Parameterization of treatment effects for meta-analysis in multi-state Markov models.

Abstract:

:Standard approaches to analysis of randomized controlled trials (RCTs) using Markov models make it difficult to generalize treatment effects to new patient groups and synthesize evidence across trials. This paper demonstrates how pair-wise and mixed treatment comparison meta-analysis can be applied to event history data for disease progression reported by RCTs. The data, in the form of aggregated discrete time transitions, have a multi-nomial likelihood. In order for evidence synthesis to be performed a structured approach to modelling the differences in the effects of the different treatments must be taken. A multi-state continuous-time Markov model similar to others used in published economic evaluations of asthma treatments is developed, with transition rates related to the likelihood via Kolmogorov's forward equations. The formulation in terms of rates allows a flexible characterization of summary treatment effects. These ideas are applied to an illustrative data set consisting of a set of five trials comparing eight different treatments for asthma. A range of models is developed in which the relative treatment effects act on forward, backward transitions, or both, and models are compared using the DIC. Bayesian inferential techniques are used and the parameters are estimated using MCMC simulation in WinBUGS. An intuitively appealing mechanism of action involving a single parameter acting on all backward transitions was identified for the relative effects of the treatments, which allowed the estimation of a pooled treatment effect, allowing us to rank the different treatment options within each connected evidence network to ascertain which were the most clinically effective.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Price MJ,Welton NJ,Ades AE

doi

10.1002/sim.4059

subject

Has Abstract

pub_date

2011-01-30 00:00:00

pages

140-51

issue

2

eissn

0277-6715

issn

1097-0258

journal_volume

30

pub_type

杂志文章
  • 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

  • Accounting for competing risks in randomized controlled trials: a review and recommendations for improvement.

    abstract::In studies with survival or time-to-event outcomes, a competing risk is an event whose occurrence precludes the occurrence of the primary event of interest. Specialized statistical methods must be used to analyze survival data in the presence of competing risks. We conducted a review of randomized controlled trials wi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/sim.7215

    authors: Austin PC,Fine JP

    更新日期:2017-04-15 00:00:00

  • Modelling of viral dynamics in hepatitis B and hepatitis C clinical trials.

    abstract::In the recent years, studies of hepatitis B (HBV) and hepatitis C virus (HCV) dynamics have drawn great attention as they provide insight into the process of virus elimination/production and of infected cells decay during antiviral treatment. Estimates of viral dynamic parameters may be used to determine the lifetime ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3457

    authors: Sypsa V,Hatzakis A

    更新日期:2008-12-30 00:00:00

  • Semiparametric Bayesian variable selection for gene-environment interactions.

    abstract::Many complex diseases are known to be affected by the interactions between genetic variants and environmental exposures beyond the main genetic and environmental effects. Study of gene-environment (G×E) interactions is important for elucidating the disease etiology. Existing Bayesian methods for G×E interaction studie...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8434

    authors: Ren J,Zhou F,Li X,Chen Q,Zhang H,Ma S,Jiang Y,Wu C

    更新日期:2020-02-28 00:00:00

  • Conflicts of interest in data monitoring of industry versus publicly financed clinical trials.

    abstract::The FDA Guidance, while highly appropriate for industry sponsored trials, need not be imposed on publicly (e.g. NIH) financed clinical trials. While the potential for conflicts of interest exist in the latter, they are in general manageable and pose an acceptable low risk of threatening the integrity of a study. Howev...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1787

    authors: Lachin JM

    更新日期:2004-05-30 00:00:00

  • Proportion cured and mean log survival time as functions of tumour size.

    abstract::We obtained maximum likelihood estimates (MLEs) of the proportion cured pi c and mean log survival time mu t for a sample of 4355 patients with intraocular melanoma whose survival times subsequent to treatment were assumed to follow a log-normal distribution. Following stratification by tumour size, MLEs of pi c and m...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780090814

    authors: Gamel JW,McLean IW,Rosenberg SH

    更新日期:1990-08-01 00:00:00

  • 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 w...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4085

    authors: Pencina MJ,D'Agostino RB Sr,Steyerberg EW

    更新日期:2011-01-15 00:00:00

  • Simple methods for checking for possible errors in reported odds ratios, relative risks and confidence intervals.

    abstract::Meta-analyses of data from epidemiological studies are often based on odds ratios (ORs) or relative risks (RRs) and their 95 per cent confidence intervals (CIs) as reported by the authors. Where possible ORs, RRs and CIs should be checked against the source data. Some simple methods are presented for checking the vali...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19990815)18:15<1973::aid-s

    authors: Lee PN

    更新日期:1999-08-15 00:00:00

  • Simultaneous estimation of intrarater and interrater agreement for multiple raters under order restrictions for a binary trait.

    abstract::It is valuable in many studies to assess both intrarater and interrater agreement. Most measures of intrarater agreement do not adjust for unequal estimates of prevalence between the separate rating occasions for a given rater and measures of interrater agreement typically ignore data from the second set of assessment...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1138

    authors: Lester Kirchner H,Lemke JH

    更新日期:2002-06-30 00:00:00

  • Adaptive increase in sample size when interim results are promising: a practical guide with examples.

    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

    authors: Mehta CR,Pocock SJ

    更新日期:2011-12-10 00:00:00

  • Measurement error in continuous endpoints in randomised trials: Problems and solutions.

    abstract::In randomised trials, continuous endpoints are often measured with some degree of error. This study explores the impact of ignoring measurement error and proposes methods to improve statistical inference in the presence of measurement error. Three main types of measurement error in continuous endpoints are considered:...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8359

    authors: Nab L,Groenwold RHH,Welsing PMJ,van Smeden M

    更新日期:2019-11-30 00:00:00

  • Estimating adjusted risk difference (RD) and number needed to treat (NNT) measures in the Cox regression model.

    abstract::In medical research, risk difference (RD) and number needed to treat (NNT) measures for survival times have been mainly proposed without consideration of covariates. In this paper, we develop adjusted RD and NNT measures for use in observational studies with survival time outcomes within the framework of the Cox propo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3793

    authors: Laubender RP,Bender R

    更新日期:2010-03-30 00:00:00

  • A boundary-optimized rejection region test for the two-sample binomial problem.

    abstract::Testing the equality of 2 proportions for a control group versus a treatment group is a well-researched statistical problem. In some settings, there may be strong historical data that allow one to reliably expect that the control proportion is one, or nearly so. While one-sample tests or comparisons to historical cont...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7579

    authors: Gabriel EE,Nason M,Fay MP,Follmann DA

    更新日期:2018-03-30 00:00:00

  • Predicting analysis times in randomized clinical trials.

    abstract::Randomized clinical trial designs commonly include one or more planned interim analyses. At these times an external monitoring committee reviews the accumulated data and determines whether it is scientifically and ethically appropriate for the study to continue. With failure-time endpoints, it is common to schedule an...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.843

    authors: Bagiella E,Heitjan DF

    更新日期:2001-07-30 00:00:00

  • Conditional power and predictive power based on right censored data with supplementary auxiliary information.

    abstract::Conditional power and predictive power provide estimates of the probability of success at the end of the trial based on the information from the interim analysis. The observed value of the time to event endpoint at the interim analysis could be biased for the true treatment effect due to early censoring, leading to a ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7673

    authors: Sun L,Wan Y

    更新日期:2018-08-15 00:00:00

  • Comparing the performance of two indices for spatial model selection: application to two mortality data.

    abstract::The statistical analysis of spatially correlated data has become an important scientific research topic lately. The analysis of the mortality or morbidity rates observed at different areas may help to decide if people living in certain locations are considered at higher risk than others. Once the statistical model for...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20000730)19:14<1915::aid-sim503>

    authors: Hsiao CK,Tzeng JY,Wang CH

    更新日期:2000-07-30 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

  • 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

  • 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

  • Joint analysis of mixed types of outcomes with latent variables.

    abstract::We propose a joint modeling approach to investigating the observed and latent risk factors of mixed types of outcomes. The proposed model comprises three parts. The first part is an exploratory factor analysis model that summarizes latent factors through multiple observed variables. The second part is a proportional h...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8840

    authors: Pan D,Wei Y,Song X

    更新日期:2020-12-09 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

  • Policy developments in regulatory approval.

    abstract::Although radical changes in drug regulation are rare (e.g., the Federal Food, Drug and Cosmetic Act of 1938 and the 1962 amendment to the Act creating an effectiveness requirement), regulations and guidance do evolve significantly in the face of new problems and accumulating experience. Recent changes have been driven...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1298

    authors: Temple R

    更新日期:2002-10-15 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

  • A recycling framework for the construction of Bonferroni-based multiple tests.

    abstract::In this paper we describe Bonferroni-based multiple testing procedures (MTPs) as strategies to split and recycle test mass. Here, 'test mass' refers to (parts of) the nominal level alpha at which the family-wise error rate is controlled. Briefly, test mass is split between different null hypotheses, and whenever a nul...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3513

    authors: Burman CF,Sonesson C,Guilbaud O

    更新日期:2009-02-28 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

  • Familial associations of lipid profiles: a generalized estimating equations approach.

    abstract::Elevated plasma levels of apolipoproteins A1 (apoA1) and B (apoB) are important protective factors and risk factors, respectively, for atherosclerosis and coronary heart disease. It is well known that both apoA1 and apoB reveal strong familial aggregation. Our goal was to investigate whether exogenous variables influe...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20001230)19:24<3345::aid-sim829>

    authors: Ziegler A,Kastner C,Brunner D,Blettner M

    更新日期:2000-12-30 00:00:00

  • Survival analysis of hierarchical learning curves in assessment of cardiac device and procedural safety.

    abstract::Many Americans rely on cardiac surgical procedures and devices such as pacemakers and thrombolytic catheters to treat or manage their cardiovascular diseases. However, the failure of these cardiac devices and procedures could have grave consequences. One reason cardiac devices tended to fail was due to physician error...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7906

    authors: Govindarajulu U,Bedi S,Kluger A,Resnic F

    更新日期:2018-12-10 00:00:00

  • Approximate multinormal probabilities applied to correlated multiple endpoints in clinical trials.

    abstract::Clinical trials with multiple endpoints incur increased familywise type I errors. The Bonferroni correction is a common method used to modify the p-values to account for multiple significance testing. For independent endpoints the Bonferroni method is slightly conservative whereas with high correlation the conservatis...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780100712

    authors: James S

    更新日期:1991-07-01 00:00:00

  • Measurement error correction for nutritional exposures with correlated measurement error: use of the method of triads in a longitudinal setting.

    abstract::Nutritional exposures are often measured with considerable error in commonly used surrogate instruments such as the food frequency questionnaire (FFQ) (denoted by Q(i) for the ith subject). The error can be both systematic and random. The diet record (DR) denoted by R(i) for the ith subject is considered an alloyed go...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3238

    authors: Rosner B,Michels KB,Chen YH,Day NE

    更新日期:2008-08-15 00:00:00

  • A score test for establishing non-inferiority with respect to short-term survival in two-sample comparisons with identical proportions of long-term survivors.

    abstract::In recent years randomized trials designed to establish non-inferiority of a new treatment as compared to a standard one have been more widely used. Two-sample statistics have been proposed for this equivalence testing problem. However, they are not suited to situations where a long-term survivor fraction is expected....

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1453

    authors: Broët P,Tubert-Bitter P,De Rycke Y,Moreau T

    更新日期:2003-03-30 00:00:00