Practical modifications to the time-to-event continual reassessment method for phase I cancer trials with fast patient accrual and late-onset toxicities.

abstract：:The relationship between association and surrogacy has been the focus of much debate in the surrogate marker literature. Recently, the individual causal association (ICA) has been introduced as a metric of surrogacy in the causal inference framework, when both the surrogate and the true endpoint are normally distribut...

journal_title：Statistics in medicine

authors： Meyvisch P,Alonso A,Van der Elst W,Molenberghs G

更新日期：2020-11-20 00:00:00

abstract：:Our objective is to develop a model to estimate the relative risk of disease in each area, Ai, i=1, ... , n, of a region and to identify areas of unusually high or low risk. We use a product partition model (PPM) in which we assume that the true relative risks can be partitioned into a number of components or sets of ...

journal_title：Statistics in medicine

authors： Hegarty A,Barry D

更新日期：2008-08-30 00:00:00

abstract：:Outcomes research often requires estimating the impact of a binary treatment on a binary outcome in a non-randomized setting, such as the effect of taking a drug on mortality. The data often come from self-selected samples, leading to a spurious correlation between the treatment and outcome when standard binary depend...

journal_title：Statistics in medicine

authors： Bhattacharya J,Goldman D,McCaffrey D

更新日期：2006-02-15 00:00:00

abstract：:In this paper we study the problem of variable selection for the proportional odds model, which is a useful alternative to the proportional hazards model and might be appropriate when the proportional hazards assumption is not satisfied. We propose to fit the proportional odds model by maximizing the marginal likeliho...

journal_title：Statistics in medicine

authors： Lu W,Zhang HH

更新日期：2007-09-10 00:00:00

abstract：:Observational studies provide a rich source of information for assessing effectiveness of treatment interventions in many situations where it is not ethical or practical to perform randomized controlled trials. However, such studies are prone to bias from hidden (unmeasured) confounding. A promising approach to identi...

journal_title：Statistics in medicine

authors： Lin NX,Henley WE

更新日期：2016-12-10 00:00:00

abstract：:Phase II trials often test the null hypothesis H(0): p or=p(1), where p is the true unknown proportion responding to the new treatment, p(0) is the greatest response proportion which is deemed clinically ineffective, and p(1) is the smallest response proportion which is deemed clinically effe...

journal_title：Statistics in medicine

authors： Ayanlowo AO,Redden DT

更新日期：2007-03-30 00:00:00

abstract：:We consider the estimation of parameters in a particular segmented generalized linear model with additive measurement error in predictors, with a focus on linear and logistic regression. In epidemiologic studies segmented regression models often occur as threshold models, where it is assumed that the exposure has no i...

journal_title：Statistics in medicine

authors： Küchenhoff H,Carroll RJ

更新日期：1997-01-15 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：:Reference ranges which take time (such as age) into account are often required in medicine, but simple, systematic and efficient statistical methods for constructing them are lacking. A method is described which is based on low order polynomial curves (linear, quadratic or occasionally cubic), together with guidelines...

journal_title：Statistics in medicine

authors： Royston P

更新日期：1991-05-01 00:00:00

abstract：:Health questionnaires are often built up from sets of questions that are totaled to obtain a sum score. An important consideration in designing questionnaires is to minimize respondent burden. An increasingly popular method for efficient measurement is computerized adaptive testing; unfortunately, many health question...

journal_title：Statistics in medicine

authors： Smits N,Finkelman MD

更新日期：2014-02-10 00:00:00

abstract：:We propose the use of Gaussian mixed models to analyse statistically 24 hour ambulatory blood pressure data from clinical trials. We develop specific models and apply them to data from a clinical study that compares two angiotensin-converting enzyme inhibitors. We investigate and discuss computing issues related to th...

journal_title：Statistics in medicine

authors： Selwyn MR,Difranco DM

更新日期：1993-09-30 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：:This paper discusses and compares several estimators of mean rate of change in unbalanced longitudinal data based on a model with randomly distributed regression coefficients across individuals. The estimators are unweighted and weighted means of these coefficients. The paper also evaluates commonly used variance esti...

journal_title：Statistics in medicine

authors： Palta M,Cook T

更新日期：1987-07-01 00:00:00

abstract：:We investigate a binary partitioning algorithm in the case of a continuous repeated measures outcome. The procedure is based on the use of the likelihood ratio statistic to evaluate the performance of individual splits. The procedure partitions a set of longitudinal data into two mutually exclusive groups based on an ...

journal_title：Statistics in medicine

authors： Abdolell M,LeBlanc M,Stephens D,Harrison RV

更新日期：2002-11-30 00:00:00

abstract：:Diseases can be interconnected. In the recent years, there has been a surge of multidisease studies. Among them, HDN (human disease network) analysis takes a system perspective, examines the interconnections among diseases along with their individual properties, and has demonstrated great potential. Most of the existi...

journal_title：Statistics in medicine

authors： Ma C,Li Y,Shia B,Ma S

更新日期：2020-04-30 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

authors： Wei J,Wu J

更新日期：2020-03-15 00:00:00

abstract：:Phase II and phase III trials play a crucial role in drug development programs. They are costly and time consuming and, because of high failure rates in late development stages, at the same time risky investments. Commonly, sample size calculation of phase III is based on the treatment effect observed in phase II. The...

journal_title：Statistics in medicine

authors： Kirchner M,Kieser M,Götte H,Schüler A

更新日期：2016-01-30 00:00:00

abstract：:Growth trends in children are often based on cross-sectional studies, in which a sample of the population is investigated at one given point in time. Estimating age-related percentiles in such studies involves fitting data distributions, each of which is specific for one age group, and a subsequent smoothing of the pe...

journal_title：Statistics in medicine

authors： Sorribas A,March J,Voit EO

更新日期：2000-03-15 00:00:00

abstract：:With reference to a real data on cataract surgery, we discuss the problem of zero-inflated circular-circular regression when both covariate and response are circular random variables and a large proportion of the responses are zeros. The regression model is proposed, and the estimation procedure for the parameters is ...

journal_title：Statistics in medicine

authors： Jha J,Biswas A

更新日期：2018-01-15 00:00:00

abstract：:A new method is proposed for the surveillance of Down's syndrome among newborn. Despite the strong dependence of overall risk of Down's syndrome on maternal age, it has been suggested that an environmentally induced increase in risk may be additive over all maternal ages. The surveillance method introduced here is spe...

journal_title：Statistics in medicine

authors： Lie RT,Heuch I,Irgens LM

更新日期：1993-01-15 00:00:00

abstract：:This paper describes compliance with the completion of a quality of life questionnaire in the Breast Cancer Prevention Trial, a large multi-centre randomized trial that is studying the efficacy of Tamoxifen in preventing breast cancer. In the first 4875 women enrolled in the control arm of the study, there was a very ...

journal_title：Statistics in medicine

authors： Ganz PA,Day R,Costantino J

更新日期：1998-03-15 00:00:00

abstract：:Day and Walter derived methods of joint maximum likelihood estimation for the sojourn time distribution and the false negative rate for a screening programme. Their methods are not directly applicable to a programme which uses alternate screening by two modalities whose sojourn times and false negative rates will diff...

journal_title：Statistics in medicine

authors： Alexander FE

更新日期：1989-06-01 00:00:00

abstract：:The lifetime risk of a clinical condition is the probability of onset of the condition during one's lifespan. Recent advances in Alzheimer's disease (AD) research have identified screening tests for biomarkers that can identify persons who are in the earliest stages of the AD process but who do not yet have any clinic...

journal_title：Statistics in medicine

authors： Brookmeyer R,Abdalla N

更新日期：2019-04-30 00:00:00

abstract：:Analyses to compare non-randomized groups are more and more common in both post hoc analyses of randomized clinical trials data and in analyses of long-term observational data. In such cases, it is quite likely that there are unknown or uncollected sources of heterogeneity in event rates. Research has shown that an un...

journal_title：Statistics in medicine

authors： Eberly LE

更新日期：2004-07-15 00:00:00

abstract：:A survey is conducted at w of K selection units or lists, e.g. health care institutions or weeks in a year, to estimate N, the total number of individuals with particular characteristics. Our estimator utilizes two items determined for each survey participant: the number, u, among the w lists in S and the number, j, a...

journal_title：Statistics in medicine

authors： Laska EM,Meisner M,Wanderling J

更新日期：2009-07-30 00:00:00

abstract：:Experimental designs with repeated measures allow response patterns over time (or dose) to be modelled and compared between different homogeneous groups. Issues in data analysis often focus on the pattern of variation of the repeated measures, the appropriateness of a univariate or multivariate analysis, and the shape...

journal_title：Statistics in medicine

authors： Stanek EJ 3rd,Kline G

更新日期：1991-01-01 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

authors： Englert S,Kieser M

更新日期：2015-06-15 00:00:00

abstract：:Interim analyses are conducted to allow for early termination of the trial, for ethical as well as economical reasons. Here we consider interim analyses in repeated measurements studies where the measurements are binary. Two methods for analysing this kind of data are compared according to their operating characterist...

journal_title：Statistics in medicine

authors： Spiessens B,Lesaffre E,Verbeke G

更新日期：2003-02-28 00:00:00

abstract：:Several relative risk models for survival time data in drug combination therapy are derived and their properties are discussed. The main intention of this paper is to clarify the differences among the models in order to help to choose the appropriate one in a given situation. The models are motivated by discussing the...

journal_title：Statistics in medicine

authors： Kübler J,Schumacher M

更新日期：1990-12-01 00:00:00

abstract：:The proposed guidelines for the assessment of the effect of new pharmaceutical agents on the QT interval (beginning of QRS complex to end of T wave on the electrocardiogram) are based on the maximum of a series over time of simple one-sided 95 per cent upper confidence bounds. This procedure is typically very conserva...

journal_title：Statistics in medicine

authors： Boos DD,Hoffman D,Kringle R,Zhang J

更新日期：2007-09-10 00:00:00