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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.

Page 1 of 10

  1. Content type: Research article

    Muscular dystrophies (MDs) are a group of inherited conditions characterized by progressive muscle degeneration and weakness. The rarity and heterogeneity of the population with MD have hindered therapeutic de...

    Authors: Xiaoxue Chen, Abiy Agiro, Ann S. Martin, Ann M. Lucas and Kevin Haynes

    Citation: BMC Medical Research Methodology 2019 19:174

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  2. Content type: Research article

    Longitudinal qualitative research is starting to be used in applied health research, having been popular in social research for several decades. There is potential for a large volume of complex data to be capt...

    Authors: Laura Sheard and Claire Marsh

    Citation: BMC Medical Research Methodology 2019 19:169

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  3. Content type: Research article

    Today we are often interested in the predictive value of a continuous marker with respect to the expected difference in outcome between a new treatment and a standard treatment. We can investigate this in a ra...

    Authors: Maren Eckert and Werner Vach

    Citation: BMC Medical Research Methodology 2019 19:168

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  4. Content type: Research article

    Patient Reported Outcomes (PRO) are gaining more and more importance in the context of clinical trials. The assessment of PRO is frequently performed by questionnaires where the multiple items of a questionnai...

    Authors: Manuel Feißt, André Hennigs, Jörg Heil, Helfried Moosbrugger, Augustin Kelava, Ilona Stolpner, Meinhard Kieser and Geraldine Rauch

    Citation: BMC Medical Research Methodology 2019 19:167

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  5. Content type: Research article

    Analysis of competing risks is commonly achieved through a cause specific or a subdistribution framework using Cox or Fine & Gray models, respectively. The estimation of treatment effects in observational data...

    Authors: Michael Andrew Barrowman, Niels Peek, Mark Lambie, Glen Philip Martin and Matthew Sperrin

    Citation: BMC Medical Research Methodology 2019 19:166

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  6. Content type: Research article

    Although criticisms regarding the dichotomisation of continuous variables are well known, applying logit model to dichotomised outcomes is the convention because the odds ratios are easily obtained and they ap...

    Authors: Ying Chen, Yilin Ning, Shih Ling Kao, Nathalie C. Støer, Falk Müller-Riemenschneider, Kavita Venkataraman, Eric Yin Hao Khoo, E-Shyong Tai and Chuen Seng Tan

    Citation: BMC Medical Research Methodology 2019 19:165

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  7. Content type: Research article

    The quality of systematic reviews and meta-analyses (SR/MAs) depends on the extent of the methods used. We investigated the methodological steps used by authors of SR/MAs of clinical trials via an author survey.

    Authors: Hoang Thi Nam Giang, Ali Mahmoud Ahmed, Reem Yousry Fala, Mohamed Magdy Khattab, Mona Hassan Ahmed Othman, Sara Attia Mahmoud Abdelrahman, Le Phuong Thao, Ahmed Elsaid Abd Elsamie Gabl, Samar Ahmed Elrashedy, Peter N. Lee, Kenji Hirayama, Hosni Salem and Nguyen Tien Huy

    Citation: BMC Medical Research Methodology 2019 19:164

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  8. Content type: Research article

    Many prodromal Alzheimer’s disease trials collect two types of data: the time until clinical diagnosis of dementia and longitudinal patient information. These data are often analysed separately, although they ...

    Authors: Floor M. van Oudenhoven, Sophie H.N. Swinkels, Tobias Hartmann, Hilkka Soininen, Anneke M.J. van Hees and Dimitris Rizopoulos

    Citation: BMC Medical Research Methodology 2019 19:163

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  9. Content type: Research article

    Omics data can be very informative in survival analysis and may improve the prognostic ability of classical models based on clinical risk factors for various diseases, for example breast cancer. Recent researc...

    Authors: Alexander Volkmann, Riccardo De Bin, Willi Sauerbrei and Anne-Laure Boulesteix

    Citation: BMC Medical Research Methodology 2019 19:162

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  10. Content type: Research article

    In many clinical trials continuous outcomes are dichotomized to compare proportions of patients who respond. A common and recommended approach to handling missing data in responder analysis is to impute as non...

    Authors: Lysbeth Floden and Melanie L. Bell

    Citation: BMC Medical Research Methodology 2019 19:161

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  11. Content type: Research article

    Sleeping under a long-lasting insecticidal net (LLIN) is recommended for all pregnant women in sub-Saharan Africa, due to the high prevalence of malaria infection and its associated complications in the region...

    Authors: Ahmed Dahiru Balami, Salmiah Md Said, Nor Afiah Mohd Zulkefli, Norsa’adah Bachok and Emmanuel Luke Balami

    Citation: BMC Medical Research Methodology 2019 19:157

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  12. Content type: Research article

    In recent years, the availability of publicly available data related to public health has significantly increased. These data have substantial potential to develop public health policy; however, this requires ...

    Authors: Paul van Schaik, Yonghong Peng, Adedokun Ojelabi and Jonathan Ling

    Citation: BMC Medical Research Methodology 2019 19:152

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  13. Content type: Research article

    Randomized controlled trials are the gold-standard for clinical trials. However, randomization is not always feasible. In this article we propose a prospective and adaptive matched case-control trial design as...

    Authors: Dorothea Weber, Lorenz Uhlmann, Silvia Schönenberger and Meinhard Kieser

    Citation: BMC Medical Research Methodology 2019 19:150

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  14. Content type: Technical advance

    In the context of environmentally influenced communicable diseases, proximity to environmental sources results in spatial heterogeneity of risk, which is sometimes difficult to measure in the field. Most preve...

    Authors: Abdoulaye Guindo, Issaka Sagara, Boukary Ouedraogo, Kankoe Sallah, Mahamadoun Hamady Assadou, Sara Healy, Patrick Duffy, Ogobara K. Doumbo, Alassane Dicko, Roch Giorgi and Jean Gaudart

    Citation: BMC Medical Research Methodology 2019 19:149

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  15. Content type: Research article

    The life expectancy of cancer patients, and the loss in expectation of life as compared to the life expectancy without cancer, is a useful measure of cancer patient survival and complement the more commonly re...

    Authors: Therese M.-L. Andersson, Mark J. Rutherford and Paul C. Lambert

    Citation: BMC Medical Research Methodology 2019 19:145

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  16. Content type: Research article

    Sequence symmetry analysis (SSA) is a signal detection method that can be used to assist with adverse drug event detection. It provides both a risk estimate and a data visualisation. Published methods provide ...

    Authors: Adrian Kym Preiss, Elizabeth Ellen Roughead and Nicole Leanne Pratt

    Citation: BMC Medical Research Methodology 2019 19:143

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  17. Content type: Research article

    Incidence rates of advanced cancer stages are important, e.g., for monitoring cancer screening programs. However, information from cancer registries on tumor stage is often incomplete. Exemplified by colorecta...

    Authors: Katja Anita Oppelt, Sabine Luttmann, Klaus Kraywinkel and Ulrike Haug

    Citation: BMC Medical Research Methodology 2019 19:142

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  18. Content type: Research article

    In most cases, the total scores from different instruments assessing the same construct are not directly comparable, but must be equated. In this study we aimed to illustrate a novel test equating methodology ...

    Authors: Núria Duran Adroher, Svend Kreiner, Carolyn Young, Roger Mills and Alan Tennant

    Citation: BMC Medical Research Methodology 2019 19:141

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  19. Content type: Research article

    Report cards on the health care system increasingly report provider-specific performance on indicators that measure the quality of health care delivered. A natural reaction to the publishing of hospital-specif...

    Authors: Peter C. Austin, Iris E. Ceyisakar, Ewout W. Steyerberg, Hester F. Lingsma and Perla J. Marang-van de Mheen

    Citation: BMC Medical Research Methodology 2019 19:131

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  20. Content type: Research article

    Tacrolimus (TAC) is an immunosuppressant drug given to kidney transplant recipients post-transplant to prevent antibody formation and kidney rejection. The optimal therapeutic dose for TAC is poorly defined an...

    Authors: Kristen R. Campbell, Elizabeth Juarez-Colunga, Gary K. Grunwald, James Cooper, Scott Davis and Jane Gralla

    Citation: BMC Medical Research Methodology 2019 19:130

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  21. Content type: Research article

    Multiple primary outcomes may be specified in randomised controlled trials (RCTs). When analysing multiple outcomes it’s important to control the family wise error rate (FWER). A popular approach to do this is...

    Authors: Victoria Vickerstaff, Rumana Z. Omar and Gareth Ambler

    Citation: BMC Medical Research Methodology 2019 19:129

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    The Correction to this article has been published in BMC Medical Research Methodology 2019 19:158

  22. Content type: Debate

    Use of big data is becoming increasingly popular in medical research. Since big data-based projects differ notably from classical research studies, both in terms of scope and quality, a debate is apt as to whe...

    Authors: Amke Caliebe, Friedhelm Leverkus, Gerd Antes and Michael Krawczak

    Citation: BMC Medical Research Methodology 2019 19:125

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  23. Content type: Research article

    Cluster randomised trials with unequal sized clusters often have lower precision than with clusters of equal size. To allow for this, sample sizes are inflated by a modified version of the design effect for cl...

    Authors: James Thomas Martin, Karla Hemming and Alan Girling

    Citation: BMC Medical Research Methodology 2019 19:123

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  24. Content type: Research article

    Accurate measurement of health literacy is essential to improve accessibility and effectiveness of health care and prevention. One measure frequently applied in international research is the Short Assessment o...

    Authors: A. J. Woudstra, C. S. Meppelink, H. Pander Maat, J. Oosterhaven, M. P. Fransen and A. L. Dima

    Citation: BMC Medical Research Methodology 2019 19:122

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  25. Content type: Research article

    Clinical prediction models are not routinely validated. To facilitate validation procedures, the online Evidencio platform (https://​www.​evidencio.​com) ...

    Authors: Cornelia D. van Steenbeek, Marissa C. van Maaren, Sabine Siesling, Annemieke Witteveen, Xander A. A. M. Verbeek and Hendrik Koffijberg

    Citation: BMC Medical Research Methodology 2019 19:117

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  26. Content type: Research article

    Comorbidity measures, such as the Charlson Comorbidity Index (CCI) and Elixhauser Method (EM), are frequently used for risk-adjustment by healthcare researchers. This study sought to create CCI and EM lists of...

    Authors: David Metcalfe, James Masters, Antonella Delmestri, Andrew Judge, Daniel Perry, Cheryl Zogg, Belinda Gabbe and Matthew Costa

    Citation: BMC Medical Research Methodology 2019 19:115

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  27. Content type: Research article

    Length of stay evaluations are very common to determine the burden of nosocomial infections. However, there exist fundamentally different methods to quantify the prolonged length of stay associated with nosoco...

    Authors: Martin Wolkewitz, Martin Schumacher, Gerta Rücker, Stephan Harbarth and Jan Beyersmann

    Citation: BMC Medical Research Methodology 2019 19:111

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  28. Content type: Research article

    This study compares an algorithm to detect acute gastroenteritis (AG) episodes from drug dispensing data to the validated data reported in a primary care surveillance system in France.

    Authors: Ana-Maria Vilcu, Thierry Blanchon, Laure Sabatte, Cécile Souty, Milka Maravic, Thomas Hanslik and Olivier Steichen

    Citation: BMC Medical Research Methodology 2019 19:110

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  29. Content type: Research article

    The likelihood ratio function (LR), the ratio of conditional probabilities of obtaining a specific marker value among those with the event of interest over those without, provides an easily interpretable way t...

    Authors: Andrew M. Smith, John P. Christodouleas and Wei-Ting Hwang

    Citation: BMC Medical Research Methodology 2019 19:108

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  30. Content type: Research article

    Antimalarial efficacy studies in patients with uncomplicated Plasmodium falciparum are confounded by a new infection (a competing risk event) since this event can potentially preclude a recrudescent event (primar...

    Authors: Prabin Dahal, Philippe J. Guerin, Ric N. Price, Julie A. Simpson and Kasia Stepniewska

    Citation: BMC Medical Research Methodology 2019 19:107

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  31. Content type: Research article

    Net survival, a measure of the survival where the patients would only die from the cancer under study, may be compared between treatment groups using either “cause-specific methods”, when the causes of death a...

    Authors: Juste Aristide Goungounga, Célia Touraine, Nathalie Grafféo and Roch Giorgi

    Citation: BMC Medical Research Methodology 2019 19:104

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  32. Content type: Research article

    Childhood acute lymphoblastic leukemia (cALL) is the most frequent pediatric cancer. Over the past decades, treatment of cALL has significantly improved, with cure rates close to 90%. However intensive chemoth...

    Authors: Miguel Caubet Fernandez, Simon Drouin, Mariia Samoilenko, Sophia Morel, Maja Krajinovic, Caroline Laverdière, Daniel Sinnett, Emile Levy, Valérie Marcil and Geneviève Lefebvre

    Citation: BMC Medical Research Methodology 2019 19:100

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  33. Content type: Research article

    Despite its popularity, issues concerning the estimation of power in multilevel logistic regression models are prevalent because of the complexity involved in its calculation (i.e., computer-simulation-based a...

    Authors: Oscar L. Olvera Astivia, Anne Gadermann and Martin Guhn

    Citation: BMC Medical Research Methodology 2019 19:97

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  34. Content type: Technical advance

    In clinical and epidemiological researches, continuous predictors are often discretized into categorical variables for classification of patients. When the relationship between a continuous predictor and log r...

    Authors: Yimin Chen, Jialing Huang, Xianying He, Yongxiang Gao, Gehendra Mahara, Zhuochen Lin and Jinxin Zhang

    Citation: BMC Medical Research Methodology 2019 19:96

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  35. Content type: Research article

    While it is widely acknowledged that family burden can be ameliorated with effective psycho-social interventions, how to measure family burden and define a valid cutoff to identify family caregivers in need of...

    Authors: Yu Yu, Zi-Wei Liu, Wei Zhou, Mei Zhao, Bing-Wei Tang and Shui-Yuan Xiao

    Citation: BMC Medical Research Methodology 2019 19:93

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  36. Content type: Research article

    Radiologic assessments of baseline and post-treatment tumor burden are subject to measurement variability, but the impact of this variability on the objective response rate (ORR) and progression rate in specif...

    Authors: Jeong-Hwa Yoon, Soon Ho Yoon and Seokyung Hahn

    Citation: BMC Medical Research Methodology 2019 19:90

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  37. Content type: Research article

    Performing well-powered randomised controlled trials (RCTs) of new treatments for rare diseases is often infeasible. However, with the increasing availability of historical data, incorporating existing informa...

    Authors: Caroline Brard, Lisa V. Hampson, Nathalie Gaspar, Marie-Cécile Le Deley and Gwénaël Le Teuff

    Citation: BMC Medical Research Methodology 2019 19:85

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  38. Content type: Research article

    Multiple neonatal and pediatric disorders have been linked to older paternal ages. Combining these findings with the evidence that many men are having children at much later ages generates considerable public ...

    Authors: James A. Thompson

    Citation: BMC Medical Research Methodology 2019 19:82

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  39. Content type: Software

    Recommended statistical methods for meta-analysis of diagnostic test accuracy studies require relatively complex bivariate statistical models which can be a barrier for non-statisticians. A further barrier exi...

    Authors: Suzanne C. Freeman, Clareece R. Kerby, Amit Patel, Nicola J. Cooper, Terry Quinn and Alex J. Sutton

    Citation: BMC Medical Research Methodology 2019 19:81

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  40. Content type: Software

    In medical research, explanatory continuous variables are frequently transformed or converted into categorical variables. If the coding is unknown, many tests can be used to identify the “optimal” transformati...

    Authors: Benoit Liquet and Jérémie Riou

    Citation: BMC Medical Research Methodology 2019 19:79

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  41. Content type: Research article

    An important part of the systematic review methodology is appraisal of the risk of bias in included studies. Cochrane systematic reviews are considered golden standard regarding systematic review methodology, ...

    Authors: Andrija Babic, Ruzica Tokalic, João Amílcar Silva Cunha, Ivana Novak, Jelena Suto, Marin Vidak, Ivana Miosic, Ivana Vuka, Tina Poklepovic Pericic and Livia Puljak

    Citation: BMC Medical Research Methodology 2019 19:76

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  42. Content type: Technical advance

    In clinical trials, study designs may focus on assessment of superiority, equivalence, or non-inferiority, of a new medicine or treatment as compared to a control. Typically, evidence in each of these paradigm...

    Authors: Don van Ravenzwaaij, Rei Monden, Jorge N. Tendeiro and John P. A. Ioannidis

    Citation: BMC Medical Research Methodology 2019 19:71

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  43. Content type: Research article

    Chronic respiratory symptoms involving bronchitis, cough and phlegm in children are underappreciated but pose a significant public health burden. Efforts for prevention and management could be supported by an ...

    Authors: Huiyu Deng, Robert Urman, Frank D. Gilliland and Sandrah P. Eckel

    Citation: BMC Medical Research Methodology 2019 19:70

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  44. Content type: Research article

    Treatment switching is common in randomised trials of oncology treatments, with control group patients switching onto the experimental treatment during follow-up. This distorts an intention-to-treat comparison...

    Authors: N. R. Latimer, K. R. Abrams and U. Siebert

    Citation: BMC Medical Research Methodology 2019 19:69

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  45. Content type: Research article

    Crowding in the emergency department (ED) is associated with increased mortality, increased treatment cost, and reduced quality of care. Crowding arises when demand exceed resources in the ED and a first sign ...

    Authors: Andreas Halgreen Eiset, Hans Kirkegaard and Mogens Erlandsen

    Citation: BMC Medical Research Methodology 2019 19:68

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  46. Content type: Research article

    Following visible successes on a wide range of predictive tasks, machine learning techniques are attracting substantial interest from medical researchers and clinicians. We address the need for capacity develo...

    Authors: Jenni A. M. Sidey-Gibbons and Chris J. Sidey-Gibbons

    Citation: BMC Medical Research Methodology 2019 19:64

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  47. Content type: Research article

    Meta-analysis is a useful tool for combining evidence from multiple studies to estimate a pooled treatment effect. An extension of meta-analysis, network meta-analysis, is becoming more commonly used as a way ...

    Authors: Martin Law, Navid Alam, Areti Angeliki Veroniki, Yi Yu and Dan Jackson

    Citation: BMC Medical Research Methodology 2019 19:61

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