Analyzing longitudinal data with patients in different disease states during follow-up and death as final state.

abstract：:This paper discusses the benefits and limitations of adaptive sample size re-estimation for phase 3 confirmatory clinical trials. Comparisons are made with more traditional fixed sample and group sequential designs. It is seen that the real benefit of the adaptive approach arises through the ability to invest sample s...

journal_title：Statistics in medicine

authors： Mehta CR,Pocock SJ

更新日期：2011-12-10 00:00:00

abstract：:Selection of dose for cancer patients treated with radiation therapy (RT) must balance the increased efficacy with the increased toxicity associated with higher dose. Historically, a single dose has been selected for a population of patients (e.g., all stage III non-small cell lung cancer). However, the availability o...

journal_title：Statistics in medicine

authors： Schipper MJ,Taylor JM,TenHaken R,Matuzak MM,Kong FM,Lawrence TS

更新日期：2014-12-30 00:00:00

abstract：:A preference trial is a special form of cross-over trial where clinical conditions determine when patients change treatment, in a prescribed order. This can be modelled using a geometric distribution. The model can be simply fitted using standard logistic regression methodology. The procedure is applied to a trial stu...

journal_title：Statistics in medicine

authors： Lindsey JK,Jones B

更新日期：1996-02-28 00:00:00

abstract：:Using both simulated and real datasets, we compared two approaches for estimating absolute risk from nested case-control (NCC) data and demonstrated the feasibility of using the NCC design for estimating absolute risk. In contrast to previously published results, we successfully demonstrated not only that data from a ...

journal_title：Statistics in medicine

authors： Salim A,Delcoigne B,Villaflores K,Koh WP,Yuan JM,van Dam RM,Reilly M

更新日期：2017-02-10 00:00:00

abstract：:The use of both sequential designs and adaptive treatment allocation are effective in reducing the number of patients receiving an inferior treatment in a clinical trial. In large samples, when the asymptotic normality of test statistics can be utilized, a standard sequential design can be combined with adaptive alloc...

journal_title：Statistics in medicine

authors： Stallard N,Rosenberger WF

更新日期：2002-02-28 00:00:00

abstract：:Geocoding a study population as completely as possible is an important data assimilation component of many spatial epidemiologic studies. Unfortunately, complete geocoding is rare in practice. The failure of a substantial proportion of study subjects' addresses to geocode has consequences for spatial analyses, some of...

journal_title：Statistics in medicine

authors： Zimmerman DL,Fang X,Mazumdar S

更新日期：2008-09-20 00:00:00

abstract：:A trial of Duchenne muscular dystrophy involved tracking boys of all ages through a one-year baseline period, followed by a one-year trial of leucine versus placebo treatment. In this paper we develop a model for a total-muscle-strength score that uses the data of the extended baseline period in the evaluation of the ...

journal_title：Statistics in medicine

authors： Madsen KS,Miller JP,Province MA

更新日期：1986-05-01 00:00:00

abstract：:This article deals with determination of a sample size that guarantees the success of a trial. We follow a Bayesian approach and we say an experiment is successful if it yields a large posterior probability that an unknown parameter of interest (an unknown treatment effect or an effects-difference) is greater than a c...

journal_title：Statistics in medicine

authors： Brutti P,De Santis F,Gubbiotti S

更新日期：2008-06-15 00:00:00

abstract：:While there has been extensive research developing gene-environment interaction (GEI) methods in case-control studies, little attention has been given to sparse and efficient modeling of GEI in longitudinal studies. In a two-way table for GEI with rows and columns as categorical variables, a conventional saturated int...

journal_title：Statistics in medicine

authors： Ko YA,Mukherjee B,Smith JA,Park SK,Kardia SL,Allison MA,Vokonas PS,Chen J,Diez-Roux AV

更新日期：2014-12-20 00:00:00

abstract：:Exploring the possible reasons for heterogeneity between studies is an important aspect of conducting a meta-analysis. This paper compares a number of methods which can be used to investigate whether a particular covariate, with a value defined for each study in the meta-analysis, explains any heterogeneity. The main ...

journal_title：Statistics in medicine

authors： Thompson SG,Sharp SJ

更新日期：1999-10-30 00:00:00

abstract：:During a course of human immunodeficiency virus (HIV-1) infection, the viral load usually increases sharply to a peak following infection and then drops rapidly to a steady state, where it remains until progression to AIDS. This steady state is often referred to as the viral set point. It is believed that the HIV vira...

journal_title：Statistics in medicine

authors： Mei Y,Wang L,Holte SE

更新日期：2008-01-15 00:00:00

abstract：:Correlation is always a concern in the analysis of clustered data. One area of interest is to develop a general correlation modelling approach for high dimensional data with unbalanced hierarchical and heterogeneous data structures, e.g. multilevel data. Commonly used correlation structures might have limitation for s...

journal_title：Statistics in medicine

authors： Chao EC

更新日期：2006-07-30 00:00:00

abstract：:Causal inference for non-censored response variables, such as binary or quantitative outcomes, is often based on either (1) direct standardization ('G-formula') or (2) inverse probability of treatment assignment weights ('propensity score'). To do causal inference in survival analysis, one needs to address right-censo...

journal_title：Statistics in medicine

authors： Andersen PK,Syriopoulou E,Parner ET

更新日期：2017-07-30 00:00:00

abstract：:A good measure of field vaccine efficacy should evaluate the direct protective effect of vaccination on the person who receives the vaccine. The conventional estimator for vaccine efficacy depends on population level factors that are either unrelated or indirectly related to the direct biological action of the vaccine...

journal_title：Statistics in medicine

authors： Longini IM Jr,Halloran ME,Haber M,Chen RT

更新日期：1993-02-01 00:00:00

abstract：:The application of Bayesian hierarchical models to measure spatial effects in time to event data has not been widely reported. This case study aims to estimate the effect of area of residence on waiting times to coronary artery bypass graft (CABG) and to assess the role of important individual specific covariates (age...

journal_title：Statistics in medicine

authors： Crook AM,Knorr-Held L,Hemingway H

更新日期：2003-09-30 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：:Sample size planning should reflect the primary objective of a trial. If the primary objective is prediction, the sample size determination should focus on prediction accuracy instead of power. We present formulas for the determination of training set sample size for survival prediction. Sample size is chosen to contr...

journal_title：Statistics in medicine

authors： Götte H,Zwiener I

更新日期：2013-02-28 00:00:00

abstract：:Biostatisticians have frequently uncritically accepted the measurements provided by their medical colleagues engaged in clinical research. Such measures often involve considerable loss of information. Particularly, unfortunate is the widespread use of the so-called 'responder analysis', which may involve not only a lo...

journal_title：Statistics in medicine

authors： Senn S,Julious S

更新日期：2009-11-20 00:00:00

abstract：:In standard time-to-event or survival analysis, occurrence times of the event of interest are observed exactly or are right-censored, meaning that it is only known that the event occurred after the last observation time. There are numerous methods available for estimating the survival curve and for testing and estimat...

journal_title：Statistics in medicine

authors： Lindsey JC,Ryan LM

更新日期：1998-01-30 00:00:00

abstract：:The use of outcome-dependent sampling with longitudinal data analysis has previously been shown to improve efficiency in the estimation of regression parameters. The motivating scenario is when outcome data exist for all cohort members but key exposure variables will be gathered only on a subset. Inference with outcom...

journal_title：Statistics in medicine

authors： Zelnick LR,Schildcrout JS,Heagerty PJ

更新日期：2018-06-15 00:00:00

abstract：:A frequent objective in medical research is the investigation of differences in patient survival between several experimental treatments and one standard treatment. In order to assess these differences statistically, we have to apply adjustments for multiple comparisons to prevent an increased number of false-positive...

journal_title：Statistics in medicine

authors： Herberich E,Hothorn T

更新日期：2012-01-13 00:00:00

abstract：:The Box-Cox power exponential (BCPE) distribution, developed in this paper, provides a model for a dependent variable Y exhibiting both skewness and kurtosis (leptokurtosis or platykurtosis). The distribution is defined by a power transformation Y(nu) having a shifted and scaled (truncated) standard power exponential ...

journal_title：Statistics in medicine

authors： Rigby RA,Stasinopoulos DM

更新日期：2004-10-15 00:00:00

abstract：:Non-linear mixed-effects models (NLMEMs) are used to improve information gathering from longitudinal studies and are applied to treatment evaluation in disease-evolution studies, such as human immunodeficiency virus (HIV) infection. The estimation of parameters and the statistical tests are critical issues in NLMEMs s...

journal_title：Statistics in medicine

authors： Samson A,Lavielle M,Mentré F

更新日期：2007-11-30 00:00:00

abstract：:The authors measure the efficacy of three methods for predicting the time to infection for susceptible individuals in a population undergoing an HIV epidemic. The methods differ in whether they require detailed information of the contact network and whether they require knowledge of the initial source of infection. Ef...

journal_title：Statistics in medicine

authors： Altmann M,Wee BC,Willard K,Peterson D,Gatewood LC

更新日期：1994-01-15 00:00:00

abstract：:We suggest measures to quantify the degrees of necessity and of sufficiency of prognostic factors for dichotomous and for survival outcomes. A cause, represented by certain values of prognostic factors, is considered necessary for an event if, without the cause, the event cannot develop. It is considered sufficient fo...

journal_title：Statistics in medicine

authors： Gleiss A,Schemper M

更新日期：2019-10-15 00:00:00

abstract：:This paper presents a general approach for simultaneously assessing, from serial data, diagnostic consistency, interrater reliability and incidence of a strictly progressive disease. Observed data are viewed as incomplete: diagnostic errors are not distinguished from true diagnoses. We introduce a broad class of model...

journal_title：Statistics in medicine

authors： Espeland MA,Murphy WC,Leverett DH

更新日期：1988-03-01 00:00:00

abstract：:In this paper we present a simple method for constructing (1- alpha)100 per cent confidence intervals for binomial proportions with near nominal coverage for all underlying proportion parameters on the unit interval. This new method uses, with a slight modification, the standard normal approximation technique taught i...

journal_title：Statistics in medicine

authors： Borkowf CB

更新日期：2006-11-15 00:00:00

abstract：:Drop-out often occurs in clinical trials with multiple visits and drop-out is often informative in the sense that the population of patients who dropped out is different from the population of patients who completed the study. To handle data with informative drop-out, an intention-to-treat analysis, which evaluates tr...

journal_title：Statistics in medicine

authors： Shao J,Zhong B

更新日期：2003-08-15 00:00:00

abstract：:We study the competing risks model and show that the cause j cumulative incidence function integrated from 0 to τ has a natural interpretation as the expected number of life years lost due to cause j before time τ. This is analogous to the τ-restricted mean lifetime, which is the survival function integrated from 0 to...

journal_title：Statistics in medicine

authors： Andersen PK

更新日期：2013-12-30 00:00:00

abstract：:Piecewise constant incidence models were developed to estimate the force of infection in women from age- and time-specific antenatal or neonatal seroprevalence data. Differential inclusion of infected women in sero-surveys compared to uninfected women was taken into account, with respect to both changes in inclusion r...

journal_title：Statistics in medicine

authors： Ades AE,Medley GF

更新日期：1994-09-30 00:00:00