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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. Content type: Research article

    Parametric distributions based on individual patient data can be used to represent both stochastic and parameter uncertainty. Although general guidance is available on how parameter uncertainty should be accou...

    Authors: Koen Degeling, Maarten J. IJzerman, Miriam Koopman and Hendrik Koffijberg

    Citation: BMC Medical Research Methodology 2017 17:170

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  2. Content type: Research article

    Diagnostic tests are performed in a subset of the population who are at higher risk, resulting in undiagnosed cases among those who do not receive the test. This poses a challenge for estimating the prevalence...

    Authors: Nanhua Zhang, Si Cheng, Lilliam Ambroggio, Todd A. Florin and Maurizio Macaluso

    Citation: BMC Medical Research Methodology 2017 17:168

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  3. Content type: Research article

    The variable “small for gestational age,” frequently defined as birth weight below the 10th percentile in a gestational age and sex-normalized population, is nowadays generally perceived as a more adequate mea...

    Authors: Geneviève Lefebvre and Mariia Samoilenko

    Citation: BMC Medical Research Methodology 2017 17:165

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    The Correction to this article has been published in BMC Medical Research Methodology 2018 18:17

  4. Content type: Research article

    The bias caused by drop-out is an important factor in large population-based epidemiological studies. Many studies account for it by weighting their longitudinal data, but to date there is no detailed final ap...

    Authors: Steffen C. E. Schmidt and Alexander Woll

    Citation: BMC Medical Research Methodology 2017 17:164

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  5. Content type: Research article

    Missing data may seriously compromise inferences from randomised clinical trials, especially if missing data are not handled appropriately. The potential bias due to missing data depends on the mechanism causi...

    Authors: Janus Christian Jakobsen, Christian Gluud, Jørn Wetterslev and Per Winkel

    Citation: BMC Medical Research Methodology 2017 17:162

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  6. Content type: Research article

    The Marginal Structural Cox Model (Cox-MSM), an alternative approach to handle time-dependent confounder, was introduced for survival analysis and applied to estimate the joint causal effect of two time-depend...

    Authors: Clovis Lusivika-Nzinga, Hana Selinger-Leneman, Sophie Grabar, Dominique Costagliola and Fabrice Carrat

    Citation: BMC Medical Research Methodology 2017 17:160

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  7. Content type: Research article

    In healthcare research, outcomes with skewed probability distributions are common. Sample size calculations for such outcomes are typically based on estimates on a transformed scale (e.g. log) which may someti...

    Authors: Aidan G. O’Keeffe, Gareth Ambler and Julie A. Barber

    Citation: BMC Medical Research Methodology 2017 17:157

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  8. Content type: Research article

    The follow-up rate, a standard index of the completeness of follow-up, is important for assessing the validity of a cohort study. A common method for estimating the follow-up rate, the “Percentage Method”, def...

    Authors: Xiaonan Xue, Ilir Agalliu, Mimi Y. Kim, Tao Wang, Juan Lin, Reza Ghavamian and Howard D. Strickler

    Citation: BMC Medical Research Methodology 2017 17:155

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  9. Content type: Research article

    Exploratory preclinical, as well as clinical trials, may involve a small number of patients, making it difficult to calculate and analyze the pharmacokinetic (PK) parameters, especially if the PK parameters sh...

    Authors: Sudeep Pradhan, Byungjeong Song, Jaeyeon Lee, Jung-woo Chae, Kyung Im Kim, Hyun-moon Back, Nayoung Han, Kwang-il Kwon and Hwi-yeol Yun

    Citation: BMC Medical Research Methodology 2017 17:154

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  10. Content type: Research article

    It is important to quantify the dose response for a drug in phase 2a clinical trials so the optimal doses can then be selected for subsequent late phase trials. In a phase 2a clinical trial of new lead drug be...

    Authors: Feng Liu, Stephen J. Walters and Steven A. Julious

    Citation: BMC Medical Research Methodology 2017 17:149

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  11. Content type: Research Article

    The use of health-related quality of life (HRQoL) as an endpoint in cancer clinical trials is growing rapidly. Hence, research into the statistical approaches used to analyze HRQoL data is of major importance,...

    Authors: Antoine Barbieri, Jean Peyhardi, Thierry Conroy, Sophie Gourgou, Christian Lavergne and Caroline Mollevi

    Citation: BMC Medical Research Methodology 2017 17:148

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  12. Content type: Research article

    It is challenging for current statistical models to predict clinical progression of Parkinson’s disease (PD) because of the involvement of multi-domains and longitudinal data.

    Authors: Ming Wang, Zheng Li, Eun Young Lee, Mechelle M. Lewis, Lijun Zhang, Nicholas W. Sterling, Daymond Wagner, Paul Eslinger, Guangwei Du and Xuemei Huang

    Citation: BMC Medical Research Methodology 2017 17:147

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  13. Content type: Debate

    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...

    Authors: Anne-Laure Boulesteix, Rory Wilson and Alexander Hapfelmeier

    Citation: BMC Medical Research Methodology 2017 17:138

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  14. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:137

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  15. Content type: Research article

    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...

    Authors: P. Saha-Chaudhuri and C.R. Weinberg

    Citation: BMC Medical Research Methodology 2017 17:136

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  16. Content type: Research article

    Multiple imputation is a popular approach to handling missing data in medical research, yet little is known about its applicability for estimating the relative risk. Standard methods for imputing incomplete bi...

    Authors: Thomas R. Sullivan, Katherine J. Lee, Philip Ryan and Amy B. Salter

    Citation: BMC Medical Research Methodology 2017 17:134

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  17. Content type: Research article

    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 ...

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

    Citation: BMC Medical Research Methodology 2017 17:130

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  18. Content type: Research article

    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.

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

    Citation: BMC Medical Research Methodology 2017 17:128

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  19. Content type: Technical advance

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:129

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  20. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:126

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  21. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:124

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  22. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:120

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  23. Content type: Research Article

    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 ...

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

    Citation: BMC Medical Research Methodology 2017 17:115

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  24. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:114

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  25. Content type: Research Article

    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...

    Authors: Odile Sauzet and Janet L. Peacock

    Citation: BMC Medical Research Methodology 2017 17:110

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  26. Content type: Research Article

    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 ...

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

    Citation: BMC Medical Research Methodology 2017 17:109

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  27. Content type: Research Article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:105

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  28. Content type: Technical advance

    The sample size required to power a study to a nominal level in a paired comparative diagnostic accuracy study, i.e. studies in which the diagnostic accuracy of two testing procedures is compared relative to a...

    Authors: Gareth P. J. McCray, Andrew C. Titman, Paula Ghaneh and Gillian A. Lancaster

    Citation: BMC Medical Research Methodology 2017 17:102

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  29. Content type: Research article

    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...

    Authors: Yu-Kang Tu and Yun-Chun Wu

    Citation: BMC Medical Research Methodology 2017 17:104

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  30. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:103

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  31. Content type: Research article

    Complex interventions such as self-management courses are difficult to evaluate due to the many interacting components. The way complex interventions are delivered can influence the effect they have for patien...

    Authors: G. Wojewodka, S. Hurley, S. J. C. Taylor, A. J. Noble, L. Ridsdale and L. H. Goldstein

    Citation: BMC Medical Research Methodology 2017 17:100

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  32. Content type: Research article

    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.

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

    Citation: BMC Medical Research Methodology 2017 17:93

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  33. Content type: Research Article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:92

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  34. Content type: Research Article

    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 ...

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

    Citation: BMC Medical Research Methodology 2017 17:90

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  35. Content type: Research article

    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...

    Authors: 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

    Citation: BMC Medical Research Methodology 2017 17:86

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  36. Content type: Research article

    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...

    Authors: 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

    Citation: BMC Medical Research Methodology 2017 17:88

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  37. Content type: Research article

    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. ...

    Authors: Nils Ternès, Federico Rotolo and Stefan Michiels

    Citation: BMC Medical Research Methodology 2017 17:83

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  38. Content type: Research article

    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...

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

    Citation: BMC Medical Research Methodology 2017 17:81

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  39. Content type: Research Article

    Relative survival analysis is a subfield of survival analysis where competing risks data are observed, but the causes of death are unknown. A first step in the analysis of such data is usually the estimation o...

    Authors: Klemen Pavlič and Maja Pohar Perme

    Citation: BMC Medical Research Methodology 2017 17:79

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  40. Content type: Research article

    Double-adjustment can be used to remove confounding if imbalance exists after propensity score (PS) matching. However, it is not always possible to include all covariates in adjustment. We aimed to find the op...

    Authors: Tri-Long Nguyen, Gary S. Collins, Jessica Spence, Jean-Pierre Daurès, P. J. Devereaux, Paul Landais and Yannick Le Manach

    Citation: BMC Medical Research Methodology 2017 17:78

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  41. Content type: Technical advance

    Health interventions fall along a spectrum from simple to more complex. There is wide interest in methods for reviewing ‘complex interventions’, but few transparent approaches for assessing intervention comple...

    Authors: Simon Lewin, Maggie Hendry, Jackie Chandler, Andrew D. Oxman, Susan Michie, Sasha Shepperd, Barnaby C. Reeves, Peter Tugwell, Karin Hannes, Eva A. Rehfuess, Vivien Welch, Joanne E. Mckenzie, Belinda Burford, Jennifer Petkovic, Laurie M. Anderson, Janet Harris…

    Citation: BMC Medical Research Methodology 2017 17:76

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  42. Content type: Research article

    Achieving adequate response rates is an ongoing challenge for longitudinal studies. The World Trade Center Health Registry is a longitudinal health study that periodically surveys a cohort of ~71,000 people ex...

    Authors: Shengchao Yu, Howard E. Alper, Angela-Maithy Nguyen, Robert M. Brackbill, Lennon Turner, Deborah J. Walker, Carey B. Maslow and Kimberly C. Zweig

    Citation: BMC Medical Research Methodology 2017 17:77

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  43. Content type: Research article

    In studies of recurrent events, it is common to consider a person who has suffered a disease episode and received curative treatment to be not at risk of suffering a new episode for a duration of time. It is a...

    Authors: Yin-Bun Cheung, Ying Xu, Matthew Cairns and Paul Milligan

    Citation: BMC Medical Research Methodology 2017 17:72

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  44. Content type: Research article

    The scientific evidence-base for policies to tackle health inequalities is limited. Natural policy experiments (NPE) have drawn increasing attention as a means to evaluating the effects of policies on health. ...

    Authors: Yannan Hu, Frank J. van Lenthe, Rasmus Hoffmann, Karen van Hedel and Johan P. Mackenbach

    Citation: BMC Medical Research Methodology 2017 17:68

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