Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease.

Introduction: The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF-McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.

Aim: Discuss missed opportunities and the methodological approach of the ISTH Guideline in contrast to how GRADE was previously applied in rare diseases.

Methods: Critically analyse the methodology of each guideline along with best practices in the use of GRADE.

International Society on Thrombosis and Haemostasis Clinical Practice Guideline for Treatment of Congenital Haemophilia-A Critical Appraisal.

Introduction: Evidence-based clinical practice guidelines drive optimal patient care and facilitate access to high-quality treatment. Creating guidelines for rare diseases such as haemophilia, where evidence does not often come from randomized controlled trials but from non-randomized and well-designed observational studies and real-world data, is challenging. The methodology used for assessing available evidence should consider this critical fact.

A core outcome set for prophylaxis and perioperative treatment of von Willebrand disease: The coreVWD initiative.

Introduction: Treatment options are expanding for von Willebrand disease (VWD). A core outcome set (COS)-a minimum set of agreed-upon outcomes to be used in every clinical trial for a given condition-provides guidance on which outcomes are most important to measure to ensure necessary data is collected for a variety of stakeholders and enable comparison across products and trials.

Aim: coreVWD aimed to develop a COS for trials for prophylaxis and perioperative treatments for VWD.

Intention to Act: Predicting Bystander Intervention in Violent Situations in South Korea.

The purpose of this study is to explore the predictors of bystanders' intentions to intervene and types of intended intervention in domestic violence (DV) and sexual violence (SV) situations in South Korea. Using nationally representative data from the 2016 National Fact-Finding Survey on Gender Equality ( = 7,399) and logistic regression-based models, this study focused on two key predictors, bystander gender and attitudes about gender equality, while controlling for age, educational level, and employment status. Our findings indicated that males and females were equally likely to state intentions to intervene in both DV and SV situations.

Small Molecule Benzothiophene with Efficacy in a Mouse Model of Drug-Resistant Infection.

Hospital-acquired infections, caused by ESKAPE bacteria, are a challenging global public health concern, in part due to the emergence of drug-resistant strains. While profiling a diverse set of compounds for activity this class of bacteria, we noted that the benzothiophene JSF-2827 exhibited promising antibacterial activity against . A hit evolution campaign ensued, involving the design, synthesis, and biological assay of analogues designed to address early issues such as a short mouse liver microsome half-life and a modest mouse pharmacokinetic profile.

Qualitative interview study of strategies to support healthcare personnel mental health through an occupational health lens.

Background: Employee Occupational Health ('occupational health') clinicians have expansive perspectives of the experience of healthcare personnel. Integrating mental health into the purview of occupational health is a newer approach that could combat historical limitations of healthcare personnel mental health programmes, which have been isolated and underused.

Objective: We aimed to document innovation and opportunities for supporting healthcare personnel mental health through occupational health clinicians.

Virology-the path forward.

In the United States (US), biosafety and biosecurity oversight of research on viruses is being reappraised. Safety in virology research is paramount and oversight frameworks should be reviewed periodically. Changes should be made with care, however, to avoid impeding science that is essential for rapidly reducing and responding to pandemic threats as well as addressing more common challenges caused by infectious diseases.

Avatrombopag for adults with early versus chronic immune thrombocytopenia.

Avatrombopag is a newer thrombopoietin receptor agonist (TPO-RA) currently approved to treat chronic ITP (duration >12 months). No studies have yet evaluated the safety and effectiveness of avatrombopag in newly diagnosed ITP (duration <3 months) or persistent ITP (duration 3-12 months), and so its use in these populations is presently off-label worldwide. We hypothesize that avatrombopag has similar safety and effectiveness irrespective of ITP disease phase.

Strategies to Improve Perioperative Palliative Care Integration for Seriously Ill Veterans.

Context: Seriously ill patients are at higher risk for adverse surgical outcomes. Palliative care (PC) interventions for seriously ill surgical patients are associated with improved quality of patient care and patient-centered outcomes, yet, they are underutilized perioperatively.

Objectives: To identify strategies for improving perioperative PC integration for seriously ill Veterans from the perspectives of PC providers and surgeons.

Real-world healthcare costs and resource utilization in patients with von Willebrand disease and angiodysplasia.

Objective: To describe the economic burden among VWD patients with angiodysplasia compared to VWD patients without angiodysplasia and the general population.

Methods: This was a retrospective analysis using the Merative MarketScan Commercial and Medicare Databases® (January 2011-September 2020). Selected patients had ≥1 medical claim for VWD or low VWF, ≥1 medical claim for AGD, and ≥3 GI-related bleeding episodes within a year.

Facilitators of palliative care quality improvement team cohesion: Lessons from a seven-site implementation project in India.

Background: The Palliative Care: Promoting Access and Improvement of the Cancer Experience (PC-PAICE) initiative is a team-based, palliative care (PC) quality improvement (QI) project working to promote high-quality PC in India. As a PC QI initiative, PC-PAICE implementation relied upon building interdisciplinary teams, providing the ideal context for understanding facilitators of team cohesion that compelled clinical, organizational, and administrative team members to work together. There is an opportunity to leverage the intersection between QI implementation and organizational theory to inform and improve implementation science.

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science.

Background: The National Hemophilia Foundation (NHF) conducted extensive all-stakeholder inherited bleeding disorder (BD) community consultations to inform a blueprint for future research. Sustaining and expanding the specialized and comprehensive Hemophilia Treatment Center care model, to better serve all people with inherited BDs (PWIBD), and increasing equitable access to optimal health emerged as top priorities.

Research Design And Methods: NHF, with the American Thrombosis and Hemostasis Network (ATHN), convened multidisciplinary expert working groups (WG) to distill priority research initiatives from consultation findings.

Beyond the guidelines: how we approach challenging scenarios in the diagnosis and management of von Willebrand disease.

Although von Willebrand disease (VWD) is the most common inherited bleeding disorder, its diagnosis and management are often challenging. Clinical practice guidelines, developed through systematic review of the medical literature and considering the best available evidence, provide guidance for common clinical scenarios. However, in the clinical setting, patients often present with characteristics and nuances that may fall outside the realm of available evidence and guidelines, and hence, shared decision-making will be essential in the evaluation and management of these patients.

Effect of β2-agonist treatment on insulin-stimulated peripheral glucose disposal in healthy men in a randomised placebo-controlled trial.

β-agonist treatment improves skeletal muscle glucose uptake and whole-body glucose homeostasis in rodents, likely via mTORC2-mediated signalling. However, human data on this topic is virtually absent. We here investigate the effects of two-weeks treatment with the β-agonist clenbuterol (40 µg/day) on glucose control as well as energy- and substrate metabolism in healthy young men (age: 18-30 years, BMI: 20-25 kg/m) in a randomised, placebo-controlled, double-blinded, cross-over study (ClinicalTrials.

Correctly Establishing and Interpreting Oxygenation Status in Sickle Cell Disease.

Background: As hypoxemia and hypoxia are central elements of disease pathophysiology and disease-related morbidity and mortality in individuals affected by sickle cell disease (SCD), clinical management aims to optimize oxygenation.

Content: Hypoxemia is primarily screened for with pulse oximetry. However, in SCD pulse oximetry can inaccurately reflect arterial saturation, posing the risk of undetected (occult) hypoxemia.