A new approach to designing phase I-II cancer trials for cytotoxic chemotherapies.

abstract：:We consider methods for selecting the joint specification of the mean and variance functions in statistical models for rates or counts. Based on analyses of diagnosis-specific hospital discharge rates in Michigan, we show that a Poisson model with an extra variance component for the systematic variation is superior to...

journal_title：Statistics in medicine

authors： Wolfe RA,Petroni GR,McLaughlin CG,McMahon LF Jr

更新日期：1991-09-01 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：:For the analysis of time to event data in contraceptive studies when individuals are subject to competing causes for discontinuation, some authors have recently advocated the use of the cumulative incidence rate as a more appropriate measure to summarize data than the complement of the Kaplan-Meier estimate of discont...

journal_title：Statistics in medicine

authors： Farley TM,Ali MM,Slaymaker E

更新日期：2001-12-15 00:00:00

abstract：:We examine the use of randomization-based inference for analyzing multiarmed randomized clinical trials, including the application of conditional randomization tests to multiple comparisons. The view is taken that the linkage of the statistical test to the experimental design (randomization procedure) should be recogn...

journal_title：Statistics in medicine

authors： Wang Y,Rosenberger WF,Uschner D

更新日期：2020-02-20 00:00:00

abstract：:The use of data from multiple studies or centers for the validation of a clinical test or a multivariable prediction model allows researchers to investigate the test's/model's performance in multiple settings and populations. Recently, meta-analytic techniques have been proposed to summarize discrimination and calibra...

journal_title：Statistics in medicine

authors： Wynants L,Riley RD,Timmerman D,Van Calster B

更新日期：2018-05-30 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：:For time-to-event outcomes, a rich literature exists on the bias introduced by covariate measurement error in regression models, such as the Cox model, and methods of analysis to address this bias. By comparison, less attention has been given to understanding the impact or addressing errors in the failure time outcome...

journal_title：Statistics in medicine

authors： Oh EJ,Shepherd BE,Lumley T,Shaw PA

更新日期：2018-04-15 00:00:00

abstract：:Although estimation and confidence intervals have become popular alternatives to hypothesis testing and p-values, statisticians usually determine sample sizes for randomized clinical trials by controlling the power of a statistical test at an appropriate alternative, even those statisticians who recommend the use of c...

journal_title：Statistics in medicine

authors： Bristol DR

更新日期：1989-07-01 00:00:00

abstract：:In the analysis of composite endpoints in a clinical trial, time to first event analysis techniques such as the logrank test and Cox proportional hazard test do not take into account the multiplicity, importance, and the severity of events in the composite endpoint. Several generalized pairwise comparison analysis met...

journal_title：Statistics in medicine

authors： Verbeeck J,Spitzer E,de Vries T,van Es GA,Anderson WN,Van Mieghem NM,Leon MB,Molenberghs G,Tijssen J

更新日期：2019-12-30 00:00:00

abstract：:A common but not necessarily logical requirement in drug development is that a 'limit of quantitation' be set for chemical assays and that observations that fall below the limit should not be treated as real data but should be labelled as below the limit and set aside for special treatment. We examine five of seven ap...

journal_title：Statistics in medicine

authors： Senn S,Holford N,Hockey H

更新日期：2012-12-30 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：:Austin Bradford Hill was once a friend to The Lancet, but, as occasionally happens, friends fall out. The great legacy of his association with the journal, however, was Principles of Medical Statistics. As each edition was succeeded by another--the first in 1937, the last in 1991--he seemed to shift his view about the...

journal_title：Statistics in medicine

authors： Horton R

更新日期：2000-12-15 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：:Ewell and Ibrahim derived the large sample distribution of the logrank statistic under general local alternatives. Their asymptotic results enable us to extend several group sequential designs which allow for early stopping in favour of the null hypothesis to the setting in which the cure rate model is appropriate. In...

journal_title：Statistics in medicine

authors： Patricia Bernardo MV,Ibrahim JG

更新日期：2000-11-30 00:00:00

abstract：:In a medical study we are often interested in graphically displaying the relationship between continuous variables and clinical events indicating disease progression. Often, it is reasonable to make the minimal assumption that the risk of progression is an arbitrary monotone function of the continuous variable. Someti...

journal_title：Statistics in medicine

authors： Ancukiewicz M,Finkelstein DM,Schoenfeld DA

更新日期：2003-10-30 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：:It is valuable in many studies to assess both intrarater and interrater agreement. Most measures of intrarater agreement do not adjust for unequal estimates of prevalence between the separate rating occasions for a given rater and measures of interrater agreement typically ignore data from the second set of assessment...

journal_title：Statistics in medicine

authors： Lester Kirchner H,Lemke JH

更新日期：2002-06-30 00:00:00

abstract：:Effective statistical help to biological and medical research demands thorough involvement of the statistician. The breadth of his activities can be illustrated by considering the questions he needs to discuss with his scientific colleagues in the course of planning a comparative experiment. The paper presents and com...

journal_title：Statistics in medicine

authors： Finney DJ

更新日期：1982-01-01 00:00:00

abstract：:We review two recent trends: the emergence of evidence-based medicine and the growing use of Bayesian statistics in medical applications. Evidence-based medicine requires an integrated assessment of the available evidence, and associated uncertainty, but there is also an emphasis on decision-making, for individual pat...

journal_title：Statistics in medicine

authors： Ashby D,Smith AF

更新日期：2000-12-15 00:00:00

abstract：:Although radical changes in drug regulation are rare (e.g., the Federal Food, Drug and Cosmetic Act of 1938 and the 1962 amendment to the Act creating an effectiveness requirement), regulations and guidance do evolve significantly in the face of new problems and accumulating experience. Recent changes have been driven...

journal_title：Statistics in medicine

authors： Temple R

更新日期：2002-10-15 00:00:00

abstract：:The intraclass correlation coefficient rho plays a key role in the design of cluster randomized trials. Estimates of rho obtained from previous cluster trials and used to inform sample size calculation in planned trials may be imprecise due to the typically small numbers of clusters in such studies. It may be useful t...

journal_title：Statistics in medicine

authors： Ukoumunne OC,Davison AC,Gulliford MC,Chinn S

更新日期：2003-12-30 00:00:00

abstract：:We propose a joint modeling approach to investigating the observed and latent risk factors of mixed types of outcomes. The proposed model comprises three parts. The first part is an exploratory factor analysis model that summarizes latent factors through multiple observed variables. The second part is a proportional h...

journal_title：Statistics in medicine

authors： Pan D,Wei Y,Song X

更新日期：2020-12-09 00:00:00

abstract：:Longitudinal studies are often concerned with estimating the recurrence rate of a non-fatal event. In many cases, only the total number of events occurring during successive time intervals is known. We compared a mixed Poisson-gamma regression method proposed by Thall and a quasi-likelihood method proposed by Zeger an...

journal_title：Statistics in medicine

authors： Stukel TA

更新日期：1993-07-30 00:00:00

abstract：:We develop reinforcement learning trials for discovering individualized treatment regimens for life-threatening diseases such as cancer. A temporal-difference learning method called Q-learning is utilized that involves learning an optimal policy from a single training set of finite longitudinal patient trajectories. A...

journal_title：Statistics in medicine

authors： Zhao Y,Kosorok MR,Zeng D

更新日期：2009-11-20 00:00:00

abstract：:This paper models monthly AIDS diagnosis counts in terms of smooth secular trend, calendar month effects, and the number of workdays per month. A parameterization of month effects allows separation of true seasonal effects from a linear trend over the calendar year and an arbitrary June effect. There is strong evidenc...

journal_title：Statistics in medicine

authors： Bacchetti P

更新日期：1994-10-15 00:00:00

abstract：:Statistical methods for testing and interval estimation of the ratio of marginal probabilities in the matched-pair setting are considered in this paper. We are especially interested in the situation where the null value is not one, as in one-sided equivalence trials. We propose a Fieller-type statistic based on constr...

journal_title：Statistics in medicine

authors： Nam JM,Blackwelder WC

更新日期：2002-03-15 00:00:00

abstract：:We describe how trends in the vaccination coverage at 19 months of age vary by race/ethnicity; explore the extent to which data required to evaluate a child's up-to-date vaccination status is missing as a result of the scattering of vaccination records among many vaccination providers; evaluate how the prevalence of t...

journal_title：Statistics in medicine

authors： Smith PJ,Stevenson J

更新日期：2008-09-10 00:00:00

abstract：:In the last decade or so, pharmaceutical drug development activities in the area of new antibacterial drugs for treating serious bacterial diseases have declined, and at the same time, there are worries that the increased prevalence of antibiotic-resistant bacterial infections, especially the increase in drug-resistan...

journal_title：Statistics in medicine

authors： Huque MF,Valappil T,Soon GG

更新日期：2014-11-10 00:00:00

abstract：:There has been substantial statistical literature in the last several decades on assessing agreement, and coverage probability approach was selected as a preferred index for assessing and improving measurement agreement in a core laboratory setting. With this approach, a satisfactory agreement is based on pre-specifie...

journal_title：Statistics in medicine

authors： Barnhart HX

更新日期：2016-08-15 00:00:00

abstract：:Suppose that two graders classify all eyes in a sample of patients for the presence or absence of a specified abnormality. In the statistical analysis of the data, possible correlation between the observations in the right and left eyes should be taken into account. Recently, general methods have been developed to ana...

journal_title：Statistics in medicine

authors： Schouten HJ

更新日期：1993-12-15 00:00:00