Investigating the presence of surgical learning in the Timing of Primary Surgery for cleft palate randomised trial.

Background/aims: When conducting a randomised controlled trial in surgery, it is important to consider surgical learning, where surgeons' familiarity with one, or both, of the interventions increases during the trial. If present, learning may compromise trial validity. We demonstrate a statistical investigation into surgical learning within a trial of cleft palate repair.

Applying Conformal Prediction to a Deep Learning Model for Intracranial Hemorrhage Detection to Improve Trustworthiness.

Purpose To apply conformal prediction to a deep learning (DL) model for intracranial hemorrhage (ICH) detection and evaluate model performance in detection as well as model accuracy in identifying challenging cases. Materials and Methods This was a retrospective (November 2017 through December 2017) study of 491 noncontrast head CT volumes from the CQ500 dataset in which three senior radiologists annotated sections containing ICH. The dataset was split into definite and challenging (uncertain) subsets, where challenging images were defined as those in which there was disagreement among readers.

Guidance to inform research recruitment processes for studies involving critically ill patients.

Clinical research in intensive care units (ICUs) is essential for improving treatments for critically ill patients. However, invitations to participate in clinical research in this situation pose numerous challenges. Studies are frequently initiated within a narrow time window when patients are often unconscious and unable to consent.

Neighborhood economic vulnerability as a predictor for patterns of care and outcomes for patients with uterine cancer.

Objective: To quantify the effect of neighborhood socioeconomic vulnerability as it relates to racial disparity in uterine cancer treatment and survival.

Methods: Patients with a diagnosis of uterine cancer who underwent hysterectomy in New York State from 2004 to 2017 were included in this retrospective cohort study. Neighborhood socioeconomic vulnerability as quantified by the Area Deprivation Index was calculated.

Real-world HbA changes and prescription characteristics among type 2 diabetes mellitus patients initiating treatment with once weekly semaglutide for diabetes.

Purpose: The purpose of this study was to evaluate patient, prescriber, and dose characteristics and evaluate changes in glycated hemoglobin (HbA) for patients prescribed once weekly semaglutide for diabetes (OW sema T2D).

Methods: This study was a retrospective claims-based study using the Optum Research Database. The sample included adult patients who had at least one claim for OW sema T2D between Jan 1, 2018, and Dec 31, 2019, were continuously enrolled in the health plan and had a diagnosis of type 2 diabetes (T2DM) during the pre-index or post-index periods.

Synthetically enhanced: unveiling synthetic data's potential in medical imaging research.

Background: Chest X-rays (CXR) are essential for diagnosing a variety of conditions, but when used on new populations, model generalizability issues limit their efficacy. Generative AI, particularly denoising diffusion probabilistic models (DDPMs), offers a promising approach to generating synthetic images, enhancing dataset diversity. This study investigates the impact of synthetic data supplementation on the performance and generalizability of medical imaging research.

Seizure control via pH manipulation: a phase II double-blind randomised controlled trial of inhaled carbogen as adjunctive treatment of paediatric convulsive status epilepticus (Carbogen for Status Epilepticus in Children Trial (CRESCENT)).

Background: Paediatric convulsive status epilepticus is the most common neurological emergency presenting to emergency departments. Risks of resultant neurological morbidity and mortality increase with seizure duration. If the seizure fails to stop within defined time-windows, standard care follows an algorithm of stepwise escalation to more intensive treatments, ultimately resorting to induction of general anaesthesia and ventilation.

Immunogenicity and Protective Efficacy of Psoralen-Inactivated SARS-CoV-2 Vaccine in Nonhuman Primates.

COVID-19 caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has significantly impacted public health and the economy worldwide. Most of the currently licensed COVID-19 vaccines act by inhibiting the receptor-binding function of the SARS-CoV-2 spike protein. The constant emergence of SARS-CoV-2 variants resulting from mutations in the receptor-binding domain (RBD) leads to vaccine immune evasion and underscores the importance of broadly acting COVID-19 vaccines.

The overlap between randomised evaluations of recruitment and retention interventions: An updated review of recruitment (Online Resource for Recruitment in Clinical triAls) and retention (Online Resource for Retention in Clinical triAls) literature.

Background: The Online Resource for Recruitment in Clinical triAls (ORRCA) and the Online Resource for Retention in Clinical triAls (ORRCA2) were established to organise and map the literature addressing participant recruitment and retention within clinical research. The two databases are updated on an ongoing basis using separate but parallel systematic reviews. However, recruitment and retention of research participants is widely acknowledged to be interconnected.

Real-World Effectiveness of Once-Weekly Glucagon-Like Peptide-1 Receptor Agonists (OW GLP-1RAs) in Comparison with Dipeptidyl Peptidase-4 Inhibitors (DPP-4is) for Glycemic Control and Weight Outcomes in Type 2 Diabetes Mellitus (RELATE).

Background: The efficacy of once-weekly (OW) glucagon-like peptide-1 receptor agonists (GLP-1RAs) has been established in several trials in people with type 2 diabetes mellitus (T2DM); however, real-world evidence on their effectiveness is limited. This study evaluated the effectiveness of OW GLP-1RA regarding glycemic and weight outcomes, and relative to DPP-4i in a comparator analysis.

Methods: This observational cohort study evaluated glycated hemoglobin (HbA) and weight outcomes in people with T2DM with two or more prescription claims for the same OW GLP-1RA using a pre-post study design (including for a semaglutide OW T2DM subgroup, hereafter referred to as semaglutide).

Questioning approaches to consent in time critical obstetric trials: findings from a mixed-methods study.

Objective: Trial legislation enables research to be conducted without prior consent (RWPC) in emergency situations, yet this approach has rarely been used in time-critical obstetric trials. This study explored views and experiences of antenatal recruitment and consent and RWPC in an emergency intrapartum randomised clinical trial.

Design: Embedded, mixed-methods study within a trial, involving questionnaires, recorded recruitment discussions, interviews and focus groups in the first 13 months of trial recruitment (December 2020-January 2022).

Good Statistical Practice-development of tailored Good Clinical Practice training for statisticians.

Background: Statisticians are fundamental in ensuring clinical research, including clinical trials, are conducted with quality, transparency, reproducibility and integrity. Good Clinical Practice (GCP) is an international quality standard for the conduct of clinical trials research. Statisticians are required to undertake training on GCP but existing training is generic and, crucially, does not cover statistical activities.

Using healthcare systems data for outcomes in clinical trials: issues to consider at the design stage.

Background: Healthcare system data (HSD) are increasingly used in clinical trials, augmenting or replacing traditional methods of collecting outcome data. This study, PRIMORANT, set out to identify, in the UK context, issues to be considered before the decision to use HSD for outcome data in a clinical trial is finalised, a methodological question prioritised by the clinical trials community.

Methods: The PRIMORANT study had three phases.

The use of healthcare systems data for RCTs.

Background: Healthcare systems data (HSD) has the potential to optimise the efficiency of randomised controlled trials (RCTs), by decreasing trial-specific data demands. Therefore, the use of HSD in trials is expected to increase. In 2019, it was estimated that 47% of NIHR-funded trials were planning to use HSD.

Should Virtual Objective Structured Clinical Examination (OSCE) Teaching Replace or Complement Face-to-Face Teaching in the Post-COVID-19 Educational Environment: An Evaluation of an Innovative National COVID-19 Teaching Programme.

Background The COVID-19 pandemic brought about drastic changes to medical education and examinations, with a shift to online lectures and webinars. Additionally, social restrictions in the United Kingdom (UK) inhibited students' ability to practice for objective structured clinical examination (OSCE) with their peers. Methods The Virtual OSCE buddy scheme (VOBS) provided a means to practice OSCE skills virtually by linking groups of 2-6 final-year medical students with a junior doctor who had recently passed their exams.

The effectiveness and acceptability of multimedia information when recruiting children and young people to trials: the TRECA meta-analysis of SWATs.

Background: The information provided to potential trial participants plays a crucial role in their decision-making. Printed participant information sheets for trials have received recurrent criticism as being too long and technical, unappealing and hard to navigate. An alternative is to provide information through multimedia (text, animations, video, audio, diagrams and photos).

Involving patients and the public In sTatistIcal Analysis pLans (INITIAL): A delphi survey.

Background: Patient and public involvement (PPI) in trials aims to enhance research by improving its relevance and transparency. Planning for statistical analysis begins at the design stage of a trial within the protocol and is refined and detailed in a Statistical Analysis Plan (SAP). While PPI is common in design and protocol development it is less common within SAPs.

Durability of Effectiveness Between Users of Once-Weekly Semaglutide and Dipeptidyl Peptidase 4 Inhibitors (DPP-4i) in US Adults with Type 2 Diabetes.

Introduction: Long-term effectiveness and durability of glucose-lowering medications are important considerations in managing type 2 diabetes (T2D). This study aimed to compare durability of treatment efficacy of once-weekly (OW) semaglutide for T2D with that of the dipeptidyl peptidase 4 inhibitor (DPP-4i) class.

Methods: This observational cohort study used 2017-2022 data from the Optum Clinformatics Data Mart to compare long-term clinical outcomes associated with semaglutide or DPP-4i in US adults with T2D.

Effectiveness of guided telerehabilitation on functional performance in community-dwelling older adults: A systematic review.

Objective: To systematically review the effectiveness of guided telerehabilitation on improving functional performance in community-dwelling older adults.

Data Sources: Articles published in PubMed, Cochrane Library and Embase (Ovid) from 01 January 2010 up to 17 October 2023.

Review Methods: Included studies had (1) a randomised controlled trial design, (2) an average population age of 65 years or older, (3) a home-based setting and (4) evaluated the effectiveness of functional performance outcome measures.

Pleural Effusion: Diagnostic Approach in Adults.

Pleural effusion affects 1.5 million patients in the United States each year. New effusions require expedited investigation because treatments range from common medical therapies to invasive surgical procedures.