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  1. With the dramatic increase in the pipeline for new sickle cell disease (SCD) therapies in recent years, the time is ripe to ensure a robust body of evidence is available for decision making by regulators, paye...

    Authors: Ellen Tambor, Matoya Robinson, Lewis Hsu, Hsing-Yuan Chang and Jennifer Al Naber
    Citation: BMC Medical Research Methodology 2021 21:219
  2. Vascular prevention trials typically use dichotomous event outcomes although this may be inefficient statistically and gives no indication of event severity. We assessed whether ordinal outcomes would be more ...

    Authors: Lisa J. Woodhouse, Alan A. Montgomery, Jonathan Mant, Barry R. Davis, Ale Algra, Jean-Louis Mas, Jan A. Staessen, Lutgarde Thijs, Andrew Tonkin, Adrienne Kirby, Stuart J. Pocock, John Chalmers, Graeme J. Hankey, J. David Spence, Peter Sandercock, Hans-Christoph Diener…
    Citation: BMC Medical Research Methodology 2021 21:218
  3. Th EQUATOR Network improves the quality and transparency in health research, primarily by promoting awareness and use of reporting guidelines. In 2018, the UK EQUATOR Centre launched

    Authors: Caroline Struthers, James Harwood, Jennifer Anne de Beyer, Paula Dhiman, Patricia Logullo and Michael Schlüssel
    Citation: BMC Medical Research Methodology 2021 21:217
  4. Risk prediction models for time-to-event outcomes play a vital role in personalized decision-making. A patient’s biomarker values, such as medical lab results, are often measured over time but traditional pred...

    Authors: Kaci L Pickett, Krithika Suresh, Kristen R Campbell, Scott Davis and Elizabeth Juarez-Colunga
    Citation: BMC Medical Research Methodology 2021 21:216
  5. Kruskal-Wallis H test from the bank of classical statistics tests is a well-known nonparametric alternative to a one-way analysis of variance. The test is extensively used in decision-making problems where one...

    Authors: Rehan Ahmad Khan Sherwani, Huma Shakeel, Wajiha Batool Awan, Maham Faheem and Muhammad Aslam
    Citation: BMC Medical Research Methodology 2021 21:215
  6. Case-crossover studies have been widely used in various fields including pharmacoepidemiology. Vines and Farrington indicated in 2001 that when within-subject exposure dependency exists, conditional logistic r...

    Authors: Kiyoshi Kubota, Thu-Lan Kelly, Tsugumichi Sato, Nicole Pratt, Elizabeth Roughead and Takuhiro Yamaguchi
    Citation: BMC Medical Research Methodology 2021 21:214
  7. The external validity of the randomized controlled trial (RCT) refers to the extent to which the results of the RCT apply to the relevant, non-trial population and is impacted by its eligibility criteria, its ...

    Authors: Neil Thivalapill, Shahin Lockman, Kathleen Powis, Rebecca Zash, Jean Leidner, Gbolahan Ajibola, Mompati Mmalane, Joseph Makhema and Roger L. Shapiro
    Citation: BMC Medical Research Methodology 2021 21:212
  8. Disease surveillance of diabetes among youth has relied mainly upon manual chart review. However, increasingly available structured electronic health record (EHR) data have been shown to yield accurate determi...

    Authors: Kristin M. Lenoir, Lynne E. Wagenknecht, Jasmin Divers, Ramon Casanova, Dana Dabelea, Sharon Saydah, Catherine Pihoker, Angela D. Liese, Debra Standiford, Richard Hamman and Brian J. Wells
    Citation: BMC Medical Research Methodology 2021 21:210
  9. Recruitment to stroke clinical trials is challenging, but consumer registers can facilitate participation. Researchers need to understand the key factors that facilitate trial involvement and improve consumer ...

    Authors: Ishanka Weerasekara, Jasmine Baye, Meredith Burke, Gary Crowfoot, Gillian Mason, Rachael Peak, Dawn Simpson, Frederick Rohan Walker, Michael Nilsson, Michael Pollack and Coralie English
    Citation: BMC Medical Research Methodology 2021 21:209
  10. In drug trials, clinical adverse events (AEs), concomitant medication and laboratory safety outcomes are repeatedly collected to support drug safety evidence. Despite the potential correlation of these outcome...

    Authors: Noel Patson, Mavuto Mukaka, Umberto D’Alessandro, Gertrude Chapotera, Victor Mwapasa, Don Mathanga, Lawrence Kazembe, Miriam K. Laufer and Tobias Chirwa
    Citation: BMC Medical Research Methodology 2021 21:208
  11. Network Meta-Analysis (NMA) is a key component of submissions to reimbursement agencies world-wide, especially when there is limited direct head-to-head evidence for multiple technologies from randomised contr...

    Authors: David A. Jenkins, Humaira Hussein, Reynaldo Martina, Pascale Dequen-O’Byrne, Keith R. Abrams and Sylwia Bujkiewicz
    Citation: BMC Medical Research Methodology 2021 21:207
  12. Identifying strategies to optimize participation in health studies is one of the major concerns for researchers. The purpose of this study was to evaluate the efficiency of different invitation strategies on p...

    Authors: Rezvan Rajabzadeh, Leila Janani and Seyed Abbas Motevalian
    Citation: BMC Medical Research Methodology 2021 21:206
  13. Parent participation in children’s health interventions is insufficiently defined and measured. This project quantified parent participation to enable future examination with outcomes in an intervention focuse...

    Authors: Barbara Lohse and Leslie Cunningham-Sabo
    Citation: BMC Medical Research Methodology 2021 21:205
  14. Many recent studies have investigated the hospital volume-outcome relationship in surgery. In some cases, the results have prompted the centralization of surgical activity. However, the methodologies and inter...

    Authors: Mathieu Levaillant, Romaric Marcilly, Lucie Levaillant, Philippe Michel, Jean-François Hamel-Broza, Benoît Vallet and Antoine Lamer
    Citation: BMC Medical Research Methodology 2021 21:204
  15. Little guidance exists regarding how best to upskill and support those delivering complex healthcare interventions to ensure robust trial outcomes and implementation fidelity. Mentoring was provided to occupat...

    Authors: Kristelle Craven, Jain Holmes, Katie Powers, Sara Clarke, Rachel L. Cripps, Rebecca Lindley, Julie Phillips, Ruth Tyerman, Christopher McKevitt, David Clarke and Kathryn Radford
    Citation: BMC Medical Research Methodology 2021 21:203
  16. The purpose of this formative study was to assess barriers and facilitators to participation of transgender and gender diverse (TGD) patients in clinical research to solicit specific feedback on perceived acce...

    Authors: Andrew Asquith, Lauren Sava, Alexander B. Harris, Asa E. Radix, Dana J. Pardee and Sari L. Reisner
    Citation: BMC Medical Research Methodology 2021 21:202
  17. To examine the value of a Sequential Multiple Assignment Randomized Trial (SMART) design compared to a conventional randomized control trial (RCT) for telemedicine strategies to support titration of insulin th...

    Authors: Xiaoxi Yan, David B. Matchar, Nirmali Sivapragasam, John P. Ansah, Aastha Goel and Bibhas Chakraborty
    Citation: BMC Medical Research Methodology 2021 21:200
  18. For finite samples with binary outcomes penalized logistic regression such as ridge logistic regression has the potential of achieving smaller mean squared errors (MSE) of coefficients and predictions than max...

    Authors: Hana Šinkovec, Georg Heinze, Rok Blagus and Angelika Geroldinger
    Citation: BMC Medical Research Methodology 2021 21:199
  19. In many clinical applications, evolution of a longitudinal marker is censored by an event occurrence, and, symmetrically, event occurrence can be influenced by the longitudinal marker evolution. In such framew...

    Authors: Maéva Kyheng, Génia Babykina, Camille Ternynck, David Devos, Julien Labreuche and Alain Duhamel
    Citation: BMC Medical Research Methodology 2021 21:198
  20. To illustrate the challenges of estimating the effect of an exposure that is bounded by duration of follow-up on all-cause 28-day mortality, whilst simultaneously addressing missing data and time-varying covar...

    Authors: Rebecca Evans, Katie Pike, Alasdair MacGowan and Chris A. Rogers
    Citation: BMC Medical Research Methodology 2021 21:197
  21. Statistical model building requires selection of variables for a model depending on the model’s aim. In descriptive and explanatory models, a common recommendation often met in the literature is to include all...

    Authors: Lorena Hafermann, Heiko Becher, Carolin Herrmann, Nadja Klein, Georg Heinze and Geraldine Rauch
    Citation: BMC Medical Research Methodology 2021 21:196
  22. Extensive literature has shown an association of Adverse Childhood Experiences (ACEs) with adverse health outcomes; however, its ability to predict events or stratify risks is less known. Individuals with ment...

    Authors: Asmita Bhattarai, Gina Dimitropoulos, Brian Marriott, Jaime Paget, Andrew G. M. Bulloch, Suzanne C. Tough and Scott B. Patten
    Citation: BMC Medical Research Methodology 2021 21:195
  23. Advance care planning (ACP) enables people to define, discuss, and record preferences for treatment and care. Measures of ACP behavior are lacking in the Netherlands. We aimed to translate, culturally adapt an...

    Authors: Doris van der Smissen, Agnes van der Heide, Rebecca L. Sudore, Judith A. C. Rietjens and Ida J. Korfage
    Citation: BMC Medical Research Methodology 2021 21:194
  24. As a hot method in machine learning field, the forests approach is an attractive alternative approach to Cox model. Random survival forests (RSF) methodology is the most popular survival forests method, wherea...

    Authors: Yingxin Liu, Shiyu Zhou, Hongxia Wei and Shengli An
    Citation: BMC Medical Research Methodology 2021 21:193
  25. In follow-up studies, the occurrence of the intermediate event may influence the risk of the outcome of interest. Existing methods estimate the effect of the intermediate event by including a time-varying cova...

    Authors: Haixia Hu, Ling Wang, Chen Li, Wei Ge and Jielai Xia
    Citation: BMC Medical Research Methodology 2021 21:192
  26. The lung allocation system in the U.S. prioritizes lung transplant candidates based on estimated pre- and post-transplant survival via the Lung Allocation Scores (LAS). However, these models do not account for...

    Authors: Erin M. Schnellinger, Edward Cantu III, Michael O. Harhay, Douglas E. Schaubel, Stephen E. Kimmel and Alisa J. Stephens-Shields
    Citation: BMC Medical Research Methodology 2021 21:191
  27. Observational studies are increasingly being used to provide supplementary evidence in addition to Randomized Control Trials (RCTs) because they provide a scale and diversity of participants and outcomes that ...

    Authors: Chinmay Belthangady, Will Stedden and Beau Norgeot
    Citation: BMC Medical Research Methodology 2021 21:190
  28. Single group data present unique challenges for synthesises of evidence. Proportional meta-analysis is becoming an increasingly common technique employed for the synthesis of single group data. Proportional me...

    Authors: Timothy Hugh Barker, Celina Borges Migliavaca, Cinara Stein, Verônica Colpani, Maicon Falavigna, Edoardo Aromataris and Zachary Munn
    Citation: BMC Medical Research Methodology 2021 21:189
  29. Patient-centered care is an essential component of health care quality. To achieve patient-centered care, health care authorities should have a clear definition and an applicable tool to measure the extent of ...

    Authors: Hana Hasan Webair, Tengku Alina Tengku Ismail, Shaiful Bahari Ismail, Azza Jameel Khaffaji, Nik Hazlina Nik Hussain, Azidah Abdul Kadir, Rosediani Muhamad, Fatin Aina Abu Bakar, Nur Raihan Ismail and Nagwa Badri
    Citation: BMC Medical Research Methodology 2021 21:188
  30. Fundamental to the success of clinical research that involves human participants is the quality of the data that is generated. To ensure data quality, clinical trials must comply with the Good Clinical Practic...

    Authors: Lauren Houston, Ping Yu, Allison Martin and Yasmine Probst
    Citation: BMC Medical Research Methodology 2021 21:187
  31. The emergence of antimicrobial resistance across populations is a global threat to public health. Surveillance programs often monitor human and animal populations to evaluate trends of emergence in these popul...

    Authors: Min Zhang, Chong Wang and Annette M. O’Connor
    Citation: BMC Medical Research Methodology 2021 21:186
  32. There are significant challenges associated with studies of people released from custodial settings, including loss to follow-up in the community. Interpretation of findings with consideration of differences b...

    Authors: Ashleigh Cara Stewart, Reece Cossar, Shelley Walker, Anna Lee Wilkinson, Brendan Quinn, Paul Dietze, Rebecca Winter, Amy Kirwan, Michael Curtis, James R. P. Ogloff, Stuart Kinner, Campbell Aitken, Tony Butler, Emma Woods and Mark Stoové
    Citation: BMC Medical Research Methodology 2021 21:185
  33. Reviews of qualitative studies allow for deeper understanding of concepts and findings beyond the single qualitative studies. Concerns on study reporting quality led to the publication of the COREQ-guidelines ...

    Authors: Y. de Jong, E. M. van der Willik, J. Milders, C. G. N. Voorend, Rachael L. Morton, F. W. Dekker, Y. Meuleman and M. van Diepen
    Citation: BMC Medical Research Methodology 2021 21:184
  34. Systematic and scoping literature searches are increasingly resource intensive. We present the results of a scoping review which combines the use of a novel artificial-intelligence-(AI)-assisted Medline search...

    Authors: Jane Whelan, Mohammad Ghoniem, Nicolas Médoc, Mike Apicella and Ekkehard Beck
    Citation: BMC Medical Research Methodology 2021 21:183

    The Correction to this article has been published in BMC Medical Research Methodology 2021 21:276

  35. Healthcare decisions are ideally based on clinical trial results, published in study registries, as journal articles or summarized in secondary research articles. In this research project, we investigated the ...

    Authors: Anette Blümle, Katharina Wollmann, Karin Bischoff, Philipp Kapp, Szimonetta Lohner, Edris Nury, Kai Nitschke, Jasmin Zähringer, Gerta Rücker and Martin Schumacher
    Citation: BMC Medical Research Methodology 2021 21:182
  36. Interrupted time series (ITS) studies are frequently used to evaluate the effects of population-level interventions or exposures. However, examination of the performance of statistical methods for this design ...

    Authors: Simon L. Turner, Andrew B. Forbes, Amalia Karahalios, Monica Taljaard and Joanne E. McKenzie
    Citation: BMC Medical Research Methodology 2021 21:181
  37. Since primary data collection can be time-consuming and expensive, surgical site infections (SSIs) could ideally be monitored using routinely collected administrative data. We derived and internally validated ...

    Authors: Yelena Petrosyan, Kednapa Thavorn, Glenys Smith, Malcolm Maclure, Roanne Preston, Carl van Walravan and Alan J. Forster
    Citation: BMC Medical Research Methodology 2021 21:179
  38. Variations in clinical practice contribute to negative outcomes for children with cancer. Research in this area is imperative to standardise practice, yet such research is challenging to undertake, and a signi...

    Authors: Natalie Bradford, Christine Cashion, Paula Condon, Shelley Rumble and Alison Bowers
    Citation: BMC Medical Research Methodology 2021 21:178
  39. Non-proportional hazards are common with time-to-event data but the majority of randomised clinical trials (RCTs) are designed and analysed using approaches which assume the treatment effect follows proportion...

    Authors: Kim Jachno, Stephane Heritier and Rory Wolfe
    Citation: BMC Medical Research Methodology 2021 21:177
  40. Participation in epidemiologic studies has been declining over the last decades. In addition to postal invitations and phone calls, home visits can be conducted to increase participation. The aim of this study...

    Authors: Lilian Krist, Ahmed Bedir, Julia Fricke, Alexander Kluttig and Rafael Mikolajczyk
    Citation: BMC Medical Research Methodology 2021 21:176
  41. Randomized controlled trials (RCT) are considered the ideal design for evaluating the efficacy of interventions. However, conducting a successful RCT has technological and logistical challenges. Defects in ran...

    Authors: Sina Kianersi, Maya Luetke, Christina Ludema, Alexander Valenzuela and Molly Rosenberg
    Citation: BMC Medical Research Methodology 2021 21:175
  42. The use of auxiliary variables with maximum likelihood parameter estimation for surveys that miss data by design is not a widespread approach, despite its documented improved efficiency over traditional approa...

    Authors: George Vamvakas, Courtenay Norbury and Andrew Pickles
    Citation: BMC Medical Research Methodology 2021 21:173
  43. While web conferencing technologies are being widely used in communication and collaboration, their uptake in conducting research field work has been relatively slow. The benefits that these technologies offer...

    Authors: Kate Anderson, Alana Gall, Tamara Butler, Brian Arley, Kirsten Howard, Alan Cass and Gail Garvey
    Citation: BMC Medical Research Methodology 2021 21:172
  44. Null hypothesis significance testing (NHST) is among the most frequently employed methods in the biomedical sciences. However, the problems of NHST and p-values have been discussed widely and various Bayesian alt...

    Authors: Riko Kelter
    Citation: BMC Medical Research Methodology 2021 21:171
  45. To model the progression of geographic atrophy (GA) in patients with age-related macular degeneration (AMD) by building a suitable statistical regression model for GA size measurements obtained from fundus aut...

    Authors: Charlotte Behning, Monika Fleckenstein, Maximilian Pfau, Christine Adrion, Lukas Goerdt, Moritz Lindner, Steffen Schmitz-Valckenberg, Frank G Holz and Matthias Schmid
    Citation: BMC Medical Research Methodology 2021 21:170

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