Re-evaluating design specifications of longitudinal clinical trials without unblinding when the key response is rate of change.

abstract：:A considerable number of the clinical reports which are presented to the Dutch Board for the Evaluation of Drugs, have deficiencies and/or shortcomings. A number of these, including loose description of the target population and sampling method, methodological flaws, incorrect treatment of withdrawals, confounding of ...

journal_title：Statistics in medicine

authors： De Jonge H

更新日期：1983-04-01 00:00:00

abstract：:The sojourn time, time spent in the preclinical detectable phase (PCDP) for chronic diseases, for example, breast cancer, plays an important role in the design and assessment of screening programmes. Traditional methods to estimate it usually assume a uniform incidence rate of preclinical disease from a randomized con...

journal_title：Statistics in medicine

authors： Duffy SW,Chen HH,Tabar L,Day NE

更新日期：1995-07-30 00:00:00

abstract：:R2 has been criticized as a measure of model performance when predicting a dichotomous outcome, both because its value is often low and because it is sensitive to the prevalence of the event of interest. The C statistic is more widely used to measure model performance in a 0/1 setting. We use a simple parametric famil...

journal_title：Statistics in medicine

authors： Ash A,Shwartz M

更新日期：1999-02-28 00:00:00

abstract：:We propose a semiparametric method for estimating ROC surfaces for continuous diagnostic tests based on two test measurements. Such a three-class diagnostic problem based on two test measurements arises naturally from some DNA amplification-related diagnostic scenarios. Simulation results show that our proposed semipa...

journal_title：Statistics in medicine

authors： Wan S,Zhang B

更新日期：2009-08-15 00:00:00

abstract：:Clinical research often involves continuous outcome measures, such as blood cholesterol, that are amenable to statistical techniques of analysis based on the mean, such as the t-test or multiple linear regression. Clinical interest, however, frequently focuses on the proportion of subjects who fall below or above a cl...

journal_title：Statistics in medicine

authors： Suissa S,Blais L

更新日期：1995-02-15 00:00:00

abstract：:The primary goal of a phase I trial is to find the maximally tolerated dose (MTD) of a treatment. The MTD is usually defined in terms of a tolerable probability, q(*), of toxicity. Our objective is to find the highest dose with toxicity risk that does not exceed q(*), a criterion that is often desired in designing pha...

journal_title：Statistics in medicine

authors： Leung DH,Wang YG

更新日期：2002-01-15 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：:Assessing the calibration of methods for estimating the probability of the occurrence of a binary outcome is an important aspect of validating the performance of risk-prediction algorithms. Calibration commonly refers to the agreement between predicted and observed probabilities of the outcome. Graphical methods are a...

journal_title：Statistics in medicine

authors： Austin PC,Steyerberg EW

更新日期：2019-09-20 00:00:00

abstract：:When estimating the probability of natural conception from observational data on couples with an unfulfilled child wish, the start of assisted reproductive therapy (ART) is a competing event that cannot be assumed to be independent of natural conception. In clinical practice, interest lies in the probability of natura...

journal_title：Statistics in medicine

authors： van Geloven N,Geskus RB,Mol BW,Zwinderman AH

更新日期：2014-11-20 00:00:00

abstract：:We applied a two-stage random effects model to pulmonary function data from 31 sarcoidosis patients to illustrate its usefulness in analysing unbalanced longitudinal data. For the first stage, repeated measurements of percentage of predicted forced vital capacity (FVC%) from an individual were modelled as a function o...

journal_title：Statistics in medicine

authors： Vacek PM,Mickey RM,Bell DY

更新日期：1989-02-01 00:00:00

abstract：:I discuss alternatives to the one compartment model, delta Yt = alpha + beta exp(- gamma t). Instead of comparing the one and two compartment models, I derive statistics for testing mixtures of the parameters (beta, gamma) in the one compartment model. I apply the proposed methods to the problem of hydrogen clearance ...

journal_title：Statistics in medicine

authors： Zelterman D

更新日期：1989-08-01 00:00:00

abstract：:Missing data arise in crossover trials, as they do in any form of clinical trial. Several papers have addressed the problems that missing data create, although almost all of these assume that the probability that a planned observation is missing does not depend on the value that would have been observed; that is, the ...

journal_title：Statistics in medicine

authors： Ho WK,Matthews JN,Henderson R,Farewell D,Rodgers LR

更新日期：2012-07-20 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：:Previous work on the consequences of regression to the mean for the interpretation of responses to treatment is extended to the situation where the response measured is the proportional change in some variable. Methods for correcting for the bias are discussed. ...

journal_title：Statistics in medicine

authors： Curnow RN

更新日期：1987-03-01 00:00:00

abstract：:Laboratory determinations on children aged 6 to 10 years obtained over a 5-year period are analysed by a method described in detail for differentiating between children from exposed and control areas of Seveso, Italy. In the analysis, stratification is employed to distinguish the separate days of laboratory measuremen...

journal_title：Statistics in medicine

authors： Mantel N,Mocarelli P,Marocchi A,Brambilla P,Baretta R

更新日期：1983-04-01 00:00:00

abstract：:For the meta-analysis of controlled clinical trials or epidemiological studies, in which the responses are at least approximately normally distributed, a refined test for the hypothesis of no overall treatment effect is proposed. The test statistic is based on a direct estimation function for the variance of the overa...

journal_title：Statistics in medicine

authors： Hartung J,Knapp G

更新日期：2001-06-30 00:00:00

abstract：:Recently, there has been much work on early phase cancer designs that incorporate both toxicity and efficacy data, called phase I-II designs because they combine elements of both phases. However, they do not explicitly address the phase II hypothesis test of H0 : p ≤ p0 , where p is the probability of efficacy at the ...

journal_title：Statistics in medicine

authors： Bartroff J,Lai TL,Narasimhan B

更新日期：2014-07-20 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：:Identification of key factors associated with the risk of developing cardiovascular disease and quantification of this risk using multivariable prediction algorithms are among the major advances made in preventive cardiology and cardiovascular epidemiology in the 20th century. The ongoing discovery of new risk markers...

journal_title：Statistics in medicine

authors： Pencina MJ,D'Agostino RB Sr,D'Agostino RB Jr,Vasan RS

更新日期：2008-01-30 00:00:00

abstract：:Statistical tests have been proposed for determining whether incident cases of adverse health effects are 'clustered' together. Several procedures, termed 'focused', specifically analyse disease surveillance data around pre-specified putative sources of environmental hazard. Little has been done to compare the perform...

journal_title：Statistics in medicine

authors： Waller LA,Lawson AB

更新日期：1995-11-15 00:00:00

abstract：:Many split-mouth trials are characterized by the pairing of site-specific outcome and baseline data within each segment of a subject's mouth. However when the response variable of interest is binary, methods of statistical analysis for this design are not well developed. In this paper we present several analytic appro...

journal_title：Statistics in medicine

authors： Donner A,Zou GY

更新日期：2007-08-15 00:00:00

abstract：:The 'landmark' and 'Simon and Makuch' non-parametric estimators of the survival function are commonly used to contrast the survival experience of time-dependent treatment groups in applications such as stem cell transplant versus chemotherapy in leukemia. However, the theoretical survival functions corresponding to th...

journal_title：Statistics in medicine

authors： Bernasconi DP,Rebora P,Iacobelli S,Valsecchi MG,Antolini L

更新日期：2016-03-30 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：:I reflect on the statistical methods of the Christakis-Fowler studies on network-based contagion of traits by checking the sensitivity of these kinds of results to various alternate specifications and generative mechanisms. Despite the honest efforts of all involved, I remain pessimistic about establishing whether bin...

journal_title：Statistics in medicine

authors： Thomas AC

更新日期：2013-02-20 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：:We present an estimate of the kappa-coefficient of agreement between two methods of rating based on matched pairs of binary responses and show that the estimate depends on the common intraclass correlation coefficient between the pairs. Via Monte Carlo simulation, we investigate power of the test of significance on ka...

journal_title：Statistics in medicine

authors： Shoukri MM,Martin SW,Mian IU

更新日期：1995-01-15 00:00:00

abstract：:In this paper, an approach to estimating the cumulative mean function for history process with time dependent covariates and right censored time-to-event variable is developed using the combined technique of joint modeling and inverse probability weighting method. The consistency of proposed estimator is derived. Theo...

journal_title：Statistics in medicine

authors： Deng D

更新日期：2016-11-10 00:00:00

abstract：:Within the rich literature on generalized linear models, substantial efforts have been devoted to models for categorical responses that are either completely ordered or completely unordered. Few studies have focused on the analysis of partially ordered outcomes, which arise in practically every area of study, includin...

journal_title：Statistics in medicine

authors： Zhang Q,Ip EH

更新日期：2012-01-13 00:00:00

abstract：:Current methods for statistical analysis of twin studies focus on continuous and dichotomous data, while only limited methodology exists for analysing multinomial data. As a consequence, investigators are often tempted to collapse multinomial data into two categories simply to facilitate the analysis. We address this ...

journal_title：Statistics in medicine

authors： Bartfay E,Donner A

更新日期：2001-01-30 00:00:00

abstract：:This paper concerns using modified weighted Schoenfeld residuals to test the proportionality of subdistribution hazards for the Fine-Gray model, similar to the tests proposed by Grambsch and Therneau for independently censored data. We develop a score test for the time-varying coefficients based on the modified Schoen...

journal_title：Statistics in medicine

authors： Zhou B,Fine J,Laird G

更新日期：2013-09-30 00:00:00