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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. 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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  2. 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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  3. 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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  4. 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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  5. 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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  6. 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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  7. 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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  8. 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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  9. 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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  10. 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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  11. 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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  12. 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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  13. 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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  14. 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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  15. 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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  16. 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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  17. 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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  18. 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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  19. 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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  20. 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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  21. 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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  22. 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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  23. 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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  24. 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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  25. Individual participant data meta-analysis (IPD-MA) is considered the gold standard for investigating subgroup effects. Frequently used regression-based approaches to detect subgroups in IPD-MA are: meta-regres...

    Authors: Michail Belias, Maroeska M. Rovers, Johannes B. Reitsma, Thomas P. A. Debray and Joanna IntHout

    Citation: BMC Medical Research Methodology 2019 19:183

    Content type: Research article

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  26. Long-term clinical outcomes are necessary to assess the cost-effectiveness of new treatments over a lifetime horizon. Without long-term clinical trial data, current practice to extrapolate survival beyond the ...

    Authors: Shannon Cope, Dieter Ayers, Jie Zhang, Katharine Batt and Jeroen P. Jansen

    Citation: BMC Medical Research Methodology 2019 19:182

    Content type: Research article

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  27. 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

    Content type: Research article

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  28. 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

    Content type: Research article

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  29. 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

    Content type: Research article

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  30. 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

    Content type: Research article

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  31. 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

    Content type: Research article

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  32. 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

    Content type: Research article

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  33. 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

    Content type: Research article

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  34. 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

    Content type: Research article

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  35. 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

    Content type: Research article

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  36. 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

    Content type: Research article

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  37. 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

    Content type: Research article

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  38. 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

    Content type: Research article

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  39. 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

    Content type: Research article

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  40. 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

    Content type: Technical advance

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  41. 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

    Content type: Research article

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  42. 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

    Content type: Research article

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  43. 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

    Content type: Research article

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  44. 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

    Content type: Research article

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  45. 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

    Content type: Research article

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  46. 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

    Content type: Research article

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  47. 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

    Content type: Research article

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

  48. 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

    Content type: Debate

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  49. 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

    Content type: Research article

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