Testing an Electronic Patient-Reported Outcome Platform in the Context of Traumatic Brain Injury: PRiORiTy Usability Study.

Background: Traumatic brain injury (TBI) is a significant public health issue and a leading cause of death and disability globally. Advances in clinical care have improved survival rates, leading to a growing population living with long-term effects of TBI, which can impact physical, cognitive, and emotional health. These effects often require continuous management and individualized care.

Implementation of patient and public involvement and engagement (PPIE) for the therapies for long COVID in non-hospitalised individuals (TLC) project.

Background: Patients, their family members and caregivers have firsthand experiences of living with or supporting someone living with a disease or medical condition. This knowledge by experience cannot be replaced by the knowledge acquired by clinicians, researchers, or other professionals through study and/or work. The Therapies for Long COVID in non-hospitalised individuals (TLC) research project was funded in the UK by the National Institute for Health and Care Research (NIHR) and UK Research and Innovation to investigate the impact of long COVID on affected individuals.

Vericiguat Global Study in Participants with Chronic Heart Failure: Design of the VICTOR trial.

Aims: In the VICTORIA (Vericiguat Global Study in Subjects with Heart Failure with Reduced Ejection Fraction) trial, the soluble guanylate cyclase stimulator vericiguat reduced the risk of hospitalization for heart failure (HHF) or cardiovascular death in patients with heart failure (HF) and reduced ejection fraction (HFrEF) with recent worsening HF. The effect of vericiguat in patients with HFrEF without recent worsening HF remains unknown. The VICTOR (Vericiguat Global Study in Participants with Chronic Heart Failure) trial was designed to assess the efficacy and safety of vericiguat in patients with ejection fraction ≤40% without recent worsening HF on a background of current foundational HFrEF therapy.

Recommendations to promote equity, diversity and inclusion in decentralized clinical trials.

Decentralized clinical trials involving the use of digital tools to facilitate remote research are gaining momentum. Rapid advancements in digital technologies have supported the adoption of these trials. These innovations facilitate virtual interactions between clinical trial teams and participants by making it easier to collect, transfer and store electronic data.

Projecting the benefit of vericiguat in PARADIGM-HF and DAPA-HF populations: Insights from the VICTORIA trial.

Aims: The VICTORIA trial demonstrated a significant reduction in the primary composite outcome of heart failure (HF) hospitalization or cardiovascular death with vericiguat relative to placebo in high-risk HF. This study aimed to contextualize treatment effects of vericiguat in populations with varying risk profiles simulated from the PARADIGM-HF and DAPA-HF trials.

Methods: Subgroups of VICTORIA participants (n = 5050) were generated to simulate PARADIGM-HF and DAPA-HF trial populations.

Key considerations for digital decentralised clinical trials from a feasibility study assessing pacing interventions for long COVID.

Post COVID-19 condition or long COVID is highly prevalent and often debilitating, with key symptoms including fatigue, breathlessness, and brain fog. There is currently a lack of evidence-based treatments for this highly complex syndrome. There is a need for clinical trial platforms to rapidly evaluate nonpharmacological treatments to support affected individuals with symptom management.

Understanding how traumatic brain injury-related changes in fluid biomarkers affect quality of life outcomes in veterans: a prospective observational trial protocol (UNTANGLE).

Introduction: Traumatic brain injury (TBI) is a major cause of disability, with annual global incidence estimated as 69 million people. Survivors can experience long-term visual changes, altered mental state, neurological deficits and long-term effects that may be associated with mental illness. TBI is prevalent in military personnel due to gunshot wounds, and blast injury.

An Evaluation of the Replacement of Animal-derived Biomaterials in Human Primary Cell Culture.

The likelihood that potential new drugs will successfully navigate the current translational pipeline is poor, with fewer than 10% of drug candidates making this transition successfully, even after their entry into clinical trials. Prior to this stage, candidate drugs are typically evaluated by using models of increasing complexity, beginning with basic cell culture studies and progressing through to animal studies, where many of these candidates are lost due to lack of efficacy or toxicology concerns. There are many reasons for this poor translation, but interspecies differences in functional and physiological parameters undoubtedly contribute to the problem.

Anther Culture Protocols for Barley and Wheat.

Doubled haploid (DH) techniques remain valuable tools for wheat (Triticum aestivum L.) and barley (Hordeum vulgare L.) genetic improvement, and DH populations are used extensively in breeding and research endeavors.

Development of a quality of life measure for left ventricular assist device recipients using a mixed methods approach.

Background: Left ventricular assist device (LVAD) recipients report symptom improvement but find adjusting to life with the LVAD challenging. These challenges are unique, and existing patient-reported outcome measures (PROMs) do not reflect their experiences. This study aimed to develop a culturally relevant quality of life PROM for use with LVAD recipients in future research, design evolutions and clinical practice.

An assessment of nurses' perceived and actual household emergency preparedness.

Nurses' household preparedness is critical if they are to avoid role conflict and report for duty during an emergency. To date, the alignment between nurses' perceived and actual household preparedness remains under examined. Investigating one of these variables in isolation fails to consider that perceived and actual household preparedness must be high and aligned.

Patient-reported outcomes in integrated health and social care: A scoping review.

Background: Patient-reported outcomes (PROs) have potential to support integrated health and social care research and practice; however, evidence of their utilisation has not been synthesised.

Objective: To identify PRO measures utilised in integrated care and adult social care research and practice and to chart the evidence of implementation factors influencing their uptake.

Design: Scoping review of peer-reviewed literature.

Diuretic use and outcomes in patients with heart failure with reduced ejection fraction: Insights from the VICTORIA trial.

Aims: In VICTORIA, vericiguat compared with placebo reduced the risk of cardiovascular death (CVD) and heart failure hospitalization (HFH) in patients enrolled after a worsening heart failure (WHF) event. We examined clinical outcomes and efficacy of vericiguat as it relates to background use of loop diuretics in patients with WHF.

Methods And Results: We calculated the total daily loop diuretic dose equivalent to furosemide dosing at randomization and categorized these as: no loop diuretic, 1-39, 41-80, 40, and >80 mg total daily dose (TDD).

Recommendations to address respondent burden associated with patient-reported outcome assessment.

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making.

Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial.

Background: Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies.

Methods: An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions.

Paving the way for patient centricity in real-world evidence (RWE): Qualitative interviews to identify considerations for wider implementation of patient-reported outcomes in RWE generation.

Objectives: Real-world evidence (RWE) generation can be enhanced by including patient-reported outcomes (PROs). Methods for collecting and using PRO data in the real-world setting are currently underdeveloped and there is no international guidance specific to its use in this context. This study explored stakeholders' perspectives and needs for using PROs in RWE generation.

Clinician survey of current global practice for sarcoma surveillance following resection of primary retroperitoneal, abdominal, and pelvic sarcoma.

Introduction: - Postoperative surveillance following resection of primary retroperitoneal, abdominal, and pelvic sarcoma (RPS) is standard of care in international sarcoma centres and has rapidly evolved without an evidence base to become highly intensive and prolonged. This clinician survey aims to capture a global, contemporary snapshot of international guidelines used to inform sarcoma surveillance following resection of primary RPS.

Materials And Methods: - Between July 2022 and March 2023, an online, anonymous, clinician survey to assess the current duration, imaging intervals and imaging modalities used for postoperative surveillance following resection of primary RPS was distributed among clinicians working at centres which are members of the TransAtlantic Australasian Retroperitoneal Working Group (TARPSWG).

Considerations for patient and public involvement and engagement in health research.

Patient and public involvement and engagement (PPIE) can provide valuable insights into the experiences of those living with and affected by a disease or health condition. Inclusive collaboration between patients, the public and researchers can lead to productive relationships, ensuring that health research addresses patient needs. Guidelines are available to support effective PPIE; however, evaluation of the impact of PPIE strategies in health research is limited.

Global use of electronic patient-reported outcome systems in nephrology: a mixed methods study.

Objectives: The use of electronic patient-reported outcome (ePRO) systems to support the management of patients with chronic kidney disease is increasing. This mixed-methods study aimed to comprehensively identify existing and developing ePRO systems, used in nephrology settings globally, ascertaining key characteristics and factors for successful implementation.

Study Design: ePRO systems and developers were identified through a scoping review of the literature and contact with field experts.

Background Medical Therapy and Clinical Outcomes From the VICTORIA Trial.

Background: We examined whether the primary composite outcome (cardiovascular death or heart failure hospitalization) was related to differences in background use and dosing of guideline-directed medical therapy in patients with heart failure with reduced ejection fraction enrolled in VICTORIA (Vericiguat Global Study in Subjects with Heart Failure with Reduced Ejection Fraction), a randomized trial of vericiguat versus placebo.

Methods: We evaluated the adherence to guideline use of angiotensin-converting enzyme inhibitors, angiotensin-receptor blockers, angiotensin receptor-neprilysin inhibitors, beta-blockers, and mineralocorticoid receptor antagonists. We assessed basic adherence; indication-corrected adherence accounting for guideline indications and contraindications; and dose-corrected adherence (indication-corrected adherence+≥50% of drug dose target).