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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 11

  1. The mortality risk among cancer patients measured from the time of diagnosis is often elevated in comparison to the general population. However, for some cancer types, the patient mortality risk will over time...

    Authors: Lasse H. Jakobsen, Therese M.-L. Andersson, Jorne L. Biccler, Laurids Ø. Poulsen, Marianne T. Severinsen, Tarec C. El-Galaly and Martin Bøgsted

    Citation: BMC Medical Research Methodology 2020 20:71

    Content type: Research Article

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  2. It is important to estimate the treatment effect of interest accurately and precisely within the analysis of randomised controlled trials. One way to increase precision in the estimate and thus improve the pow...

    Authors: Hanaya Raad, Victoria Cornelius, Susan Chan, Elizabeth Williamson and Suzie Cro

    Citation: BMC Medical Research Methodology 2020 20:70

    Content type: Research article

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  3. With the growth in use of biotherapic drugs in various medical fields, the occurrence of anti-drug antibodies represents nowadays a serious issue. This immune response against a drug can be due either to pre-e...

    Authors: Julianne Duhazé, Miguel Caubet, Signe Hässler, Delphine Bachelet, Matthieu Allez, Florian Deisenhammer, Anna Fogdell-Hahn, Aude Gleizes, Salima Hacein-Bey-Abina, Xavier Mariette, Marc Pallardy and Philippe Broët

    Citation: BMC Medical Research Methodology 2020 20:69

    Content type: Research Article

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  4. Missing data are an inevitable challenge in Randomised Controlled Trials (RCTs), particularly those with Patient Reported Outcome Measures. Methodological guidance suggests that to avoid incorrect conclusions,...

    Authors: Alexina J. Mason, Richard D. Grieve, Alvin Richards-Belle, Paul R. Mouncey, David A. Harrison and James R. Carpenter

    Citation: BMC Medical Research Methodology 2020 20:66

    Content type: Technical Advance

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  5. Sero- prevalence studies often have a problem of missing data. Few studies report the proportion of missing data and even fewer describe the methods used to adjust the results for missing data. The objective o...

    Authors: Neema R. Mosha, Omololu S. Aluko, Jim Todd, Rhoderick Machekano and Taryn Young

    Citation: BMC Medical Research Methodology 2020 20:65

    Content type: Research article

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  6. Cox proportional hazards regression models are used to evaluate associations between exposures of interest and time-to-event outcomes in observational data. When exposures are measured on only a sample of part...

    Authors: Colleen M. Sitlani, Thomas Lumley, Barbara McKnight, Kenneth M. Rice, Nels C. Olson, Margaret F. Doyle, Sally A. Huber, Russell P. Tracy, Bruce M. Psaty and Joseph A. C. Delaney

    Citation: BMC Medical Research Methodology 2020 20:62

    Content type: Research Article

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  7. Attention deficit hyperactivity disorder (ADHD) is one of the most common childhood mental health disorders. Stimulant drugs as the most commonly used treatment and first-line therapy for ADHD have side effect...

    Authors: Hoda Shirafkan, Javad Mahmoudi-Gharaei, Akbar Fotouhi, Seyyed Ali Mozaffarpur, Mehdi Yaseri and Mostafa Hoseini

    Citation: BMC Medical Research Methodology 2020 20:56

    Content type: Research article

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  8. Claims-based algorithms are commonly used to identify sepsis in health services research because the laboratory features required to define clinical criteria may not be available in administrative data.

    Authors: John P. Donnelly, Yuling Dai, Lisandro D. Colantonio, Hong Zhao, Monika M. Safford, John W. Baddley, Paul Muntner and Henry E. Wang

    Citation: BMC Medical Research Methodology 2020 20:54

    Content type: Research article

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  9. The health regulatory focus is an application of Higgins’ regulatory focus theory to a health-specific context. It explains individual differences in health motivation, strategies, and behavior. Previous resea...

    Authors: Bjarne Schmalbach, Markus Zenger, Elmar Brähler and Katja Petrowski

    Citation: BMC Medical Research Methodology 2020 20:51

    Content type: Research article

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  10. Longitudinal randomized controlled trials (RCTs) often aim to test and measure the effect of treatment between arms at a single time point. A two-sample χ2 test is a common statistical approach when outcome data ...

    Authors: Mary L. Miller, Denise J. Roe, Chengcheng Hu and Melanie L. Bell

    Citation: BMC Medical Research Methodology 2020 20:50

    Content type: Research article

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  11. Missing participant outcome data (MOD) are ubiquitous in systematic reviews with network meta-analysis (NMA) as they invade from the inclusion of clinical trials with reported participant losses. There are ava...

    Authors: Loukia M. Spineli and Chrysostomos Kalyvas

    Citation: BMC Medical Research Methodology 2020 20:48

    Content type: Research article

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  12. The main goal of this study is to explore the use of features representing patient-level electronic health record (EHR) data, generated by the unsupervised deep learning algorithm autoencoder, in predictive mo...

    Authors: Lei Wang, Liping Tong, Darcy Davis, Tim Arnold and Tina Esposito

    Citation: BMC Medical Research Methodology 2020 20:37

    Content type: Research article

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  13. Network meta-analysis (NMA) is becoming increasingly popular in systematic reviews and health technology assessments. However, there is still ambiguity concerning the properties of the estimation approaches as...

    Authors: Corinna Kiefer, Sibylle Sturtz and Ralf Bender

    Citation: BMC Medical Research Methodology 2020 20:36

    Content type: Research Article

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  14. There is broad recognition of the importance of evidence in informing clinical decisions. When information from all studies included in a systematic review (“review”) does not contribute to a meta-analysis, de...

    Authors: Ian J. Saldanha, Kristina B. Lindsley, Sarah Money, Hannah J. Kimmel, Bryant T. Smith and Kay Dickersin

    Citation: BMC Medical Research Methodology 2020 20:30

    Content type: Research article

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  15. Personalized healthcare promises to successfully advance the treatment of heterogeneous neurological disorders such as relapsing remitting multiple sclerosis by addressing the caveats of traditional healthcare...

    Authors: E. Stühler, S. Braune, F. Lionetto, Y. Heer, E. Jules, C. Westermann, A. Bergmann and P. van Hövell

    Citation: BMC Medical Research Methodology 2020 20:24

    Content type: Research article

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  16. Cohen’s κ coefficient is often used as an index to measure the agreement of inter-rater determinations. However, κ varies greatly depending on the marginal distribution of the target population and overestimates ...

    Authors: Chikara Honda and Tetsuji Ohyama

    Citation: BMC Medical Research Methodology 2020 20:20

    Content type: Research article

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  17. With the increased empirical interest in the positive significance of improving nurses’ sense of professional benefits, there is a requirement for measures of nurses’ perceived professional benefit (NPPB). Our...

    Authors: Yanli Hu, Jing Hu, Liping Li, Bin Zhao, Xiaohong Liu and Fan Li

    Citation: BMC Medical Research Methodology 2020 20:18

    Content type: Research article

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  18. Patients infected with the Human Immunodeficiency Virus (HIV) are susceptible to many diseases. In these patients, the occurrence of one disease alters the chance of contracting another. Under such circumstanc...

    Authors: Sahar Nouri, Mahmood Mahmoudi, Kazem Mohammad, Mohammad Ali Mansournia, Mahdi Yaseri and Noori Akhtar-Danesh

    Citation: BMC Medical Research Methodology 2020 20:17

    Content type: Research article

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  19. Use of generalized linear models with continuous, non-linear functions for age, period and cohort makes it possible to estimate these effects so they are interpretable, reliable and easily displayed graphicall...

    Authors: Annette Dobson, Richard Hockey, Hsiu-Wen Chan and Gita Mishra

    Citation: BMC Medical Research Methodology 2020 20:16

    Content type: Research article

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  20. Regression analyses of time series of disease counts on environmental determinants are a prominent component of environmental epidemiology. For planning such studies, it can be useful to predict the precision ...

    Authors: Ben G. Armstrong, Antonio Gasparrini, Aurelio Tobias and Francesco Sera

    Citation: BMC Medical Research Methodology 2020 20:15

    Content type: Research article

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  21. Drug safety in children is a major concern; however, there is still a lack of methods for quantitatively measuring, let alone to improving, drug safety in children under different clinical conditions. To asses...

    Authors: Gang Yu, Xian Zeng, Shaoqing Ni, Zheng Jia, Weihong Chen, Xudong Lu, Jiye An, Huilong Duan, Qiang Shu and Haomin Li

    Citation: BMC Medical Research Methodology 2020 20:9

    Content type: Research article

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  22. Clinical research and medical practice can be advanced through the prediction of an individual’s health state, trajectory, and responses to treatments. However, the majority of current clinical risk prediction...

    Authors: Shannon Wongvibulsin, Katherine C. Wu and Scott L. Zeger

    Citation: BMC Medical Research Methodology 2019 20:1

    Content type: Technical Advance

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  23. This study aimed to address the current limitations of the use of composite endpoints in orthopaedic trauma research by quantifying the relative importance of clinical outcomes common to orthopaedic trauma pat...

    Authors: Ugochukwu N. Udogwu, Andrea Howe, Katherine Frey, Marckenley Isaac, Daniel Connelly, Dimitrius Marinos, Mitchell Baker, Renan C. Castillo, Gerard P. Slobogean, Robert V. O’Toole and Nathan N. O’Hara

    Citation: BMC Medical Research Methodology 2019 19:242

    Content type: Research article

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  24. Verbal autopsy (VA) is increasingly being considered as a cost-effective method to improve cause of death information in countries with low quality vital registration. VA algorithms that use empirical data hav...

    Authors: Hafizur Rahman Chowdhury, Abraham D. Flaxman, Jonathan C. Joseph, Riley H. Hazard, Nurul Alam, Ian Douglas Riley and Alan D. Lopez

    Citation: BMC Medical Research Methodology 2019 19:232

    Content type: Research article

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  25. In low-income settings, key outcomes such as biomarkers or clinical assessments are often missing for a substantial proportion of the study population. The aim of this study was to assess the extent to which H...

    Authors: Siaka Koné, Bassirou Bonfoh, Daouda Dao, Inza Koné and Günther Fink

    Citation: BMC Medical Research Methodology 2019 19:231

    Content type: Technical advance

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  26. Patient-Reported Outcome Measures (PROMs) have been proposed for benchmarking health care quality across hospitals, which requires extensive case-mix adjustment. The current study’s aim was to develop and comp...

    Authors: Arvind Oemrawsingh, Nikki van Leeuwen, Esmee Venema, Martien Limburg, Frank-Erik de Leeuw, Markus P. Wijffels, Aafke J. de Groot, Pieter H. E. Hilkens, Jan A. Hazelzet, Diederik W. J. Dippel, Carla H. Bakker, Helene R. Voogdt-Pruis and Hester F. Lingsma

    Citation: BMC Medical Research Methodology 2019 19:229

    Content type: Research article

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  27. Multi-center studies can generate robust and generalizable evidence, but privacy considerations and legal restrictions often make it challenging or impossible to pool individual-level data across data-contribu...

    Authors: Di Shu, Jessica G. Young and Sengwee Toh

    Citation: BMC Medical Research Methodology 2019 19:228

    Content type: Research article

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  28. Attrition due to death and non-attendance are common sources of bias in studies of age-related diseases. A simulation study is presented to compare two methods for estimating the survivor average causal effect...

    Authors: Myra B. McGuinness, Jessica Kasza, Amalia Karahalios, Robyn H. Guymer, Robert P. Finger and Julie A. Simpson

    Citation: BMC Medical Research Methodology 2019 19:223

    Content type: Research article

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    The Correction to this article has been published in BMC Medical Research Methodology 2020 20:40

  29. To assess cross-cultural validity between Dutch and English versions of the FVQ_CYP, a patient-reported outcome measure developed in the United Kingdom (UK) for children and adolescents with (severe) visual im...

    Authors: Ellen B. M. Elsman, Valerija Tadić, Carel F. W. Peeters, Ger H. M. B. van Rens, Jugnoo S. Rahi and Ruth M. A. van Nispen

    Citation: BMC Medical Research Methodology 2019 19:221

    Content type: Research article

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  30. Until recently a typical rule that has often been used for the endorsement of new medications by the Food and Drug Administration has been the existence of at least two statistically significant clinical trial...

    Authors: Don van Ravenzwaaij and John P. A. Ioannidis

    Citation: BMC Medical Research Methodology 2019 19:218

    Content type: Technical advance

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  31. Continuous monitoring of surgical outcomes after joint replacement is needed to detect which brands’ components have a higher than expected failure rate and are therefore no longer recommended to be used in su...

    Authors: Alexander Begun, Elena Kulinskaya and Alexander J MacGregor

    Citation: BMC Medical Research Methodology 2019 19:217

    Content type: Research Article

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  32. Antiretroviral therapy (ART) has significantly reduced HIV-related morbidity and mortality. However, therapeutic benefit of ART is often limited by delayed drug-associated toxicity. Nucleoside reverse transcri...

    Authors: Jong Soo Lee, Elijah Paintsil, Vivek Gopalakrishnan and Musie Ghebremichael

    Citation: BMC Medical Research Methodology 2019 19:216

    Content type: Research article

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  33. Antimalarial clinical efficacy studies for uncomplicated Plasmodium falciparum malaria frequently encounter situations in which molecular genotyping is unable to discriminate between parasitic recurrence, either ...

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

    Citation: BMC Medical Research Methodology 2019 19:215

    Content type: Research article

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  34. We recently introduced a system of partial differential equations (PDEs) to model the prevalence of chronic diseases with a possibly prolonged state of asymptomatic, undiagnosed disease preceding a diagnosis. ...

    Authors: Ralph Brinks, Sophie Kaufmann, Annika Hoyer, Edward W Gregg and Jürgen Saal

    Citation: BMC Medical Research Methodology 2019 19:213

    Content type: Research Article

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  35. Large and complex population-based cancer data are becoming broadly available, thanks to purposeful linkage between cancer registry data and health electronic records. Aiming at understanding the explanatory p...

    Authors: Camille Maringe, Aurélien Belot, Francisco Javier Rubio and Bernard Rachet

    Citation: BMC Medical Research Methodology 2019 19:210

    Content type: Research article

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  36. The analysis of twin data presents a unique challenge. Second-born twins on average weigh less than first-born twins and have an elevated risk of perinatal mortality. It is not clear whether the risk differenc...

    Authors: Muhammad Abu Shadeque Mullah, James A. Hanley and Andrea Benedetti

    Citation: BMC Medical Research Methodology 2019 19:209

    Content type: Research article

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  37. Recently, there has been a heightened interest in developing and evaluating different methods for analysing observational data. This has been driven by the increased availability of large data resources such a...

    Authors: E. R. John, K. R. Abrams, C. E. Brightling and N. A. Sheehan

    Citation: BMC Medical Research Methodology 2019 19:207

    Content type: Research article

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  38. Clinical study reports (CSRs) have been increasingly utilised within academic research in recent years. European Medicines Agency (EMA) Policy 0070 ‘Phase 1,’ which came into effect in January 2015, requires t...

    Authors: Jean-Marc Ferran and Sarah J. Nevitt

    Citation: BMC Medical Research Methodology 2019 19:204

    Content type: Research article

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  39. It is unclear whether weighted or unweighted regression is preferred in the analysis of data derived from respondent driven sampling. Our objective was to evaluate the validity of various regression models, wi...

    Authors: Lisa Avery, Nooshin Rotondi, Constance McKnight, Michelle Firestone, Janet Smylie and Michael Rotondi

    Citation: BMC Medical Research Methodology 2019 19:202

    Content type: Research article

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  40. New emerging diseases are public health concerns in which policy makers have to make decisions in the presence of enormous uncertainty. This is an important challenge in terms of emergency preparation requirin...

    Authors: Chawarat Rotejanaprasert, Andrew B. Lawson and Sopon Iamsirithaworn

    Citation: BMC Medical Research Methodology 2019 19:200

    Content type: Research article

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  41. The incorporation of repeated measurements into multivariable prediction research may greatly enhance predictive performance. However, the methodological possibilities vary widely and a structured overview of ...

    Authors: Joost D. J. Plate, Rutger R. van de Leur, Luke P. H. Leenen, Falco Hietbrink, Linda M. Peelen and M. J. C. Eijkemans

    Citation: BMC Medical Research Methodology 2019 19:199

    Content type: Research article

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  42. When investigating subgroup effects in meta-analysis, it is unclear whether accounting in meta-regression for between-trial variation in treatment effects, but not between-trial variation in treatment interact...

    Authors: Bruno R. da Costa and Alex J. Sutton

    Citation: BMC Medical Research Methodology 2019 19:198

    Content type: Research article

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  43. Several reviews have noted shortcomings regarding the quality and reporting of network meta-analyses (NMAs). We suspect that this issue may be partially attributable to limitations in current NMA software whic...

    Authors: Audrey Béliveau, Devon J. Boyne, Justin Slater, Darren Brenner and Paul Arora

    Citation: BMC Medical Research Methodology 2019 19:196

    Content type: Software

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  44. Validation studies on an ICD-10-based algorithm to identify major bleeding events are scarce, and mostly focused on positive predictive values.

    Authors: Emmanuel Oger, Marie-Anne Botrel, Catherine Juchault and Jacques Bouget

    Citation: BMC Medical Research Methodology 2019 19:194

    Content type: Research article

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  45. The importance of adjusting for cross-study heterogeneity in control group response rates when conducting network meta-analyses (NMA) was demonstrated using a case study involving a comparison of biologics for...

    Authors: Chris Cameron, Abhishek Varu, Arthur Lau, Mahdi Gharaibeh, Marcelo Paulino and Raina Rogoza

    Citation: BMC Medical Research Methodology 2019 19:193

    Content type: Research article

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  46. Online clinical risk prediction tools built on data from multiple cohorts are increasingly being utilized for contemporary doctor-patient decision-making and validation. This report outlines a comprehensive da...

    Authors: Johanna Tolksdorf, Michael W. Kattan, Stephen A. Boorjian, Stephen J. Freedland, Karim Saba, Cedric Poyet, Lourdes Guerrios, Amanda De Hoedt, Michael A. Liss, Robin J. Leach, Javier Hernandez, Emily Vertosick, Andrew J. Vickers and Donna P. Ankerst

    Citation: BMC Medical Research Methodology 2019 19:191

    Content type: Research article

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  47. When conducing Phase-III trial, regulatory agencies and investigators might want to get reliable information about rare but serious safety outcomes during the trial. Bayesian non-inferiority approaches have be...

    Authors: Camille Aupiais, Corinne Alberti, Thomas Schmitz, Olivier Baud, Moreno Ursino and Sarah Zohar

    Citation: BMC Medical Research Methodology 2019 19:187

    Content type: Research Article

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  48. Population attributable fractions (PAF) measure the proportion of disease prevalence that would be avoided in a hypothetical population, similar to the population of interest, but where a particular risk facto...

    Authors: John Ferguson, Neil O’Leary, Fabrizio Maturo, Salim Yusuf and Martin O’Donnell

    Citation: BMC Medical Research Methodology 2019 19:186

    Content type: Research article

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  49. An overarching objective in research comparing different sample groups is to ensure that the reported differences in outcomes are not affected by differences between groups in the functioning of the measuremen...

    Authors: Curt Hagquist

    Citation: BMC Medical Research Methodology 2019 19:185

    Content type: Research article

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