to failure data. In this paper we propose a joint model when the repeated measures are semi-continuous, characterized by the presence of a large portion of zero values, as well as right skewness of non zero (positive) values. Examples...

journal_title：Statistics in medicine

authors： Liu L

更新日期：2009-03-15 00:00:00

abstract：:This paper investigates the design and analysis of dose-finding trials with two agents. The set of doses for each agent is fixed in advance. The goal of the trial is to find the set of dose combinations with probability of toxicity closest to a pre-specified value. For each of the two agents we assume that the probabi...

journal_title：Statistics in medicine

authors： Ivanova A,Wang K

更新日期：2004-06-30 00:00:00

abstract：:The Wilcoxon-Mann-Whitney (WMW) test is often used to compare the means or medians of two independent, possibly nonnormal distributions. For this problem, the true significance level of the large sample approximate version of the WMW test is known to be sensitive to differences in the shapes of the distributions. Base...

journal_title：Statistics in medicine

authors： Fagerland MW,Sandvik L

更新日期：2009-05-01 00:00:00

abstract：:The generalized estimating equations (GEE) approach is commonly used to model incomplete longitudinal binary data. When drop-outs are missing at random through dependence on observed responses (MAR), GEE may give biased parameter estimates in the model for the marginal means. A weighted estimating equations approach g...

journal_title：Statistics in medicine

authors： Preisser JS,Lohman KK,Rathouz PJ

更新日期：2002-10-30 00:00:00

abstract：:Although statistical methodology is well-developed for comparing diagnostic tests in terms of their sensitivity and specificity, comparative inference about predictive values is not. In this paper, we consider the analysis of studies comparing operating characteristics of two diagnostic tests that are measured on all ...

journal_title：Statistics in medicine

authors： Kwak M,Um SW,Jung SH

更新日期：2015-07-10 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：:For clinical trials with multiple treatment arms or endpoints a variety of sequentially rejective, weighted Bonferroni-type tests have been proposed, such as gatekeeping procedures, fixed sequence tests, and fallback procedures. They allow to map the difference in importance as well as the relationship between the var...

journal_title：Statistics in medicine

authors： Bretz F,Maurer W,Brannath W,Posch M

更新日期：2009-02-15 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：: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：:Regression to the mean arises often in statistical applications where the units chosen for study relate to some observed characteristic in the extreme of its distribution. Gardner and Heady attribute the effect of regression to the mean to measurement errors. They assume the model Yi = U + ei, where U is a fixed withi...

journal_title：Statistics in medicine

authors： Johnson WD,George VT

更新日期：1991-08-01 00:00:00

abstract：:In phase 3 clinical trials, ethical and financial concerns motivate sequential analyses in which the data are analyzed prior to completion of the entire planned study. Existing group sequential software accounts for the effects of these interim analyses on the sampling density by assuming that the contribution of subs...

journal_title：Statistics in medicine

authors： Shoben AB,Emerson SS

更新日期：2014-12-20 00:00:00

abstract：:The Cardiac Arrythmia Suppression Trial was stopped much earlier than planned. Statistical considerations played a very important role in the decision. Flexible group sequential testing was developed for the trial by implementing a Lan and DeMets procedure with use of the permutation test. We compute P-values from the...

journal_title：Statistics in medicine

authors： Pawitan Y,Hallstrom A

更新日期：1990-09-01 00:00:00

abstract：:Performance of a diagnostic test is ideally evaluated by a comparison of the test results to a gold standard for all the patients in a study. In practice, however, it is common for a subset of study patients to have the gold standard not verified (missing) due to ethical or expense considerations. Sensitivity and spec...

journal_title：Statistics in medicine

authors： Kosinski AS,Chen Y,Lyles RH

更新日期：2010-07-10 00:00:00

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

authors： Douglas JA

更新日期：1999-11-15 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：:In England and Wales, a large-scale multiple statistical surveillance system for infectious disease outbreaks has been in operation for nearly two decades. This system uses a robust quasi-Poisson regression algorithm to identify abberrances in weekly counts of isolates reported to the Health Protection Agency. In this...

journal_title：Statistics in medicine

authors： Noufaily A,Enki DG,Farrington P,Garthwaite P,Andrews N,Charlett A

更新日期：2013-03-30 00:00:00

abstract：:Population heterogeneity is frequently observed among patients' treatment responses in clinical trials because of various factors such as clinical background, environmental, and genetic factors. Different subpopulations defined by those baseline factors can lead to differences in the benefit or safety profile of a the...

journal_title：Statistics in medicine

authors： Chiu YD,Koenig F,Posch M,Jaki T

更新日期：2018-12-20 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 longitudinal studies with incomplete data, where the number of time points can become numerous, it is often advantageous to model the covariance matrix. We describe several covariance models (for example, mixed models, compound symmetry, AR(1)-type models, and combination models) that offer parsimonious alternative...

journal_title：Statistics in medicine

authors： Grady JJ,Helms RW

更新日期：1995-07-15 00:00:00

abstract：:A measure of similarity for response curves is presented and its potential use is discussed. The distribution of a suitable test statistic for testing the independence of the course of two curves is derived. The method proposed is compared with other proposals in the literature for the analysis of paired response curv...

journal_title：Statistics in medicine

authors： Schulgen G

更新日期：1989-11-01 00:00:00

abstract：:In this paper, an approach to estimating the cumulative mean function for history process with time dependent covariates and right censored time-to-event variable is developed using the combined technique of joint modeling and inverse probability weighting method. The consistency of proposed estimator is derived. Theo...

journal_title：Statistics in medicine

authors： Deng D

更新日期：2016-11-10 00:00:00

abstract：:Estimating the causal effect of quitting smoking on time to death or first myocardial infarction requires that one control for the differences in risk factors between individuals who elect to quite at each time t versus those who elect to continue smoking at time t. In this paper we examine the limitations of standard...

journal_title：Statistics in medicine

authors： Mark SD,Robins JM

更新日期：1993-09-15 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：: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：: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：:Down's syndrome screening is currently carried out using a combination of biochemical markers measured in maternal serum samples; these include MSAFP, Total hCG, uE3 and Free beta-hCG. Recently a number of papers have compared the effectiveness of different combinations of these markers. Some recommend MSAFP, Total hC...

journal_title：Statistics in medicine

authors： Dunstan FD,Gray JC,Nix AB,Reynolds T

更新日期：1997-07-15 00:00:00

abstract：:In failure time studies involving a chronic disease such as cancer, several competing causes of mortality may be operating. Commonly, the conventional statistical technique of Kaplan-Meier, which is only meaningfully interpreted by assuming independence of failure types and the censoring mechanism, is employed in clin...

journal_title：Statistics in medicine

authors： Tai BC,Machin D,White I,Gebski V,EOI (The European Osteosarcoma Intergroup).

更新日期：2001-03-15 00:00:00