Abstract:
:Missing values in the covariates are a widespread complication in the statistical inference of regression models. The maximum likelihood principle requires specification of the distribution of the covariates, at least in part. For categorical covariates, log-linear models can be used. Additionally, the missing at random assumption is necessary, which excludes a dependence of the occurrence of missing values on the unobserved covariate values. This assumption is often highly questionable. We present a framework to specify alternative missing value mechanisms such that maximum likelihood estimation of the regression parameters under a specified alternative is possible. This allows investigation of the sensitivity of a single estimate against violations of the missing at random assumption. The possible results of a sensitivity analysis are illustrated by artificial examples. The practical application is demonstrated by the analysis of two case-control studies.
journal_name
Stat Medjournal_title
Statistics in medicineauthors
Vach W,Blettner Mdoi
10.1002/sim.4780141205subject
Has Abstractpub_date
1995-06-30 00:00:00pages
1315-29issue
12eissn
0277-6715issn
1097-0258journal_volume
14pub_type
杂志文章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::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::An observed confidence distribution is proposed as a measure of strength of evidence for practically equivalent efficacies of two treatments. The concept is independent of prior opinions about relevant sizes of a difference in efficacy. It also avoids retrospective power calculations for trials with missed recruitment...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780071207
更新日期:1988-12-01 00:00:00
abstract::Comparative studies of the accuracy of diagnostic procedures often use a paired design to gain in efficiency. Standard methods for analysing data from paired designs require complete observations. In many studies, however, one of the test results may be missing for some patients. In this paper, we propose a simple cor...
journal_title:Statistics in medicine
pub_type: 临床试验,杂志文章
doi:10.1002/(SICI)1097-0258(19960815)15:15<1687::AID-S
更新日期:1996-08-15 00:00:00
abstract::This article deals with determination of a sample size that guarantees the success of a trial. We follow a Bayesian approach and we say an experiment is successful if it yields a large posterior probability that an unknown parameter of interest (an unknown treatment effect or an effects-difference) is greater than a c...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3175
更新日期:2008-06-15 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 calibration line is used to define the relationship between a new clinical technique and a standard in vitro laboratory methodology. Discrimination intervals quantify the reliability of inverse estimates obtained from the calibration line. Applied to transcutaneous PCO2 monitoring, a new in vivo measurement, discrim...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780050407
更新日期:1986-07-01 00:00:00
abstract::The effective dose (ED) is the pharmaceutical dosage required to produce a therapeutic response in a fixed proportion of the patients. When only one drug is considered, the problem is a univariate one and has been well-studied. However, in the multidimensional setting, that is, in the presence of combinations of agent...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6226
更新日期:2014-10-30 00:00:00
abstract::This paper examines several methods for deriving standardized morbidity ratios (SMR) and attributable fraction (attributable risk percentage) estimates. We show that some of the proposed methods will, in general, produce biased estimators, although the low variance of certain estimators sometimes compensates for their...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780030206
更新日期:1984-04-01 00:00:00
abstract::Pattern-mixture models (PMM) and selection models (SM) are alternative approaches for statistical analysis when faced with incomplete data and a nonignorable missing-data mechanism. Both models make empirically unverifiable assumptions and need additional constraints to identify the parameters. Here, we first introduc...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.6302
更新日期:2014-11-30 00:00:00
abstract::This paper discusses the benefits and limitations of adaptive sample size re-estimation for phase 3 confirmatory clinical trials. Comparisons are made with more traditional fixed sample and group sequential designs. It is seen that the real benefit of the adaptive approach arises through the ability to invest sample s...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4102
更新日期:2011-12-10 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::There is considerable public concern about health disparities among different cultural/racial/ethnic groups. Important process measures that might reflect inequities are treated prevalence and the service utilization rate in a defined period of time. We have previously described a method for estimating N, the distinct...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3904
更新日期:2010-07-20 00:00:00
abstract::Bayesian methods are proposed for analysing matched case-control studies in which a binary exposure variable is sometimes measured with error, but whose correct values have been validated for a random sample of the matched case-control sets. Three models are considered. Model 1 makes few assumptions other than randomn...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2000
更新日期:2005-02-15 00:00:00
abstract::Piecewise constant incidence models were developed to estimate the force of infection in women from age- and time-specific antenatal or neonatal seroprevalence data. Differential inclusion of infected women in sero-surveys compared to uninfected women was taken into account, with respect to both changes in inclusion r...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780131809
更新日期:1994-09-30 00:00:00
abstract::To compare the survival functions based on right-truncated data, Lagakos et al. proposed a weighted logrank test based on a reverse time scale. This is in contrast to Bilker and Wang, who suggested a semi-parametric version of the Mann-Whitney test by assuming that the distribution of truncation times is known or can ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2556
更新日期:2007-02-20 00:00:00
abstract::Generalized relative and absolute risk models, in which various functions of time and age modify the excess relative or absolute risk of radiation-induced cancer, are fitted to the Japanese atomic bomb survivor cancer incidence data set. Among generalized relative risk models, those in which a product of powers of tim...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/(sici)1097-0258(19990115)18:1<17::aid-sim9
更新日期:1999-01-15 00:00:00
abstract::Several relative risk models for survival time data in drug combination therapy are derived and their properties are discussed. The main intention of this paper is to clarify the differences among the models in order to help to choose the appropriate one in a given situation. The models are motivated by discussing the...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780091216
更新日期:1990-12-01 00:00:00
abstract::A general method is presented that allows the researcher to change statistical design elements such as the residual sample size during the course of an experiment, to include an interim analysis for early stopping when no formal rule for early stopping was foreseen, to increase or reduce the number of planned interim ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.1852
更新日期:2004-08-30 00:00:00
abstract::The Response Evaluation Criteria in Solid Tumors are used as standard guidelines for the clinical evaluation of cancer treatments. The assessment is based on the anatomical tumor burden: change in size of target lesions and evolution of nontarget lesions (NTL). Despite unquestionable advantages of this standard tool, ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.7640
更新日期:2018-06-15 00:00:00
abstract::The problem of testing symmetry about zero has a long and rich history in the statistical literature. We introduce a new test that sequentially discards observations whose absolute value is below increasing thresholds defined by the data. McNemar's statistic is obtained at each threshold and the largest is used as the...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5384
更新日期:2012-11-20 00:00:00
abstract::We consider Cox proportional hazards regression when the covariate vector includes error-prone discrete covariates along with error-free covariates, which may be discrete or continuous. The misclassification in the discrete error-prone covariates is allowed to be of any specified form. Building on the work of Nakamura...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3159
更新日期:2008-05-20 00:00:00
abstract::Few methods are currently available for group sequential analysis of recurrent events data subject to a terminal event in the clinical trial setting. This research helps fill this gap by developing a completely nonparametric group sequential monitoring procedure for use with the two-sample Tayob and Murray statistic. ...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.8389
更新日期:2019-12-30 00:00:00
abstract::This paper presents a procedure for obtaining short-term projections and lower bounds on the size of the acquired immunodeficiency syndrome (AIDS) epidemic. The method is similar to that proposed by Brookmeyer and Gail but adapted to the situation where individual dates of AIDS diagnosis are available. It gives result...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.4780081210
更新日期:1989-12-01 00:00:00
abstract::The time-dependent change of HIV particle load, i.e. HIV dynamics, is likely to be controlled by a multitude of quantitative trait loci (QTL) that interact with each other as well as with various developmental and environmental factors in a coordinated manner. In this article, we have derived a new statistical model f...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2489
更新日期:2006-11-30 00:00:00
abstract::Geocoding a study population as completely as possible is an important data assimilation component of many spatial epidemiologic studies. Unfortunately, complete geocoding is rare in practice. The failure of a substantial proportion of study subjects' addresses to geocode has consequences for spatial analyses, some of...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3288
更新日期:2008-09-20 00:00:00
abstract::Providing personalized treatments designed to maximize benefits and minimizing harms is of tremendous current medical interest. One problem in this area is the evaluation of the interaction between the treatment and other predictor variables. Treatment effects in subgroups having the same direction but different magni...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.5429
更新日期:2012-12-10 00:00:00
abstract::The 'at least as good as' criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non-inferiority is not taken as a fixed difference; it varies as a function of the positive control re...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2476
更新日期:2006-04-15 00:00:00
abstract::Case-control studies are usually defined to investigate risk factors for a single disease of interest. However, subsequent to data collection, investigators may wish to examine as an 'outcome' a variable that was an exposure in the original study. A naive analysis that disregards the sampling strategy that gave rise t...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.2398
更新日期:2005-12-30 00:00:00
abstract::We propose a semiparametric method for estimating ROC surfaces for continuous diagnostic tests based on two test measurements. Such a three-class diagnostic problem based on two test measurements arises naturally from some DNA amplification-related diagnostic scenarios. Simulation results show that our proposed semipa...
journal_title:Statistics in medicine
pub_type: 杂志文章
doi:10.1002/sim.3625
更新日期:2009-08-15 00:00:00