Sample size calculation for stepped wedge and other longitudinal cluster randomised trials.

abstract：:Models for infant growth have usually been based on parametric forms, commonly an exponential or similar model, which have been shown to fit poorly especially during the first year of life. An alternative approach is to use a non-parametric model, based on a shape invariant model (SIM), where a single function is tran...

journal_title：Statistics in medicine

authors： Beath KJ

更新日期：2007-05-30 00:00:00

abstract：:In many biomedical and epidemiological studies, data are often clustered due to longitudinal follow up or repeated sampling. While in some clustered data the cluster size is pre-determined, in others it may be correlated with the outcome of subunits, resulting in informative cluster size. When the cluster size is info...

journal_title：Statistics in medicine

authors： Chen Z,Zhang B,Albert PS

更新日期：2011-07-10 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：:This paper examines several methods for deriving standardized morbidity ratios (SMR) and attributable fraction (attributable risk percentage) estimates. We show that some of the proposed methods will, in general, produce biased estimators, although the low variance of certain estimators sometimes compensates for their...

journal_title：Statistics in medicine

authors： Greenland S

更新日期：1984-04-01 00:00:00

abstract：:We propose a goodness-of-fit test statistic for linear regression with heterogeneous variance, which is asymptotically chi-square if the given model is correct. The test statistic is computed as a quadratic form of observed minus predicted responses. We apply the method to a linear regression for an ordinal categorica...

journal_title：Statistics in medicine

authors： Lipsitz SR,Buoncristiani JF

更新日期：1994-01-30 00:00:00

abstract：:Flexible survival models are in need when modelling data from long term follow-up studies. In many cases, the assumption of proportionality imposed by a Cox model will not be valid. Instead, a model that can identify time varying effects of fixed covariates can be used. Although there are several approaches that deal ...

journal_title：Statistics in medicine

authors： Perperoglou A

更新日期：2016-12-10 00:00:00

abstract：:Graphical methods based on the analysis of differences between log cumulative hazard functions are considered for a two-group semi-proportional hazard model which allows for interaction between treatments and covariates. Confidence procedures and test statistics that can be used to test for interaction and for main ef...

journal_title：Statistics in medicine

authors： Dabrowska DM,Doksum KA,Feduska NJ,Husing R,Neville P

更新日期：1992-08-01 00:00:00

abstract：:There is a rich literature that considers whether an observed relation between treatment and response is due to an unobserved covariate. In order to quantify this unmeasured bias, an assumption is made about the distribution of this unobserved covariate; typically that it is either binary or at least confined to the u...

journal_title：Statistics in medicine

authors： Wang L,Krieger AM

更新日期：2006-07-15 00:00:00

abstract：:Although prostate cancer and benign prostatic hyperplasia are major health problems in U.S. men, little is known about the early stages of the natural history of prostate disease. A molecular biomarker called prostate specific antigen (PSA), together with a unique longitudinal bank of frozen serum, now allows a histor...

journal_title：Statistics in medicine

authors： Pearson JD,Morrell CH,Landis PK,Carter HB,Brant LJ

更新日期：1994-03-15 00:00:00

abstract：:We consider counting process methods for analysing time-to-event data with multiple or recurrent outcomes, using the models developed by Anderson and Gill, Wei, Lin and Weissfeld and Prentice, Williams and Peterson. We compare the methods, and show how to implement them using popular statistical software programs. By ...

journal_title：Statistics in medicine

authors： Therneau TM,Hamilton SA

更新日期：1997-09-30 00:00:00

abstract：:When estimating the average effect of a binary treatment (or exposure) on an outcome, methods that incorporate propensity scores, the G-formula, or targeted maximum likelihood estimation (TMLE) are preferred over naïve regression approaches, which are biased under misspecification of a parametric outcome model. In con...

journal_title：Statistics in medicine

authors： Luque-Fernandez MA,Schomaker M,Rachet B,Schnitzer ME

更新日期：2018-07-20 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：:In this commentary, we revisit Sir Austin Bradford Hill's seminal Alfred Watson Memorial Lecture in 1962 through the eyes of two practicing biostatisticians of the current era. We summarize some eternal takeaway messages from Hill's lecture regarding observations and experiments translated through the modern lexicon o...

journal_title：Statistics in medicine

authors： Dempsey W,Mukherjee B

更新日期：2021-01-15 00:00:00

abstract：:In observational studies with censored data, exposure-outcome associations are commonly measured with adjusted hazard ratios from multivariable Cox proportional hazards models. The difference in restricted mean survival times (RMSTs) up to a pre-specified time point is an alternative measure that offers a clinically m...

journal_title：Statistics in medicine

authors： Conner SC,Sullivan LM,Benjamin EJ,LaValley MP,Galea S,Trinquart L

更新日期：2019-09-10 00:00:00

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：:Genetic networks for gene expression data are often built by graphical models, which in turn are built from pair-wise correlations of gene expression levels. A key feature of building graphical models is the evaluation of conditional independence of two traits, given other traits. When conditional independence can be ...

journal_title：Statistics in medicine

authors： Yu Z,Wang L,Hildebrandt MA,Schaid DJ

更新日期：2008-08-30 00:00:00

abstract：:In multivariate network meta-analysis (NMA), the piecemeal nature of the evidence base means that there may be treatment-outcome combinations for which no data is available. Most existing multivariate evidence synthesis models are either unable to estimate the missing treatment-outcome combinations, or can only do so ...

journal_title：Statistics in medicine

authors： Waddingham E,Matthews PM,Ashby D

更新日期：2020-10-30 00:00:00

abstract：:The use of classification and regression tree (CART) methodology is explored for the diagnosis of patients complaining of anterior chest pain. The results are compared with those previously obtained using correspondence analysis and independent Bayes classification. The technique is shown to be of potential value for ...

journal_title：Statistics in medicine

authors： Crichton NJ,Hinde JP,Marchini J

更新日期：1997-04-15 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：:In many community-based studies on the incidence of dementia, a target population is screened and a subsample is clinically evaluated at baseline and follow-up. Incidence rates are affected by missed cases at both exams and this complicates the estimation of these rates. Recent work proposes a regression-based techniq...

journal_title：Statistics in medicine

authors： Izmirlian G,Brock D,White L

更新日期：2000-06-15 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：: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：:The mutation and natural selection phenomenon can and often does cause the failure of antimicrobial, herbicidal, pesticide and cancer treatments selection pressures. This phenomenon operates in a mathematically predictable behavior, which when understood leads to approaches to reduce and prevent the failure of the use...

journal_title：Statistics in medicine

authors： Kleinman A

更新日期：2014-12-20 00:00:00

abstract：:Antimicrobial resistance has become one of the main public health burdens of the last decades, and monitoring the development and spread of non-wild-type isolates has therefore gained increased interest. Monitoring is performed based on the minimum inhibitory concentration (MIC) values, which are collected through the...

journal_title：Statistics in medicine

authors： Jaspers S,Aerts M,Verbeke G,Beloeil PA

更新日期：2014-01-30 00:00:00

abstract：:During phase III clinical trials in life-threatening disease settings, it is important to ensure that the Data Monitoring Committee (DMC) has exclusive access to the interim efficacy and safety data generated by the data analysis centre, in order to minimize the risk of widespread prejudgement of unreliable trial resu...

journal_title：Statistics in medicine

authors： Fleming TR,Ellenberg S,DeMets DL

更新日期：2002-10-15 00:00:00

abstract：:The standard analysis of clinical trials stratified by centre is to include centres as fixed effects, but if many centres contribute small numbers of patients, this approach results in a loss of power. Assuming no treatment by centre interaction, we used simulation to examine power and coverage of confidence intervals...

journal_title：Statistics in medicine

authors： Pickering RM,Weatherall M

更新日期：2007-12-30 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：:We present a new approach to training back-propagation artificial neural nets (BP-ANN) based on regularization and cross-validation and on initialization by a logistic regression (LR) model. The new approach is expected to produce a BP-ANN predictor at least as good as the LR-based one. We have applied the approach to...

journal_title：Statistics in medicine

authors： Ciampi A,Zhang F

更新日期：2002-05-15 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：:The effective dose (ED) is the pharmaceutical dosage required to produce a therapeutic response in a fixed proportion of the patients. When only one drug is considered, the problem is a univariate one and has been well-studied. However, in the multidimensional setting, that is, in the presence of combinations of agent...

journal_title：Statistics in medicine

authors： Jankowski H,Ji X,Stanberry L

更新日期：2014-10-30 00:00:00