Skip to content

Advertisement

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.

Page 2 of 9

  1. Content type: Research article

    Restricted mean survival time (RMST) is an underutilized estimand in time-to-event analyses. Herein, we highlight its strengths by comparing time to (1) all-cause mortality and (2) initiation of antiretroviral...

    Authors: Keri L. Calkins, Chelsea E. Canan, Richard D. Moore, Catherine R. Lesko and Bryan Lau

    Citation: BMC Medical Research Methodology 2018 18:27

    Published on:

  2. Content type: Research article

    Rigorous, informative meta-analyses rely on availability of appropriate summary statistics or individual participant data. For continuous outcomes, especially those with naturally skewed distributions, summary...

    Authors: Christopher J. Weir, Isabella Butcher, Valentina Assi, Stephanie C. Lewis, Gordon D. Murray, Peter Langhorne and Marian C. Brady

    Citation: BMC Medical Research Methodology 2018 18:25

    Published on:

  3. Content type: Research article

    Medical practitioners use survival models to explore and understand the relationships between patients’ covariates (e.g. clinical and genetic features) and the effectiveness of various treatment options. Stand...

    Authors: Jared L. Katzman, Uri Shaham, Alexander Cloninger, Jonathan Bates, Tingting Jiang and Yuval Kluger

    Citation: BMC Medical Research Methodology 2018 18:24

    Published on:

  4. Content type: Research article

    Standard survival analysis fails to give insight into what happens to a patient after a first outcome event (like first relapse of a disease). Multi-state models are a useful tool for analyzing survival data w...

    Authors: G. Manzini, T. J. Ettrich, M. Kremer, M. Kornmann, D. Henne-Bruns, D. A. Eikema, P. Schlattmann and L. C. de Wreede

    Citation: BMC Medical Research Methodology 2018 18:23

    Published on:

  5. Content type: Research article

    Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare disease with extreme between-subject variability, especially with respect to rate of disease progression. This makes modelling...

    Authors: Alex G. Karanevich, Jeffrey M. Statland, Byron J. Gajewski and Jianghua He

    Citation: BMC Medical Research Methodology 2018 18:19

    Published on:

  6. Content type: Research article

    Defeat and entrapment have been shown to be of central relevance to the development of different disorders. However, it remains unclear whether they represent two distinct constructs or one overall latent vari...

    Authors: Thomas Forkmann, Tobias Teismann, Jana-Sophie Stenzel, Heide Glaesmer and Derek de Beurs

    Citation: BMC Medical Research Methodology 2018 18:16

    Published on:

  7. 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

    Published on:

  8. 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

    Published on:

  9. 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

    Published on:

  10. 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

    Published on:

  11. 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

    Published on:

  12. 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

    Published on:

  13. 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

    Published on:

  14. 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

    Published on:

  15. 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

    Published on:

    The Correction to this article has been published in BMC Medical Research Methodology 2018 18:21

  16. 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

    Published on:

  17. 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

    Published on:

  18. 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

    Published on:

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

  19. 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

    Published on:

  20. 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

    Published on:

  21. 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

    Published on:

  22. 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

    Published on:

  23. 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

    Published on:

  24. 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

    Published on:

  25. 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

    Published on:

  26. 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

    Published on:

  27. 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

    Published on:

  28. 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

    Published on:

  29. 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

    Published on:

  30. 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

    Published on:

  31. 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

    Published on:

  32. 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

    Published on:

  33. 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

    Published on:

  34. 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

    Published on:

  35. 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

    Published on:

  36. 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

    Published on:

  37. 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

    Published on:

  38. 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

    Published on:

  39. 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

    Published on:

  40. 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

    Published on:

  41. 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

    Published on:

  42. 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

    Published on:

  43. 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

    Published on:

  44. 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

    Published on:

  45. 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

    Published on:

  46. 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

    Published on: