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Data analysis, statistics and modelling

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 14

  1. Ninety-day hometime, the number of days a patient is living in the community in the first 90 after stroke, exhibits a non-normal bucket-shaped distribution, with lower and upper constraints making its analysis...

    Authors: Jessalyn K. Holodinsky, Amy Y. X. Yu, Moira K. Kapral and Peter C. Austin

    Citation: BMC Medical Research Methodology 2021 21:102

    Content type: Research article

    Published on:

  2. Advances in machine learning (ML) provide great opportunities in the prediction of hospital readmission. This review synthesizes the literature on ML methods and their performance for predicting hospital readm...

    Authors: Yinan Huang, Ashna Talwar, Satabdi Chatterjee and Rajender R. Aparasu

    Citation: BMC Medical Research Methodology 2021 21:96

    Content type: Research article

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  3. Diagnosis performances of case-identifying algorithms developed in healthcare database are usually assessed by comparing identified cases with an external data source. When this is not feasible, intra-database...

    Authors: Nicolas H. Thurin, Pauline Bosco-Levy, Patrick Blin, Magali Rouyer, Jérémy Jové, Stéphanie Lamarque, Séverine Lignot, Régis Lassalle, Abdelilah Abouelfath, Emmanuelle Bignon, Pauline Diez, Marine Gross-Goupil, Michel Soulié, Mathieu Roumiguié, Sylvestre Le Moulec, Marc Debouverie…

    Citation: BMC Medical Research Methodology 2021 21:95

    Content type: Research article

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  4. When quantifying the probability of survival in cancer patients using cancer registration data, it is common to estimate marginal relative survival, which under assumptions can be interpreted as marginal net s...

    Authors: Paul C. Lambert, Elisavet Syriopoulou and Mark R. Rutherford

    Citation: BMC Medical Research Methodology 2021 21:84

    Content type: Research article

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  5. Generalized linear mixed models (GLMMs), typically used for analyzing correlated data, can also be used for smoothing by considering the knot coefficients from a regression spline as random effects. The result...

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

    Citation: BMC Medical Research Methodology 2021 21:83

    Content type: Research article

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  6. An assumption in many analyses of longitudinal patient-reported outcome (PRO) data is that there is a single population following a single health trajectory. One approach that may help researchers move beyond ...

    Authors: Jae-Yung Kwon, Richard Sawatzky, Jennifer Baumbusch, Sandra Lauck and Pamela A. Ratner

    Citation: BMC Medical Research Methodology 2021 21:79

    Content type: Research article

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  7. Patient-reported outcome measures (PROMs) are commonly-used surrogates for clinical outcomes in cancer research. When researching severe diseases such as cancer, it is difficult to avoid the problem of incompl...

    Authors: Pimrapat Gebert, Daniel Schindel, Johann Frick, Liane Schenk and Ulrike Grittner

    Citation: BMC Medical Research Methodology 2021 21:77

    Content type: Research article

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  8. In non-inferiority trials, there is a concern that intention-to-treat (ITT) analysis, by including participants who did not receive the planned interventions, may bias towards making the treatment and control ...

    Authors: Anthony D. Bai, Adam S. Komorowski, Carson K. L. Lo, Pranav Tandon, Xena X. Li, Vaibhav Mokashi, Anna Cvetkovic, Aidan Findlater, Laurel Liang, George Tomlinson, Mark Loeb and Dominik Mertz

    Citation: BMC Medical Research Methodology 2021 21:75

    Content type: Research article

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  9. Chest pain is among the most common presenting complaints in the emergency department (ED). Swift and accurate risk stratification of chest pain patients in the ED may improve patient outcomes and reduce unnec...

    Authors: Nan Liu, Marcel Lucas Chee, Zhi Xiong Koh, Su Li Leow, Andrew Fu Wah Ho, Dagang Guo and Marcus Eng Hock Ong

    Citation: BMC Medical Research Methodology 2021 21:74

    Content type: Research article

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  10. Missing data are common in randomised controlled trials (RCTs) and can bias results if not handled appropriately. A statistically valid analysis under the primary missing-data assumptions should be conducted, ...

    Authors: Ping-Tee Tan, Suzie Cro, Eleanor Van Vogt, Matyas Szigeti and Victoria R. Cornelius

    Citation: BMC Medical Research Methodology 2021 21:72

    Content type: Research article

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  11. Conventional methods for phase I dose-escalation trials in oncology are based on a single treatment schedule only. More recently, however, multiple schedules are more frequently investigated in the same trial.

    Authors: Burak Kürsad Günhan, Sebastian Weber, Abdelkader Seroutou and Tim Friede

    Citation: BMC Medical Research Methodology 2021 21:69

    Content type: Research article

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  12. Cancer patients’ prognoses are complicated by comorbidities. Prognostic prediction models with inappropriate comorbidity adjustments yield biased survival estimates. However, an appropriate claims-based comorb...

    Authors: Sanghee Lee, Yoon Jung Chang and Hyunsoon Cho

    Citation: BMC Medical Research Methodology 2021 21:66

    Content type: Research article

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  13. The best way to calculate statistics from medical data is to use the data of individual patients. In some settings, this data is difficult to obtain due to privacy restrictions. In Germany, for example, it is ...

    Authors: Stefan Lenz, Moritz Hess and Harald Binder

    Citation: BMC Medical Research Methodology 2021 21:64

    Content type: Technical advance

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  14. Interrupted time series analysis is increasingly used to evaluate the impact of large-scale health interventions. While segmented regression is a common approach, it is not always adequate, especially in the p...

    Authors: Andrea L. Schaffer, Timothy A. Dobbins and Sallie-Anne Pearson

    Citation: BMC Medical Research Methodology 2021 21:58

    Content type: Technical advance

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  15. Estimation that employs instrumental variables (IV) can reduce or eliminate bias due to confounding. In observational studies, instruments result from natural experiments such as the effect of clinician prefer...

    Authors: Todd A. MacKenzie, Pablo Martinez-Camblor and A. James O’Malley

    Citation: BMC Medical Research Methodology 2021 21:56

    Content type: Research article

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  16. Beginning in 2019, stepped-wedge designs (SWDs) were being used in the investigation of interventions to reduce opioid-related deaths in communities across the United States. However, these interventions are c...

    Authors: Lior Rennert, Moonseong Heo, Alain H. Litwin and Victor De Gruttola

    Citation: BMC Medical Research Methodology 2021 21:53

    Content type: Research article

    Published on:

  17. Royston-Parmar flexible parametric survival models (FPMs) can be fitted on either the cause-specific hazards or cumulative incidence scale in the presence of competing risks. An advantage of modelling within t...

    Authors: Sarwar I. Mozumder, Mark J. Rutherford and Paul C. Lambert

    Citation: BMC Medical Research Methodology 2021 21:52

    Content type: Research article

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  18. We propose a new measure of treatment effect based on the expected reduction in the number of patients to treat (RNT) which is defined as the difference of the reciprocals of clinical measures of interest betw...

    Authors: Chenyang Zhang and Guosheng Yin

    Citation: BMC Medical Research Methodology 2021 21:48

    Content type: Research article

    Published on:

  19. Waist circumference is becoming recognized as a useful predictor of health risks in clinical research. However, clinical datasets tend to lack this measurement and self-reported values tend to be inaccurate. P...

    Authors: Weihong Zhou, Spencer Eckler, Andrew Barszczyk, Alex Waese-Perlman, Yingjie Wang, Xiaoping Gu, Zhong-Ping Feng, Yuzhu Peng and Kang Lee

    Citation: BMC Medical Research Methodology 2021 21:47

    Content type: Technical advance

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  20. This study investigated the ArmA-TH sub-scale measurement properties based on item response theory using the Rasch model.

    Authors: Montana Buntragulpoontawee, Jeeranan Khunachiva, Patreeya Euawongyarti, Nahathai Wongpakaran, Tinakon Wongpakaran, Atcharee Kaewma and Stephen Ashford

    Citation: BMC Medical Research Methodology 2021 21:46

    Content type: Research article

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  21. The case-crossover design is suited to medication safety studies but is vulnerable to exposure misclassification. Using the example of tricyclic antidepressants and the risk of hip fracture, we present a data ...

    Authors: Michael J. Leach, Elizabeth E. Roughead and Nicole L. Pratt

    Citation: BMC Medical Research Methodology 2021 21:43

    Content type: Technical advance

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  22. Dynamic treatment regimens (DTRs) formalise the multi-stage and dynamic decision problems that clinicians often face when treating chronic or progressive medical conditions. Compared to randomised controlled t...

    Authors: Robert K. Mahar, Myra B. McGuinness, Bibhas Chakraborty, John B. Carlin, Maarten J. IJzerman and Julie A. Simpson

    Citation: BMC Medical Research Methodology 2021 21:39

    Content type: Research article

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  23. Time-to-event data that is subject to interval censoring is common in the practice of medical research and versatile statistical methods for estimating associations in such settings have been limited. For righ...

    Authors: Martin Nygård Johansen, Søren Lundbye-Christensen, Jacob Moesgaard Larsen and Erik Thorlund Parner

    Citation: BMC Medical Research Methodology 2021 21:36

    Content type: Technical advance

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  24. Scale-up BP was a quasi-experimental implementation study, following a successful randomised controlled trial of the roll-out of telemonitoring in primary care across Lothian, Scotland. Our primary objective w...

    Authors: Richard A. Parker, Paul Padfield, Janet Hanley, Hilary Pinnock, John Kennedy, Andrew Stoddart, Vicky Hammersley, Aziz Sheikh and Brian McKinstry

    Citation: BMC Medical Research Methodology 2021 21:31

    Content type: Research article

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  25. Statistical methods for modeling longitudinal and time-to-event data has received much attention in medical research and is becoming increasingly useful. In clinical studies, such as cancer and AIDS, longitudi...

    Authors: Julius S. Ngwa, Howard J. Cabral, Debbie M. Cheng, David R. Gagnon, Michael P. LaValley and L. Adrienne Cupples

    Citation: BMC Medical Research Methodology 2021 21:29

    Content type: Research article

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  26. Ethnographic approaches offer a method and a way of thinking about implementation. This manuscript applies a specific case study method to describe the impact of the longitudinal interplay between implementati...

    Authors: Jennifer M. Van Tiem, Heather Schacht Reisinger, Julia E. Friberg, Jaime R. Wilson, Lynn Fitzwater, Ralph J. Panos and Jane Moeckli

    Citation: BMC Medical Research Methodology 2021 21:27

    Content type: Research article

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  27. Over 2 billion people suffer from vision impairment or blindness globally, and access to validated visual measurement tools in imperative in accurately describing and managing the burden of eye disease. The pr...

    Authors: William Mitchell, Srinivas Marmamula, Nazlee Zebardast, Weiwen Ng, Joseph J. Locascio, Thirupathi Kumbam, Satya Brahmanandam and Navya Rekha Barrenkala

    Citation: BMC Medical Research Methodology 2021 21:26

    Content type: Research article

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  28. Early pregnancy weights are needed to quantify gestational weight gain accurately. Different methods have been used in previous studies to impute early-pregnancy weights. However, no studies have systematicall...

    Authors: Jiaxi Yang, Dongqing Wang, Anne Marie Darling, Enju Liu, Nandita Perumal, Wafaie W. Fawzi and Molin Wang

    Citation: BMC Medical Research Methodology 2021 21:24

    Content type: Research article

    Published on:

  29. The U.S. lacks a stroke surveillance system. This study develops a method to transform an existing registry into a nationally representative database to evaluate acute ischemic stroke care quality.

    Authors: Boback Ziaeian, Haolin Xu, Roland A. Matsouaka, Ying Xian, Yosef Khan, Lee S. Schwamm, Eric E. Smith and Gregg C. Fonarow

    Citation: BMC Medical Research Methodology 2021 21:23

    Content type: Research article

    Published on:

  30. Trials of novel agents are required to improve the care of patients with rare diseases, but trial feasibility may be uncertain due to concerns over insufficient patient numbers. We aimed to determine the size ...

    Authors: M. S. M. Persson, K. E. Harman, K. S. Thomas, J. R. Chalmers, Y. Vinogradova, S. M. Langan, J. Hippisley-Cox and S. Gran

    Citation: BMC Medical Research Methodology 2021 21:22

    Content type: Research article

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  31. The use of individual patient data (IPD) in network meta-analyses (NMA) is rapidly growing. This study aimed to determine, through simulations, the impact of select factors on the validity and precision of NMA...

    Authors: Steve Kanters, Mohammad Ehsanul Karim, Kristian Thorlund, Aslam Anis and Nick Bansback

    Citation: BMC Medical Research Methodology 2021 21:21

    Content type: Research article

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  32. Semi-competing risks arise when interest lies in the time-to-event for some non-terminal event, the observation of which is subject to some terminal event. One approach to assessing the impact of covariates on...

    Authors: Catherine Lee, Paola Gilsanz and Sebastien Haneuse

    Citation: BMC Medical Research Methodology 2021 21:18

    Content type: Research article

    Published on:

  33. Recurrent episodes of pneumonia are frequently modeled using extensions of the Cox proportional hazards model with the underlying assumption of time-constant relative risks measured by the hazard ratio. We aim...

    Authors: Jordache Ramjith, Kit C.B. Roes, Heather J. Zar and Marianne A. Jonker

    Citation: BMC Medical Research Methodology 2021 21:17

    Content type: Research article

    Published on:

  34. Multi-state models are being increasingly used to capture complex disease pathways. The convenient formula of the exponential multi-state model can facilitate a quick and accessible understanding of the data. ...

    Authors: Micki Hill, Paul C. Lambert and Michael J. Crowther

    Citation: BMC Medical Research Methodology 2021 21:16

    Content type: Research article

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  35. The rising burden of the ongoing COVID-19 epidemic in South Africa has motivated the application of modeling strategies to predict the COVID-19 cases and deaths. Reliable and accurate short and long-term forec...

    Authors: Tarylee Reddy, Ziv Shkedy, Charl Janse van Rensburg, Henry Mwambi, Pravesh Debba, Khangelani Zuma and Samuel Manda

    Citation: BMC Medical Research Methodology 2021 21:15

    Content type: Research article

    Published on:

  36. As cancer treatment, biotherapies can be as effective as chemotherapy while reducing the risk of secondary effects, so that they can be taken over longer periods than conventional chemotherapy. Thus, some tria...

    Authors: Adrien Guilloteau, Michal Abrahamowicz, Olayide Boussari, Valérie Jooste, Thomas Aparicio, Catherine Quantin, Karine Le Malicot and Christine Binquet

    Citation: BMC Medical Research Methodology 2021 21:14

    Content type: Research article

    Published on:

  37. Missing data are common in end-of-life care studies, but there is still relatively little exploration of which is the best method to deal with them, and, in particular, if the missing at random (MAR) assumptio...

    Authors: Giulia Carreras, Guido Miccinesi, Andrew Wilcock, Nancy Preston, Daan Nieboer, Luc Deliens, Mogensm Groenvold, Urska Lunder, Agnes van der Heide and Michela Baccini

    Citation: BMC Medical Research Methodology 2021 21:13

    Content type: Research article

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  38. Trials with binary outcomes can be synthesised using within-trial exact likelihood or approximate normal likelihood in one-stage or two-stage approaches, respectively. The performance of the one-stage and the ...

    Authors: Loukia M. Spineli, Katerina Papadimitropoulou and Chrysostomos Kalyvas

    Citation: BMC Medical Research Methodology 2021 21:12

    Content type: Research article

    Published on:

  39. Multivariable prediction models are important statistical tools for providing synthetic diagnosis and prognostic algorithms based on patients’ multiple characteristics. Their apparent measures for predictive a...

    Authors: Katsuhiro Iba, Tomohiro Shinozaki, Kazushi Maruo and Hisashi Noma

    Citation: BMC Medical Research Methodology 2021 21:9

    Content type: Research article

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  40. In increasingly ageing populations, there is an emergent need to develop a robust prediction model for estimating an individual absolute risk for all-cause mortality, so that relevant assessments and intervent...

    Authors: Olesya Ajnakina, Deborah Agbedjro, Ryan McCammon, Jessica Faul, Robin M. Murray, Daniel Stahl and Andrew Steptoe

    Citation: BMC Medical Research Methodology 2021 21:8

    Content type: Research article

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  41. Genital warts are a common and highly contagious sexually transmitted disease. They have a large economic burden and affect several aspects of quality of life. Incidence data underestimate the real occurrence ...

    Authors: David Moriña, Amanda Fernández-Fontelo, Alejandra Cabaña, Pedro Puig, Laura Monfil, Maria Brotons and Mireia Diaz

    Citation: BMC Medical Research Methodology 2021 21:6

    Content type: Research article

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  42. Questionnaires used in longitudinal studies may have questions added or removed over time for numerous reasons. Data missing completely at a follow-up survey is a unique issue for longitudinal studies. While s...

    Authors: Claire A. Kolaja, Ben Porter, Teresa M. Powell and Rudolph P. Rull

    Citation: BMC Medical Research Methodology 2021 21:5

    Content type: Research article

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  43. There is a growing interest in assessment of the quality of hospital care, based on outcome measures. Many quality of care comparisons rely on binary outcomes, for example mortality rates. Due to low numbers, ...

    Authors: I. E. Ceyisakar, N. van Leeuwen, Diederik W. J. Dippel, Ewout W. Steyerberg and H. F. Lingsma

    Citation: BMC Medical Research Methodology 2021 21:4

    Content type: Research article

    Published on:

  44. Time-series analysis with case-only data is a prominent method for the effect of environmental determinants on disease events in environmental epidemiology. In this analysis, adjustment for seasonality and lon...

    Authors: Honghyok Kim, Jong-Tae Lee, Kelvin C. Fong and Michelle L. Bell

    Citation: BMC Medical Research Methodology 2021 21:2

    Content type: Research article

    Published on:

  45. Although a variety of instruments are available that capture stress experience, the assessment of chronic stress has been hindered by the lack of economical screening instruments. Recently, an English-language...

    Authors: K. Petrowski, E. Braehler, B. Schmalbach, A. Hinz, C. Bastianon and T. Ritz

    Citation: BMC Medical Research Methodology 2020 20:306

    Content type: Research article

    Published on:

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