Abstract:
:There is a single-minded focus on events in survival analysis, and we often ignore longitudinal data that are collected together with the event data. This is due to a lack of methodology but also a result of the artificial distinction between survival and longitudinal data analyses. Understanding the dynamics of such processes is important but has been hampered by a lack of appreciation of the difference between confirmatory and exploratory causal inferences. The latter represents an attempt at elucidating mechanisms by applying mediation analysis to statistical data and will usually be of a more tentative character than a confirmatory analysis. The concept of local independence and the associated graphs are useful. This is related to Granger causality, an important method from econometrics that is generally undervalued by statisticians. This causality concept is different from the counterfactual one since it lacks lacks the intervention aspect. The notion that one can intervene at will in naturally occurring processes, which seems to underly much of modern causal inference, is problematic when studying mediation and mechanisms. It is natural to assume a stochastic process point of view when analyzing dynamic relationships. We present some examples to illustrate this. It is not clear how survival analysis must be developed to handle the complex life-history data that are increasingly being collected today. We give some suggestions.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Aalen OOdoi
10.1002/sim.5324subject
Has Abstractpub_date
2012-08-15 00:00:00pages
1903-17issue
18eissn
0277-6715issn
1097-0258journal_volume
31pub_type
杂志文章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::Methodology for the meta-analysis of individual patient data with survival end-points is proposed. Motivated by questions about the reliance on hazard ratios as summary measures of treatment effects, a parametric approach is considered and percentile ratios are introduced as an alternative to hazard ratios. The genera...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4086
更新日期:2010-12-20 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::Interval-censored data are commonly found in studies of diseases that progress without symptoms, which require clinical evaluation for detection. Several techniques have been suggested with independent assumption. However, the assumption will not be valid if observations come from clusters. Furthermore, when the clust...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4042
更新日期:2010-12-10 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::Existing methods for power analysis for longitudinal study designs are limited in that they do not adequately address random missing data patterns. Although the pattern of missing data can be assessed during data analysis, it is unknown during the design phase of a study. The random nature of the missing data pattern ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2773
更新日期:2007-07-10 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
pub_type: 杂志文章
doi:10.1002/sim.4403
更新日期:2012-01-13 00:00:00
abstract::Many split-mouth trials are characterized by the pairing of site-specific outcome and baseline data within each segment of a subject's mouth. However when the response variable of interest is binary, methods of statistical analysis for this design are not well developed. In this paper we present several analytic appro...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2782
更新日期:2007-08-15 00:00:00
abstract::Meta-analyses of clinical trials are increasingly seeking to go beyond estimating the effect of a treatment and may also aim to investigate the effect of other covariates and how they alter treatment effectiveness. This requires the estimation of treatment-covariate interactions. Meta-regression can be used to estimat...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2768
更新日期:2007-07-10 00:00:00
abstract::Longitudinal biomarker data are often collected in studies, providing important information regarding the probability of an outcome of interest occurring at a future time. With many new and evolving technologies for biomarker discovery, the number of biomarker measurements available for analysis of disease progression...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8687
更新日期:2020-11-20 00:00:00
abstract::Although the literature on contraceptive failure is vast and is expanding rapidly, our understanding of the relative efficacy of methods is quite limited because of defects in the research design and in the analytical tools used by investigators. Errors in the literature range from simple arithmetical mistakes to outr...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.4780100206
更新日期:1991-02-01 00:00:00
abstract::Although population-based molecular association studies are becoming increasingly popular, methodology for the meta-analysis of these studies has been neglected, particularly with regard to two issues: testing Hardy-Weinberg equilibrium (HWE), and pooling results in a manner that reflects a biological model of gene ef...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2010
更新日期:2005-05-15 00:00:00
abstract::Three-arm trials including the experimental treatment, an active reference treatment and a placebo are recommended in the guidelines of the ICH and EMEA/CPMP as a useful approach to the assessment of assay sensitivity. Generally, the acceptable non-inferiority margin Δ has been defined as the maximum clinically irrele...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4099
更新日期:2011-02-10 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
pub_type: 杂志文章
doi:10.1002/sim.5515
更新日期:2012-12-30 00:00:00
abstract::Estimation and group comparison of survival curves are two very common issues in survival analysis. In practice, the Kaplan-Meier estimates of survival functions may be biased due to unbalanced distribution of confounders. Here we develop an adjusted Kaplan-Meier estimator (AKME) to reduce confounding effects using in...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2174
更新日期:2005-10-30 00:00:00
abstract::We have formulated the problem of determining whether there has been an upturn in HIV-1 seroconversion incidence over the first five years of follow-up in the Multicenter AIDS Cohort Study (MACS) as that of locating the minimum of a quadratic regression or examination of two-knot piecewise spline models. Under a quadr...
journal_title:Statistics in medicine
pub_type: 杂志文章,多中心研究
doi:10.1002/sim.4780120207
更新日期:1993-01-30 00:00:00
abstract::This paper discusses the various philosophies that influence the selection of patients for entry into randomized controlled trials. Although a number of different and often competing issues have to be considered depending upon the trial, keeping entry criteria simple, wide and at times even flexible is usually prefera...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780090114
更新日期:1990-01-01 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::Recent studies have indicated variation in the infectivity beta of HIV among heterosexual couples. We represent this heterogeneity by modelling beta as a random variable. Using data on the number of contacts and seroconversion of couples, we fit the model by maximum-likelihood estimation with a beta distribution and a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780080110
更新日期:1989-01-01 00:00:00
abstract::We consider the problem of identifying subgroups of participants in a clinical trial that have enhanced treatment effect. Recursive partitioning methods that recursively partition the covariate space based on some measure of between groups treatment effect difference are popular for such subgroup identification. The m...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8214
更新日期:2019-09-20 00:00:00
abstract::This paper introduces a new spatial scan statistic designed to adjust cluster detection for longitudinal confounding factors indexed in space. The functional-model-adjusted statistic was developed using generalized functional linear models in which longitudinal confounding factors were considered to be functional cova...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8459
更新日期:2020-04-15 00:00:00
abstract::Multi-type recurrent event data arise when two or more different kinds of events may occur repeatedly over a period of observation. The scientific objectives in such settings are often to describe features of the marginal processes and to study the association between the different types of events. Interval-censored m...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1936
更新日期:2005-03-15 00:00:00
abstract::Although the frequentist paradigm has been the predominant approach to clinical trial design since the 1940s, it has several notable limitations. Advancements in computational algorithms and computer hardware have greatly enhanced the alternative Bayesian paradigm. Compared with its frequentist counterpart, the Bayesi...
journal_title:Statistics in medicine
pub_type: 杂志文章,评审
doi:10.1002/sim.5404
更新日期:2012-11-10 00:00:00
abstract::In this paper, we define a modified version τ(b) of Kendall's tau to measure the association in a pair (X,Y) of random variables subject to fixed left censoring due to known lower detection limits. We provide a nonparametric estimator of τ(b) and investigate its asymptotic properties. We then assume an Archimedean cop...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4319
更新日期:2011-11-20 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::Assessing the calibration of methods for estimating the probability of the occurrence of a binary outcome is an important aspect of validating the performance of risk-prediction algorithms. Calibration commonly refers to the agreement between predicted and observed probabilities of the outcome. Graphical methods are a...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8281
更新日期:2019-09-20 00:00:00
abstract::Bioequivalence or interaction trials are commonly studied in crossover design and can be analysed by nonlinear mixed effects models as an alternative to noncompartmental approach. We propose an extension of the population Fisher information matrix in nonlinear mixed effects models to design crossover pharmacokinetic t...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4390
更新日期:2012-05-20 00:00:00
abstract::We analyze data obtained from a study designed to evaluate training effects on the performance of certain motor activities of Parkinson's disease patients. Maximum likelihood methods were used to fit beta-binomial/Poisson regression models tailored to evaluate the effects of training on the numbers of attempted and su...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3303
更新日期:2008-07-30 00:00:00
abstract::In an individually matched case-control study, effects of potential risk factors are ascertained through conditional logistic regression (CLR). Extension of CLR to situations with multiple disease or reference categories has been made through polychotomous CLR and is shown to be more efficient than carrying out separa...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2790
更新日期:2007-07-30 00:00:00
abstract::Random forest is a supervised learning method that combines many classification or regression trees for prediction. Here we describe an extension of the random forest method for building event risk prediction models in survival analysis with competing risks. In case of right-censored data, the event status at the pred...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5775
更新日期:2013-08-15 00:00:00