Abstract：
:This work presents a brief overview of Markov models in cancer screening evaluation and focuses on two specific models. A three-state model was first proposed to estimate jointly the sensitivity of the screening procedure and the average duration in the preclinical phase, i.e. the period when the cancer is asymptomatic but detectable by screening. A five-state model, incorporating lymph node involvement as a prognostic factor, was later proposed combined with a survival analysis to predict the mortality reduction associated with screening. The strengths and limitations of these two models are illustrated using data from French breast cancer service screening programmes. The three-state model is a useful frame but parameter estimates should be interpreted with caution. They are highly correlated and depend heavily on the parametric assumptions of the model. Our results pointed out a serious limitation to the five-state model, due to implicit assumptions which are not always verified. Although it may still be useful, there is a need for more flexible models. Over-diagnosis is an important issue for both models and induces bias in parameter estimates. It can be addressed by adding a non-progressive state, but this may provide an uncertain estimation of over-diagnosis. When the primary goal is to avoid bias, rather than to estimate over-diagnosis, it may be more appropriate to correct for over-diagnosis assuming different levels in a sensitivity analysis. This would be particularly relevant in a perspective of mortality reduction estimation.
journal_name
Stat Methods Med Resjournal_title
Statistical methods in medical researchauthors
Uhry Z,Hédelin G,Colonna M,Asselain B,Arveux P,Rogel A,Exbrayat C,Guldenfels C,Courtial I,Soler-Michel P,Molinié F,Eilstein D,Duffy SWdoi
10.1177/0962280209359848subject
Has Abstractpub_date
2010-10-01 00:00:00pages
463-86issue
5eissn
0962-2802issn
1477-0334pii
0962280209359848journal_volume
19pub_type
杂志文章,评审abstract：:Joint models for recurrent and terminal events have not been yet developed for clustered data. The goals of our study are to develop a statistical framework for modelling clustered recurrent and terminal events and to perform dynamic predictions of the terminal event in family studies. We propose a joint nested frailt...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280219863076
更新日期：2020-05-01 00:00:00
abstract：:In many longitudinal studies, evaluating the effect of a binary or continuous predictor variable on the rate of change of the outcome, i.e. slope, is often of primary interest. Sample size determination of these studies, however, is complicated by the expectation that missing data will occur due to missed visits, earl...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280212437452
更新日期：2015-12-01 00:00:00
abstract：:Methods for the analysis of reliability of ordered categorical scales are discussed, focussing on the limitation of the single summary-weighted kappa coefficients. A symmetric matrix of kappa-type coefficients is suggested as an alternative. The method is proposed as being suitable for ordinal scale where there is no ...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1191/0962280205sm413oa
更新日期：2005-10-01 00:00:00
abstract：:Age-period-cohort models are a popular tool for studying population-level rates; for example, trends in cancer incidence and mortality. Age-period-cohort models decompose observed trends into age effects that correlate with natural history, period effects that reveal factors impacting all ages simultaneously (e.g. inn...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280217713033
更新日期：2019-01-01 00:00:00
abstract：:The analysis of fecundity data is challenging and requires consideration of both highly timed and interrelated biologic processes in the context of essential behaviors such as sexual intercourse during the fertile window. Understanding human fecundity is further complicated by presence of a sterile population, i.e. co...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280212438646
更新日期：2016-02-01 00:00:00
abstract：:Cluster randomized and multicentre trials evaluate the effect of a treatment on persons nested within clusters, for instance patients within clinics or pupils within schools. Although equal sample sizes per cluster are generally optimal for parameter estimation, they are rarely feasible. This paper addresses the relat...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280206079018
更新日期：2008-08-01 00:00:00
abstract：:Adherence to medication is often measured as a continuous outcome but analyzed as a dichotomous outcome due to lack of appropriate tools. In this paper, we illustrate the use of the temporal kernel canonical correlation analysis (tkCCA) as a method to analyze adherence measurements and symptom levels on a continuous s...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280215598805
更新日期：2017-10-01 00:00:00
abstract：:Many statistical studies report p-values for inferential purposes. In several scenarios, the stochastic aspect of p-values is neglected, which may contribute to drawing wrong conclusions in real data experiments. The stochastic nature of p-values makes their use to examine the performance of given testing procedures o...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280217704451
更新日期：2018-12-01 00:00:00
abstract：:Recently, the joint analysis of longitudinal and survival data has been an active research area. Most joint models focus on survival data with only one type of failure. The research on joint modeling of longitudinal and competing risks survival data is sparse. Even so, many joint models for this type of data assume pa...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280215597939
更新日期：2017-10-01 00:00:00
abstract：:Longitudinal zero-inflated count data are encountered frequently in substance-use research when assessing the effects of covariates and risk factors on outcomes. Often, both the time to a terminal event such as death or dropout and repeated measure count responses are collected for each subject. In this setting, the l...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280216659312
更新日期：2018-04-01 00:00:00
abstract：:Publication bias frequently appears in meta-analyses when the included studies' results (e.g., p-values) influence the studies' publication processes. Some unfavorable studies may be suppressed from publication, so the meta-analytic results may be biased toward an artificially favorable direction. Many statistical tes...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280220910172
更新日期：2020-10-01 00:00:00
abstract：:Colorectal cancer is the second leading cause of death from cancer in the United States. To facilitate the efficiency of colorectal cancer screening, there is a need to stratify risk for colorectal cancer among the 90% of US residents who are considered "average risk." In this article, we investigate such risk stratif...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280213497432
更新日期：2016-08-01 00:00:00
abstract：:Trials run in either rare diseases, such as rare cancers, or rare sub-populations of common diseases are challenging in terms of identifying, recruiting and treating sufficient patients in a sensible period. Treatments for rare diseases are often designed for other disease areas and then later proposed as possible tre...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280216662070
更新日期：2018-05-01 00:00:00
abstract：:In medical experiments with the objective of testing the equality of two means, data are often partially paired by design or because of missing data. The partially paired data represent a combination of paired and unpaired observations. In this article, we review and compare nine methods for analyzing partially paired...
journal_title：Statistical methods in medical research
pub_type： 杂志文章,评审
doi：10.1177/0962280215577111
更新日期：2017-06-01 00:00:00
abstract：:Accelerated failure time model is a popular model to analyze censored time-to-event data. Analysis of this model without assuming any parametric distribution for the model error is challenging, and the model complexity is enhanced in the presence of large number of covariates. We developed a nonparametric Bayesian met...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280215626947
更新日期：2018-04-01 00:00:00
abstract：:It is a common practice to analyze longitudinal data frequently arisen in medical studies using various mixed-effects models in the literature. However, the following issues may standout in longitudinal data analysis: (i) In clinical practice, the profile of each subject's response from a longitudinal study may follow...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280214544207
更新日期：2017-02-01 00:00:00
abstract：:In many experiments and especially in translational and preclinical research, sample sizes are (very) small. In addition, data designs are often high dimensional, i.e. more dependent than independent replications of the trial are observed. The present paper discusses the applicability of max t-test-type statistics (mu...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280220970228
更新日期：2020-11-24 00:00:00
abstract：:The maximal procedure is a restricted randomization method that maximizes the number of feasible allocation sequences under the constraints of the maximum tolerated imbalance and the allocation sequence length. It assigns an equal probability to all feasible sequences. However, its implementation is not easy due to th...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280216677107
更新日期：2018-07-01 00:00:00
abstract：:Dependent censoring arises in biomedical studies when the survival outcome of interest is censored by competing risks. In survival data with microarray gene expressions, gene selection based on the univariate Cox regression analyses has been used extensively in medical research, which however, is only valid under the ...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280214533378
更新日期：2016-12-01 00:00:00
abstract：:The estimation of population parameters using complex survey data requires careful statistical modelling to account for the design features. This is further complicated by unit and item nonresponse for which a number of methods have been developed in order to reduce estimation bias. In this paper, we address some issu...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280213484401
更新日期：2016-08-01 00:00:00
abstract：:Immunotherapy, gene therapy or adoptive cell therapies, such as the chimeric antigen receptor+ T-cell therapies, have demonstrated promising therapeutic effects in oncology patients. We consider statistical designs for dose-finding adoptive cell therapy trials, in which the monotonic dose-response relationship assumed...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280220977009
更新日期：2020-12-16 00:00:00
abstract：:In recent years, there has been a prominent discussion in the literature about the potential for overestimation of the treatment effect when a clinical trial stops at an interim analysis due to the experimental treatment showing a benefit over the control. However, there has been much less attention paid to the conver...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280218795320
更新日期：2019-10-01 00:00:00
abstract：:One of the main advantages of Bayesian analyses of clinical trials is their ability to formally incorporate skepticism about large treatment effects through the use of informative priors. We conducted a simulation study to assess the performance of informative normal, Student- t, and beta distributions in estimating r...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280215620828
更新日期：2018-01-01 00:00:00
abstract：:In the clinical development of some new infectious disease drugs, early clinical pharmacology trials may predict with high confidence that the efficacious doses are well below the range of the safety margin. In this case, a dose-ranging study may be unnecessary after a proof-of-concept (PoC) study testing the highest ...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280218807950
更新日期：2019-12-01 00:00:00
abstract：:Conditional two-part random-effects models have been proposed for the analysis of healthcare cost panel data that contain both zero costs from the non-users of healthcare facilities and positive costs from the users. These models have been extended to accommodate more flexible data structures when using the generalize...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280217690770
更新日期：2018-10-01 00:00:00
abstract：:Stochastic transmission dynamic models are needed to quantify the uncertainty in estimates and predictions during outbreaks of infectious diseases. We previously developed a calibration method for stochastic epidemic compartmental models, called Multiple Shooting for Stochastic Systems (MSS), and demonstrated its comp...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280218805780
更新日期：2019-12-01 00:00:00
abstract：:To project national hepatitis C virus (HCV) burden, unbiased estimation of HCV progression to liver cirrhosis is required for the whole community of HCV-infected individuals. However, widely varying estimates of progression rates to cirrhosis have been produced. This disparity is partly associated with the statistical...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280208094688
更新日期：2009-06-01 00:00:00
abstract：:We propose a hierarchical Bayesian methodology to model spatially or spatio-temporal clustered survival data with possibility of cure. A flexible continuous transformation class of survival curves indexed by a single parameter is used. This transformation model is a larger class of models containing two special cases ...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280212445658
更新日期：2016-02-01 00:00:00
abstract：:Guarding against false positive selections is important in many applications. We discuss methods based on subsampling and sample splitting for controlling the expected number of false positives and assigning p-values. They are generic and especially useful for high-dimensional settings. We review encouraging results f...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280211428371
更新日期：2013-10-01 00:00:00
abstract：:The Cochran-Armitage (CA) test is commonly used in both epidemiology and genetics to test for linear trend in two-way tables with a binary outcome. There has been increasing interest in the power and size of the test and in determination of sample size, especially when there is potential misclassification in the 'expo...
journal_title：Statistical methods in medical research
pub_type： 杂志文章
doi：10.1177/0962280211406424
更新日期：2014-06-01 00:00:00
