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. Debate

    Towards evidence-based computational statistics: lessons from clinical research on the role and design of real-data benchmark studies

    The goal of medical research is to develop interventions that are in some sense superior, with respect to patient outcome, to interventions currently in use. Similarly, the goal of research in methodological c...

    Anne-Laure Boulesteix, Rory Wilson and Alexander Hapfelmeier

    BMC Medical Research Methodology 2017 17:138

    Published on: 9 September 2017

  2. Research article

    Monte Carlo simulation-based estimation for the minimum mortality temperature in temperature-mortality association study

    Rich literature has reported that there exists a nonlinear association between temperature and mortality. One important feature in the temperature-mortality association is the minimum mortality temperature (MM...

    Whanhee Lee, Ho Kim, Sunghee Hwang, Antonella Zanobetti, Joel D. Schwartz and Yeonseung Chung

    BMC Medical Research Methodology 2017 17:137

    Published on: 7 September 2017

  3. Research article

    Addressing data privacy in matched studies via virtual pooling

    Data confidentiality and shared use of research data are two desirable but sometimes conflicting goals in research with multi-center studies and distributed data. While ideal for straightforward analysis, conf...

    P. Saha-Chaudhuri and C.R. Weinberg

    BMC Medical Research Methodology 2017 17:136

    Published on: 7 September 2017

  4. Research article

    Trends in the number and the quality of trial protocols involving children submitted to a French Institutional Review Board

    There is a great need for high quality clinical research for children. The European Pediatric Regulation aimed to improve the quality of clinical trials in order to increase the availability of treatments for ...

    Isabelle Gautier, Perrine Janiaud, Nelly Rollet, Nicolas André, Michel Tsimaratos, Catherine Cornu, Salma Malik, Stéphanie Gentile and Behrouz Kassaï

    BMC Medical Research Methodology 2017 17:130

    Published on: 23 August 2017

  5. Research article

    Using Bayesian statistics to estimate the likelihood a new trial will demonstrate the efficacy of a new treatment

    The common frequentist approach is limited in providing investigators with appropriate measures for conducting a new trial. To answer such important questions and one has to look at Bayesian statistics.

    David J. Biau, Samuel Boulezaz, Laurent Casabianca, Moussa Hamadouche, Philippe Anract and Sylvie Chevret

    BMC Medical Research Methodology 2017 17:128

    Published on: 22 August 2017

  6. Technical advance

    Methods for significance testing of categorical covariates in logistic regression models after multiple imputation: power and applicability analysis

    Multiple imputation is a recommended method to handle missing data. For significance testing after multiple imputation, Rubin’s Rules (RR) are easily applied to pool parameter estimates. In a logistic regressi...

    Iris Eekhout, Mark A. van de Wiel and Martijn W. Heymans

    BMC Medical Research Methodology 2017 17:129

    Published on: 22 August 2017

  7. Research article

    Statistical methods for elimination of guarantee-time bias in cohort studies: a simulation study

    Aspirin has been considered to be beneficial in preventing cardiovascular diseases and cancer. Several pharmaco-epidemiology cohort studies have shown protective effects of aspirin on diseases using various st...

    In Sung Cho, Ye Rin Chae, Ji Hyeon Kim, Hae Rin Yoo, Suk Yong Jang, Gyu Ri Kim and Chung Mo Nam

    BMC Medical Research Methodology 2017 17:126

    Published on: 22 August 2017

  8. 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

  9. 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

  10. 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

  11. 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

  12. 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

  13. 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

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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

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