Analyzing longitudinal data to characterize the accuracy of markers used to select treatment.

Abstract:

:With the increasing availability of detailed clinical information, there is optimism that treatment choices can be selectively directed to those individuals most likely to benefit. While standard clinical trials can establish whether a treatment appears to be effective on average, subsequent work is needed to determine whether there are identifiable subgroups of subjects for whom treatment is either particularly beneficial or harmful. Molecular assays and modern imaging technology now allow numerous candidate measures to be used as potential determinants of treatment choice. In this manuscript, we focus on novel measures of decision accuracy that reflect the treatment marker objective. Specifically, we define longitudinal individual-level potential outcomes (principal strata) that characterize patient outcomes under treated and untreated states. We propose generalizations of sensitivity and specificity that measure the accuracy with which a marker can distinguish those subjects who are expected to have a more favorable outcome under a specific treatment choice from those subjects who are expected to have a more favorable outcome under alternative treatment options. For quantitative markers, we propose principal receiver operating characteristic curves that display the full range of potential sensitivity and specificity. We use simulations to demonstrate the properties of proposed estimators, and we illustrate the methods using candidate neuroimaging and electrodiagnostic markers that could be used to select patients for carpal tunnel surgery.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Sitlani CM,Heagerty PJ

doi

10.1002/sim.6138

subject

Has Abstract

pub_date

2014-07-30 00:00:00

pages

2881-96

issue

17

eissn

0277-6715

issn

1097-0258

journal_volume

33

pub_type

杂志文章
  • The multiple-record systems estimator when registrations refer to different but overlapping populations.

    abstract::In multiple-record systems estimation it is usually assumed that all registration relate to the same population. In this paper, we develop a method which can be used when the registrations relate to different populations, in the sense that they cover, for example, different time periods or regions. We show that under ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1818

    authors: Zwane EN,van der Pal-de Bruin K,van der Heijden PG

    更新日期:2004-07-30 00:00:00

  • Adjusting for drop-out in clinical trials with repeated measures: design and analysis issues.

    abstract::Recently, Wu and Follmann developed summary measures to adjust for informative drop-out in longitudinal studies where drop-out depends on the underlying true value of the response. In this paper we evaluate these procedures in the common situation where drop-out depends on the observed responses. We also discuss vario...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20010115)20:1<93::aid-sim655>3.0

    authors: Wu MC,Albert PS,Wu BU

    更新日期:2001-01-15 00:00:00

  • The effect of salvage therapy on survival in a longitudinal study with treatment by indication.

    abstract::We consider using observational data to estimate the effect of a treatment on disease recurrence, when the decision to initiate treatment is based on longitudinal factors associated with the risk of recurrence. The effect of salvage androgen deprivation therapy (SADT) on the risk of recurrence of prostate cancer is in...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4017

    authors: Kennedy EH,Taylor JM,Schaubel DE,Williams S

    更新日期:2010-11-10 00:00:00

  • Non-parametric methods for comparing multiple treatment groups to a control group, based on incomplete non-decreasing repeated measurements.

    abstract::In the comparison of two or more treatment groups to a control group, consider a study with non-decreasing repeated measurements of the same characteristic taken over a common set of time points for each subject. Based on the vector of possibly incomplete responses from each subject, this paper considers asymptoticall...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(SICI)1097-0258(19961215)15:23<2509::AID-S

    authors: Davis CS

    更新日期:1996-12-15 00:00:00

  • Comparing performance of surgeons using risk-adjusted procedures.

    abstract::It is naive and incorrect to use the proportions of successful operations to compare the performance of surgeons because the patients' risk profiles are different. In this paper, we explore the use of risk-adjusted procedures to compare the performance of surgeons. One such risk-adjusted statistic is the standardized ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7310

    authors: Tang X,Gan FF

    更新日期:2017-07-20 00:00:00

  • A simple correction for ties when censoring times depend on covariates.

    abstract::We point out that the conventional methods for ties correction may be seriously biased when censoring times depend on covariates. A simple modification to the Efron correction method is suggested which works remarkably well in simulation studies. The method corresponds closely to breaking ties by random ordering. The ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2173

    authors: Samuelsen SO,Wisløff TF,Skrondal A

    更新日期:2005-10-30 00:00:00

  • Analysis of matched case-control data with multiple ordered disease states: possible choices and comparisons.

    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

    authors: Mukherjee B,Liu I,Sinha S

    更新日期:2007-07-30 00:00:00

  • Detection rates and false positive rates for Down's syndrome screening: how precisely can they be estimated and what factors influence their value?

    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

    authors: Dunstan FD,Gray JC,Nix AB,Reynolds T

    更新日期:1997-07-15 00:00:00

  • Maximum likelihood estimation of the kappa coefficient from models of matched binary responses.

    abstract::We present an estimate of the kappa-coefficient of agreement between two methods of rating based on matched pairs of binary responses and show that the estimate depends on the common intraclass correlation coefficient between the pairs. Via Monte Carlo simulation, we investigate power of the test of significance on ka...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780140109

    authors: Shoukri MM,Martin SW,Mian IU

    更新日期:1995-01-15 00:00:00

  • Estimating a survival curve with unlinked entry and failure times.

    abstract::In monitoring a clinical trial or other observational study with a survival endpoint, sometimes the numbers of patients entering and dying at each time point are presented, but the connections between them are kept confidential. Hence, the exact time to failure or censoring for each individual is missing. We refer to ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2819

    authors: Wu Y,Shih WJ,Moore DF

    更新日期:2007-08-30 00:00:00

  • Determining the size of a cross-sectional sample to estimate the age-specific incidence of an irreversible disease.

    abstract::The design of a cross-sectional survey to estimate the age-specific incidence of an irreversible disease is considered, where the incidence rate is not changing over time and the risk of mortality is not affected by the onset of disease. The sample is assumed to give information on the current age and disease status o...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780132208

    authors: Marschner IC

    更新日期:1994-11-30 00:00:00

  • Robust estimation for linear panel data models.

    abstract::In different fields of applications including, but not limited to, behavioral, environmental, medical sciences, and econometrics, the use of panel data regression models has become increasingly popular as a general framework for making meaningful statistical inferences. However, when the ordinary least squares (OLS) m...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8732

    authors: Hamiye Beyaztas B,Bandyopadhyay S

    更新日期:2020-12-20 00:00:00

  • Optimal designs for Michaelis-Menten kinetic studies.

    abstract::Many reactions in enzymology are governed by the Michaelis-Menten equation. Characterising these reactions requires the estimation of the parameters K(M) and V(max) which determine the Michaelis-Menten equation and this is done by observing rates of reactions at a set of substrate concentrations. The choice of substra...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1612

    authors: Matthews JN,Allcock GC

    更新日期:2004-02-15 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

  • Assessing the robustness of sisVIVE in a Mendelian randomization study to estimate the causal effect of body mass index on income using multiple SNPs from understanding society.

    abstract::The "some invalid, some valid instrumental variable estimator" (sisVIVE) is a lasso-based method for instrumental variables (IVs) regression of outcome on an exposure. In principle, sisVIVE is robust to some of the IVs in the analysis being invalid, in the sense of being related to the outcome variable through pathway...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8066

    authors: Bao Y,Clarke PS,Smart M,Kumari M

    更新日期:2019-04-30 00:00:00

  • Measuring spatial effects in time to event data: a case study using months from angiography to coronary artery bypass graft (CABG).

    abstract::The application of Bayesian hierarchical models to measure spatial effects in time to event data has not been widely reported. This case study aims to estimate the effect of area of residence on waiting times to coronary artery bypass graft (CABG) and to assess the role of important individual specific covariates (age...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1535

    authors: Crook AM,Knorr-Held L,Hemingway H

    更新日期:2003-09-30 00:00:00

  • Comparison of trends in HIV infection for two risk categories.

    abstract::Sensible plans for health-care needs and determination of priorities for expenditure require regular assessment of trends in HIV incidences. In particular, trends in the relative HIV incidences of different risk categories are useful when assessing whether current control strategies are working equally well for all ri...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(SICI)1097-0258(19960830)15:16<1779::AID-S

    authors: Becker NG,Cui JS

    更新日期:1996-08-30 00:00:00

  • The Integrated Calibration Index (ICI) and related metrics for quantifying the calibration of logistic regression models.

    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

    authors: Austin PC,Steyerberg EW

    更新日期:2019-09-20 00:00:00

  • Hypothesis testing in the polychotomous logistic model with an application to detecting gastrointestinal cancer.

    abstract::We discuss the use of the trichotomous logistic model to discriminate between patients with gastrointestinal (GI) cancer, patients with benign GI disease and 'normal' subjects, using symptoms and the concentrations of some serum proteins that are potentially indicative of malignancy as covariates. A parsimonious model...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780040313

    authors: Marshall RJ,Chisholm EM

    更新日期:1985-07-01 00:00:00

  • Semiparametric mixture cure model analysis with competing risks data: Application to vascular access thrombosis data.

    abstract::The article is motivated by a nephrology study in Taiwan, which enrolled hemodialysis patients who suffered from vascular access thrombosis. After treatment, some patients were cured of thrombosis, while some may experience recurrence of either type (acute or nonacute) of vascular access thrombosis. Our major interest...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8711

    authors: Chen CM,Shen PS,Lin CC,Wu CC

    更新日期:2020-11-30 00:00:00

  • A Markov mixed effect regression model for drug compliance.

    abstract::Patient compliance (adherence) with prescribed medication is often erratic, while clinical outcomes are causally linked to actual, rather than nominal medication dosage. We propose here a hierarchical Markov model for patient compliance. At the first stage, conditional upon individual random effects and a set of indiv...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19981030)17:20<2313::aid-s

    authors: Girard P,Blaschke TF,Kastrissios H,Sheiner LB

    更新日期:1998-10-30 00:00:00

  • A multivariate Bayesian model for embryonic growth.

    abstract::Most longitudinal growth curve models evaluate the evolution of each of the anthropometric measurements separately. When applied to a 'reference population', this exercise leads to univariate reference curves against which new individuals can be evaluated. However, growth should be evaluated in totality, that is, by e...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6411

    authors: Willemsen SP,Eilers PH,Steegers-Theunissen RP,Lesaffre E

    更新日期:2015-04-15 00:00:00

  • Testing for publication bias in diagnostic meta-analysis: a simulation study.

    abstract::The present study investigates the performance of several statistical tests to detect publication bias in diagnostic meta-analysis by means of simulation. While bivariate models should be used to pool data from primary studies in diagnostic meta-analysis, univariate measures of diagnostic accuracy are preferable for t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6177

    authors: Bürkner PC,Doebler P

    更新日期:2014-08-15 00:00:00

  • Behind closed doors: the data monitoring board in randomized clinical trials.

    abstract::Many randomized clinical trials include a data and safety monitoring board (DSMB) that is responsible for reviewing accruing data, monitoring performance of the trial, assuring safety of the participants in the trial, and assessing the efficacy of treatment. The DSMB often makes recommendations about continuation of t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780120504

    authors: Wittes J

    更新日期:1993-03-01 00:00:00

  • Descriptive statistical analyses of serial dilution data.

    abstract::The serial dilution assay (for example, an in vitro antimicrobic susceptibility test or a serum antibody titer assay) is an important technique in biomedical research. The structure of the experiment forces grouping of the threshold concentrations into intervals. Statistical methods to analyse threshold concentrations...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780070410

    authors: Hamilton MA,Rinaldi MG

    更新日期:1988-04-01 00:00:00

  • The use of an extended baseline period in the evaluation of treatment in a longitudinal Duchenne muscular dystrophy trial.

    abstract::A trial of Duchenne muscular dystrophy involved tracking boys of all ages through a one-year baseline period, followed by a one-year trial of leucine versus placebo treatment. In this paper we develop a model for a total-muscle-strength score that uses the data of the extended baseline period in the evaluation of the ...

    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.4780050304

    authors: Madsen KS,Miller JP,Province MA

    更新日期:1986-05-01 00:00:00

  • Boosting for detection of gene-environment interactions.

    abstract::In genetic association studies, it is typically thought that genetic variants and environmental variables jointly will explain more of the inheritance of a phenotype than either of these two components separately. Traditional methods to identify gene-environment interactions typically consider only one measured enviro...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5444

    authors: Pashova H,LeBlanc M,Kooperberg C

    更新日期:2013-01-30 00:00:00

  • Smooth bootstrap methods for analysis of longitudinal data.

    abstract::In analysis of longitudinal data, the variance matrix of the parameter estimates is usually estimated by the 'sandwich' method, in which the variance for each subject is estimated by its residual products. We propose smooth bootstrap methods by perturbing the estimating functions to obtain 'bootstrapped' realizations ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3027

    authors: Li Y,Wang YG

    更新日期:2008-03-30 00:00:00

  • Hierarchical nested trial design (HNTD) for demonstrating treatment efficacy of new antibacterial drugs in patient populations with emerging bacterial resistance.

    abstract::In the last decade or so, pharmaceutical drug development activities in the area of new antibacterial drugs for treating serious bacterial diseases have declined, and at the same time, there are worries that the increased prevalence of antibiotic-resistant bacterial infections, especially the increase in drug-resistan...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6233

    authors: Huque MF,Valappil T,Soon GG

    更新日期:2014-11-10 00:00:00

  • Modelling age-specific risk: application to dementia.

    abstract::We give up-to-date methods for estimating the age-specific incidence of a disease and for estimating the effect of risk factors. We recommend taking age as the basic time scale of the analysis; then, the hazard function can be interpreted as the age-specific incidence of the disease. This choice raises a delayed entry...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19980915)17:17<1973::aid-s

    authors: Commenges D,Letenneur L,Joly P,Alioum A,Dartigues JF

    更新日期:1998-09-15 00:00:00