Flexible design clinical trial methodology in regulatory applications.

abstract：:Methods for dealing with tied event times in the Cox proportional hazards model are well developed. Also, the partial likelihood provides a natural way to handle covariates that change over time. However, ties between event times and the times that discrete time-varying covariates change have not been systematically s...

journal_title：Statistics in medicine

authors： Xin X,Horrocks J,Darlington GA

更新日期：2013-06-30 00:00:00

abstract：:This paper gives a standard error for Cohen's Kappa, conditional on the margins of the observed r x r table. An explicit formula is given for the 2 x 2 table, and a procedure for the more general situation. A parsimonious log-linear model is suggested for the general case and an approximate confidence interval for kap...

journal_title：Statistics in medicine

authors： Garner JB

更新日期：1991-05-01 00:00:00

abstract：:A relationship between baseline risk and treatment effect is increasingly investigated as a possible explanation of between-study heterogeneity in clinical trial meta-analysis. An approach that is still often applied in the medical literature is to plot the estimated treatment effects against the estimated measures of...

journal_title：Statistics in medicine

authors： Arends LR,Hoes AW,Lubsen J,Grobbee DE,Stijnen T

更新日期：2000-12-30 00:00:00

abstract：:Obtaining accurate estimates of the performance of a diagnostic test for some population of patients might be difficult when the sample of subjects used for this purpose is not representative for the whole population. Thus, in the motivating example of this paper a test is evaluated by comparing its results with those...

journal_title：Statistics in medicine

authors： Buzoianu M,Kadane JB

更新日期：2008-06-15 00:00:00

abstract：:A personalized treatment strategy formalizes evidence-based treatment selection by mapping patient information to a recommended treatment. Personalized treatment strategies can produce better patient outcomes while reducing cost and treatment burden. Thus, among clinical and intervention scientists, there is a growing...

journal_title：Statistics in medicine

authors： Laber EB,Zhao YQ,Regh T,Davidian M,Tsiatis A,Stanford JB,Zeng D,Song R,Kosorok MR

更新日期：2016-04-15 00:00:00

abstract：:Identification of the latency period for the effect of a time-varying exposure is key when assessing many environmental, nutritional, and behavioral risk factors. A pre-specified exposure metric involving an unknown latency parameter is often used in the statistical model for the exposure-disease relationship. Likelih...

journal_title：Statistics in medicine

authors： Peskoe SB,Spiegelman D,Wang M

更新日期：2019-03-30 00:00:00

abstract：:The design of clinical trials often requires knowledge of quantities such as between- and within subject variances about which only imprecise information exists. To provide assurance that the study has the desired power to detect a minimum clinically meaningful difference between treatment groups. Gould, Gould and Shi...

journal_title：Statistics in medicine

authors： Shih WJ,Gould AL

更新日期：1995-10-30 00:00:00

abstract：:In comparing two treatments under a typical sequential clinical trial setting, a 50-50 randomization design generates reliable data for making efficient inferences about the treatment difference for the benefit of patients in the general population. However, if the treatment difference is large and the endpoint of the...

journal_title：Statistics in medicine

authors： Yao Q,Wei LJ

更新日期：1996-11-15 00:00:00

abstract：:Proportional odds regression models for multinomial probabilities based on ordered categories have been generalized in two somewhat different directions. Models having scale as well as location parameters for adjustment of boundaries (on an unobservable, underlying continuum) between categories have been employed in t...

journal_title：Statistics in medicine

authors： Cox C

更新日期：1995-06-15 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：: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：:Lung function tests are used both clinically, in assessing disease, and epidemiologically, in identifying those factors which influence the growth and aging process of the lungs. The user must beware of several common pitfalls in the use of these tests, however. First, the commonly used tests of lung function can only...

journal_title：Statistics in medicine

authors： Buist AS,Vollmer WM

更新日期：1988-01-01 00:00:00

abstract：:Among many other uses, positron emission tomography (PET) can be used in studies to estimate the density of a neuroreceptor at each location throughout the brain by measuring the concentration of a radiotracer over time and modeling its kinetics. There are a variety of kinetic models in common usage and these typicall...

journal_title：Statistics in medicine

authors： Chang C,Ogden RT

更新日期：2009-03-15 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：:Studies based on aggregated hospital outcome data have established that there is a relationship between nurse staffing and adverse events. However, this result could not be confirmed in Belgium where 96 per cent of the variability of nurse staffing levels over nursing units (belonging to different hospitals) is explai...

journal_title：Statistics in medicine

authors： Diya L,Lesaffre E,Van den Heede K,Sermeus W,Vleugels A

更新日期：2010-03-30 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：:Nutritional exposures are often measured with considerable error in commonly used surrogate instruments such as the food frequency questionnaire (FFQ) (denoted by Q(i) for the ith subject). The error can be both systematic and random. The diet record (DR) denoted by R(i) for the ith subject is considered an alloyed go...

journal_title：Statistics in medicine

authors： Rosner B,Michels KB,Chen YH,Day NE

更新日期：2008-08-15 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：:In this paper, we develop a method for the simultaneous estimation of spectral density functions (SDFs) for a collection of stationary time series that share some common features. Due to the similarities among the SDFs, the log-SDF can be represented using a common set of basis functions. The basis shared by the colle...

journal_title：Statistics in medicine

authors： Maadooliat M,Sun Y,Chen T

更新日期：2018-12-30 00:00:00

abstract：:The purpose of this paper is to show that the sensitivity and specificity estimates obtained by 'discrepant analysis' are biased. Discrepant analysis is a widely used technique that attempts to provide estimates of sensitivity and specificity in the presence of an imperfect gold standard. Many researchers have applied...

journal_title：Statistics in medicine

authors： Hadgu A

更新日期：1997-06-30 00:00:00

abstract：:Non-tumor cell-based model systems have recently gained interest in pharmacogenetic research as a hypothesis generating tool. The hypotheses generated from these model systems can be followed up in functional studies, or tested in individuals taking the same investigational agents. The current cellular phenotypes (e.g...

journal_title：Statistics in medicine

authors： Fridley BL,Jenkins G,Schaid DJ,Wang L

更新日期：2009-09-20 00:00:00

abstract：:The randomized clinical trial, LU19, conducted by the Medical Research Council Lung Cancer Working Party, was designed to compare ACE (doxorubicin, cyclophosphamide and etoposide) chemotherapy plus G-CSF (granulocyte colony-stimulating factor) at 2-week intervals versus ACE chemotherapy alone at standard 3-week interv...

journal_title：Statistics in medicine

authors： Qian W,Parmar MK,Sambrook RJ,Fayers PM,Girling DJ,Stephens RJ

更新日期：2000-10-15 00:00:00

abstract：:Bivariate survival data arise, for example, in twin studies and studies of both eyes or ears of the same individual. Often it is of interest to regress the survival times on a set of predictors. In this paper we extend Wei and Tanner's multiple imputation approach for linear regression with univariate censored data to...

journal_title：Statistics in medicine

authors： Pan W,Kooperberg C

更新日期：1999-11-30 00:00:00

abstract：:Conditional power (CP) is the probability that the final study result will be statistically significant, given the data observed thus far and a specific assumption about the pattern of the data to be observed in the remainder of the study, such as assuming the original design effect, or the effect estimated from the c...

journal_title：Statistics in medicine

authors： Lachin JM

更新日期：2005-09-30 00:00:00

abstract：:We compared eight nominal 95 per cent confidence intervals for the ratio of two Poisson parameters, both assumed small, on their true coverage (the probability that the interval includes the ratio of Poisson parameters) and median width. The commonly used log-linear interval, justified by asymptotic considerations, pr...

journal_title：Statistics in medicine

authors： Barker L,Cadwell BL

更新日期：2008-09-10 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：: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：:In medical research, risk difference (RD) and number needed to treat (NNT) measures for survival times have been mainly proposed without consideration of covariates. In this paper, we develop adjusted RD and NNT measures for use in observational studies with survival time outcomes within the framework of the Cox propo...

journal_title：Statistics in medicine

authors： Laubender RP,Bender R

更新日期：2010-03-30 00:00:00

abstract：:Conventional seamless phase 2/3 design with fixed sample size determination (SSD) has gained its popularity in oncology drug development due to attractive features such as significantly shortening the development timeline, minimizing sample size, as well as early decision making. However, this design is not immune to ...

journal_title：Statistics in medicine

authors： Teng Z,Tian Y,Liu Y,Liu G

更新日期：2020-08-15 00:00:00

abstract：:The aim of this paper is to demonstrate the effect of excluding incomplete observations and competing events when calculating cross-sectional measures of NHS waiting times, and to obtain a more accurate estimate of the 'time-to-admission' of those listed on NHS waiting lists using life-table methods. The official 'tim...

journal_title：Statistics in medicine

authors： Armstrong PW

更新日期：2000-08-15 00:00:00