Abstract:
:Clinical trials utilizing predictive biomarkers have become a research focus in personalized medicine. We investigate the effects of biomarker misclassification on the design and analysis of stratified biomarker clinical trials. For a variety of inference problems including marker-treatment interaction in particular, we show that marker misclassification may have profound adverse effects on the coverage of confidence intervals, power of the tests, and required sample sizes. For each inferential problem, we propose methods to adjust for the classification errors.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Liu C,Liu A,Hu J,Yuan V,Halabi Sdoi
10.1002/sim.6164subject
Has Abstractpub_date
2014-08-15 00:00:00pages
3100-13issue
18eissn
0277-6715issn
1097-0258journal_volume
33pub_type
杂志文章abstract::Recently, Wu and Follmann developed summary measures to adjust for informative drop-out in longitudinal studies where drop-out depends on the underlying true value of the response. In this paper we evaluate these procedures in the common situation where drop-out depends on the observed responses. We also discuss vario...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/1097-0258(20010115)20:1<93::aid-sim655>3.0
更新日期:2001-01-15 00:00:00
abstract::Phase II studies in oncology are frequently conducted as two-stage single-arm trials with a binary endpoint indicating tumor response. As a common feature of these designs, the sample sizes of the two stages and the decision rules for the interim and the final analysis have to be pre-specified and adhered to strictly ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6479
更新日期:2015-06-15 00:00:00
abstract::The power prior has been widely used in many applications covering a large number of disciplines. The power prior is intended to be an informative prior constructed from historical data. It has been used in clinical trials, genetics, health care, psychology, environmental health, engineering, economics, and business. ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6728
更新日期:2015-12-10 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::Many epidemiologic investigations are designed to study the effects of multiple exposures. Most of these studies are analysed either by fitting a risk-regression model with all exposures forced in the model, or by using a preliminary-testing algorithm, such as stepwise regression, to produce a smaller model. Research ...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.4780120802
更新日期:1993-04-30 00:00:00
abstract::In behavioral, biomedical, and social-psychological sciences, it is common to encounter latent variables and heterogeneous data. Mixture structural equation models (SEMs) are very useful methods to analyze these kinds of data. Moreover, the presence of missing data, including both missing responses and missing covaria...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3915
更新日期:2010-08-15 00:00:00
abstract::Although statistical methodology is well-developed for comparing diagnostic tests in terms of their sensitivity and specificity, comparative inference about predictive values is not. In this paper, we consider the analysis of studies comparing operating characteristics of two diagnostic tests that are measured on all ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6485
更新日期:2015-07-10 00:00:00
abstract::Vaccination in populations can have several kinds of effects. Establishing that vaccination produces population-level effects beyond the direct effects in the vaccinated individuals can have important consequences for public health policy. Formal methods have been developed for study designs and analysis that can esti...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7392
更新日期:2018-01-30 00:00:00
abstract::Residuals in normal regression are used to assess a model's goodness-of-fit (GOF) and discover directions for improving the model. However, there is a lack of residuals with a characterized reference distribution for censored regression. In this article, we propose to diagnose censored regression with normalized rando...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8852
更新日期:2020-12-13 00:00:00
abstract::Unmeasured confounding is a common concern when researchers attempt to estimate a treatment effect using observational data or randomized studies with nonperfect compliance. To address this concern, instrumental variable methods, such as 2-stage predictor substitution (2SPS) and 2-stage residual inclusion (2SRI), have...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7636
更新日期:2018-05-30 00:00:00
abstract::It is not uncommon for a continuous outcome variable Y to be dichotomized and analysed using logistic regression. Moser and Coombs (Statist. Med. 2004; 23:1843-1860) provide a method for converting the output from a standard linear regression analysis using the original continuous outcome Y to give much more efficient...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3474
更新日期:2009-01-30 00:00:00
abstract::Quality of life (QOL) assessment is a key component of many clinical studies and frequently requires the use of single global summary measures that capture the overall balance of findings from a potentially wide-ranging assessment of QOL issues. We propose and evaluate an irregular multilevel latent variable model sui...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4455
更新日期:2012-05-20 00:00:00
abstract::A mixture model incorporating long-term survivors has been adopted in the field of biostatistics where some individuals may never experience the failure event under study. The surviving fractions may be considered as cured. In most applications, the survival times are assumed to be independent. However, when the survi...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.932
更新日期:2001-06-15 00:00:00
abstract::Biostatisticians have frequently uncritically accepted the measurements provided by their medical colleagues engaged in clinical research. Such measures often involve considerable loss of information. Particularly, unfortunate is the widespread use of the so-called 'responder analysis', which may involve not only a lo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3603
更新日期:2009-11-20 00:00:00
abstract::In longitudinal studies, missing observations occur commonly. It has been well known that biased results could be produced if missingness is not properly handled in the analysis. Authors have developed many methods with the focus on either incomplete response or missing covariate observations, but rarely on both. The ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5536
更新日期:2013-02-28 00:00:00
abstract::We develop a simulation-based procedure for determining the required sample size in binomial regression risk assessment studies when response data are subject to misclassification. A Bayesian average power criterion is used to determine a sample size that provides high probability, averaged over the distribution of po...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3505
更新日期:2009-02-28 00:00:00
abstract::Intraclass correlation coefficient (ICC) estimates must be provided when reporting the results of a cluster randomized trial. This study demonstrates that estimating this parameter with one-way ANOVA and an underlying mixed-effects statistical model leads to biased estimates. The bias depends on the effect size of the...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2260
更新日期:2006-03-30 00:00:00
abstract::In the presence of confounding, the consistency assumption required for identification of causal effects may be violated due to misclassification of the outcome variable. We introduce an inverse probability weighted approach to rebalance covariates across treatment groups while mitigating the influence of differential...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7522
更新日期:2018-02-10 00:00:00
abstract::Patient reported outcome and observer evaluative studies in clinical trials and post-hoc analyses often use instruments that measure responses on ordinal-rating or Likert scales. We propose a flexible distributional approach by modeling the change scores from the baseline to the end of the study using independent beta...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4012
更新日期:2010-10-30 00:00:00
abstract::Motivated by a study about prompt coronary angiography in myocardial infarction, we propose a method to estimate the causal effect of a treatment in two-arm experimental studies with possible noncompliance in both treatment and control arms. We base the method on a causal model for repeated binary outcomes (before and...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5856
更新日期:2013-11-10 00:00:00
abstract::We present some practical extensions and applications of a strategy proposed by Thall, Simon and Estey for designing and monitoring single-arm clinical trials with multiple outcomes. We show by application how the strategy may be applied to construct designs for phase IIA activity trials and phase II equivalence trial...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19980730)17:14<1563::aid-s
更新日期:1998-07-30 00:00:00
abstract::The statistical analysis of spatially correlated data has become an important scientific research topic lately. The analysis of the mortality or morbidity rates observed at different areas may help to decide if people living in certain locations are considered at higher risk than others. Once the statistical model for...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/1097-0258(20000730)19:14<1915::aid-sim503>
更新日期:2000-07-30 00:00:00
abstract::We discuss some of the fundamental concepts underlying the development of frailty and random effects models in survival. One of these fundamental concepts was the idea of a frailty model where each subject has his or her own disposition to failure, their so-called frailty, additional to any effects we wish to quantify...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1259
更新日期:2002-11-15 00:00:00
abstract::The identification of new adverse drug reactions is often tricky. For a given case, the relationship between drug exposure and symptom occurrence is usually questionable. It could be investigated statistically from a series of drug-event association cases with an independence test between the two variables. Analysing ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780100621
更新日期:1991-06-01 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::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
pub_type: 杂志文章
doi:10.1002/sim.7653
更新日期:2018-05-30 00:00:00
abstract::In multiple-record systems estimation it is usually assumed that all registration relate to the same population. In this paper, we develop a method which can be used when the registrations relate to different populations, in the sense that they cover, for example, different time periods or regions. We show that under ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1818
更新日期:2004-07-30 00:00:00
abstract::In standard time-to-event or survival analysis, occurrence times of the event of interest are observed exactly or are right-censored, meaning that it is only known that the event occurred after the last observation time. There are numerous methods available for estimating the survival curve and for testing and estimat...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/(sici)1097-0258(19980130)17:2<219::aid-sim
更新日期:1998-01-30 00:00:00
abstract::Variation in heart disease (HD) mortality rates across census tracts is greater than expected given binomial error and available explanatory variables. We extended an extra-binomial variation model for rates standardized by the direct method. The overdispersion parameter accounted for 36 per cent of the observed varia...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780091009
更新日期:1990-10-01 00:00:00
abstract::In this paper we present a simple method for constructing (1- alpha)100 per cent confidence intervals for binomial proportions with near nominal coverage for all underlying proportion parameters on the unit interval. This new method uses, with a slight modification, the standard normal approximation technique taught i...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2469
更新日期:2006-11-15 00:00:00