Group sequential designs for cure rate models with early stopping in favour of the null hypothesis.

abstract：:Researchers in clinical science and bioinformatics frequently aim to learn which of a set of candidate biomarkers is important in determining a given outcome, and to rank the contributions of the candidates accordingly. This article introduces a new approach to research questions of this type, based on targeted maximu...

journal_title：Statistics in medicine

authors： Bembom O,Petersen ML,Rhee SY,Fessel WJ,Sinisi SE,Shafer RW,van der Laan MJ

更新日期：2009-01-15 00:00:00

abstract：:Surrogate endpoint validation has been well established by the meta-analytical correlation-based approach as outlined in the seminal work of Buyse et al. (Biostatistics, 2000). Surrogacy can be assumed if strong associations on individual and study levels can be demonstrated. Alternatively, if an effect on a true endp...

journal_title：Statistics in medicine

authors： Schürmann C,Sieben W

更新日期：2016-03-30 00:00:00

abstract：:We propose a joint model for a time-to-event outcome and a quantile of a continuous response repeatedly measured over time. The quantile and survival processes are associated via shared latent and manifest variables. Our joint model provides a flexible approach to handle informative dropout in quantile regression. A M...

journal_title：Statistics in medicine

authors： Farcomeni A,Viviani S

更新日期：2015-03-30 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：:The power to detect a treatment effect in cluster randomized trials can be increased by increasing the number of clusters. An alternative is to include covariates into the regression model that relates treatment condition to outcome. In this paper, formulae are derived in order to evaluate both strategies on basis of ...

journal_title：Statistics in medicine

authors： Moerbeek M

更新日期：2006-08-15 00:00:00

abstract：BACKGROUND:It has been recommended that onset of antidepressant action be assessed using survival analyses with assessments taken at least twice per week. However, such an assessment schedule is problematic to implement. The present study assessed the feasibility of comparing onset of action between treatments using a ...

journal_title：Statistics in medicine

authors： Mallinckrodt CH,Detke MJ,Kaiser CJ,Watkin JG,Molenberghs G,Carroll RJ

更新日期：2006-07-30 00:00:00

abstract：:The objective of this report is to provide a basis to inform decisions about priorities for developing statistical research initiatives in the field of public health surveillance for emerging threats. Rapid information system advances have created a vast opportunity of secondary data sources for information to enhance...

journal_title：Statistics in medicine

authors： Rolka H,Burkom H,Cooper GF,Kulldorff M,Madigan D,Wong WK

更新日期：2007-04-15 00:00:00

abstract：:Sam Greenhouse joined the Census Bureau as a clerk at an interesting time period for the agency. The first use of sampling in the decennial census occurred in 1940. There was a major expansion of the amount of data collected. The organization of the Census Bureau underwent radical changes, including the growth of the ...

journal_title：Statistics in medicine

authors： Keller J,Clark CZ

更新日期：2003-11-15 00:00:00

abstract：:In a variety of biomedical applications, particularly those involving screening for infectious diseases, testing individuals (e.g. blood/urine samples, etc.) in pools has become a standard method of data collection. This experimental design, known as group testing (or pooled testing), can provide a large reduction in ...

journal_title：Statistics in medicine

authors： Chen P,Tebbs JM,Bilder CR

更新日期：2009-10-15 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：:In this paper we give an informal introduction to a robust method for survival analysis which is based on a modification of the usual partial likelihood estimator (PLE). Large sample results lead us to expect reduced bias for this robust estimator compared with the PLE whenever there are even slight violations of the ...

journal_title：Statistics in medicine

authors： Minder CE,Bednarski T

更新日期：1996-05-30 00:00:00

abstract：:This paper presents combinatorial (exact) methods that are useful in the analysis of disease cluster data obtained from small environments, such as buildings and neighbourhoods. Maxwell-Boltzmann and Fermi-Dirac occupancy models are compared in terms of appropriateness of representation of disease incidence patterns (...

journal_title：Statistics in medicine

authors： Grimson RC

更新日期：1993-10-01 00:00:00

abstract：:A decision-theoretic framework is proposed for designing sequential dose-finding trials with multiple outcomes. The optimal strategy is solvable theoretically via backward induction. However, for dose-finding studies involving k doses, the computational complexity is the same as the bandit problem with k-dependent arm...

journal_title：Statistics in medicine

authors： Fan SK,Wang YG

更新日期：2006-05-30 00:00:00

abstract：:Correlated response data arise often in biomedical studies. The generalized estimation equation (GEE) approach is widely used in regression analysis for such data. However, there are few methods available to check the adequacy of regression models in GEE. In this paper, a graphical method is proposed based on Cook and...

journal_title：Statistics in medicine

authors： Pan W,Connett JE,Porzio GC,Weisberg S

更新日期：2001-10-15 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：:Treatment effects for multiple outcomes can be meta-analyzed separately or jointly, but no systematic empirical comparison of the two approaches exists. From the Cochrane Library of Systematic Reviews, we identified 45 reviews, including 1473 trials and 258,675 patients, that contained two or three univariate meta-ana...

journal_title：Statistics in medicine

authors： Trikalinos TA,Hoaglin DC,Schmid CH

更新日期：2014-04-30 00:00:00

abstract：:We obtained maximum likelihood estimates (MLEs) of the proportion cured pi c and mean log survival time mu t for a sample of 4355 patients with intraocular melanoma whose survival times subsequent to treatment were assumed to follow a log-normal distribution. Following stratification by tumour size, MLEs of pi c and m...

journal_title：Statistics in medicine

authors： Gamel JW,McLean IW,Rosenberg SH

更新日期：1990-08-01 00:00:00

abstract：:Residuals in normal regression are used to assess a model's goodness-of-fit (GOF) and discover directions for improving the model. However, there is a lack of residuals with a characterized reference distribution for censored regression. In this article, we propose to diagnose censored regression with normalized rando...

journal_title：Statistics in medicine

authors： Li L,Wu T,Feng C

更新日期：2020-12-13 00:00:00

abstract：:Dependence between observations on a dichotomous variable renders invalid the usual chi-square tests of independence and inflates the variances of parameter estimates. Such a situation occurs, for example, when subjects consist of members of the same family or with repeated observations on the same person. In this pap...

journal_title：Statistics in medicine

authors： Donald A,Donner A

更新日期：1987-06-01 00:00:00

abstract：:A study of long term survival of 1487 patients given an allogeneic bone marrow transplant for acute myelogenous leukaemia and 729 patients given a transplant for severe aplastic anaemia was conducted by the International Bone Marrow Transplant Registry. One aim of this study is to determine if the mortality rates of t...

journal_title：Statistics in medicine

authors： Andersen PK,Horowitz MM,Klein JP,Socie G,Stone JV,Zhang MJ

更新日期：1999-06-30 00:00:00

abstract：:When the accrual rate is low and the treatment period is long, a long observational period is required before information concerning the primary end point, such as binary response, becomes available in the study. Simon's two-stage designs are often employed in Phase II clinical trials to avoid giving patient an ineffe...

journal_title：Statistics in medicine

authors： Chi Y,Chen CM

更新日期：2008-12-20 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

abstract：:In randomised trials, continuous endpoints are often measured with some degree of error. This study explores the impact of ignoring measurement error and proposes methods to improve statistical inference in the presence of measurement error. Three main types of measurement error in continuous endpoints are considered:...

journal_title：Statistics in medicine

authors： Nab L,Groenwold RHH,Welsing PMJ,van Smeden M

更新日期：2019-11-30 00:00:00

abstract：:In the analysis of survival data, there are often competing events that preclude an event of interest from occurring. Regression analysis with competing risks is typically undertaken using a cause-specific proportional hazards model. However, modern alternative methods exist for the analysis of the subdistribution haz...

journal_title：Statistics in medicine

authors： Lau B,Cole SR,Gange SJ

更新日期：2011-03-15 00:00:00

abstract：:The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...

journal_title：Statistics in medicine

authors： Tran TMP,Abrams S,Braekers R

更新日期：2020-05-30 00:00:00

abstract：:Epidermal nerve fibre (ENF) density and morphology are used to study small fibre involvement in diabetic, HIV, chemotherapy induced and other neuropathies. ENF density and summed length of ENFs per epidermal surface area are reduced, and ENFs may appear more clustered within the epidermis in subjects with small fibre ...

journal_title：Statistics in medicine

authors： Andersson C,Guttorp P,Särkkä A

更新日期：2016-10-30 00:00:00

abstract：:Important differences between explanatory and pragmatic studies were originally argued by Schwartz and Lellouch. Three important differences between the two types of study involve study control, study violators and inclusion criteria. It was originally argued that explanatory studies are highly controlled, and pragmat...

journal_title：Statistics in medicine

authors： McMahon AD

更新日期：2002-05-30 00:00:00

abstract：:The identification of counties burdened by exceptionally high rates of mortality is a fundamental step in the development of state-based intervention and prevention strategies. However, the estimation of rates from small geographic areas presents special problems, especially for rare events. This paper compares the us...

journal_title：Statistics in medicine

authors： Stevenson JM,Olson DR

更新日期：1993-02-01 00:00:00

abstract：:In the pharmaceutical industry, an assay method is considered validated if the accuracy and precision for an assay meet some acceptable limits. This paper discusses the assessment of assay precision in terms of the estimation of total variability of an assay from a one-way random effects model which is often considere...

journal_title：Statistics in medicine

authors： Chow SC,Tse SK

更新日期：1991-10-01 00:00:00

abstract：:Longitudinal studies are often concerned with estimating the recurrence rate of a non-fatal event. In many cases, only the total number of events occurring during successive time intervals is known. We compared a mixed Poisson-gamma regression method proposed by Thall and a quasi-likelihood method proposed by Zeger an...

journal_title：Statistics in medicine

authors： Stukel TA

更新日期：1993-07-30 00:00:00