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BMC Medical Research Methodology

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: Technical advance

    Investigating the impact of a time-dependent intervention on the probability of long-term survival is statistically challenging. A typical example is stem-cell transplantation performed after successful donor ...

    Authors: Ulrike Pötschger, Harald Heinzl, Maria Grazia Valsecchi and Martina Mittlböck

    Citation: BMC Medical Research Methodology 2018 18:14

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  2. Content type: Research article

    In patient-based studies, biomarker data are often subject to left censoring due to the detection limits, or to incomplete sample or data collection. In the context of longitudinal regression analysis, inappro...

    Authors: MinJae Lee, Mohammad H. Rahbar, Matthew Brown, Lianne Gensler, Michael Weisman, Laura Diekman and John D. Reveille

    Citation: BMC Medical Research Methodology 2018 18:8

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  3. Content type: Research article

    We wished to compare the nuisance parameters of pediatric vs. adult randomized-trials (RCTs) and determine if the latter can be used in sample size computations of the former.

    Authors: Ben Vandermeer, Ingeborg van der Tweel, Marijke C. Jansen-van der Weide, Stephanie S. Weinreich, Despina G. Contopoulos-Ioannidis, Dirk Bassler, Ricardo M. Fernandes, Lisa Askie, Haroon Saloojee, Paola Baiardi, Susan S. Ellenberg and Johanna H. van der Lee

    Citation: BMC Medical Research Methodology 2018 18:7

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  4. Content type: Research article

    Despite the frequent use of self-controlled methods in pharmacoepidemiological studies, the factors that may bias the estimates from these methods have not been adequately compared in real-world settings. Here...

    Authors: Yoshinori Takeuchi, Tomohiro Shinozaki and Yutaka Matsuyama

    Citation: BMC Medical Research Methodology 2018 18:4

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  5. Content type: Technical advance

    Many clinical trials focus on the comparison of the treatment effect between two or more groups concerning a rarely occurring event. In this situation, showing a relevant effect with an acceptable power requir...

    Authors: Ann-Kathrin Ozga, Meinhard Kieser and Geraldine Rauch

    Citation: BMC Medical Research Methodology 2018 18:2

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  6. Content type: Research article

    Hepatocellular carcinoma (HCC) has limited treatment options in patients with advanced stage disease and early detection of HCC through surveillance programs is a key component towards reducing mortality. The ...

    Authors: Nabihah Tayob, Peter Richardson, Donna L. White, Xiaoying Yu, Jessica A. Davila, Fasiha Kanwal, Ziding Feng and Hashem B. El-Serag

    Citation: BMC Medical Research Methodology 2018 18:1

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  7. Content type: Research article

    Different confounder adjustment strategies were used to estimate odds ratios (ORs) in case-control study, i.e. how many confounders original studies adjusted and what the variables are. This secondary data ana...

    Authors: Tianyi Liu, Xiaolu Nie, Zehao Wu, Ying Zhang, Guoshuang Feng, Siyu Cai, Yaqi Lv and Xiaoxia Peng

    Citation: BMC Medical Research Methodology 2017 17:179

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  8. Content type: Research article

    Confounders can produce spurious associations between exposure and outcome in observational studies. For majority of epidemiologists, adjusting for confounders using logistic regression model is their habitual...

    Authors: Yuanyuan Yu, Hongkai Li, Xiaoru Sun, Ping Su, Tingting Wang, Yi Liu, Zhongshang Yuan, Yanxun Liu and Fuzhong Xue

    Citation: BMC Medical Research Methodology 2017 17:177

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  9. Content type: Research article

    Prediction models and prognostic scores have been increasingly popular in both clinical practice and clinical research settings, for example to aid in risk-based decision making or control for confounding. In ...

    Authors: Sarah R. Haile, Beniamino Guerra, Joan B. Soriano and Milo A. Puhan

    Citation: BMC Medical Research Methodology 2017 17:172

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  10. 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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  11. 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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  12. 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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  13. 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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  14. 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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  15. 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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  16. 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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  17. 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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  18. 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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  19. 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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