Data analysis, statistics and modelling

Section edited by Chiu-Hsieh (Paul) Hsu

This section considers manuscripts that investigate how statistical techniques can impact study results along with manuscripts that propose new statistical methods that have an application in medical research.

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  1. Research article

    Estimating correlation between multivariate longitudinal data in the presence of heterogeneity

    Estimating correlation coefficients among outcomes is one of the most important analytical tasks in epidemiological and clinical research. Availability of multivariate longitudinal data presents a unique oppor...

    Feng Gao, J. Philip Miller, Chengjie Xiong, Jingqin Luo, Julia A. Beiser, Ling Chen and Mae O. Gordon

    BMC Medical Research Methodology 2017 17:124

    Published on: 17 August 2017

  2. Research article

    Using classification and regression tree modelling to investigate response shift patterns in dentine hypersensitivity

    Dentine hypersensitivity (DH) affects people’s quality of life (QoL). However changes in the internal meaning of QoL, known as Response shift (RS) may undermine longitudinal assessment of QoL. This study aimed...

    Carolina Machuca, Mario V. Vettore, Marta Krasuska, Sarah R. Baker and Peter G. Robinson

    BMC Medical Research Methodology 2017 17:120

    Published on: 14 August 2017

  3. Research Article

    Choice of futility boundaries for group sequential designs with two endpoints

    In clinical trials, the opportunity for an early stop during an interim analysis (either for efficacy or for futility) may relevantly save time and financial resources. This is especially important, if the pla...

    Svenja Schüler, Meinhard Kieser and Geraldine Rauch

    BMC Medical Research Methodology 2017 17:119

    Published on: 8 August 2017

  4. Research Article

    A comparison of the conditional inference survival forest model to random survival forests based on a simulation study as well as on two applications with time-to-event data

    Random survival forest (RSF) models have been identified as alternative methods to the Cox proportional hazards model in analysing time-to-event data. These methods, however, have been criticised for the bias ...

    Justine B. Nasejje, Henry Mwambi, Keertan Dheda and Maia Lesosky

    BMC Medical Research Methodology 2017 17:115

    Published on: 28 July 2017

  5. Research article

    A comparison of multiple imputation methods for handling missing values in longitudinal data in the presence of a time-varying covariate with a non-linear association with time: a simulation study

    Missing data is a common problem in epidemiological studies, and is particularly prominent in longitudinal data, which involve multiple waves of data collection. Traditional multiple imputation (MI) methods (f...

    Anurika Priyanjali De Silva, Margarita Moreno-Betancur, Alysha Madhu De Livera, Katherine Jane Lee and Julie Anne Simpson

    BMC Medical Research Methodology 2017 17:114

    Published on: 25 July 2017

  6. Research Article

    Basic parametric analysis for a multi-state model in hospital epidemiology

    The extended illness-death model is a useful tool to study the risks and consequences of hospital-acquired infections (HAIs). The statistical quantities of interest are the transition-specific hazard rates and...

    Maja von Cube, Martin Schumacher and Martin Wolkewitz

    BMC Medical Research Methodology 2017 17:111

    Published on: 20 July 2017

  7. Research Article

    Binomial outcomes in dataset with some clusters of size two: can the dependence of twins be accounted for? A simulation study comparing the reliability of statistical methods based on a dataset of preterm infants

    The analysis of perinatal outcomes often involves datasets with some multiple births. These are datasets mostly formed of independent observations and a limited number of clusters of size two (twins) and maybe...

    Odile Sauzet and Janet L. Peacock

    BMC Medical Research Methodology 2017 17:110

    Published on: 20 July 2017

  8. Research Article

    A random effects meta-analysis model with Box-Cox transformation

    In a random effects meta-analysis model, true treatment effects for each study are routinely assumed to follow a normal distribution. However, normality is a restrictive assumption and the misspecification of ...

    Yusuke Yamaguchi, Kazushi Maruo, Christopher Partlett and Richard D. Riley

    BMC Medical Research Methodology 2017 17:109

    Published on: 19 July 2017

  9. Research Article

    Nonlinear joint models for individual dynamic prediction of risk of death using Hamiltonian Monte Carlo: application to metastatic prostate cancer

    Joint models of longitudinal and time-to-event data are increasingly used to perform individual dynamic prediction of a risk of event. However the difficulty to perform inference in nonlinear models and to cal...

    Solène Desmée, France Mentré, Christine Veyrat-Follet, Bernard Sébastien and Jérémie Guedj

    BMC Medical Research Methodology 2017 17:105

    Published on: 17 July 2017

  10. Research article

    Using structural equation modeling for network meta-analysis

    Network meta-analysis overcomes the limitations of traditional pair-wise meta-analysis by incorporating all available evidence into a general statistical framework for simultaneous comparisons of several treat...

    Yu-Kang Tu and Yun-Chun Wu

    BMC Medical Research Methodology 2017 17:104

    Published on: 14 July 2017

  11. Research article

    Accounting for treatment use when validating a prognostic model: a simulation study

    Prognostic models often show poor performance when applied to independent validation data sets. We illustrate how treatment use in a validation set can affect measures of model performance and present the uses...

    Romin Pajouheshnia, Linda M. Peelen, Karel G. M. Moons, Johannes B. Reitsma and Rolf H. H. Groenwold

    BMC Medical Research Methodology 2017 17:103

    Published on: 14 July 2017

  12. Research article

    Inverse probability of treatment-weighted competing risks analysis: an application on long-term risk of urinary adverse events after prostate cancer treatments

    To illustrate the 10-year risks of urinary adverse events (UAEs) among men diagnosed with prostate cancer and treated with different types of therapy, accounting for the competing risk of death.

    Charlotte A. Bolch, Haitao Chu, Stephanie Jarosek, Stephen R. Cole, Sean Elliott and Beth Virnig

    BMC Medical Research Methodology 2017 17:93

    Published on: 10 July 2017

  13. Research Article

    A DAG-based comparison of interventional effect underestimation between composite endpoint and multi-state analysis in cardiovascular trials

    Composite endpoints comprising hospital admissions and death are the primary outcome in many cardiovascular clinical trials. For statistical analysis, a Cox proportional hazards model for the time to first eve...

    Antje Jahn-Eimermacher, Katharina Ingel, Stella Preussler, Antoni Bayes-Genis and Harald Binder

    BMC Medical Research Methodology 2017 17:92

    Published on: 4 July 2017

  14. Research Article

    Simulation of complex data structures for planning of studies with focus on biomarker comparison

    There are a growing number of observational studies that do not only focus on single biomarkers for predicting an outcome event, but address questions in a multivariable setting. For example, when quantifying ...

    Andreas Schulz, Daniela Zöller, Stefan Nickels, Manfred E. Beutel, Maria Blettner, Philipp S. Wild and Harald Binder

    BMC Medical Research Methodology 2017 17:90

    Published on: 13 June 2017

  15. Research article

    Bayesian hierarchical piecewise regression models: a tool to detect trajectory divergence between groups in long-term observational studies

    Bayesian hierarchical piecewise regression (BHPR) modeling has not been previously formulated to detect and characterise the mechanism of trajectory divergence between groups of participants that have longitud...

    Marie-jeanne Buscot, Simon S. Wotherspoon, Costan G. Magnussen, Markus Juonala, Matthew A. Sabin, David P. Burgner, Terho Lehtimäki, Jorma S. A. Viikari, Nina Hutri-Kähönen, Olli T. Raitakari and Russell J. Thomson

    BMC Medical Research Methodology 2017 17:86

    Published on: 6 June 2017

  16. Research article

    Methodologic considerations in the design and analysis of nested case-control studies: association between cytokines and postoperative delirium

    The nested case-control study (NCC) design within a prospective cohort study is used when outcome data are available for all subjects, but the exposure of interest has not been collected, and is difficult or p...

    Long H. Ngo, Sharon K. Inouye, Richard N. Jones, Thomas G. Travison, Towia A. Libermann, Simon T. Dillon, George A. Kuchel, Sarinnapha M. Vasunilashorn, David C. Alsop and Edward R. Marcantonio

    BMC Medical Research Methodology 2017 17:88

    Published on: 6 June 2017

  17. Research article

    Robust estimation of the expected survival probabilities from high-dimensional Cox models with biomarker-by-treatment interactions in randomized clinical trials

    Thanks to the advances in genomics and targeted treatments, more and more prediction models based on biomarkers are being developed to predict potential benefit from treatments in a randomized clinical trial. ...

    Nils Ternès, Federico Rotolo and Stefan Michiels

    BMC Medical Research Methodology 2017 17:83

    Published on: 22 May 2017

  18. Research article

    Changes in affect after completing a mailed survey about trauma: two pre- and post-test studies in former disability applicants for posttraumatic stress disorder

    One potential concern with using mailed surveys containing trauma-related content is the possibility of re-traumatizing survivors without a trained mental health professional present. Prior research provides i...

    Maureen Murdoch, Shannon Marie Kehle-Forbes and Melissa Ruth Partin

    BMC Medical Research Methodology 2017 17:81

    Published on: 10 May 2017

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