Abstract:
:The use of the continual reassessment method (CRM) and other model-based approaches to design Phase I clinical trials has increased owing to the ability of the CRM to identify the maximum tolerated dose better than the 3 + 3 method. However, the CRM can be sensitive to the variance selected for the prior distribution of the model parameter, especially when a small number of patients are enrolled. Although methods have emerged to adaptively select skeletons and to calibrate the prior variance only at the beginning of a trial, there has not been any approach developed to adaptively calibrate the prior variance throughout a trial. We propose three systematic approaches to adaptively calibrate the prior variance during a trial and compare them via simulation with methods proposed to calibrate the variance at the beginning of a trial.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Zhang J,Braun TM,Taylor JMdoi
10.1002/sim.5621subject
Has Abstractpub_date
2013-06-15 00:00:00pages
2221-34issue
13eissn
0277-6715issn
1097-0258journal_volume
32pub_type
杂志文章abstract::We consider the problem of comparing cumulative incidence functions of non-mortality events in the presence of informative coarsening and the competing risk of death. We extend frequentist-based hypothesis tests previously developed for non-informative coarsening and propose a novel Bayesian method based on comparing ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3397
更新日期:2008-12-10 00:00:00
abstract::To study the effect of a mega hydropower dam in southwest Ethiopia on malaria incidence, we have set up a longitudinal study. To gain insight in temporal and spatial aspects, that is, in time (period = year-season combination) and location (village), we need models that account for these effects. The frailty model w...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5752
更新日期:2013-08-15 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
pub_type: 杂志文章
doi:10.1002/sim.4780141105
更新日期:1995-06-15 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
pub_type: 杂志文章
doi:10.1002/sim.6765
更新日期:2016-03-30 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
pub_type: 临床试验,杂志文章
doi:10.1002/1097-0258(20001015)19:19<2657::aid-sim557>
更新日期:2000-10-15 00:00:00
abstract::When data are dichotomous, this paper notes the utility of inverse sampling in establishing equivalence with respect to the risk ratio. This paper develops an exact equivalence test that accounts for the risk ratio under inverse sampling and further discusses the relationship between the exact equivalence test and the...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970815)16:15<1777::aid-s
更新日期:1997-08-15 00:00:00
abstract::The article is motivated by a nephrology study in Taiwan, which enrolled hemodialysis patients who suffered from vascular access thrombosis. After treatment, some patients were cured of thrombosis, while some may experience recurrence of either type (acute or nonacute) of vascular access thrombosis. Our major interest...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8711
更新日期:2020-11-30 00:00:00
abstract::Modelling disease clustering over space and time can be helpful in providing indications of possible exposures and planning corresponding public health practices. Though a considerable number of studies focus on modelling spatio-temporal patterns of disease, most of them do not directly model a spatio-temporal cluster...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2424
更新日期:2006-03-15 00:00:00
abstract::There are many settings in which the distribution of error in a mismeasured covariate varies with the value of another covariate. Take, for example, the case of HIV phylogenetic cluster size, large values of which are an indication of rapid HIV transmission. Researchers wish to find behavioral correlates of HIV phylog...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7289
更新日期:2017-07-30 00:00:00
abstract::Clinical trials, especially the randomized clinical trial, have been and will remain the gold standard for the evaluation of new interventions, including pharmaceuticals, biologics, medical devices, procedures, or behavioral modifications. Despite more than five decades of experience, there are still challenges in the...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5405
更新日期:2012-11-10 00:00:00
abstract::The focus of this paper is the development of a range of cluster detection diagnostics that can be used to assess the degree to which a clustering method recovers the true clustering behaviour of small area data. The diagnostics proposed range from individual region specific diagnostics to neighbourhood diagnostics, a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2401
更新日期:2006-03-15 00:00:00
abstract::Multivariate outcomes measured longitudinally over time are common in medicine, public health, psychology and sociology. The typical (saturated) longitudinal multivariate regression model has a separate set of regression coefficients for each outcome. However, multivariate outcomes are often quite similar and many out...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3589
更新日期:2009-06-15 00:00:00
abstract::A new exponentially weighted moving average (EWMA) control chart well suited for 'online' routine surveillance of medical procedures is introduced. The chart is based on inter-event counts for failures recorded when the failures occur. The method can be used for many types of hospital procedures and activities, such a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2520
更新日期:2007-01-15 00:00:00
abstract::Some clinical trials aim to demonstrate therapeutic equivalence on multiple primary endpoints. For example, therapeutic equivalence studies of agents for the treatment of osteoarthritis use several primary endpoints including investigator's global assessment of disease activity, patient's global assessment of response...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.985
更新日期:2001-11-15 00:00:00
abstract::Meta-analysis is the methodology for combining findings from similar research studies asking the same question. When the question of interest involves multiple outcomes, multivariate meta-analysis is used to synthesize the outcomes simultaneously taking into account the correlation between the outcomes. Likelihood-bas...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4327
更新日期:2011-10-30 00:00:00
abstract::Cox proportional hazard regression model is a popular tool to analyze the relationship between a censored lifetime variable with other relevant factors. The semiparametric Cox model is widely used to study different types of data arising from applied disciplines such as medical science, biology, and reliability studie...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8377
更新日期:2019-11-30 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
pub_type: 临床试验,杂志文章
doi:10.1002/sim.4780010103
更新日期:1982-01-01 00:00:00
abstract::Growth trends in children are often based on cross-sectional studies, in which a sample of the population is investigated at one given point in time. Estimating age-related percentiles in such studies involves fitting data distributions, each of which is specific for one age group, and a subsequent smoothing of the pe...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(20000315)19:5<697::aid-sim
更新日期:2000-03-15 00:00:00
abstract::Assessing the QT prolongation potential of a drug is typically done based on pivotal safety studies called thorough QT studies. Model-based estimation of the drug-induced QT prolongation at the estimated mean maximum drug concentration could increase efficiency over the currently used intersection-union test. However,...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7395
更新日期:2017-10-30 00:00:00
abstract::Toxicity probability interval designs have received increasing attention as a dose-finding method in recent years. In this study, we compared the two-stage, likelihood-based continual reassessment method (CRM), modified toxicity probability interval (mTPI), and the Bayesian optimal interval design (BOIN) in order to e...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7043
更新日期:2017-01-30 00:00:00
abstract::The Rvachev-Baroyan-Longini model is a space-time predictive model of the spread of influenza epidemics. It has been applied to 128 cities of the USSR, and more recently, to forecasting the spread of the pandemic of 1968-1969 throughout 52 large cities. It is a deterministic, mass-action, space and time continuous mod...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780071107
更新日期:1988-11-01 00:00:00
abstract::Inference for randomized clinical trials is generally based on the assumption that outcomes are independently and identically distributed under the null hypothesis. In some trials, particularly in infectious disease, outcomes may be correlated. This may be known in advance (e.g. allowing randomization of family member...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2977
更新日期:2008-03-15 00:00:00
abstract::For many HIV-infected patients, use of antiretroviral therapy (ART) results in a sustained suppression of plasma viral load to undetectable levels. However, due to lack of antigenic stimulation, this may also result in a gradual loss of cell-mediated immune (CMI) responses that help control HIV infection. In concept, ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2555
更新日期:2007-02-10 00:00:00
abstract::Data on alcohol availability and problems in all cities in Los Angeles County were collected from several different sources and linked together to form a Local Alcohol Availability Database (LAAD). The two major purposes of the project are to provide a city-level alcohol availability and alcohol-related problems datab...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780140517
更新日期:1995-03-15 00:00:00
abstract::Repeated cross-sectional samples are common in national surveys of health like the National Health Interview Survey (NHIS). Because population health outcomes generally evolve slowly, pooling data across years can improve the precision of current-year annual estimates of disease prevalence and other health outcomes. P...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3897
更新日期:2011-02-28 00:00:00
abstract::Often multiple outcomes are of interest in each study identified by a systematic review, and in this situation a separate univariate meta-analysis is usually applied to synthesize the evidence for each outcome independently; an alternative approach is a single multivariate meta-analysis model that utilizes any correla...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2524
更新日期:2007-01-15 00:00:00
abstract::A statistical analysis is presented of the intensities of admissions to psychiatric hospitals among women in Denmark who either gave birth to a child in the year 1975 or had an induced abortion in the same year. The models used are time-continuous Markov and semi-Markov processes, and the methods employed include non-...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780050305
更新日期:1986-05-01 00:00:00
abstract::With considerable current interest in longitudinal epidemiologic studies, little is available regarding sample size requirements. This paper considers a method for analysis of longitudinal data, where one compares the mean rates of change for two or more groups, and proposes a statistic for use in determining sample s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780090414
更新日期:1990-04-01 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
pub_type: 杂志文章
doi:10.1002/sim.4780110708
更新日期:1992-05-01 00:00:00
abstract::A decision-theoretic framework is proposed for designing sequential dose-finding trials with multiple outcomes. The optimal strategy is solvable theoretically via backward induction. However, for dose-finding studies involving k doses, the computational complexity is the same as the bandit problem with k-dependent arm...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2322
更新日期:2006-05-30 00:00:00