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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

    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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  2. 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

  3. 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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  4. 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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  5. 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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  6. 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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  7. 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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  8. 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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  9. 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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  10. 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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  11. 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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  12. 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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  13. 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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  14. 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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  15. 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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  16. 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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  17. 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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  18. 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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  19. 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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