Articles

Response rate of patient reported outcomes: the delivery method matters

Patient Reported Outcomes (PROs) are subjective outcomes of disease and/or treatment in clinical research. For effective evaluations of PROs, high response rates are crucial. This study assessed the impact of ...

Authors: Olaf M. Neve, Peter Paul G. van Benthem, Anne M. Stiggelbout and Erik F. Hensen

coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials

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

Statistical reanalysis of vascular event outcomes in primary and secondary vascular prevention trials

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…

GoodReports: developing a website to help health researchers find and use reporting guidelines

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

Random survival forests for dynamic predictions of a time-to-event outcome using a longitudinal biomarker

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

Analysis of COVID-19 data using neutrosophic Kruskal Wallis H test

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

A novel weighting method to remove bias from within-subject exposure dependency in case-crossover studies

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

Predictive P-score for treatment ranking in Bayesian network meta-analysis

Network meta-analysis (NMA) is a widely used tool to compare multiple treatments by synthesizing different sources of evidence. Measures such as the surface under the cumulative ranking curve (SUCRA) and the P...

Authors: Kristine J. Rosenberger, Rui Duan, Yong Chen and Lifeng Lin

Are morbidity and mortality estimates from randomized controlled trials externally valid? A comparison of outcomes among infants enrolled into an RCT or a cohort study in Botswana

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

Employing multiple synchronous outcome samples per subject to improve study efficiency

Accuracy can be improved by taking multiple synchronous samples from each subject in a study to estimate the endpoint of interest if sample values are not highly correlated. If feasible, it is useful to assess...

Authors: Roger P. A’Hern

Determining diagnosis date of diabetes using structured electronic health record (EHR) data: the SEARCH for diabetes in youth study

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

What do stroke survivors’ value about participating in research and what are the most important research problems related to stroke or transient ischemic attack (TIA)? A survey

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

Joint modelling of multivariate longitudinal clinical laboratory safety outcomes, concomitant medication and clinical adverse events: application to artemisinin-based treatment during pregnancy clinical trial

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

Methods for the inclusion of real-world evidence in network meta-analysis

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

Effects of different invitation strategies on participation in a cohort study of Iranian public sector employees: a cluster randomized trial

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

Quantifying parent engagement in the randomized Fuel for Fun impact study identified design considerations and BMI relationships

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

Assessing the hospital volume-outcome relationship in surgery: a scoping review

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

Embedding mentoring to support trial processes and implementation fidelity in a randomised controlled trial of vocational rehabilitation for stroke survivors

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

Patient-centered practices for engaging transgender and gender diverse patients in clinical research studies

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

Chi-square test under indeterminacy: an application using pulse count data

The data obtained from the counting process is known as the count data. In practice, the counting can be done at the same time or the time of the count is not the same. To test either the K counts are differed...

Authors: Muhammad Aslam

Sequential Multiple Assignment Randomized Trial (SMART) to identify optimal sequences of telemedicine interventions for improving initiation of insulin therapy: A simulation study

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

To tune or not to tune, a case study of ridge logistic regression in small or sparse datasets

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

Joint latent class model: Simulation study of model properties and application to amyotrophic lateral sclerosis disease

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

Analytical challenges in estimating the effect of exposures that are bounded by follow-up time: experiences from the Blood Stream Infection—Focus on Outcomes study

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

Statistical model building: Background “knowledge” based on inappropriate preselection causes misspecification

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

Can the adverse childhood experiences (ACEs) checklist be utilized to predict emergency department visits among children and adolescents?

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

Measuring advance care planning behavior in Dutch adults: translation, cultural adaptation and validation of the Advance Care Planning Engagement Survey

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

A comparative study of forest methods for time-to-event data: variable selection and predictive performance

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

An improved method for the effect estimation of the intermediate event on the outcome based on the susceptible pre-identification

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

Mitigating selection bias in organ allocation models

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

Minimizing bias in massive multi-arm observational studies with BCAUS: balancing covariates automatically using supervision

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

Conducting proportional meta-analysis in different types of systematic reviews: a guide for synthesisers of evidence

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

Patient-centered infertility questionnaire for female clients (PCIQ-F): part I: questionnaire development

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

Clinical researchers’ lived experiences with data quality monitoring in clinical trials: a qualitative study

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

A Bayesian latent class mixture model with censoring for correlation analysis in antimicrobial resistance across populations

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

Strategies to maximise study retention and limit attrition bias in a prospective cohort study of men reporting a history of injecting drug use released from prison: the prison and transition health study

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

A meta-review demonstrates improved reporting quality of qualitative reviews following the publication of COREQ- and ENTREQ-checklists, regardless of modest uptake

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

Applying a novel approach to scoping review incorporating artificial intelligence: mapping the natural history of gonorrhoea

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

Investigator initiated trials versus industry sponsored trials - translation of randomized controlled trials into clinical practice (IMPACT)

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

Evaluation of statistical methods used in the analysis of interrupted time series studies: a simulation study

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

Evaluating the impact of covariate lookback times on performance of patient-level prediction models

The goal of our study is to examine the impact of the lookback length when engineering features to use in developing predictive models using observational healthcare data. Using a longer lookback for feature e...

Authors: Jill Hardin and Jenna M. Reps

Predicting postoperative surgical site infection with administrative data: a random forests algorithm

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

Recruitment principles and strategies for supportive care research in pediatric oncology

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

Impact of a non-constant baseline hazard on detection of time-dependent treatment effects: a simulation study

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

The effect of home visits as an additional recruitment step on the composition of the final sample: a cross-sectional analysis in two study centers of the German National Cohort (NAKO)

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

Use of research electronic data capture (REDCap) in a COVID-19 randomized controlled trial: a practical example

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

Correction to: Applied comparison of large-scale propensity score matching and cardinality matching for causal inference in observational research

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

Two-stage sampling in the estimation of growth parameters and percentile norms: sample weights versus auxiliary variable estimation

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

Bayesian Hodges-Lehmann tests for statistical equivalence in the two-sample setting: Power analysis, type I error rates and equivalence boundary selection in biomedical research

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

Modeling of atrophy size trajectories: variable transformation, prediction and age-of-onset estimation

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