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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 15

  1. Linking independent sources of data describing the same individuals enable innovative epidemiological and health studies but require a robust record linkage approach. We describe a hybrid record linkage proces...

    Authors: Yue Jiao, Fabienne Lesueur, Chloé-Agathe Azencott, Maïté Laurent, Noura Mebirouk, Lilian Laborde, Juana Beauvallet, Marie-Gabrielle Dondon, Séverine Eon-Marchais, Anthony Laugé, Catherine Noguès, Nadine Andrieu, Dominique Stoppa-Lyonnet and Sandrine M. Caputo

    Citation: BMC Medical Research Methodology 2021 21:155

    Content type: Research article

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  2. Converting electronic health record (EHR) entries to useful clinical inferences requires one to address the poor scalability of existing implementations of Generalized Linear Mixed Models (GLMM) for repeated m...

    Authors: Cristian G. Bologa, Vernon Shane Pankratz, Mark L. Unruh, Maria Eleni Roumelioti, Vallabh Shah, Saeed Kamran Shaffi, Soraya Arzhan, John Cook and Christos Argyropoulos

    Citation: BMC Medical Research Methodology 2021 21:151

    Content type: Technical advance

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  3. Already at hospital admission, clinicians require simple tools to identify hospitalized COVID-19 patients at high risk of mortality. Such tools can significantly improve resource allocation and patient managem...

    Authors: Hamid Reza Marateb, Maja von Cube, Ramin Sami, Shaghayegh Haghjooy Javanmard, Marjan Mansourian, Babak Amra, Forogh Soltaninejad, Mojgan Mortazavi, Peyman Adibi, Nilufar Khademi, Nastaran Sadat Hosseini, Arash Toghyani, Razieh Hassannejad, Miquel Angel Mañanas, Harald Binder and Martin Wolkewitz

    Citation: BMC Medical Research Methodology 2021 21:146

    Content type: Research article

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  4. A large multi-center survey was conducted to understand patients’ perspectives on biobank study participation with particular focus on racial and ethnic minorities. In order to enrich the study sample with rac...

    Authors: Aya A. Mitani, Nathaniel D. Mercaldo, Sebastien Haneuse and Jonathan S. Schildcrout

    Citation: BMC Medical Research Methodology 2021 21:145

    Content type: Research article

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  5. Case–control studies based on pharmaco-epidemiological databases typically use decision rules to determine exposure status from information on dates of prescription redemptions, although this induces misclassi...

    Authors: Henrik Støvring, Anton Pottegård and Jesper Hallas

    Citation: BMC Medical Research Methodology 2021 21:144

    Content type: Research article

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  6. Various interacting and interdependent components comprise complex interventions. These components create difficulty in assessing the true impact of interventions designed to improve patient-centered outcomes....

    Authors: Maricela Cruz, Marco A. Pinto-Orellana, Daniel L. Gillen and Hernando C. Ombao

    Citation: BMC Medical Research Methodology 2021 21:143

    Content type: Software

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  7. Clustering of observations is a common phenomenon in epidemiological and clinical research. Previous studies have highlighted the importance of using multilevel analysis to account for such clustering, but in ...

    Authors: Georgia Ntani, Hazel Inskip, Clive Osmond and David Coggon

    Citation: BMC Medical Research Methodology 2021 21:139

    Content type: Research article

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  8. Hometime, the total number of days a person is living in the community (not in a healthcare institution) in a defined period of time after a hospitalization, is a patient-centred outcome metric increasingly us...

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

    Citation: BMC Medical Research Methodology 2021 21:138

    Content type: Research article

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  9. Clustered data arise in research when patients are clustered within larger units. Generalised Estimating Equations (GEE) and Generalised Linear Models (GLMM) can be used to provide marginal and cluster-specifi...

    Authors: Menelaos Pavlou, Gareth Ambler and Rumana Z. Omar

    Citation: BMC Medical Research Methodology 2021 21:135

    Content type: Research article

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  10. Despite experimental evidence suggesting that pain sensitivity is not impaired by cognitive impairment, observational studies in nursing home residents have observed an inverse association between cognitive im...

    Authors: Anthony P. Nunes, Danni Zhao, William M. Jesdale and Kate L. Lapane

    Citation: BMC Medical Research Methodology 2021 21:132

    Content type: Research article

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  11. Patient-Reported Outcomes (PROs) are standardized questionnaires used to measure subjective outcomes such as quality of life in healthcare. They are considered paramount to assess the results of therapeutic in...

    Authors: Antoine Vanier, Véronique Sébille, Myriam Blanchin and Jean-Benoit Hardouin

    Citation: BMC Medical Research Methodology 2021 21:128

    Content type: Research article

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  12. In a star-shaped network, pairwise comparisons link treatments with a reference treatment (often placebo or standard care), but not with each other. Thus, comparisons between non-reference treatments rely on i...

    Authors: Jeong-Hwa Yoon, Sofia Dias and Seokyung Hahn

    Citation: BMC Medical Research Methodology 2021 21:113

    Content type: Technical advance

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  13. Cardinality matching (CM), a novel matching technique, finds the largest matched sample meeting prespecified balance criteria thereby overcoming limitations of propensity score matching (PSM) associated with l...

    Authors: Stephen P. Fortin, Stephen S Johnston and Martijn J Schuemie

    Citation: BMC Medical Research Methodology 2021 21:109

    Content type: Research article

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  14. Sparse relative effectiveness evidence is a frequent problem in Health Technology Assessment (HTA). Where evidence directly pertaining to the decision problem is sparse, it may be feasible to expand the eviden...

    Authors: Georgios F. Nikolaidis, Beth Woods, Stephen Palmer and Marta O. Soares

    Citation: BMC Medical Research Methodology 2021 21:107

    Content type: Research article

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  15. Previous research has shown that chronic disease case definitions constructed using population-based administrative health data may have low accuracy for ascertaining cases of episodic diseases such as rheumat...

    Authors: Allison Feely, Lily SH Lim, Depeng Jiang and Lisa M. Lix

    Citation: BMC Medical Research Methodology 2021 21:105

    Content type: Research article

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  16. 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

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  17. 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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  18. 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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  19. 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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  20. 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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  21. 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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  22. 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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  23. 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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  24. 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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  25. 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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  26. 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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  27. 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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  28. 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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  29. 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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  30. 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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  31. 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:

  32. 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:

  33. 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:

  34. 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:

  35. 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

    Published on:

  36. 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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  37. 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:

  38. 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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  39. 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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  40. 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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  41. 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

    Published on:

  42. 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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  43. 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:

  44. 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:

  45. 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

    Published on:

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