Assessing the effect of interventions in the context of mixture distributions with detection limits.

Abstract:

:Many quantitative assay measurements of metabolites of environmental toxicants in clinical investigations are subject to left censoring due to values falling below assay detection limits. Moreover, when observations occur in both unexposed individuals and exposed individuals who reflect a mixture of two distributions due to differences in exposure, metabolism, response to intervention and other factors, the measurements of these biomarkers can be bimodally distributed with an extra spike below the limit of detection. Therefore, estimating the effect of interventions on these biomarkers becomes an important and challenging problem. In this article, we present maximum likelihood methods to estimate the effect of intervention in the context of mixture distributions when a large proportion of observations are below the limit of detection. The selection of the number of components of mixture distributions was carried out using both bootstrap-based and cross-validation-based information criterion. We illustrate our methods using data from a randomized clinical trial conducted in Qidong, People's Republic of China.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Chu H,Kensler TW,Muñoz A

doi

10.1002/sim.2079

subject

Has Abstract

pub_date

2005-07-15 00:00:00

pages

2053-67

issue

13

eissn

0277-6715

issn

1097-0258

journal_volume

24

pub_type

临床试验,杂志文章,随机对照试验
  • Estimation of haemophilia-associated AIDS incidence in Japan using individual dates of diagnosis.

    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

    authors: Tango T

    更新日期:1989-12-01 00:00:00

  • Global goodness-of-fit tests for group testing regression models.

    abstract::In a variety of biomedical applications, particularly those involving screening for infectious diseases, testing individuals (e.g. blood/urine samples, etc.) in pools has become a standard method of data collection. This experimental design, known as group testing (or pooled testing), can provide a large reduction in ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3678

    authors: Chen P,Tebbs JM,Bilder CR

    更新日期:2009-10-15 00:00:00

  • Recommended tests for association in 2 x 2 tables.

    abstract::The asymptotic Pearson's chi-squared test and Fisher's exact test have long been the most used for testing association in 2x2 tables. Unconditional tests preserve the significance level and generally are more powerful than Fisher's exact test for moderate to small samples, but previously were disadvantaged by being co...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3531

    authors: Lydersen S,Fagerland MW,Laake P

    更新日期:2009-03-30 00:00:00

  • Statistical inferences for a twin correlation with multinomial outcomes.

    abstract::Current methods for statistical analysis of twin studies focus on continuous and dichotomous data, while only limited methodology exists for analysing multinomial data. As a consequence, investigators are often tempted to collapse multinomial data into two categories simply to facilitate the analysis. We address this ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20010130)20:2<249::aid-sim641>3.

    authors: Bartfay E,Donner A

    更新日期:2001-01-30 00:00:00

  • Comparison of operational characteristics for binary tests with clustered data.

    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

    authors: Kwak M,Um SW,Jung SH

    更新日期:2015-07-10 00:00:00

  • Circular-circular regression model with a spike at zero.

    abstract::With reference to a real data on cataract surgery, we discuss the problem of zero-inflated circular-circular regression when both covariate and response are circular random variables and a large proportion of the responses are zeros. The regression model is proposed, and the estimation procedure for the parameters is ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7496

    authors: Jha J,Biswas A

    更新日期:2018-01-15 00:00:00

  • Adjusting for verification bias in diagnostic test evaluation: a Bayesian approach.

    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

    authors: Buzoianu M,Kadane JB

    更新日期:2008-06-15 00:00:00

  • A metastasis or a second independent cancer? Evaluating the clonal origin of tumors using array copy number data.

    abstract::When a cancer patient develops a new tumor it is necessary to determine if it is a recurrence (metastasis) of the original cancer, or an entirely new occurrence of the disease. This is accomplished by assessing the histo-pathology of the lesions. However, there are many clinical scenarios in which this pathological di...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3866

    authors: Ostrovnaya I,Olshen AB,Seshan VE,Orlow I,Albertson DG,Begg CB

    更新日期:2010-07-10 00:00:00

  • M. D. deB. Edwardes, 'The generalization of the odds ratio, risk ratio and risk difference to r x k tables'. Statistics in Medicine 2000; 19(14): 1901-1914.

    abstract::The original article to which this Correction refers was published in Statistics in Medicine 2000 19(14): 1901-1914. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20001115)19:21<3017::aid-sim785>

    authors: Edwardes MD

    更新日期:2000-11-15 00:00:00

  • An index of disease activity in rheumatoid arthritis.

    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

    authors: Jones PW,Ziade MF,Davis MJ,Dawes PT

    更新日期:1993-06-30 00:00:00

  • Tutorial in biostatistics methods for interval-censored data.

    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

    authors: Lindsey JC,Ryan LM

    更新日期:1998-01-30 00:00:00

  • Measurement error in dietary assessment: an investigation using covariance structure models. Part II.

    abstract::In Part I we presented a covariance structure model for analysing measurement error in the assessment of nitrogen intake. In this paper we include data on urine nitrogen excretion which allows a critical assessment of the model proposed. Inclusion of urine nitrogen data produces more pessimistic estimates of the quali...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780121005

    authors: Plummer M,Clayton D

    更新日期:1993-05-30 00:00:00

  • Binary regression with continuous outcomes.

    abstract::Clinical research often involves continuous outcome measures, such as blood cholesterol, that are amenable to statistical techniques of analysis based on the mean, such as the t-test or multiple linear regression. Clinical interest, however, frequently focuses on the proportion of subjects who fall below or above a cl...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780140303

    authors: Suissa S,Blais L

    更新日期:1995-02-15 00:00:00

  • Analyzing survival curves at a fixed point in time.

    abstract::A common problem encountered in many medical applications is the comparison of survival curves. Often, rather than comparison of the entire survival curves, interest is focused on the comparison at a fixed point in time. In most cases, the naive test based on a difference in the estimates of survival is used for this ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2864

    authors: Klein JP,Logan B,Harhoff M,Andersen PK

    更新日期:2007-10-30 00:00:00

  • Model averaging for robust assessment of QT prolongation by concentration-response analysis.

    abstract::Assessing the QT prolongation potential of a drug is typically done based on pivotal safety studies called thorough QT studies. Model-based estimation of the drug-induced QT prolongation at the estimated mean maximum drug concentration could increase efficiency over the currently used intersection-union test. However,...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7395

    authors: Dosne AG,Bergstrand M,Karlsson MO,Renard D,Heimann G

    更新日期:2017-10-30 00:00:00

  • A regression model for multivariate random length data.

    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

    authors: Barnhart HX,Kosinski AS,Sampson AR

    更新日期:1999-01-30 00:00:00

  • Level-adjusted funnel plots based on predicted marginal expectations: an application to prophylactic antibiotics in gallstone surgery.

    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

    authors: Lindhagen L,Darkahi B,Sandblom G,Berglund L

    更新日期:2014-09-20 00:00:00

  • An extension of the continual reassessment methods using a preliminary up-and-down design in a dose finding study in cancer patients, in order to investigate a greater range of doses.

    abstract::In a phase I clinical trial in cancer patients, the drug involved had one known main adverse effect, which also occurs spontaneously in cancer patients with a fairly high frequency. Experiments in rats have shown marked effects of the drug on tumour growth in high doses, but also dose-dependent toxicity. Consequently,...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780140909

    authors: Møller S

    更新日期:1995-05-15 00:00:00

  • The analysis of incomplete data in the three-period two-treatment cross-over design for clinical trials.

    abstract::The additional time to complete a three-period two-treatment (3P2T) cross-over trial may cause a greater number of patient dropouts than with a two-period trial. This paper develops maximum likelihood (ML), single imputation and multiple imputation missing data analysis methods for the 3P2T cross-over designs. We use ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(SICI)1097-0258(19960130)15:2<127::AID-SIM

    authors: Richardson BA,Flack VF

    更新日期:1996-01-30 00:00:00

  • Psychiatric admissions and choice of abortion.

    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

    authors: Andersen PK,Rasmussen NK

    更新日期:1986-05-01 00:00:00

  • Nonparametric collective spectral density estimation with an application to clustering the brain signals.

    abstract::In this paper, we develop a method for the simultaneous estimation of spectral density functions (SDFs) for a collection of stationary time series that share some common features. Due to the similarities among the SDFs, the log-SDF can be represented using a common set of basis functions. The basis shared by the colle...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7972

    authors: Maadooliat M,Sun Y,Chen T

    更新日期:2018-12-30 00:00:00

  • Minimum sample size for developing a multivariable prediction model: PART II - binary and time-to-event outcomes.

    abstract::When designing a study to develop a new prediction model with binary or time-to-event outcomes, researchers should ensure their sample size is adequate in terms of the number of participants (n) and outcome events (E) relative to the number of predictor parameters (p) considered for inclusion. We propose that the mini...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7992

    authors: Riley RD,Snell KI,Ensor J,Burke DL,Harrell FE Jr,Moons KG,Collins GS

    更新日期:2019-03-30 00:00:00

  • The effect of unbalanced randomization on the progressively censored Savage test.

    abstract::Equal allocation of patients to treatment in a randomized clinical trial may have disadvantages ethically if the new treatment is believed to be at least as beneficial as the standard treatment. Others have considered, in a non-sequential setting, unbalanced randomized designs which allocate fewer patients to the pote...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780010309

    authors: Lesser ML

    更新日期:1982-07-01 00:00:00

  • Estimation of the time-dependent vaccine efficacy from a measles epidemic.

    abstract::We present a method to estimate the time-dependent vaccine efficacy from the cohort-specific vaccination coverage and from data on the vaccination status of cases and apply it to a measles epidemic in Germany which involved 529 cases, 88 of whom were vaccinated and 370 unvaccinated (for the remaining 71 cases the vacc...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1043

    authors: Eichner M,Diebner HH,Schubert C,Kreth HW,Dietz K

    更新日期:2002-08-30 00:00:00

  • On estimation of the variance in Cochran-Armitage trend tests for genetic association using case-control studies.

    abstract::The Cochran-Armitage trend test has been used in case-control studies for testing genetic association. As the variance of the test statistic is a function of unknown parameters, e.g. disease prevalence and allele frequency, it must be estimated. The usual estimator combining data for cases and controls assumes they fo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2250

    authors: Zheng G,Gastwirth JL

    更新日期:2006-09-30 00:00:00

  • A joint model of recurrent events and a terminal event with a nonparametric covariate function.

    abstract::We extend the shared frailty model of recurrent events and a dependent terminal event to allow for a nonparametric covariate function. We include a Gaussian random effect (frailty) in the intensity functions of both the recurrent and terminal events to capture correlation between the two processes. We employ the penal...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4297

    authors: Zhangsheng Y,Liu L

    更新日期:2011-09-30 00:00:00

  • Joint modeling longitudinal semi-continuous data and survival, with application to longitudinal medical cost data.

    abstract::It has been increasingly common to analyze simultaneously repeated measures and time to failure data. In this paper we propose a joint model when the repeated measures are semi-continuous, characterized by the presence of a large portion of zero values, as well as right skewness of non zero (positive) values. Examples...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3497

    authors: Liu L

    更新日期:2009-03-15 00:00:00

  • True verification probabilities should not be used in estimating the area under receiver operating characteristic curve.

    abstract::In medical research, a two-phase study is often used for the estimation of the area under the receiver operating characteristic curve (AUC) of a diagnostic test. However, such a design introduces verification bias. One of the methods to correct verification bias is inverse probability weighting (IPW). Since the probab...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8700

    authors: Wu Y

    更新日期:2020-11-30 00:00:00

  • Last observation carry-forward and last observation analysis.

    abstract::Drop-out often occurs in clinical trials with multiple visits and drop-out is often informative in the sense that the population of patients who dropped out is different from the population of patients who completed the study. To handle data with informative drop-out, an intention-to-treat analysis, which evaluates tr...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1519

    authors: Shao J,Zhong B

    更新日期:2003-08-15 00:00:00

  • Combining biomarkers for classification with covariate adjustment.

    abstract::Combining multiple markers can improve classification accuracy compared with using a single marker. In practice, covariates associated with markers or disease outcome can affect the performance of a biomarker or biomarker combination in the population. The covariate-adjusted receiver operating characteristic (ROC) cur...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7274

    authors: Kim S,Huang Y

    更新日期:2017-07-10 00:00:00