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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. 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

    Published on:

  2. 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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  3. 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

    Published on:

  4. 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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  5. 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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  6. 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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  7. 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

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  8. 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

    Published on:

  9. 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:

  10. 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

    Published on:

  11. 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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  12. 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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  13. 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

    Published on:

  14. 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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  15. 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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  16. 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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  17. 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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  18. 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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  19. 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:

  20. 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:

  21. 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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  22. 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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  23. 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:

  24. 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

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  25. 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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  26. 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:

  27. 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:

  28. 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

    Published on:

  29. 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:

  30. 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

    Published on:

  31. 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

    Published on:

  32. 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

    Published on:

  33. 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

    Published on:

  34. 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:

  35. 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:

  36. Long-term effectiveness of asthma control medication has been shown in clinical trials but results from observational studies with children and adolescents are lacking. Marginal structural models estimated usi...

    Authors: Carolin Veit, Ronald Herrera, Gudrun Weinmayr, Jon Genuneit, Doris Windstetter, Christian Vogelberg, Erika von Mutius, Dennis Nowak, Katja Radon, Jessica Gerlich and Tobias Weinmann

    Citation: BMC Medical Research Methodology 2020 20:307

    Content type: Research article

    Published on:

  37. 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:

  38. In most countries, the official statistics for the coronavirus disease 2019 (COVID-19) take account of in-hospital deaths but not those that occur at home. The study’s objective was to introduce a methodology ...

    Authors: Hervé Hubert, Valentine Baert, Jean-Baptiste Beuscart and Emmanuel Chazard

    Citation: BMC Medical Research Methodology 2020 20:305

    Content type: Research article

    Published on:

  39. To assess the agreement of continuous measurements between a number of observers, Jones et al. introduced limits of agreement with the mean (LOAM) for multiple observers, representing how much an individual ob...

    Authors: Heidi S. Christensen, Jens Borgbjerg, Lars Børty and Martin Bøgsted

    Citation: BMC Medical Research Methodology 2020 20:304

    Content type: Technical advance

    Published on:

  40. Records of medication prescriptions can be used in conjunction with pharmacy dispensing records to investigate the incidence of adherence, which is defined as observing the treatment plans agreed between a patien...

    Authors: Holly Tibble, James Lay-Flurrie, Aziz Sheikh, Rob Horne, Mehrdad A. Mizani and Athanasios Tsanas

    Citation: BMC Medical Research Methodology 2020 20:303

    Content type: Research article

    Published on:

  41. Social-environmental data obtained from the US Census is an important resource for understanding health disparities, but rarely is the full dataset utilized for analysis. A barrier to incorporating the full da...

    Authors: Elizabeth Handorf, Yinuo Yin, Michael Slifker and Shannon Lynch

    Citation: BMC Medical Research Methodology 2020 20:302

    Content type: Research article

    Published on:

  42. Typically, subgroup analyses in clinical trials are conducted by comparing the intervention effect in each subgroup by means of an interaction test. However, trials are rarely, if ever, adequately powered for ...

    Authors: Russell Thirard, Raimondo Ascione, Jane M. Blazeby and Chris A. Rogers

    Citation: BMC Medical Research Methodology 2020 20:300

    Content type: Technical advance

    Published on:

  43. Precise predictions of incidence and mortality rates due to breast cancer (BC) are required for planning of public health programs as well as for clinical services. A number of approaches has been established ...

    Authors: Sumaira Mubarik, Ying Hu and Chuanhua Yu

    Citation: BMC Medical Research Methodology 2020 20:299

    Content type: Research article

    Published on:

  44. In recent months, multiple efforts have sought to characterize COVID-19 social distancing policy responses. These efforts have used various coding frameworks, but many have relied on coding methodologies that ...

    Authors: Jeff Lane, Michelle M. Garrison, James Kelley, Priya Sarma and Aaron Katz

    Citation: BMC Medical Research Methodology 2020 20:298

    Content type: Research article

    Published on:

  45. Even though investigating predictors of intervention success (e.g Cognitive Training, CT) is gaining more and more interest in the light of an individualized medicine, results on specific predictors of interve...

    Authors: André Mattes and Mandy Roheger

    Citation: BMC Medical Research Methodology 2020 20:296

    Content type: Research article

    Published on:

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