Articles

Modelling seizure rates rather than time to an event within clinical trials of antiepileptic drugs

Predictive models within epilepsy are frequently developed via Cox’s proportional hazards models. These models estimate risk of a specified event such as 12-month remission. They are relatively simple to produ...

Authors: Laura J. Bonnett, Jane L. Hutton and Anthony G. Marson

Clustering of continuous and binary outcomes at the general practice level in individually randomised studies in primary care - a review of 10 years of primary care trials

In randomised controlled trials, the assumption of independence of individual observations is fundamental to the design, analysis and interpretation of studies. However, in individually randomised trials in pr...

Authors: Beth Stuart, Taeko Becque, Michael Moore and Paul Little

Correction to: Estimation of treatment preference effects in clinical trials when some participants are indifferent to treatment choice

In the original publication of this article [1], the number “− 0.49” in the below sentence in the Results section should be changed to “-3.23”, and this typo does not affect the wider conclusions.

Authors: Stephen D. Walter, Robin M. Turner, Petra Macaskill, Kirsten J. McCaffery and Les Irwig

Methodological challenges of analysing COVID-19 data during the pandemic

Authors: Martin Wolkewitz and Livia Puljak

Agreement between the Schedule for the Evaluation of Individual Quality of Life-Direct Weighting (SEIQoL-DW) interview and a paper-administered adaption

The Schedule for the Evaluation of Individual Quality of Life-Direct Weighting (SEIQoL-DW) is a prevalent face-to-face interview method for measuring quality of life by integrating respondent-generated dimensi...

Authors: Marion Burckhardt, Steffen Fleischer and Almuth Berg

Assessing the quality of health research from an Indigenous perspective: the Aboriginal and Torres Strait Islander quality appraisal tool

The lack of attention to Indigenous epistemologies and, more broadly, Indigenous values in primary research, is mirrored in the standardised critical appraisal tools used to guide evidence-based practice and s...

Authors: Stephen Harfield, Odette Pearson, Kim Morey, Elaine Kite, Karla Canuto, Karen Glover, Judith Streak Gomersall, Drew Carter, Carol Davy, Edoardo Aromataris and Annette Braunack-Mayer

Telephone versus web panel National Survey for monitoring adoption of preventive behaviors to climate change in populations: a case study of Lyme disease in Québec, Canada

To monitor the adoption of climate change adaptive behaviors in the population, public health authorities have to conduct national surveys, which can help them target vulnerable subpopulations. To ensure relia...

Authors: Grâce Ngambo Domche, Pierre Valois, Magalie Canuel, Denis Talbot, Maxime Tessier, Cécile Aenishaenslin, Catherine Bouchard and Sandie Briand

Prevalence estimation by joint use of big data and health survey: a demonstration study using electronic health records in New York city

Electronic Health Records (EHR) has been increasingly used as a tool to monitor population health. However, subject-level errors in the records can yield biased estimates of health indicators. There is an urge...

Authors: Ryung S. Kim and Viswanathan Shankar

When piloting health services interventions, what predicts real world behaviours? A systematic concept mapping review

Modeling studies to inform the design of complex health services interventions often involves elements that differ from the intervention’s ultimate real-world use. These “hypothetical” elements include pilot p...

Authors: Tavis Hayes, Natasha Hudek, Ian D. Graham, Doug Coyle and Jamie C. Brehaut

Validation of an algorithm based on administrative data to detect new onset of atrial fibrillation after cardiac surgery

Postoperative atrial fibrillation (POAF) is a frequent complication of cardiac surgery associated with important morbidity, mortality, and costs. To assess the effectiveness of preventive interventions, an imp...

Authors: Jonathan Bourgon Labelle, Paul Farand, Christian Vincelette, Myriam Dumont, Mathilde Le Blanc and Christian M. Rochefort

Using estimated probability of pre-diagnosis behavior as a predictor of cancer survival time: an example in esophageal cancer

Information on the associations between pre-diagnosis health behavior and post-diagnosis survival time in esophageal cancer could assist in planning health services but can be difficult to obtain using establi...

Authors: Paul P. Fahey, Andrew Page, Glenn Stone and Thomas Astell-Burt

A factorial cluster-randomised controlled trial combining home-environmental and early child development interventions to improve child health and development: rationale, trial design and baseline findings

Exposure to unhealthy environments and inadequate child stimulation are main risk factors that affect children’s health and wellbeing in low- and middle-income countries. Interventions that simultaneously addr...

Authors: Stella M. Hartinger, Nestor Nuño, Jan Hattendorf, Hector Verastegui, Walter Karlen, Mariela Ortiz and Daniel Mäusezahl

Multiple imputation by predictive mean matching in cluster-randomized trials

Random effects regression imputation has been recommended for multiple imputation (MI) in cluster randomized trials (CRTs) because it is congenial to analyses that use random effects regression. This method re...

Authors: Brittney E. Bailey, Rebecca Andridge and Abigail B. Shoben

On estimating the time to statistical cure

The mortality risk among cancer patients measured from the time of diagnosis is often elevated in comparison to the general population. However, for some cancer types, the patient mortality risk will over time...

Authors: Lasse H. Jakobsen, Therese M.-L. Andersson, Jorne L. Biccler, Laurids Ø. Poulsen, Marianne T. Severinsen, Tarec C. El-Galaly and Martin Bøgsted

An evaluation of inverse probability weighting using the propensity score for baseline covariate adjustment in smaller population randomised controlled trials with a continuous outcome

It is important to estimate the treatment effect of interest accurately and precisely within the analysis of randomised controlled trials. One way to increase precision in the estimate and thus improve the pow...

Authors: Hanaya Raad, Victoria Cornelius, Susan Chan, Elizabeth Williamson and Suzie Cro

Assessing the effect of genetic markers on drug immunogenicity from a mechanistic model-based approach

With the growth in use of biotherapic drugs in various medical fields, the occurrence of anti-drug antibodies represents nowadays a serious issue. This immune response against a drug can be due either to pre-e...

Authors: Julianne Duhazé, Miguel Caubet, Signe Hässler, Delphine Bachelet, Matthieu Allez, Florian Deisenhammer, Anna Fogdell-Hahn, Aude Gleizes, Salima Hacein-Bey-Abina, Xavier Mariette, Marc Pallardy and Philippe Broët

Non-communicable diseases research output in the Eastern Mediterranean region: an overview of systematic reviews

Rates of non-communicable diseases (NCDs) are rapidly rising in the Eastern Mediterranean Region (EMR). Systematic reviews satisfy the demand from practitioners and policy makers for prompt comprehensive evide...

Authors: Alaa Akkawi, Joanne Khabsa, Aya Noubani, Sarah Jamali, Abla M. Sibai and Tamara Lotfi

Factors associated with patient willingness to participate in anaesthesia clinical trials: a vignette-based cross-sectional study

Clinical trials are essential to improve knowledge of anesthesia and perioperative medicine. Unfortunately, many studies face participant-recruitment issues and fail to include the planned number of participan...

Authors: Caroline Noirmain, Béatrice Gil-Wey, Isabelle Pichon, Pauline Brindel and Guy Haller

A framework for extending trial design to facilitate missing data sensitivity analyses

Missing data are an inevitable challenge in Randomised Controlled Trials (RCTs), particularly those with Patient Reported Outcome Measures. Methodological guidance suggests that to avoid incorrect conclusions,...

Authors: Alexina J. Mason, Richard D. Grieve, Alvin Richards-Belle, Paul R. Mouncey, David A. Harrison and James R. Carpenter

Research methodology used in the 50 most cited articles in the field of pediatrics: types of studies that become citation classics

One of the frequently used methods for assessing research trends and the impact of published scientific literature in a particular discipline is citation analysis. Journals may strive to improve their metrics ...

Authors: Antonia Jelicic Kadic, Tanja Kovacevic, Edita Runjic, Ana Simicic Majce, Josko Markic, Branka Polic, Julije Mestrovic and Livia Puljak

Analytical methods used in estimating the prevalence of HIV/AIDS from demographic and cross-sectional surveys with missing data: a systematic review

Sero- prevalence studies often have a problem of missing data. Few studies report the proportion of missing data and even fewer describe the methods used to adjust the results for missing data. The objective o...

Authors: Neema R. Mosha, Omololu S. Aluko, Jim Todd, Rhoderick Machekano and Taryn Young

Evaluation of confounding in epidemiologic studies assessing alcohol consumption on the risk of ischemic heart disease

Among different investigators studying the same exposures and outcomes, there may be a lack of consensus about potential confounders that should be considered as matching, adjustment, or stratification variabl...

Authors: Joshua D. Wallach, Stylianos Serghiou, Lingzhi Chu, Alexander C. Egilman, Vasilis Vasiliou, Joseph S. Ross and John P. A. Ioannidis

Non-participation and attrition in a longitudinal study of civilians exposed to the January 2015 terrorist attacks in Paris, France

Non-participation and attrition are rarely studied despite being important methodological issues when performing post-disaster studies. A longitudinal survey of civilians exposed to the January 2015 terrorist ...

Authors: Cécile Vuillermoz, Lise Eilin Stene, Lydéric Aubert, Yvon Motreff, Philippe Pirard, Thierry Baubet, Sophie Lesieur, Pierre Chauvin and Stéphanie Vandentorren

Incorporating sampling weights into robust estimation of Cox proportional hazards regression model, with illustration in the Multi-Ethnic Study of Atherosclerosis

Cox proportional hazards regression models are used to evaluate associations between exposures of interest and time-to-event outcomes in observational data. When exposures are measured on only a sample of part...

Authors: Colleen M. Sitlani, Thomas Lumley, Barbara McKnight, Kenneth M. Rice, Nels C. Olson, Margaret F. Doyle, Sally A. Huber, Russell P. Tracy, Bruce M. Psaty and Joseph A. C. Delaney

Hidden analyses: a review of reporting practice and recommendations for more transparent reporting of initial data analyses

In the data pipeline from the data collection process to the planned statistical analyses, initial data analysis (IDA) typically takes place between the end of the data collection and do not touch the research...

Authors: Marianne Huebner, Werner Vach, Saskia le Cessie, Carsten Oliver Schmidt and Lara Lusa

Comparison of alternative approaches for difference, noninferiority, and equivalence testing of normal percentiles

Percentiles are widely used in scientific research for determining the comparative magnitude and reference limit of quantitative measurements. The investigations for point and interval estimation of normal per...

Authors: Gwowen Shieh

A novel approach to conducting clinical trials in the community setting: utilizing patient-driven platforms and social media to drive web-based patient recruitment

Participant recruitment for clinical research studies remains a significant challenge for researchers. Novel approaches to recruitment are necessary to ensure that populations are easier to reach. In the conte...

Authors: Janelle Applequist, Cristina Burroughs, Artemio Ramirez Jr, Peter A. Merkel, Marc E. Rothenberg, Bruce Trapnell, Robert J. Desnick, Mustafa Sahin and Jeffrey P. Krischer

Protocol registration improves reporting quality of systematic reviews in dentistry

The aims of this study were to assess whether the previous registration of a systematic review (SR) is associated with the improvement of the quality of the report of SRs and whether SR registration reduced ou...

Authors: Mateus Bertolini Fernandes dos Santos, Bernardo Antônio Agostini, Rafaela Bassani, Gabriel Kalil Rocha Pereira and Rafael Sarkis-Onofre

Individualizing the dosage of Methylphenidate in children with attention deficit hyperactivity disorder

Attention deficit hyperactivity disorder (ADHD) is one of the most common childhood mental health disorders. Stimulant drugs as the most commonly used treatment and first-line therapy for ADHD have side effect...

Authors: Hoda Shirafkan, Javad Mahmoudi-Gharaei, Akbar Fotouhi, Seyyed Ali Mozaffarpur, Mehdi Yaseri and Mostafa Hoseini

Identifying long-term psychological distress from single measures: evidence from a nationally representative longitudinal survey of the Australian population

Single time-point assessments of psychological distress are often used to indicate chronic mental health problems, but the validity of this approach is unclear. The aims of this study were to investigate how a...

Authors: J. Welsh, R. J. Korda, E. Banks, L. Strazdins, G. Joshy and P. Butterworth

Agreement of claims-based methods for identifying sepsis with clinical criteria in the REasons for Geographic and Racial Differences in Stroke (REGARDS) cohort

Claims-based algorithms are commonly used to identify sepsis in health services research because the laboratory features required to define clinical criteria may not be available in administrative data.

Authors: John P. Donnelly, Yuling Dai, Lisandro D. Colantonio, Hong Zhao, Monika M. Safford, John W. Baddley, Paul Muntner and Henry E. Wang

Assembling and validating a heart failure-free cohort from the Reasons for Geographic and Racial Differences in Stroke (REGARDS) study

Studies examining incident heart failure (HF) have been limited to select populations. To examine incident HF with broader generalizability, there is need to assemble a HF-free cohort using a geographically-di...

Authors: Parag Goyal, Matthew T. Mefford, Ligong Chen, Madeline R. Sterling, Raegan W. Durant, Monika M. Safford and Emily B. Levitan

Questionnaire development for the Lolland-Falster Health Study, Denmark: an iterative and incremental process

In composing multi-thematic questionnaires for the Lolland-Falster Health Study (LOFUS), we faced a range of challenges, for which we found limited guidance in the literature. LOFUS is a household-based popula...

Authors: Cecilie Lindström Egholm, Aake Packness, Jakob Stokholm, Knud Rasmussen, Christina Ellervik, Erik Simonsen, Anne Illemann Christensen and Randi Jepsen

Norm values and psychometric properties for the German health regulatory focus scale – results of a representative survey

The health regulatory focus is an application of Higgins’ regulatory focus theory to a health-specific context. It explains individual differences in health motivation, strategies, and behavior. Previous resea...

Authors: Bjarne Schmalbach, Markus Zenger, Elmar Brähler and Katja Petrowski

Power difference in a χ² test vs generalized linear mixed model in the presence of missing data – a simulation study

Longitudinal randomized controlled trials (RCTs) often aim to test and measure the effect of treatment between arms at a single time point. A two-sample χ² test is a common statistical approach when outcome data ...

Authors: Mary L. Miller, Denise J. Roe, Chengcheng Hu and Melanie L. Bell

Comparison of exclusion, imputation and modelling of missing binary outcome data in frequentist network meta-analysis

Missing participant outcome data (MOD) are ubiquitous in systematic reviews with network meta-analysis (NMA) as they invade from the inclusion of clinical trials with reported participant losses. There are ava...

Authors: Loukia M. Spineli and Chrysostomos Kalyvas

Contamination in complex healthcare trials: the falls in care homes (FinCH) study experience

Trials are at risk of contamination bias which can occur when participants in the control group are inadvertently exposed to the intervention. This is a particular risk in rehabilitation studies where it is ea...

Authors: K. Robinson, F. Allen, J. Darby, C. Fox, A. L. Gordon, J. C. Horne, P. Leighton, E. Sims and P. A. Logan

Nurse effects on measurement error in household biosocial surveys

Biosocial survey data are in high demand, yet little is known about the measurement quality of health measures collected by nurses in respondents’ homes. Our objective was to analyze the degree to which nurses...

Authors: Alexandru Cernat and Joseph W. Sakshaug

The ability of different imputation methods for missing values in mental measurement questionnaires

Incomplete data are of particular important influence in mental measurement questionnaires. Most experts, however, mostly focus on clinical trials and cohort studies and generally pay less attention to this de...

Authors: Xueying Xu, Leizhen Xia, Qimeng Zhang, Shaoning Wu, Mingcheng Wu and Hongbo Liu

A scoping review of core outcome sets and their ‘mapping’ onto real-world data using prostate cancer as a case study

A Core Outcomes Set (COS) is an agreed minimum set of outcomes that should be reported in all clinical studies related to a specific condition. Using prostate cancer as a case study, we identified, summarized,...

Authors: Michela Meregaglia, Oriana Ciani, Helen Banks, Maximilian Salcher-Konrad, Caroline Carney, Sahan Jayawardana, Paula Williamson and Giovanni Fattore

Correction to: A comparison of methods to estimate the survivor average causal effect in the presence of missing data: a simulation study

In the original publication of this article [1], the incorrect causal diagram was submitted as Fig. 1.

Authors: Myra B. McGuinness, Jessica Kasza, Amalia Karahalios, Robyn H. Guymer, Robert P. Finger and Julie A. Simpson

The application of unsupervised deep learning in predictive models using electronic health records

The main goal of this study is to explore the use of features representing patient-level electronic health record (EHR) data, generated by the unsupervised deep learning algorithm autoencoder, in predictive mo...

Authors: Lei Wang, Liping Tong, Darcy Davis, Tim Arnold and Tina Esposito

A simulation study to compare different estimation approaches for network meta-analysis and corresponding methods to evaluate the consistency assumption

Network meta-analysis (NMA) is becoming increasingly popular in systematic reviews and health technology assessments. However, there is still ambiguity concerning the properties of the estimation approaches as...

Authors: Corinna Kiefer, Sibylle Sturtz and Ralf Bender

Development and validation of algorithms to classify type 1 and 2 diabetes according to age at diagnosis using electronic health records

Validated algorithms to classify type 1 and 2 diabetes (T1D, T2D) are mostly limited to white pediatric populations. We conducted a large study in Hong Kong among children and adults with diabetes to develop a...

Authors: Calvin Ke, Thérèse A. Stukel, Andrea Luk, Baiju R. Shah, Prabhat Jha, Eric Lau, Ronald C. W. Ma, Wing-Yee So, Alice P. Kong, Elaine Chow and Juliana C. N. Chan

Mechanisms and pathways to impact in public health research: a preliminary analysis of research funded by the National Institute for Health Research (NIHR)

The mechanisms and pathways to impacts from public health research in the UK have not been widely studied. Through the lens of one funder (NIHR), our aims are to map the diversity of public health research, in...

Authors: Harriet Boulding, Adam Kamenetzky, Ioana Ghiga, Becky Ioppolo, Facundo Herrera, Sarah Parks, Catriona Manville, Susan Guthrie and Saba Hinrichs-Krapels

The methods and baseline characteristics of a VA randomized controlled study evaluating supported employment provided in primary care patient aligned care teams

This article describes the design and baseline sample of a single-site trial comparing Individual Placement and Support (IPS) supported employment delivered within a Veterans Health Administration (VHA) primar...

Authors: Lori L. Davis, Catherine M. Blansett, Mercy N. Mumba, David MacVicar, Richard Toscano, Patricia Pilkinton, Whitney Gay and Al Bartolucci

Comparison of retention in observational cohorts and nested simulated HIV vaccine efficacy trials in the key populations in Uganda

Outcomes in observational studies may not best estimate those expected in the HIV vaccine efficacy trials. We compared retention in Simulated HIV Vaccine Efficacy Trials (SiVETs) and observational cohorts draw...

Authors: Andrew Abaasa, Jim Todd, Stephen Nash, Yunia Mayanja, Pontiano Kaleebu, Patricia E. Fast and Matt Price

Outcome choice and definition in systematic reviews leads to few eligible studies included in meta-analyses: a case study

There is broad recognition of the importance of evidence in informing clinical decisions. When information from all studies included in a systematic review (“review”) does not contribute to a meta-analysis, de...

Authors: Ian J. Saldanha, Kristina B. Lindsley, Sarah Money, Hannah J. Kimmel, Bryant T. Smith and Kay Dickersin

Patient and Public Involvement (PPI) in evidence synthesis: how the PatMed study approached embedding audience responses into the expression of a meta-ethnography

Patient and public involvement (PPI) has become enshrined as an important pillar of health services empirical research, including PPI roles during stages of research development and analysis and co-design appr...

Authors: S. Park, N. Khan, F. Stevenson and A. Malpass

Comparison of different rating scales for the use in Delphi studies: different scales lead to different consensus and show different test-retest reliability

Consensus-orientated Delphi studies are increasingly used in various areas of medical research using a variety of different rating scales and criteria for reaching consensus. We explored the influence of using...

Authors: Toni Lange, Christian Kopkow, Jörg Lützner, Klaus-Peter Günther, Sascha Gravius, Hanns-Peter Scharf, Johannes Stöve, Richard Wagner and Jochen Schmitt