Abstract:
:An important problem in oncology is comparing chemotherapy (chemo) agents in terms of their effects on survival or progression-free survival time. When the goal is to evaluate individual agents, a difficulty commonly encountered with observational data is that many patients receive a chemo combination including two or more agents. Because agents given in combination may interact, quantifying the contribution of each individual agent to the combination's overall effect is problematic. Still, if on average combinations including a particular agent confer longer survival, then that agent may be considered superior to agents whose combinations confer shorter survival. Motivated by this idea, we propose a definition of individual agent effects based on observational survival data from patients treated with many different chemo combinations. We define an individual agent effect as the average of the effects of the chemo combinations that include the agent. Similarly, we define the effect of each pair of agents as the average of the effects of the combinations including the pair. Under a Bayesian regression model for survival time in which the chemo combination effects follow a hierarchical structure, these definitions are used as a basis for estimating the posterior effects and ranks of the individual agents, and of all pairs of agents. The methods are illustrated by a data set arising from 224 pediatric brain tumor patients treated with over 27 different chemo combinations involving seven chemo agents.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Thall PF,Liu DD,Berrak SG,Wolff JEdoi
10.1002/sim.4249subject
Has Abstractpub_date
2011-07-10 00:00:00pages
1777-94issue
15eissn
0277-6715issn
1097-0258journal_volume
30pub_type
杂志文章,评审abstract::To construct a confidence interval for the mean of a log-normal distribution in small samples, we propose likelihood-based approaches - the signed log-likelihood ratio and modified signed log-likelihood ratio methods. Extensive Monte Carlo simulation results show the advantages of the modified signed log-likelihood ra...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1381
更新日期:2003-06-15 00:00:00
abstract::The principles and historical development of clinical trials are outlined briefly. Some issues that extend the basic philosophy first put forward by Sir Austin Bradford Hill 40 years ago and that will need consideration in the future application of clinical trials are discussed. ...
journal_title:Statistics in medicine
pub_type: 传,历史文章,杂志文章
doi:10.1002/sim.4780010414
更新日期:1982-10-01 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::The relative importance of prognostic factors in regression can be measured either by standardized regression coefficients or by percentages of explained variation in a dependent variable. One advantage of using explained variation is the direct comparability of qualitative prognostic factors with others, or of groups...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780122413
更新日期:1993-12-30 00:00:00
abstract::Disease incidence predictions are useful for a number of administrative and scientific purposes. The simplest ones are made using trend extrapolation, on either an arithmetic or a logarithmic scale. This paper shows how approximate confidence prediction intervals can be calculated for such predictions, both for the to...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780131503
更新日期:1994-08-15 00:00:00
abstract::In many studies, a K degree of freedom large sample chi2 test is used to assess the effect of treatment on a multivariate response, such as an omnibus T2-like test of a difference between two treatment groups in any of K repeated measures. Alternately, a K df chi2 test may be used to test the equality of K+1 groups in...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1637
更新日期:2003-11-15 00:00:00
abstract::We consider the use of the assurance method in clinical trial planning. In the assurance method, which is an alternative to a power calculation, we calculate the probability of a clinical trial resulting in a successful outcome, via eliciting a prior probability distribution about the relevant treatment effect. This i...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5916
更新日期:2014-01-15 00:00:00
abstract::Drop-out often occurs in clinical trials with multiple visits and drop-out is often informative in the sense that the population of patients who dropped out is different from the population of patients who completed the study. To handle data with informative drop-out, an intention-to-treat analysis, which evaluates tr...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1519
更新日期:2003-08-15 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
pub_type: 杂志文章
doi:10.1002/sim.8066
更新日期:2019-04-30 00:00:00
abstract::In time-to-event analysis, the traditional summary measures have been based on the hazard function, survival function, quantile event time, restricted mean event time, and residual lifetime. Under competing risks, furthermore, typical summary measures have been the cause-specific hazard function and cumulative inciden...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8871
更新日期:2021-01-06 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
pub_type: 杂志文章
doi:10.1002/sim.4204
更新日期:2011-06-30 00:00:00
abstract::Log-linear models for capture-recapture type data are widely used for estimating sizes of populations. Log-linear methods model conditional interactions between the sources. Often, however, the marginal associations are more appropriate and easier for the practitioner to conceptualize. Analyses here of previously publ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19980115)17:1<69::aid-sim7
更新日期:1998-01-15 00:00:00
abstract::The two-period cross-over design with two sequences of drug administration is a standard experimental design when bioequivalence of one test formulation is to be assessed in comparison with a reference formulation. Previously, an approach based on Fieller's confidence interval has been presented for the assessment of ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19971015)16:19<2229::aid-s
更新日期:1997-10-15 00:00:00
abstract::Biomedical studies often measure variables with error. Examples in the literature include investigation of the association between the change in some outcome variable (blood pressure, cholesterol level etc.) and a set of explanatory variables (age, smoking status etc.). Typically, one fits linear regression models to ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19981130)17:22<2597::aid-s
更新日期:1998-11-30 00:00:00
abstract::Equal allocation of patients to treatment in a randomized clinical trial may have disadvantages ethically if the new treatment is believed to be at least as beneficial as the standard treatment. Others have considered, in a non-sequential setting, unbalanced randomized designs which allocate fewer patients to the pote...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780010309
更新日期:1982-07-01 00:00:00
abstract::The National Children's Study is a national household probability sample designed to identify 100,000 children at birth and follow the sampled children for 21 years. Data from the study will support examining numerous hypotheses concerning genetic and environmental effects on the health and development of children. Th...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3891
更新日期:2010-06-15 00:00:00
abstract::Girardeau, Ravaud and Donner in 2008 presented a formula for sample size calculations for cluster randomised crossover trials, when the intracluster correlation coefficient, interperiod correlation coefficient and mean cluster size are specified in advance. However, in many randomised trials, the number of clusters is...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8191
更新日期:2019-08-15 00:00:00
abstract::We present a new approach to training back-propagation artificial neural nets (BP-ANN) based on regularization and cross-validation and on initialization by a logistic regression (LR) model. The new approach is expected to produce a BP-ANN predictor at least as good as the LR-based one. We have applied the approach to...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1107
更新日期:2002-05-15 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
pub_type: 杂志文章
doi:10.1002/1097-0258(20001230)19:24<3497::aid-sim830>
更新日期:2000-12-30 00:00:00
abstract::The continual reassessment method (CRM) is an adaptive design for Phase I trials whose operating characteristics, including appropriate sample size, probability of correctly identifying the maximum tolerated dose, and the expected proportion of participants assigned to each dose, can only be determined via simulation....
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8746
更新日期:2020-09-16 00:00:00
abstract::A good measure of field vaccine efficacy should evaluate the direct protective effect of vaccination on the person who receives the vaccine. The conventional estimator for vaccine efficacy depends on population level factors that are either unrelated or indirectly related to the direct biological action of the vaccine...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780120309
更新日期:1993-02-01 00:00:00
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
pub_type: 杂志文章
doi:10.1002/sim.4780100908
更新日期:1991-09-01 00:00:00
abstract::We examine the properties of several tests for goodness-of-fit for multinomial logistic regression. One test is based on a strategy of sorting the observations according to the complement of the estimated probability for the reference outcome category and then grouping the subjects into g equal-sized groups. A g x c c...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3202
更新日期:2008-09-20 00:00:00
abstract::The setting of a quarantine time for an emerging infectious disease will depend on current knowledge concerning incubation times. Methods for the analysis of information on incubation times are investigated with a particular focus on inference regarding a possible maximum incubation time, after which an exposed indivi...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2206
更新日期:2005-11-30 00:00:00
abstract::Funnel plots are widely used to visualize grouped data, for example, in institutional comparison. This paper extends the concept to a multi-level setting, displaying one level at a time, adjusted for the other levels, as well as for covariates at all levels. These level-adjusted funnel plots are based on a Markov chai...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5677
更新日期:2014-09-20 00:00:00
abstract::Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8813
更新日期:2021-02-20 00:00:00
abstract::In the comparison of two or more treatment groups to a control group, consider a study with non-decreasing repeated measurements of the same characteristic taken over a common set of time points for each subject. Based on the vector of possibly incomplete responses from each subject, this paper considers asymptoticall...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(SICI)1097-0258(19961215)15:23<2509::AID-S
更新日期:1996-12-15 00:00:00
abstract::In clinical trials, treatment comparisons are often performed by models that incorporate important prognostic factors. Since these models require complete covariate information on all patients, statisticians frequently resort to complete case analysis or to omission of an important covariate. A probability imputation ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780090707
更新日期:1990-07-01 00:00:00
abstract::Hospital length of stay (LOS) is an important measure of healthcare utilization and is generally positively skewed and heterogeneous. We fit a Coxian phase-type distribution to LOS and identify the hidden states of the underlying latent homogeneous Markov model. We demonstrate that selecting an appropriate number of p...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4490
更新日期:2012-06-30 00:00:00
abstract::Slow recruitment in clinical trials leads to increased costs and resource utilization, which includes both the clinic staff and patient volunteers. Careful planning and monitoring of the accrual process can prevent the unnecessary loss of these resources. We propose two hierarchical extensions to the existing Bayesian...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6359
更新日期:2015-02-20 00:00:00