Parametric multistate survival models: Flexible modelling allowing transition-specific distributions with application to estimating clinically useful measures of effect differences.

Abstract:

:Multistate models are increasingly being used to model complex disease profiles. By modelling transitions between disease states, accounting for competing events at each transition, we can gain a much richer understanding of patient trajectories and how risk factors impact over the entire disease pathway. In this article, we concentrate on parametric multistate models, both Markov and semi-Markov, and develop a flexible framework where each transition can be specified by a variety of parametric models including exponential, Weibull, Gompertz, Royston-Parmar proportional hazards models or log-logistic, log-normal, generalised gamma accelerated failure time models, possibly sharing parameters across transitions. We also extend the framework to allow time-dependent effects. We then use an efficient and generalisable simulation method to calculate transition probabilities from any fitted multistate model, and show how it facilitates the simple calculation of clinically useful measures, such as expected length of stay in each state, and differences and ratios of proportion within each state as a function of time, for specific covariate patterns. We illustrate our methods using a dataset of patients with primary breast cancer. User-friendly Stata software is provided.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Crowther MJ,Lambert PC

doi

10.1002/sim.7448

subject

Has Abstract

pub_date

2017-12-20 00:00:00

pages

4719-4742

issue

29

eissn

0277-6715

issn

1097-0258

journal_volume

36

pub_type

杂志文章
  • The questioning statistician.

    abstract::Effective statistical help to biological and medical research demands thorough involvement of the statistician. The breadth of his activities can be illustrated by considering the questions he needs to discuss with his scientific colleagues in the course of planning a comparative experiment. The paper presents and com...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章

    doi:10.1002/sim.4780010103

    authors: Finney DJ

    更新日期:1982-01-01 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

  • Technical uncertainty in the back-calculation of occupational exposure to dioxins.

    abstract::Members of a cohort of workers in chemical industry (the so-called Boehringer cohort) exposed to 2, 3, 7, 8-tetrachlorodibenzo-para-dioxin (TCDD) from 1950 to 1984 were subject in the years 1985-1986 and 1992-1994 to an extensive biomonitoring programme on the TCDD levels of the individual workers. For establishing a ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3074

    authors: Heinzl H,Mittlböck M,Edler L

    更新日期:2008-05-30 00:00:00

  • Computational methods in medical decision making: to screen or not to screen?

    abstract::Screening for a disease such as cancer is often regarded as a beneficial and successful strategy for reducing mortality. However, as with any clinical treatment or intervention, benefit cannot be assumed, and screening can entail both costs and harms, so the screening as a 'treatment' must undergo evaluation. An evalu...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2037

    authors: Kafadar K,Prorok PC

    更新日期:2005-02-28 00:00:00

  • Constrained S-estimators for linear mixed effects models with covariance components.

    abstract::Linear mixed effects (LME) models are increasingly used for analyses of biological and biomedical data. When the multivariate normal assumption is not adequate for an LME model, then a robust estimation approach is preferable to the maximum likelihood one. M-estimators were considered before for robust estimation of t...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4169

    authors: Chervoneva I,Vishnyakov M

    更新日期:2011-06-30 00:00:00

  • Flexible longitudinal linear mixed models for multiple censored responses data.

    abstract::In biomedical studies and clinical trials, repeated measures are often subject to some upper and/or lower limits of detection. Hence, the responses are either left or right censored. A complication arises when more than one series of responses is repeatedly collected on each subject at irregular intervals over a perio...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8017

    authors: Lachos VH,A Matos L,Castro LM,Chen MH

    更新日期:2019-03-15 00:00:00

  • Joint analysis of multi-level repeated measures data and survival: an application to the end stage renal disease (ESRD) data.

    abstract::Shared random effects models have been increasingly common in the joint analyses of repeated measures (e.g. CD4 counts, hemoglobin levels) and a correlated failure time such as death. In this paper we study several shared random effects models in the multi-level repeated measures data setting with dependent failure ti...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3392

    authors: Liu L,Ma JZ,O'Quigley J

    更新日期:2008-11-29 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

  • Seasonal and other short-term influences on United States AIDS incidence.

    abstract::This paper models monthly AIDS diagnosis counts in terms of smooth secular trend, calendar month effects, and the number of workdays per month. A parameterization of month effects allows separation of true seasonal effects from a linear trend over the calendar year and an arbitrary June effect. There is strong evidenc...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780131905

    authors: Bacchetti P

    更新日期:1994-10-15 00:00:00

  • Methods for epidemiologic analyses of multiple exposures: a review and comparative study of maximum-likelihood, preliminary-testing, and empirical-Bayes regression.

    abstract::Many epidemiologic investigations are designed to study the effects of multiple exposures. Most of these studies are analysed either by fitting a risk-regression model with all exposures forced in the model, or by using a preliminary-testing algorithm, such as stepwise regression, to produce a smaller model. Research ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/sim.4780120802

    authors: Greenland S

    更新日期:1993-04-30 00:00:00

  • Study control, violators, inclusion criteria and defining explanatory and pragmatic trials.

    abstract::Important differences between explanatory and pragmatic studies were originally argued by Schwartz and Lellouch. Three important differences between the two types of study involve study control, study violators and inclusion criteria. It was originally argued that explanatory studies are highly controlled, and pragmat...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1120

    authors: McMahon AD

    更新日期:2002-05-30 00:00:00

  • Multivariate test power approximations for balanced linear mixed models in studies with missing data.

    abstract::Multilevel and longitudinal studies are frequently subject to missing data. For example, biomarker studies for oral cancer may involve multiple assays for each participant. Assays may fail, resulting in missing data values that can be assumed to be missing completely at random. Catellier and Muller proposed a data ana...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6811

    authors: Ringham BM,Kreidler SM,Muller KE,Glueck DH

    更新日期:2016-07-30 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

  • Identifying optimal risk windows for self-controlled case series studies of vaccine safety.

    abstract::In vaccine safety studies, subjects are considered at increased risk for adverse events for a period of time after vaccination known as risk window. To our knowledge, risk windows for vaccine safety studies have tended to be pre-defined and not to use information from the current study. Inaccurate specification of the...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4125

    authors: Xu S,Zhang L,Nelson JC,Zeng C,Mullooly J,McClure D,Glanz J

    更新日期:2011-03-30 00:00:00

  • Establishing the relationship between nurse staffing and hospital mortality using a clustered discrete-time logistic model.

    abstract::Studies based on aggregated hospital outcome data have established that there is a relationship between nurse staffing and adverse events. However, this result could not be confirmed in Belgium where 96 per cent of the variability of nurse staffing levels over nursing units (belonging to different hospitals) is explai...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3756

    authors: Diya L,Lesaffre E,Van den Heede K,Sermeus W,Vleugels A

    更新日期:2010-03-30 00:00:00

  • A random forest approach for competing risks based on pseudo-values.

    abstract::Random forest is a supervised learning method that combines many classification or regression trees for prediction. Here we describe an extension of the random forest method for building event risk prediction models in survival analysis with competing risks. In case of right-censored data, the event status at the pred...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5775

    authors: Mogensen UB,Gerds TA

    更新日期:2013-08-15 00:00:00

  • Identifying the types of missingness in quality of life data from clinical trials.

    abstract::This paper discusses methods of identifying the types of missingness in quality of life (QOL) data in cancer clinical trials. The first approach involves collecting information on why the QOL questionnaires were not completed. Based on the reasons provided one may be able to distinguish the mechanisms causing missing ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,评审

    doi:10.1002/(sici)1097-0258(19980315/15)17:5/7<739::ai

    authors: Curran D,Bacchi M,Schmitz SF,Molenberghs G,Sylvester RJ

    更新日期:1998-03-15 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

  • An alternative index for assessing profile similarity in bioequivalence trials.

    abstract::In a typical bioequivalence trial, summary measures of the plasma concentration versus time profile are used to compare two formulations of a drug product. Commonly used measures include area under the curve (AUC), maximum plasma concentration (C(max)) and time to maximum concentration (T(max)). Equivalence of these s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20001030)19:20<2855::aid-sim550>

    authors: Mauger DT,Chinchilli VM

    更新日期:2000-10-30 00:00:00

  • Scientific considerations for assessing biosimilar products.

    abstract::The problem for assessing biosimilarity and drug interchangeability of follow-on biologics (biosimilar products) is studied. Unlike the generic products, the development of biosimilar products is much more complicated because of fundamental differences in functional structures and manufacturing processes. As a result,...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5571

    authors: Chow SC,Wang J,Endrenyi L,Lachenbruch PA

    更新日期:2013-02-10 00:00:00

  • The effects of measurement error in response variables and tests of association of explanatory variables in change models.

    abstract::Biomedical studies often measure variables with error. Examples in the literature include investigation of the association between the change in some outcome variable (blood pressure, cholesterol level etc.) and a set of explanatory variables (age, smoking status etc.). Typically, one fits linear regression models to ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19981130)17:22<2597::aid-s

    authors: Yanez ND 3rd,Kronmal RA,Shemanski LR

    更新日期:1998-11-30 00:00:00

  • Multilevel latent variable models for global health-related quality of life assessment.

    abstract::Quality of life (QOL) assessment is a key component of many clinical studies and frequently requires the use of single global summary measures that capture the overall balance of findings from a potentially wide-ranging assessment of QOL issues. We propose and evaluate an irregular multilevel latent variable model sui...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4455

    authors: Kifley A,Heller GZ,Beath KJ,Bulger D,Ma J,Gebski V

    更新日期:2012-05-20 00:00:00

  • Analysis of mortality rates via marginal extended quasi-likelihood.

    abstract::We use a mixed Poisson regression model with extra variation to analyse mortality data cross-classified by age and geographic region. We use estimates of dispersion parameter and fixed effects parameters, obtained by maximizing a marginal quasi-likelihood function, to estimate mortality rates in an empirical Bayes man...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(SICI)1097-0258(19960715)15:13<1397::AID-S

    authors: Lu WS,Tsutakawa RK

    更新日期:1996-07-15 00:00:00

  • Investigating the prediction ability of survival models based on both clinical and omics data: two case studies.

    abstract::In biomedical literature, numerous prediction models for clinical outcomes have been developed based either on clinical data or, more recently, on high-throughput molecular data (omics data). Prediction models based on both types of data, however, are less common, although some recent studies suggest that a suitable c...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6246

    authors: De Bin R,Sauerbrei W,Boulesteix AL

    更新日期:2014-12-30 00:00:00

  • Clinical trials: some reflections.

    abstract::The principles and historical development of clinical trials are outlined briefly. Some issues that extend the basic philosophy first put forward by Sir Austin Bradford Hill 40 years ago and that will need consideration in the future application of clinical trials are discussed. ...

    journal_title:Statistics in medicine

    pub_type: 传,历史文章,杂志文章

    doi:10.1002/sim.4780010414

    authors: Holland WW,Breeze E,Swan AV

    更新日期:1982-10-01 00:00:00

  • On the estimation of total variability in assay validation.

    abstract::In the pharmaceutical industry, an assay method is considered validated if the accuracy and precision for an assay meet some acceptable limits. This paper discusses the assessment of assay precision in terms of the estimation of total variability of an assay from a one-way random effects model which is often considere...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780101006

    authors: Chow SC,Tse SK

    更新日期:1991-10-01 00:00:00

  • A multiple imputation strategy for incomplete longitudinal data.

    abstract::Longitudinal studies are commonly used to study processes of change. Because data are collected over time, missing data are pervasive in longitudinal studies, and complete ascertainment of all variables is rare. In this paper a new imputation strategy for completing longitudinal data sets is proposed. The proposed met...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.740

    authors: Landrum MB,Becker MP

    更新日期:2001-09-15 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

  • Sample size calculation for stepped wedge and other longitudinal cluster randomised trials.

    abstract::The sample size required for a cluster randomised trial is inflated compared with an individually randomised trial because outcomes of participants from the same cluster are correlated. Sample size calculations for longitudinal cluster randomised trials (including stepped wedge trials) need to take account of at least...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7028

    authors: Hooper R,Teerenstra S,de Hoop E,Eldridge S

    更新日期:2016-11-20 00:00:00

  • A method to test for a recent increase in HIV-1 seroconversion incidence: results from the Multicenter AIDS Cohort Study (MACS).

    abstract::We have formulated the problem of determining whether there has been an upturn in HIV-1 seroconversion incidence over the first five years of follow-up in the Multicenter AIDS Cohort Study (MACS) as that of locating the minimum of a quadratic regression or examination of two-knot piecewise spline models. Under a quadr...

    journal_title:Statistics in medicine

    pub_type: 杂志文章,多中心研究

    doi:10.1002/sim.4780120207

    authors: Zhou SY,Kingsley LA,Taylor JM,Chmiel JS,He DY,Hoover DR

    更新日期:1993-01-30 00:00:00