Integrating data from randomized controlled trials and observational studies to predict the response to pregabalin in patients with painful diabetic peripheral neuropathy

More patient-specific medical care is expected as more is learned about variations in patient responses to medical treatments. Analytical tools enable insights by linking treatment responses from different typ...

Authors: Joe Alexander, Roger A. Edwards, Alberto Savoldelli, Luigi Manca, Roberto Grugni, Birol Emir, Ed Whalen, Stephen Watt, Marina Brodsky and Bruce Parsons

Basic parametric analysis for a multi-state model in hospital epidemiology

The extended illness-death model is a useful tool to study the risks and consequences of hospital-acquired infections (HAIs). The statistical quantities of interest are the transition-specific hazard rates and...

Authors: Maja von Cube, Martin Schumacher and Martin Wolkewitz

A random effects meta-analysis model with Box-Cox transformation

In a random effects meta-analysis model, true treatment effects for each study are routinely assumed to follow a normal distribution. However, normality is a restrictive assumption and the misspecification of ...

Authors: Yusuke Yamaguchi, Kazushi Maruo, Christopher Partlett and Richard D. Riley

A literature review of applied adaptive design methodology within the field of oncology in randomised controlled trials and a proposed extension to the CONSORT guidelines

The application of adaptive design methodology within a clinical trial setting is becoming increasingly popular. However the application of these methods within trials is not being reported as adaptive designs...

Authors: Pankaj Mistry, Janet A Dunn and Andrea Marshall

Nonlinear joint models for individual dynamic prediction of risk of death using Hamiltonian Monte Carlo: application to metastatic prostate cancer

Joint models of longitudinal and time-to-event data are increasingly used to perform individual dynamic prediction of a risk of event. However the difficulty to perform inference in nonlinear models and to cal...

Authors: Solène Desmée, France Mentré, Christine Veyrat-Follet, Bernard Sébastien and Jérémie Guedj

Protocol adherence for continuously titrated interventions in randomized trials: an overview of the current methodology and case study

The standard definition for protocol adherence is the proportion of all scheduled doses that are delivered. In clinical research, this definition has several limitations when evaluating protocol adherence in t...

Authors: F. Lauzier, N. K. Adhikari, A. Seely, K. K. Y. Koo, E. P. Belley-Côté, K. E. A. Burns, D. J. Cook, F. D’Aragon, B. Rochwerg, M. E. Kho, S. J. W. Oczkowksi, E. H. Duan, M. O. Meade, A. G. Day and F. Lamontagne

Clarifying the distinction between case series and cohort studies in systematic reviews of comparative studies: potential impact on body of evidence and workload

Distinguishing cohort studies from case series is difficult.

Authors: Tim Mathes and Dawid Pieper

Sample size re-estimation in paired comparative diagnostic accuracy studies with a binary response

The sample size required to power a study to a nominal level in a paired comparative diagnostic accuracy study, i.e. studies in which the diagnostic accuracy of two testing procedures is compared relative to a...

Authors: Gareth P. J. McCray, Andrew C. Titman, Paula Ghaneh and Gillian A. Lancaster

Using structural equation modeling for network meta-analysis

Network meta-analysis overcomes the limitations of traditional pair-wise meta-analysis by incorporating all available evidence into a general statistical framework for simultaneous comparisons of several treat...

Authors: Yu-Kang Tu and Yun-Chun Wu

Accounting for treatment use when validating a prognostic model: a simulation study

Prognostic models often show poor performance when applied to independent validation data sets. We illustrate how treatment use in a validation set can affect measures of model performance and present the uses...

Authors: Romin Pajouheshnia, Linda M. Peelen, Karel G. M. Moons, Johannes B. Reitsma and Rolf H. H. Groenwold

Cost and yield considerations when expanding recruitment for genetic studies: the primary open-angle African American glaucoma genetics study

African Americans have been historically under-represented in genetic studies. More research is needed on effective recruitment strategies for this population, especially on approaches that supplement traditio...

Authors: Rebecca Salowe, Laura O’Keefe, Sayaka Merriam, Roy Lee, Naira Khachatryan, Prithvi Sankar, Eydie Miller-Ellis, Amanda Lehman, Victoria Addis, Windell Murphy, Jeffrey Henderer, Maureen Maguire and Joan O’Brien

Inter-rater reliability of AMSTAR is dependent on the pair of reviewers

Inter-rater reliability (IRR) is mainly assessed based on only two reviewers of unknown expertise. The aim of this paper is to examine differences in the IRR of the Assessment of Multiple Systematic Reviews (AMST...

Authors: Dawid Pieper, Anja Jacobs, Beate Weikert, Alba Fishta and Uta Wegewitz

Implementation fidelity of a self-management course for epilepsy: method and assessment

Complex interventions such as self-management courses are difficult to evaluate due to the many interacting components. The way complex interventions are delivered can influence the effect they have for patien...

Authors: G. Wojewodka, S. Hurley, S. J. C. Taylor, A. J. Noble, L. Ridsdale and L. H. Goldstein

Data cleaning and management protocols for linked perinatal research data: a good practice example from the Smoking MUMS (Maternal Use of Medications and Safety) Study

Data cleaning is an important quality assurance in data linkage research studies. This paper presents the data cleaning and preparation process for a large-scale cross-jurisdictional Australian study (the Smok...

Authors: Duong Thuy Tran, Alys Havard and Louisa R. Jorm

Awareness of wearing an accelerometer does not affect physical activity in youth

This study aimed to investigate whether awareness of being monitored by an accelerometer has an effect on physical activity in young people.

Authors: Jérémy Vanhelst, Laurent Béghin, Elodie Drumez, Stéphanie Coopman and Frédéric Gottrand

Estimating parameters for probabilistic linkage of privacy-preserved datasets

Probabilistic record linkage is a process used to bring together person-based records from within the same dataset (de-duplication) or from disparate datasets using pairwise comparisons and matching probabilit...

Authors: Adrian P. Brown, Sean M. Randall, Anna M. Ferrante, James B. Semmens and James H. Boyd

Selection bias and subject refusal in a cluster-randomized controlled trial

Selection bias and non-participation bias are major methodological concerns which impact external validity. Cluster-randomized controlled trials are especially prone to selection bias as it is impractical to b...

Authors: Rochelle Yang, Barry L. Carter, Tyler H. Gums, Brian M. Gryzlak, Yinghui Xu and Barcey T. Levy

Barriers to publishing in biomedical journals perceived by a sample of French researchers: results of the DIAzePAM study

As publishing is essential but competitive for researchers, difficulties in writing and submitting medical articles to biomedical journals are disabling. The DIAzePAM (Difficultés des Auteurs à la Publication d’A...

Authors: Martin Duracinsky, Christophe Lalanne, Laurence Rous, Aichata Fofana Dara, Lesya Baudoin, Claire Pellet, Alexandre Descamps, Fabienne Péretz and Olivier Chassany

Inverse probability of treatment-weighted competing risks analysis: an application on long-term risk of urinary adverse events after prostate cancer treatments

To illustrate the 10-year risks of urinary adverse events (UAEs) among men diagnosed with prostate cancer and treated with different types of therapy, accounting for the competing risk of death.

Authors: Charlotte A. Bolch, Haitao Chu, Stephanie Jarosek, Stephen R. Cole, Sean Elliott and Beth Virnig

A DAG-based comparison of interventional effect underestimation between composite endpoint and multi-state analysis in cardiovascular trials

Composite endpoints comprising hospital admissions and death are the primary outcome in many cardiovascular clinical trials. For statistical analysis, a Cox proportional hazards model for the time to first eve...

Authors: Antje Jahn-Eimermacher, Katharina Ingel, Stella Preussler, Antoni Bayes-Genis and Harald Binder

Erratum to: Standardizing effect size from linear regression models with log-transformed variables for meta-analysis

Authors: Miguel Rodríguez-Barranco, Aurelio Tobías, Daniel Redondo, Elena Molina-Portillo and María José Sánchez

Simulation of complex data structures for planning of studies with focus on biomarker comparison

There are a growing number of observational studies that do not only focus on single biomarkers for predicting an outcome event, but address questions in a multivariable setting. For example, when quantifying ...

Authors: Andreas Schulz, Daniela Zöller, Stefan Nickels, Manfred E. Beutel, Maria Blettner, Philipp S. Wild and Harald Binder

Limitations of pulmonary embolism ICD-10 codes in emergency department administrative data: let the buyer beware

Administrative data is a useful tool for research and quality improvement; however, validity of research findings based on these data depends on their reliability. Diagnoses assigned by physicians are subseque...

Authors: Kristin Burles, Grant Innes, Kevin Senior, Eddy Lang and Andrew McRae

A nonparametric multiple imputation approach for missing categorical data

Incomplete categorical variables with more than two categories are common in public health data. However, most of the existing missing-data methods do not use the information from nonresponse (missingness) pro...

Authors: Muhan Zhou, Yulei He, Mandi Yu and Chiu-Hsieh Hsu

Methodologic considerations in the design and analysis of nested case-control studies: association between cytokines and postoperative delirium

The nested case-control study (NCC) design within a prospective cohort study is used when outcome data are available for all subjects, but the exposure of interest has not been collected, and is difficult or p...

Authors: Long H. Ngo, Sharon K. Inouye, Richard N. Jones, Thomas G. Travison, Towia A. Libermann, Simon T. Dillon, George A. Kuchel, Sarinnapha M. Vasunilashorn, David C. Alsop and Edward R. Marcantonio