Extensions of net reclassification improvement calculations to measure usefulness of new biomarkers.

Abstract:

:Appropriate quantification of added usefulness offered by new markers included in risk prediction algorithms is a problem of active research and debate. Standard methods, including statistical significance and c statistic are useful but not sufficient. Net reclassification improvement (NRI) offers a simple intuitive way of quantifying improvement offered by new markers and has been gaining popularity among researchers. However, several aspects of the NRI have not been studied in sufficient detail. In this paper we propose a prospective formulation for the NRI which offers immediate application to survival and competing risk data as well as allows for easy weighting with observed or perceived costs. We address the issue of the number and choice of categories and their impact on NRI. We contrast category-based NRI with one which is category-free and conclude that NRIs cannot be compared across studies unless they are defined in the same manner. We discuss the impact of differing event rates when models are applied to different samples or definitions of events and durations of follow-up vary between studies. We also show how NRI can be applied to case-control data. The concepts presented in the paper are illustrated in a Framingham Heart Study example. In conclusion, NRI can be readily calculated for survival, competing risk, and case-control data, is more objective and comparable across studies using the category-free version, and can include relative costs for classifications. We recommend that researchers clearly define and justify the choices they make when choosing NRI for their application.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Pencina MJ,D'Agostino RB Sr,Steyerberg EW

doi

10.1002/sim.4085

subject

Has Abstract

pub_date

2011-01-15 00:00:00

pages

11-21

issue

1

eissn

0277-6715

issn

1097-0258

journal_volume

30

pub_type

杂志文章
  • A general frailty model to accommodate individual heterogeneity in the acquisition of multiple infections: An application to bivariate current status data.

    abstract::The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8506

    authors: Tran TMP,Abrams S,Braekers R

    更新日期:2020-05-30 00:00:00

  • Prognostic modelling with logistic regression analysis: a comparison of selection and estimation methods in small data sets.

    abstract::Logistic regression analysis may well be used to develop a prognostic model for a dichotomous outcome. Especially when limited data are available, it is difficult to determine an appropriate selection of covariables for inclusion in such models. Also, predictions may be improved by applying some sort of shrinkage in t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(20000430)19:8<1059::aid-si

    authors: Steyerberg EW,Eijkemans MJ,Harrell FE Jr,Habbema JD

    更新日期:2000-04-30 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

  • A comparison of likelihood-based and marginal estimating equation methods for analysing repeated ordered categorical responses with missing data: application to an intervention trial of vitamin prophylaxis for oesophageal dysplasia.

    abstract::The purpose of this research was to develop appropriate methods for analysing repeated ordinal categorical data that arose in an intervention trial to prevent oesophageal cancer. The measured response was the degree of oesophageal dysplasia at 2.5 and 6 years after randomization. An important feature was that some res...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780130511

    authors: Mark SD,Gail MH

    更新日期:1994-03-15 00:00:00

  • A method to estimate the variance of an endpoint from an on-going blinded trial.

    abstract::Blinded estimation of variance allows for changing the sample size without compromising the integrity of the trial. Some of the methods that estimate the variance in a blinded manner either make untenable assumptions or are only applicable to two-treatment trials. We propose a new method for continuous endpoints that ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2070

    authors: Xing B,Ganju J

    更新日期:2005-06-30 00:00:00

  • On the association between variables with lower detection limits.

    abstract::In this paper, we define a modified version τ(b) of Kendall's tau to measure the association in a pair (X,Y) of random variables subject to fixed left censoring due to known lower detection limits. We provide a nonparametric estimator of τ(b) and investigate its asymptotic properties. We then assume an Archimedean cop...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4319

    authors: Romdhani H,Lakhal-Chaieb L

    更新日期:2011-11-20 00:00:00

  • Bayesian methods to compare dose levels with placebo in a small n, sequential, multiple assignment, randomized trial.

    abstract::Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8813

    authors: Fang F,Hochstedler KA,Tamura RN,Braun TM,Kidwell KM

    更新日期:2021-02-20 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

  • 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

  • Cluster detection diagnostics for small area health data: with reference to evaluation of local likelihood models.

    abstract::The focus of this paper is the development of a range of cluster detection diagnostics that can be used to assess the degree to which a clustering method recovers the true clustering behaviour of small area data. The diagnostics proposed range from individual region specific diagnostics to neighbourhood diagnostics, a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2401

    authors: Hossain MM,Lawson AB

    更新日期:2006-03-15 00:00:00

  • Estimating heterogeneous treatment effects for latent subgroups in observational studies.

    abstract::Individuals may vary in their responses to treatment, and identification of subgroups differentially affected by a treatment is an important issue in medical research. The risk of misleading subgroup analyses has become well known, and some exploratory analyses can be helpful in clarifying how covariates potentially i...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7970

    authors: Kim HJ,Lu B,Nehus EJ,Kim MO

    更新日期:2019-02-10 00:00:00

  • The use of lung function tests in identifying factors that affect lung growth and aging.

    abstract::Lung function tests are used both clinically, in assessing disease, and epidemiologically, in identifying those factors which influence the growth and aging process of the lungs. The user must beware of several common pitfalls in the use of these tests, however. First, the commonly used tests of lung function can only...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780070106

    authors: Buist AS,Vollmer WM

    更新日期:1988-01-01 00:00:00

  • Measurement in clinical trials: a neglected issue for statisticians?

    abstract::Biostatisticians have frequently uncritically accepted the measurements provided by their medical colleagues engaged in clinical research. Such measures often involve considerable loss of information. Particularly, unfortunate is the widespread use of the so-called 'responder analysis', which may involve not only a lo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3603

    authors: Senn S,Julious S

    更新日期:2009-11-20 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

  • How serious is bias in effect estimation in randomised trials with survival data given risk heterogeneity and informative censoring?

    abstract::It is often assumed that randomisation will prevent bias in estimation of treatment effects from clinical trials, but this is not true of the semiparametric Proportional Hazards model for survival data when there is underlying risk heterogeneity. Here, a new formula is proposed for estimation of this bias, improving o...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7343

    authors: McNamee R

    更新日期:2017-09-20 00:00:00

  • 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

  • Group sequential designs for clinical trials with bivariate endpoints.

    abstract::Although all clinical trials are designed and monitored using more than one endpoint, methods are needed to assure that decision criteria are chosen to reflect the clinically relevant tradeoffs that assure the trial's scientific integrity. This article presents a framework for the design and monitoring clinical trials...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8696

    authors: Hu J,Blatchford PJ,Goldenberg NA,Kittelson JM

    更新日期:2020-11-20 00:00:00

  • Risk-adjusted CUSUM charts under model error.

    abstract::In recent years, quality control charts have been increasingly applied in the healthcare environment, for example, to monitor surgical performance. Risk-adjusted cumulative (CUSUM) charts that utilize risk scores like the Parsonnet score to estimate the probability of death of a patient from an operation turn out to b...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8104

    authors: Knoth S,Wittenberg P,Gan FF

    更新日期:2019-05-30 00:00:00

  • Assessing neural activity related to decision-making through flexible odds ratio curves and their derivatives.

    abstract::It is well established that neural activity is stochastically modulated over time. Therefore, direct comparisons across experimental conditions and determination of change points or maximum firing rates are not straightforward. This study sought to compare temporal firing probability curves that may vary across groups...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4220

    authors: Roca-Pardiñas J,Cadarso-Suárez C,Pardo-Vazquez JL,Leboran V,Molenberghs G,Faes C,Acuña C

    更新日期:2011-06-30 00:00:00

  • Ratio of geometric means to analyze continuous outcomes in meta-analysis: comparison to mean differences and ratio of arithmetic means using empiric data and simulation.

    abstract::Meta-analyses pooling continuous outcomes can use mean differences (MD), standardized MD (MD in pooled standard deviation units, SMD), or ratio of arithmetic means (RoM). Recently, ratio of geometric means using ad hoc (RoGM (ad hoc) ) or Taylor series (RoGM (Taylor) ) methods for estimating variances have been propos...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4501

    authors: Friedrich JO,Adhikari NK,Beyene J

    更新日期:2012-07-30 00:00:00

  • A restricted mixture model for dietary pattern analysis in small samples.

    abstract::Multivariate finite mixture models have been applied to the identification of dietary patterns. These models are known to have many parameters, and consequently large samples are usually required. We present a special case of a multivariate mixture model that reduces the number of parameters to be estimated and seems ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5336

    authors: Rita Gaio A,Costa JP,Santos AC,Ramos E,Lopes C

    更新日期:2012-08-30 00:00:00

  • Meta-analysis combining parallel and cross-over clinical trials. II: Binary outcomes.

    abstract::We examine different methods to pool binary outcomes used both in parallel and cross-over trials. Odds ratio (OR) estimators obtained from joint conditional probabilities in cross-over trials, such as the Mantel-Haenszel and Peto methods, are compared to an OR estimator using marginal results of cross-over trials. Whe...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1206

    authors: Curtin F,Elbourne D,Altman DG

    更新日期:2002-08-15 00:00:00

  • Cause-specific quantile regression on inactivity time.

    abstract::In time-to-event analysis, the traditional summary measures have been based on the hazard function, survival function, quantile event time, restricted mean event time, and residual lifetime. Under competing risks, furthermore, typical summary measures have been the cause-specific hazard function and cumulative inciden...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8871

    authors: Jia Y,Jeong JH

    更新日期:2021-01-06 00:00:00

  • Robust Bayesian sample size determination in clinical trials.

    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

    authors: Brutti P,De Santis F,Gubbiotti S

    更新日期:2008-06-15 00:00:00

  • Bayesian approach to average power calculations for binary regression models with misclassified outcomes.

    abstract::We develop a simulation-based procedure for determining the required sample size in binomial regression risk assessment studies when response data are subject to misclassification. A Bayesian average power criterion is used to determine a sample size that provides high probability, averaged over the distribution of po...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3505

    authors: Cheng D,Stamey JD,Branscum AJ

    更新日期:2009-02-28 00:00:00

  • The Peto odds ratio viewed as a new effect measure.

    abstract::Meta-analysis has generally been accepted as a fundamental tool for combining effect estimates from several studies. For binary studies with rare events, the Peto odds ratio (POR) method has become the relative effect estimator of choice. However, the POR leads to biased estimates for the OR when treatment effects are...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6301

    authors: Brockhaus AC,Bender R,Skipka G

    更新日期:2014-12-10 00:00:00

  • A missing composite covariate in survival analysis: a case study of the Chinese Longitudinal Health and Longevity Survey.

    abstract::We estimate a Cox proportional hazards model where one of the covariates measures the level of a subject's cognitive functioning by grading the total score obtained by the subject on the items of a questionnaire. A case study is presented where the sample includes partial respondents, who did not answer some questionn...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3773

    authors: Lagona F,Zhang Z

    更新日期:2010-01-30 00:00:00

  • A frailty model approach for regression analysis of multivariate current status data.

    abstract::This paper discusses regression analysis of multivariate current status failure time data (The Statistical Analysis of Interval-censoring Failure Time Data. Springer: New York, 2006), which occur quite often in, for example, tumorigenicity experiments and epidemiologic investigations of the natural history of a diseas...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3715

    authors: Chen MH,Tong X,Sun J

    更新日期:2009-11-30 00:00:00

  • Human disease cost network analysis.

    abstract::Diseases can be interconnected. In the recent years, there has been a surge of multidisease studies. Among them, HDN (human disease network) analysis takes a system perspective, examines the interconnections among diseases along with their individual properties, and has demonstrated great potential. Most of the existi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8472

    authors: Ma C,Li Y,Shia B,Ma S

    更新日期:2020-04-30 00:00:00

  • Quantifying the impact of between-study heterogeneity in multivariate meta-analyses.

    abstract::Measures that quantify the impact of heterogeneity in univariate meta-analysis, including the very popular I(2) statistic, are now well established. Multivariate meta-analysis, where studies provide multiple outcomes that are pooled in a single analysis, is also becoming more commonly used. The question of how to quan...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5453

    authors: Jackson D,White IR,Riley RD

    更新日期:2012-12-20 00:00:00