Efficient adaptive designs with mid-course sample size adjustment in clinical trials.

abstract：:Motivated by a study for soft tissue sarcoma, this article considers the analysis of diseases recurrence and survival. A multivariate frailty hazard model is established for joint modeling of three correlated time-to-event outcomes: local disease recurrence, distant disease recurrence (metastasis), and death. The goal...

journal_title：Statistics in medicine

authors： Wen S,Huang X,Frankowski RF,Cormier JN,Pisters P

更新日期：2016-11-20 00:00:00

abstract：:Population dynamic models, commonly used tools in the study of epidemics and other complex population processes, are implicit non-linear mathematical equations. Inference based on such models can be difficult due to the problems associated with high dimensional parameters that may be non-identified and complex likelih...

journal_title：Statistics in medicine

authors： Brookhart MA,Hubbard AE,van der Laan MJ,Colford JM Jr,Eisenberg JN

更新日期：2002-12-15 00:00:00

abstract：:We discuss the use of the trichotomous logistic model to discriminate between patients with gastrointestinal (GI) cancer, patients with benign GI disease and 'normal' subjects, using symptoms and the concentrations of some serum proteins that are potentially indicative of malignancy as covariates. A parsimonious model...

journal_title：Statistics in medicine

authors： Marshall RJ,Chisholm EM

更新日期：1985-07-01 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：:A general method is presented that allows the researcher to change statistical design elements such as the residual sample size during the course of an experiment, to include an interim analysis for early stopping when no formal rule for early stopping was foreseen, to increase or reduce the number of planned interim ...

journal_title：Statistics in medicine

authors： Müller HH,Schäfer H

更新日期：2004-08-30 00:00:00

abstract：:Identification of treatment selection biomarkers has become very important in cancer drug development. Adaptive enrichment designs have been developed for situations where a unique treatment selection biomarker is not apparent based on the mechanism of action of the drug. With such designs, the eligibility rules may b...

journal_title：Statistics in medicine

authors： Simon R,Simon N

更新日期：2017-11-20 00:00:00

abstract：:Combinatorial drugs have been widely applied in disease treatment, especially chemotherapy for cancer, due to its improved efficacy and reduced toxicity compared with individual drugs. The study of combinatorial drugs requires efficient experimental designs and proper follow-up statistical modeling techniques. Linear ...

journal_title：Statistics in medicine

authors： Xiao Q,Wang L,Xu H

更新日期：2019-01-30 00:00:00

abstract：:In psychiatry, clinicians use criteria sets from the Diagnostic and Statistical Manual of Mental Disorders to diagnose mental disorders. Most criteria sets have several symptom domains, and in order to be diagnosed, an individual must meet the minimum number of symptoms required by each domain. Some efforts are now fo...

journal_title：Statistics in medicine

authors： Mauro C,Shear MK,Wang Y

更新日期：2019-07-20 00:00:00

abstract：:We have formulated the problem of determining whether there has been an upturn in HIV-1 seroconversion incidence over the first five years of follow-up in the Multicenter AIDS Cohort Study (MACS) as that of locating the minimum of a quadratic regression or examination of two-knot piecewise spline models. Under a quadr...

journal_title：Statistics in medicine

authors： Zhou SY,Kingsley LA,Taylor JM,Chmiel JS,He DY,Hoover DR

更新日期：1993-01-30 00:00:00

abstract：:This paper describes the Stoke Index which has been designed to give a global measure of disease activity in rheumatoid arthritis. The index is based on two objective laboratory measurements, one subjective and two semi-objective clinical measurements, chosen from 13 measurements using clinical judgement. Variable sel...

journal_title：Statistics in medicine

authors： Jones PW,Ziade MF,Davis MJ,Dawes PT

更新日期：1993-06-30 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：:We propose to use a very simple model to test whether a cancer cluster is due to chance alone. We focus on the acute childhood leukaemia cluster in Columbus, Ohio. In 1975, 12 leukaemia cases were observed in Columbus while the expected number is 6 cases per year. According to our simple model, the probability of such...

journal_title：Statistics in medicine

authors： Schinazi RB

更新日期：2000-08-30 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：:A common but not necessarily logical requirement in drug development is that a 'limit of quantitation' be set for chemical assays and that observations that fall below the limit should not be treated as real data but should be labelled as below the limit and set aside for special treatment. We examine five of seven ap...

journal_title：Statistics in medicine

authors： Senn S,Holford N,Hockey H

更新日期：2012-12-30 00:00:00

abstract：:The first randomized clinical trial at the National Cancer Institute (NCI), planned in 1954, commenced in 1955 for the treatment of patients with acute leukaemia. The programme in clinical trials at NCI had strong influence from the clinician and administrator, C. Gordon Zubrod, who introduced the randomized clinical ...

journal_title：Statistics in medicine

authors： Gehan EA,Schneiderman MA

更新日期：1990-08-01 00:00:00

abstract：:Methodology for causal inference based on propensity scores has been developed and popularized in the last two decades. However, the majority of the methodology has concentrated on binary treatments. Only recently have these methods been extended to settings with multi-valued treatments. We propose a number of discret...

journal_title：Statistics in medicine

authors： Tchernis R,Horvitz-Lennon M,Normand SL

更新日期：2005-07-30 00:00:00

abstract：:Estimation and group comparison of survival curves are two very common issues in survival analysis. In practice, the Kaplan-Meier estimates of survival functions may be biased due to unbalanced distribution of confounders. Here we develop an adjusted Kaplan-Meier estimator (AKME) to reduce confounding effects using in...

journal_title：Statistics in medicine

authors： Xie J,Liu C

更新日期：2005-10-30 00:00:00

abstract：:Subjects in randomized controlled trials do not always comply to the treatment condition they have been assigned to. This may cause the estimated effect of the intervention to be biased and also affect efficiency, coverage of confidence intervals, and statistical power. In cluster randomized trials non-compliance may ...

journal_title：Statistics in medicine

authors： Moerbeek M,Schie SV

更新日期：2019-11-20 00:00:00

abstract：:In this paper, we discuss statistical inference for a 2 × 2 table under inverse sampling, where the total number of cases is fixed by design. We demonstrate that the exact unconditional distributions of some relevant statistics differ from the distributions under conventional sampling, where the sample size is fixed b...

journal_title：Statistics in medicine

authors： Heimann G,Von Tress M,Gasparini M

更新日期：2015-08-30 00:00:00

abstract：:Joint models initially dedicated to a single longitudinal marker and a single time-to-event need to be extended to account for the rich longitudinal data of cohort studies. Multiple causes of clinical progression are indeed usually observed, and multiple longitudinal markers are collected when the true latent trait of...

journal_title：Statistics in medicine

authors： Proust-Lima C,Dartigues JF,Jacqmin-Gadda H

更新日期：2016-02-10 00:00:00

abstract：:Linear mixed effects (LME) models are increasingly used for analyses of biological and biomedical data. When the multivariate normal assumption is not adequate for an LME model, then a robust estimation approach is preferable to the maximum likelihood one. M-estimators were considered before for robust estimation of t...

journal_title：Statistics in medicine

authors： Chervoneva I,Vishnyakov M

更新日期：2011-06-30 00:00:00

abstract：:This paper investigates the impact on area life tables of the specification of unobserved frailty. Frailty specification may affect both the regression effects of area and individual level covariates, and lead to changes in the value of summary mortality parameters, such as life expectancy. The paper also investigates...

journal_title：Statistics in medicine

authors： Congdon P

更新日期：1995-09-15 00:00:00

abstract：:Standard approaches to analysis of randomized controlled trials (RCTs) using Markov models make it difficult to generalize treatment effects to new patient groups and synthesize evidence across trials. This paper demonstrates how pair-wise and mixed treatment comparison meta-analysis can be applied to event history da...

journal_title：Statistics in medicine

authors： Price MJ,Welton NJ,Ades AE

更新日期：2011-01-30 00:00:00

abstract：:The principles and historical development of clinical trials are outlined briefly. Some issues that extend the basic philosophy first put forward by Sir Austin Bradford Hill 40 years ago and that will need consideration in the future application of clinical trials are discussed. ...

journal_title：Statistics in medicine

authors： Holland WW,Breeze E,Swan AV

更新日期：1982-10-01 00:00:00

abstract：:Blinded estimation of variance allows for changing the sample size without compromising the integrity of the trial. Some of the methods that estimate the variance in a blinded manner either make untenable assumptions or are only applicable to two-treatment trials. We propose a new method for continuous endpoints that ...

journal_title：Statistics in medicine

authors： Xing B,Ganju J

更新日期：2005-06-30 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：:Genome-wide association studies are characterized by a huge number of statistical tests performed to discover new disease-related genetic variants [in the form of single-nucleotide polymorphisms (SNPs)] in human DNA. Many SNPs have been identified for cross-sectionally measured phenotypes. However, there is a growing ...

journal_title：Statistics in medicine

authors： Sikorska K,Rivadeneira F,Groenen PJ,Hofman A,Uitterlinden AG,Eilers PH,Lesaffre E

更新日期：2013-01-15 00:00:00

abstract：:We propose a new weighted hurdle regression method for modeling count data, with particular interest in modeling cardiovascular events in patients on dialysis. Cardiovascular disease remains one of the leading causes of hospitalization and death in this population. Our aim is to jointly model the relationship/associat...

journal_title：Statistics in medicine

authors： Sentürk D,Dalrymple LS,Mu Y,Nguyen DV

更新日期：2014-11-10 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：:Age-period-cohort (APC) analysis is widely used in cancer epidemiology to model trends in cancer rates. We develop methods for comparative APC analysis of two independent cause-specific hazard rates assuming that an APC model holds for each one. We construct linear hypothesis tests to determine whether the two hazards...

journal_title：Statistics in medicine

authors： Rosenberg PS,Anderson WF

更新日期：2010-05-20 00:00:00