Abstract:
:In this paper, we discuss family-based association test (FBATs) relating genetic data to survival and time-to-onset data. We show how the standard logrank and Wilcoxon statistics can be used with family data to develop tests of association. We prove that the FBAT-logrank approach can be identical to the proportional hazard approach discussed in Mokliatchouk et al. (2000). Further, using simulation studies, we compare the power of the logrank, Wilcoxon and an approach developed for censored exponential data (Euro J Hum Gen 2001; 9:301-306). Based on the results of the simulation study, we suggest rules of thumb about which statistics to use in a given situation. An application of all three tests to an Alzheimer study illustrates the practical relevance of our discussion.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Lange C,Blacker D,Laird NMdoi
10.1002/sim.1707subject
Has Abstractpub_date
2004-01-30 00:00:00pages
179-89issue
2eissn
0277-6715issn
1097-0258journal_volume
23pub_type
杂志文章abstract::The percentile-finding experimental design known variously as 'forced-choice fixed-staircase', 'geometric up-and-down' or 'k-in-a-row' (KR) was introduced by Wetherill four decades ago. To date, KR has been by far the most widely used up-and-down (U&D) design for estimating non-median percentiles; it is implemented mo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3590
更新日期:2009-06-15 00:00:00
abstract::Multivariate random length data occur when we observe multiple measurements of a quantitative variable and the variable number of these measurements is also an observed outcome for each experimental unit. For example, for a patient with coronary artery disease, we may observe a number of lesions in that patient's coro...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990130)18:2<199::aid-sim
更新日期:1999-01-30 00:00:00
abstract::This article concerns construction of a confidence surface for tangential slopes of the dose-response surface of a combination therapy to identify where response increases as a function of drug dosage. This approach extends to the assessment of the effectiveness of the combination therapy. ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780110513
更新日期:1992-03-01 00:00:00
abstract::In many biomedical and epidemiological studies, data are often clustered due to longitudinal follow up or repeated sampling. While in some clustered data the cluster size is pre-determined, in others it may be correlated with the outcome of subunits, resulting in informative cluster size. When the cluster size is info...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4239
更新日期:2011-07-10 00:00:00
abstract::This paper reviews Bayesian strategies for monitoring clinical trial data. It focuses on a Bayesian stochastic curtailment method based on the predictive probability of observing a clinically significant outcome at the scheduled end of the study given the observed data. The proposed method is applied to derive efficac...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2204
更新日期:2006-07-15 00:00:00
abstract::We develop reinforcement learning trials for discovering individualized treatment regimens for life-threatening diseases such as cancer. A temporal-difference learning method called Q-learning is utilized that involves learning an optimal policy from a single training set of finite longitudinal patient trajectories. A...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3720
更新日期:2009-11-20 00:00:00
abstract::Down's syndrome screening is currently carried out using a combination of biochemical markers measured in maternal serum samples; these include MSAFP, Total hCG, uE3 and Free beta-hCG. Recently a number of papers have compared the effectiveness of different combinations of these markers. Some recommend MSAFP, Total hC...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19970715)16:13<1481::aid-s
更新日期:1997-07-15 00:00:00
abstract::The aim of this paper is to demonstrate the effect of excluding incomplete observations and competing events when calculating cross-sectional measures of NHS waiting times, and to obtain a more accurate estimate of the 'time-to-admission' of those listed on NHS waiting lists using life-table methods. The official 'tim...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/1097-0258(20000815)19:15<2037::aid-sim606>
更新日期:2000-08-15 00:00:00
abstract::Quantifying socioeconomic disparities and understanding the roots of inequalities are growing topics in cancer research. However, socioeconomic differences are challenging to investigate mainly due to the lack of accurate data at individual-level, while aggregate indicators are only partially informative. We implement...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8392
更新日期:2020-01-15 00:00:00
abstract::Interpretation of the Mantoux test for tuberculous infection can be complicated by cross-reactions caused by infection with non-specific mycobacteria. Thus, the distribution of positive indurations is a mixture of two distributions. To estimate tuberculous infection prevalence, the marginal distribution of indurations...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.745
更新日期:2001-04-15 00:00:00
abstract::This paper describes the Stoke Index which has been designed to give a global measure of disease activity in rheumatoid arthritis. The index is based on two objective laboratory measurements, one subjective and two semi-objective clinical measurements, chosen from 13 measurements using clinical judgement. Variable sel...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780121206
更新日期:1993-06-30 00:00:00
abstract::In this paper, we consider pooling schemes in which samples are to be tested in two-stages. We show that when batch size is limited as well as pool size, selection schemes tend to be more efficient and flexible. Formulae for the efficiencies of square arrays in all dimensions and for all selection schemes are given in...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3965
更新日期:2010-09-20 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::Reference ranges which take time (such as age) into account are often required in medicine, but simple, systematic and efficient statistical methods for constructing them are lacking. A method is described which is based on low order polynomial curves (linear, quadratic or occasionally cubic), together with guidelines...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章,多中心研究
doi:10.1002/sim.4780100502
更新日期:1991-05-01 00:00:00
abstract::When interpreting screening mammograms radiologists decide whether suspicious abnormalities exist that warrant the recall of the patient for further testing. Previous work has found significant differences in interpretation among radiologists; their false-positive and false-negative rates have been shown to vary widel...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2633
更新日期:2007-03-30 00:00:00
abstract::Paired data from matched case-control studies are commonly used to estimate the association between the exposure to a risk factor and the occurrence of a disease. The odds ratio is typically used to quantify this association. Difficulties in estimating the true odds ratio with matched pairs arise, however, when the ex...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5355
更新日期:2012-11-30 00:00:00
abstract::Sample size planning should reflect the primary objective of a trial. If the primary objective is prediction, the sample size determination should focus on prediction accuracy instead of power. We present formulas for the determination of training set sample size for survival prediction. Sample size is chosen to contr...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5550
更新日期:2013-02-28 00:00:00
abstract::In this article we develop flexible regression models in two respects to evaluate the influence of the covariate variables on the mixed Poisson and continuous responses and to evaluate how the correlation between Poisson response and continuous response changes over time. A scenario for dealing with regression models ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2776
更新日期:2007-09-10 00:00:00
abstract::The log-rank test is the most powerful non-parametric test for detecting a proportional hazards alternative and thus is the most commonly used testing procedure for comparing time-to-event distributions between different treatments in clinical trials. When the log-rank test is used for the primary data analysis, the s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3501
更新日期:2009-02-28 00:00:00
abstract::Between-community variance or community-by-time variance is one of the key factors driving the cost of conducting group randomized trials, which are often very expensive. We investigated empirically whether between-community variance could be reduced by controlling individual- and/or community-level covariates and ide...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990315)18:5<539::aid-sim
更新日期:1999-03-15 00:00:00
abstract::For bivariate meta-analysis of diagnostic studies, likelihood approaches are very popular. However, they often run into numerical problems with possible non-convergence. In addition, the construction of confidence intervals is controversial. Bayesian methods based on Markov chain Monte Carlo (MCMC) sampling could be u...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3858
更新日期:2010-05-30 00:00:00
abstract::When the event time of interest depends on the censoring time, conventional two-sample test methods, such as the log-rank and Wilcoxon tests, can produce an invalid test result. We extend our previous work on estimation using auxiliary variables to adjust for dependent censoring via multiple imputation, to the compari...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3480
更新日期:2009-02-01 00:00:00
abstract::In this paper, an approach to estimating the cumulative mean function for history process with time dependent covariates and right censored time-to-event variable is developed using the combined technique of joint modeling and inverse probability weighting method. The consistency of proposed estimator is derived. Theo...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6998
更新日期:2016-11-10 00:00:00
abstract::This paper presents a mixture model which combines features of the usual Cox proportional hazards model with those of a class of models, known as mixtures-of-experts. The resulting model is more flexible than the usual Cox model in the sense that the log hazard ratio is allowed to vary non-linearly as a function of th...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990515)18:9<1119::aid-si
更新日期:1999-05-15 00:00:00
abstract::Case-control studies are prone to low power for testing gene-environment interactions (GXE) given the need for a sufficient number of individuals on each strata of disease, gene, and environment. We propose a new study design to increase power by strategically pooling biospecimens. Pooling biospecimens allows us to in...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5357
更新日期:2012-11-20 00:00:00
abstract::Cancer immunotherapy trials have two special features: a delayed treatment effect and a cure rate. Both features violate the proportional hazard model assumption and ignoring either one of the two features in an immunotherapy trial design will result in substantial loss of statistical power. To properly design immunot...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8440
更新日期:2020-03-15 00:00:00
abstract::Appropriate methods for meta-regression applied to a set of clinical trials, and the limitations and pitfalls in interpretation, are insufficiently recognized. Here we summarize recent research focusing on these issues, and consider three published examples of meta-regression in the light of this work. One principal m...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1187
更新日期:2002-06-15 00:00:00
abstract::Most statistical methodology for phase III clinical trials focuses on the comparison of a single experimental treatment with a control. An increasing desire to reduce the time before regulatory approval of a new drug is sought has led to development of two-stage or sequential designs for trials that combine the defini...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1362
更新日期:2003-03-15 00:00:00
abstract::In this commentary, we revisit Sir Austin Bradford Hill's seminal Alfred Watson Memorial Lecture in 1962 through the eyes of two practicing biostatisticians of the current era. We summarize some eternal takeaway messages from Hill's lecture regarding observations and experiments translated through the modern lexicon o...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8830
更新日期:2021-01-15 00:00:00
abstract::Obtaining accurate estimates of the performance of a diagnostic test for some population of patients might be difficult when the sample of subjects used for this purpose is not representative for the whole population. Thus, in the motivating example of this paper a test is evaluated by comparing its results with those...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3099
更新日期:2008-06-15 00:00:00