Exact and asymptotic inference in clinical trials with small event rates under inverse sampling.

abstract：:We consider several sources of heterogeneity in a clinical trial with patients' survival time as the main response criterion: differences in prognosis which can be attributed to a latent or ignored prognostic factor; differences in treatment efficacy in subgroups of patients, and differences in treatment combinations ...

journal_title：Statistics in medicine

authors： Schumacher M,Olschewski M,Schmoor C

更新日期：1987-10-01 00:00:00

abstract：:Algorithms for identifying public health threats or disease outbreaks are vulnerable to false alarms arising from sudden shifts in health-care utilization or data participation. This paper describes a method of reducing false alerts in automated public health surveillance algorithms, and in particular, automated syndr...

journal_title：Statistics in medicine

authors： Peter W,Najmi AH,Burkom HS

更新日期：2011-06-30 00:00:00

abstract：:Evaluating treatment effects within different subsets of patients is a common practice in the analysis of individual randomized clinical trials. Such analyses are limited, however, by the number of patients available. Overviews, by providing evidence based on large numbers of patients, can be useful for overcoming the...

journal_title：Statistics in medicine

authors： Gelber RD,Goldhirsch A

更新日期：1987-04-01 00:00:00

abstract：:We survey models for analysing repeated observations on an ordered categorical response variable. The models presented are univariate models that permit correlation among repeated measurements. The models describe simultaneously the dependence of marginal response distributions on values of explanatory variables and o...

journal_title：Statistics in medicine

authors： Agresti A

更新日期：1989-10-01 00:00:00

abstract：:Delay in the outcome variable is challenging for outcome-adaptive randomization, as it creates a lag between the number of subjects accrued and the information known at the time of the analysis. Motivated by a real-life pediatric ulcerative colitis trial, we consider a case where a short-term predictor is available fo...

journal_title：Statistics in medicine

authors： Kim MO,Liu C,Hu F,Lee JJ

更新日期：2014-10-15 00:00:00

abstract：:Studies involving longitudinal binary responses are widely applied in the health and biomedical sciences research and frequently analyzed by generalized estimating equations (GEE) method. This article proposes an alternative goodness-of-fit test based on the nonparametric smoothing approach for assessing the adequacy ...

journal_title：Statistics in medicine

authors： Lin KC,Chen YJ,Shyr Y

更新日期：2008-09-30 00:00:00

abstract：:In many practical applications, count data often exhibit greater or less variability than allowed by the equality of mean and variance, referred to as overdispersion/underdispersion, and there are several reasons that may lead to the overdispersion/underdispersion such as zero inflation and mixture. Moreover, if the c...

journal_title：Statistics in medicine

authors： Chen XD,Fu YZ,Wang XR

更新日期：2013-04-15 00:00:00

abstract：:We present methods for binomial regression when the outcome is determined using the results of a single diagnostic test with imperfect sensitivity and specificity. We present our model, illustrate it with the analysis of real data, and provide an example of WinBUGS program code for performing such an analysis. Conditi...

journal_title：Statistics in medicine

authors： McInturff P,Johnson WO,Cowling D,Gardner IA

更新日期：2004-04-15 00:00:00

abstract：:Various aspects of portfolio management and project prioritization within the pharmaceutical industry are examined. It is shown that the cost and probability architecture of a project is a crucial aspect of its value. An appropriate simple tool for ranking projects is the Pearson index. Various difficulties are consid...

journal_title：Statistics in medicine

authors： Senn S

更新日期：1996-12-30 00:00:00

abstract：:This paper presents tables analogous to T-tables for use in the 2 x 2 multinomial trial, where the continuity corrected Z-statistic is used to make exact unconditional inference. This is the first solution of a discrete exact unconditional inference problem involving a multivariate nuisance parameter for which no anci...

journal_title：Statistics in medicine

authors： Shuster JJ

更新日期：1992-05-01 00:00:00

abstract：:In noninferiority studies, a limit of indifference is used to express a tolerance in results such that the clinician would regard such results as being acceptable or 'not worse'. We applied this concept to a measure of accuracy, the Receiver Operating Characteristic (ROC) curve, for a sequence of tests. We expressed a...

journal_title：Statistics in medicine

authors： Ahmed AE,Schubert CM,McClish DK

更新日期：2013-07-20 00:00:00

abstract：:In vaccine safety studies, subjects are considered at increased risk for adverse events for a period of time after vaccination known as risk window. To our knowledge, risk windows for vaccine safety studies have tended to be pre-defined and not to use information from the current study. Inaccurate specification of the...

journal_title：Statistics in medicine

authors： Xu S,Zhang L,Nelson JC,Zeng C,Mullooly J,McClure D,Glanz J

更新日期：2011-03-30 00:00:00

abstract：:For bivariate meta-analysis of diagnostic studies, likelihood approaches are very popular. However, they often run into numerical problems with possible non-convergence. In addition, the construction of confidence intervals is controversial. Bayesian methods based on Markov chain Monte Carlo (MCMC) sampling could be u...

journal_title：Statistics in medicine

authors： Paul M,Riebler A,Bachmann LM,Rue H,Held L

更新日期：2010-05-30 00:00:00

abstract：:In observational studies, many continuous or categorical covariates may be related to an outcome. Various spline-based procedures or the multivariable fractional polynomial (MFP) procedure can be used to identify important variables and functional forms for continuous covariates. This is the main aim of an explanatory...

journal_title：Statistics in medicine

authors： Binder H,Sauerbrei W,Royston P

更新日期：2013-06-15 00:00:00

abstract：:Sexually transmitted infections (STIs) with Chlamydia trachomatis, Neisseria gonorrhoeae, and Trichomonas vaginalis are among the most common infectious diseases in the United States, disproportionately affecting young women. Because a significant portion of the infections present no symptoms, infection control relies...

journal_title：Statistics in medicine

authors： Li Z,Liu H,Tu W

更新日期：2015-09-10 00:00:00

abstract：:Both traditional phase I designs and the increasingly popular continual reassessment method (CRM) designs select an estimate of maximum tolerable dose (MTD) from among a set of prespecified dose levels. Although CRM designs use an implied dose-response model to select the next dose level, in general it is neither assu...

journal_title：Statistics in medicine

authors： Storer BE

更新日期：2001-08-30 00:00:00

abstract：:In studies with survival or time-to-event outcomes, a competing risk is an event whose occurrence precludes the occurrence of the primary event of interest. Specialized statistical methods must be used to analyze survival data in the presence of competing risks. We conducted a review of randomized controlled trials wi...

journal_title：Statistics in medicine

authors： Austin PC,Fine JP

更新日期：2017-04-15 00:00:00

abstract：:A calibration line is used to define the relationship between a new clinical technique and a standard in vitro laboratory methodology. Discrimination intervals quantify the reliability of inverse estimates obtained from the calibration line. Applied to transcutaneous PCO2 monitoring, a new in vivo measurement, discrim...

journal_title：Statistics in medicine

authors： Kost GJ

更新日期：1986-07-01 00:00:00

abstract：:The need for resource-intensive laboratory assays to assess exposures in many epidemiologic studies provides ample motivation to consider study designs that incorporate pooled samples. In this paper, we consider the case in which specimens are combined for the purpose of determining the presence or absence of a pool-w...

journal_title：Statistics in medicine

authors： Lyles RH,Tang L,Lin J,Zhang Z,Mukherjee B

更新日期：2012-09-28 00:00:00

abstract：:We present an approach for inference on contrasts of clinically meaningful functionals of a survivor distribution (e.g., restricted mean, quantiles) that can avoid strong parametric or semiparametric assumptions on the underlying structure of the data. In this multistage approach, we first use an adaptive predictive m...

journal_title：Statistics in medicine

authors： Rudser KD,LeBlanc ML,Emerson SS

更新日期：2012-07-20 00:00:00

abstract：:The construction, validation and updating of a prognostic model for kidney graft survival is reported using data from the Eurotransplant database. First, a model is constructed for data from transplantations in the period 1984 to 1987. The model is later updated for the 1988 1990 data. The first data set was randomly ...

journal_title：Statistics in medicine

authors： Van Houwelingen HC,Thorogood J

更新日期：1995-09-30 00:00:00

abstract：:In recent years randomized trials designed to establish non-inferiority of a new treatment as compared to a standard one have been more widely used. Two-sample statistics have been proposed for this equivalence testing problem. However, they are not suited to situations where a long-term survivor fraction is expected....

journal_title：Statistics in medicine

authors： Broët P,Tubert-Bitter P,De Rycke Y,Moreau T

更新日期：2003-03-30 00:00:00

abstract：:Weak measurement of epidemiologic exposures is an impediment to appreciation of the effects of those exposures. This paper discusses two strategies to assess the true effects of weakly measured exposure. The first is to use external information about the extent of mismeasurement to adjust estimates of the effects of e...

journal_title：Statistics in medicine

authors： Marshall JR

更新日期：1989-09-01 00:00:00

abstract：:The incremental life expectancy, defined as the difference in mean survival times between two treatment groups, is a crucial quantity of interest in cost-effectiveness analyses. Usually, this quantity is very difficult to estimate from censored survival data with a limited follow-up period. The paper develops estimati...

journal_title：Statistics in medicine

authors： Titman AC

更新日期：2016-09-10 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：: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

abstract：:The coefficient of individual equivalence is a permutation-based measure of agreement between two observers making replicated readings on each subject. It compares the observed disagreement between the observers to the expected disagreement under individual equivalence. Individual equivalence of observers requires tha...

journal_title：Statistics in medicine

authors： Pan Y,Haber M,Gao J,Barnhart HX

更新日期：2012-09-10 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

abstract：:The "some invalid, some valid instrumental variable estimator" (sisVIVE) is a lasso-based method for instrumental variables (IVs) regression of outcome on an exposure. In principle, sisVIVE is robust to some of the IVs in the analysis being invalid, in the sense of being related to the outcome variable through pathway...

journal_title：Statistics in medicine

authors： Bao Y,Clarke PS,Smart M,Kumari M

更新日期：2019-04-30 00:00:00

abstract：:Few methods are currently available for group sequential analysis of recurrent events data subject to a terminal event in the clinical trial setting. This research helps fill this gap by developing a completely nonparametric group sequential monitoring procedure for use with the two-sample Tayob and Murray statistic. ...

journal_title：Statistics in medicine

authors： Xia M,Murray S,Tayob N

更新日期：2019-12-30 00:00:00