Positing, fitting, and selecting regression models for pooled biomarker data.

Abstract:

:Pooling biospecimens prior to performing lab assays can help reduce lab costs, preserve specimens, and reduce information loss when subject to a limit of detection. Because many biomarkers measured in epidemiological studies are positive and right-skewed, proper analysis of pooled specimens requires special methods. In this paper, we develop and compare parametric regression models for skewed outcome data subject to pooling, including a novel parameterization of the gamma distribution that takes full advantage of the gamma summation property. We also develop a Monte Carlo approximation of Akaike's Information Criterion applied to pooled data in order to guide model selection. Simulation studies and analysis of motivating data from the Collaborative Perinatal Project suggest that using Akaike's Information Criterion to select the best parametric model can help ensure valid inference and promote estimate precision.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Mitchell EM,Lyles RH,Schisterman EF

doi

10.1002/sim.6496

subject

Has Abstract

pub_date

2015-07-30 00:00:00

pages

2544-58

issue

17

eissn

0277-6715

issn

1097-0258

journal_volume

34

pub_type

杂志文章
  • Using pilot data to size a two-arm randomized trial to find a nearly optimal personalized treatment strategy.

    abstract::A personalized treatment strategy formalizes evidence-based treatment selection by mapping patient information to a recommended treatment. Personalized treatment strategies can produce better patient outcomes while reducing cost and treatment burden. Thus, among clinical and intervention scientists, there is a growing...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6783

    authors: Laber EB,Zhao YQ,Regh T,Davidian M,Tsiatis A,Stanford JB,Zeng D,Song R,Kosorok MR

    更新日期:2016-04-15 00:00:00

  • Multiple imputation analysis of case-cohort studies.

    abstract::The usual methods for analyzing case-cohort studies rely on sometimes not fully efficient weighted estimators. Multiple imputation might be a good alternative because it uses all the data available and approximates the maximum partial likelihood estimator. This method is based on the generation of several plausible co...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4130

    authors: Marti H,Chavance M

    更新日期:2011-06-15 00:00:00

  • Assessing local influence in principal component analysis with application to haematology study data.

    abstract::In many medical and health studies, high-dimensional data are often encountered. Principal component analysis (PCA) is a commonly used technique to reduce such data to a few components that includes most of the information provided by the original data. However, PCA is known to be very sensitive to some abnormal obser...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2747

    authors: Fung WK,Gu H,Xiang L,Yau KK

    更新日期:2007-06-15 00:00:00

  • What are the statistical implications of treatment non-compliance in cluster randomized trials: A simulation study.

    abstract::Subjects in randomized controlled trials do not always comply to the treatment condition they have been assigned to. This may cause the estimated effect of the intervention to be biased and also affect efficiency, coverage of confidence intervals, and statistical power. In cluster randomized trials non-compliance may ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8351

    authors: Moerbeek M,Schie SV

    更新日期:2019-11-20 00:00:00

  • An extension of the continual reassessment method using decision theory.

    abstract::The primary goal of a phase I trial is to find the maximally tolerated dose (MTD) of a treatment. The MTD is usually defined in terms of a tolerable probability, q(*), of toxicity. Our objective is to find the highest dose with toxicity risk that does not exceed q(*), a criterion that is often desired in designing pha...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.970

    authors: Leung DH,Wang YG

    更新日期:2002-01-15 00:00:00

  • Estimating the stenosis probabilities in arteriosclerosis obliterans using generalized estimating equations.

    abstract::For each of 211 arteriosclerosis obliterans patients, the degree of stenosis of arteries at four sites were examined at Hiroshima University Hospital to analyse the relationship between the degree of stenosis and age, sex and site. The generalized estimating equations using a proportional odds model for the stenosis p...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1363

    authors: Nakashima E,Tsuji S,Fukuoka H,Ohtaki M,Ito K

    更新日期:2003-07-15 00:00:00

  • Construction, validation and updating of a prognostic model for kidney graft survival.

    abstract::The construction, validation and updating of a prognostic model for kidney graft survival is reported using data from the Eurotransplant database. First, a model is constructed for data from transplantations in the period 1984 to 1987. The model is later updated for the 1988 1990 data. The first data set was randomly ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780141806

    authors: Van Houwelingen HC,Thorogood J

    更新日期:1995-09-30 00:00:00

  • Genetic association studies with bivariate mixed responses subject to measurement error and misclassification.

    abstract::In genetic association studies, mixed effects models have been widely used in detecting the pleiotropy effects which occur when one gene affects multiple phenotype traits. In particular, bivariate mixed effects models are useful for describing the association of a gene with a continuous trait and a binary trait. Howev...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8688

    authors: Zhang Q,Yi GY

    更新日期:2020-11-20 00:00:00

  • Extending the c-statistic to nominal polytomous outcomes: the Polytomous Discrimination Index.

    abstract::Diagnostic problems in medicine are sometimes polytomous, meaning that the outcome has more than two distinct categories. For example, ovarian tumors can be benign, borderline, primary invasive, or metastatic. Extending the main measure of binary discrimination, the c-statistic or area under the ROC curve, to nominal ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5321

    authors: Van Calster B,Van Belle V,Vergouwe Y,Timmerman D,Van Huffel S,Steyerberg EW

    更新日期:2012-10-15 00:00:00

  • Practical issues in data monitoring of clinical trials: summary of responses to a questionnaire at NIH.

    abstract::A targeted poll was undertaken to compare and contrast models of data monitoring of randomized clinical trials sponsored by the National Institutes of Health. In an attempt to represent the institutes which conduct clinical trials, twelve individuals were selected and asked to respond to a questionnaire specifically p...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780120520

    authors: Geller NL,Stylianou M

    更新日期:1993-03-01 00:00:00

  • Interval estimation for rank correlation coefficients based on the probit transformation with extension to measurement error correction of correlated ranked data.

    abstract::The Spearman (rho(s)) and Kendall (tau) rank correlation coefficient are routinely used as measures of association between non-normally distributed random variables. However, confidence limits for rho(s) are only available under the assumption of bivariate normality and for tau under the assumption of asymptotic norma...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2547

    authors: Rosner B,Glynn RJ

    更新日期:2007-02-10 00:00:00

  • Non-inferiority trials: the 'at least as good as' criterion with dichotomous data.

    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

    authors: Laster LL,Johnson MF,Kotler ML

    更新日期:2006-04-15 00:00:00

  • Model-based phase I designs incorporating toxicity and efficacy for single and dual agent drug combinations: methods and challenges.

    abstract::Novel therapies are challenging the standards of drug development. Agents with specific biologic targets, unknown dose-efficacy curves, and limited toxicity mandate novel designs to identify biologically optimal doses. We review two model-based designs that utilize either a proportional odds model or a continuation ra...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3706

    authors: Mandrekar SJ,Qin R,Sargent DJ

    更新日期:2010-05-10 00:00:00

  • Ties between event times and jump times in the Cox model.

    abstract::Methods for dealing with tied event times in the Cox proportional hazards model are well developed. Also, the partial likelihood provides a natural way to handle covariates that change over time. However, ties between event times and the times that discrete time-varying covariates change have not been systematically s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5683

    authors: Xin X,Horrocks J,Darlington GA

    更新日期:2013-06-30 00:00:00

  • A refined method for the meta-analysis of controlled clinical trials with binary outcome.

    abstract::For the meta-analysis of controlled clinical trials with binary outcome a test statistic for testing an overall treatment effect is proposed, which is based on a refined estimator for the variance of the treatment effect estimator usually used in the random-effects model of meta-analysis. In simulation studies it is s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1009

    authors: Hartung J,Knapp G

    更新日期:2001-12-30 00:00:00

  • Modelling covariate adjusted mortality relative to a standard population.

    abstract::A study of long term survival of 1487 patients given an allogeneic bone marrow transplant for acute myelogenous leukaemia and 729 patients given a transplant for severe aplastic anaemia was conducted by the International Bone Marrow Transplant Registry. One aim of this study is to determine if the mortality rates of t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19990630)18:12<1529::aid-s

    authors: Andersen PK,Horowitz MM,Klein JP,Socie G,Stone JV,Zhang MJ

    更新日期:1999-06-30 00:00:00

  • Sam Greenhouse's years at the Census Bureau and the UNRRA.

    abstract::Sam Greenhouse joined the Census Bureau as a clerk at an interesting time period for the agency. The first use of sampling in the decennial census occurred in 1940. There was a major expansion of the amount of data collected. The organization of the Census Bureau underwent radical changes, including the growth of the ...

    journal_title:Statistics in medicine

    pub_type: 传,历史文章,杂志文章

    doi:10.1002/sim.1627

    authors: Keller J,Clark CZ

    更新日期:2003-11-15 00:00:00

  • Design and sample size considerations for valuation studies of multi-attribute utility instruments.

    abstract::The EQ-5D, a widely used multiattribute utility instrument, is commonly used in health economic evaluations where the goal is to decide on which treatments to reimburse. Like other instruments, value sets of the EQ-5D are constructed using valuation studies typically valuing a subset of the health states and using pre...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8592

    authors: Shams S,Pullenayegum E

    更新日期:2020-10-15 00:00:00

  • Stratified analysis of multivariate clinical data: application of a Mantel-Haenszel approach.

    abstract::Laboratory determinations on children aged 6 to 10 years obtained over a 5-year period are analysed by a method described in detail for differentiating between children from exposed and control areas of Seveso, Italy. In the analysis, stratification is employed to distinguish the separate days of laboratory measuremen...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780020221

    authors: Mantel N,Mocarelli P,Marocchi A,Brambilla P,Baretta R

    更新日期:1983-04-01 00:00:00

  • A community trial strategy for evaluating treatment for symptomatic conditions.

    abstract::A method has been developed for simultaneously comparing the usefulness of many treatments of established value for symptomatic medical conditions. Medical assessment of outcome is not employed. Instead patients are required to assess treatments prescribed during the course of ordinary general practice rather than und...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1002/sim.4780040104

    authors: Charlton JR,D'Souza MF,Tooley M,Silver R

    更新日期:1985-01-01 00:00:00

  • Choice of test for association in small sample unordered r x c tables.

    abstract::Pearson's chi-squared, the likelihood-ratio, and Fisher-Freeman-Halton's test statistics are often used to test the association of unordered r x c tables. Asymptotical, exact conditional, or exact conditional with mid-p adjustment methods are commonly used to compute the p-value. We have compared test power and signif...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2839

    authors: Lydersen S,Pradhan V,Senchaudhuri P,Laake P

    更新日期:2007-10-15 00:00:00

  • Violations of the independent increment assumption when using generalized estimating equation in longitudinal group sequential trials.

    abstract::In phase 3 clinical trials, ethical and financial concerns motivate sequential analyses in which the data are analyzed prior to completion of the entire planned study. Existing group sequential software accounts for the effects of these interim analyses on the sampling density by assuming that the contribution of subs...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6306

    authors: Shoben AB,Emerson SS

    更新日期:2014-12-20 00:00:00

  • Model selection in logistic joinpoint regression with applications to analyzing cohort mortality patterns.

    abstract::We consider a general model for anomaly detection in a longitudinal cohort mortality pattern based on logistic joinpoint regression with unknown joinpoints. We discuss backward and forward sequential procedures for selecting both the locations and the number of joinpoints. Estimation of the model parameters and the se...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3017

    authors: Czajkowski M,Gill R,Rempala G

    更新日期:2008-04-30 00:00:00

  • Statistical methods for multivariate interval-censored recurrent events.

    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

    authors: Chen BE,Cook RJ,Lawless JF,Zhan M

    更新日期:2005-03-15 00:00:00

  • Using mark-recapture methodology to estimate the size of a population at risk for sexually transmitted diseases.

    abstract::To study the spread of sexually transmitted diseases (STDs) using social/sexual mixing models, one must have quantitative information about sexual mixing. An unavoidable complication in gathering such information by survey is that members of the surveyed population will almost certainly have sexual contacts outside th...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780111202

    authors: Rubin G,Umbach D,Shyu SF,Castillo-Chavez C

    更新日期:1992-09-15 00:00:00

  • Precision of incidence predictions based on Poisson distributed observations.

    abstract::Disease incidence predictions are useful for a number of administrative and scientific purposes. The simplest ones are made using trend extrapolation, on either an arithmetic or a logarithmic scale. This paper shows how approximate confidence prediction intervals can be calculated for such predictions, both for the to...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780131503

    authors: Hakulinen T,Dyba T

    更新日期:1994-08-15 00:00:00

  • Modification of the sample size and the schedule of interim analyses in survival trials based on data inspections.

    abstract::A method is presented which allows us to adapt the sample size as well as the number and time points of interim analyses to the treatment difference observed at an interim look during the course of a clinical trial with censored survival time as the endpoint. The method allows the inclusion of data inspections during ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1136

    authors: Schäfer H,Müller HH

    更新日期:2001-12-30 00:00:00

  • Model mis-specification and overestimation of the intraclass correlation coefficient in cluster randomized trials.

    abstract::Intraclass correlation coefficient (ICC) estimates must be provided when reporting the results of a cluster randomized trial. This study demonstrates that estimating this parameter with one-way ANOVA and an underlying mixed-effects statistical model leads to biased estimates. The bias depends on the effect size of the...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2260

    authors: Giraudeau B

    更新日期:2006-03-30 00:00:00

  • Bias resulting from the use of 'assay sensitivity' as an inclusion criterion for meta-analysis.

    abstract::Assay sensitivity has been proposed as a criterion for including psychiatric clinical outcome studies in meta-analyses. The authors assess the performance of assay sensitivity as a method for determining study appropriateness for meta-analysis by calculating expected standard drug vs placebo effect sizes for various c...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2240

    authors: Gelfand LA,Strunk DR,Tu XM,Noble RE,Derubeis RJ

    更新日期:2006-03-30 00:00:00

  • Likelihood-based analysis of outcome-dependent sampling designs with longitudinal data.

    abstract::The use of outcome-dependent sampling with longitudinal data analysis has previously been shown to improve efficiency in the estimation of regression parameters. The motivating scenario is when outcome data exist for all cohort members but key exposure variables will be gathered only on a subset. Inference with outcom...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7633

    authors: Zelnick LR,Schildcrout JS,Heagerty PJ

    更新日期:2018-06-15 00:00:00