Global prophylaxis trends in hemophilia: a macroeconomic analysis and its association with world development indicators.

Introduction: Prophylaxis is the recommended management strategy for all persons with hemophilia (PwH), yet its adoption is uneven worldwide.

Areas Covered: This analysis examines global disparities in hemophilia care, focusing on global prophylactic coverage and its correlation with the World Bank's world development indicators. It outlines the disproportionate consumption of clotting factors and non-factor concentrates in high-income countries compared to lower-income counterparts and the challenges of expanding prophylaxis coverage in under-resourced settings.

Development of the World Federation of Hemophilia Shared Decision-Making Tool.

Introduction: The use of shared decision-making (SDM) in clinical settings is becoming more prevalent. The evolving and increasingly complex treatment landscape of haemophilia management has augmented the need and desire for SDM between patients and their healthcare team. SDM tools have been used in other chronic conditions and can be an effective form of education for patients and clinicians.

Development of the coreHEM mental health patient-reported outcome measure - A novel mental health outlook measure for people with haemophilia.

Introduction: Currently, no quality-of-life instrument exists that captures the full experience of the mental health outlook (MHO), a coreHEM core outcome, in people with haemophilia, including the potential transformational experience of receiving gene therapy.

Aim: To describe the methods used to develop a content validated patient-reported outcome measure (PROM) that measures MHO for people with haemophilia.

Methods: A conceptual framework, developed from a literature/evidence review, was used to create an interview guide and draft a questionnaire.

The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients.

Background: The prevalence of hemophilia varies globally, with close to 100% of patients diagnosed in high-income countries and as low as 12% diagnosed in lower-income countries. These inequalities in the care of people with hemophilia exist across various care indicators.

Objectives: This analysis aims to describe the clinical care outcomes of patients in the World Bleeding Disorders Registry (WBDR).

Reported prevalence of von Willebrand disease worldwide in relation to income classification.

Introduction: The diagnosis of von Willebrand disease (VWD) is complex and challenging, especially when diagnostic resources are limited. This results in a lack of consistency in identifying and reporting the number of people with VWD and variations in the VWD prevalence worldwide.

Aim: To analyze the reported prevalence of VWD worldwide in relation to income classification.

Von Willebrand Disease: Gaining a global perspective.

Introduction: Recent guidelines for von Willebrand Disease (VWD) highlighted the challenges in diagnosis and management. Identifying the number of persons with VWD (PwVWD) internationally will help target support to aid diagnosis of PwVWD.

Aim: To examine international registration rates of PwVWD, the influence of income status, geographical region and the age and sex profile.

Theory of change and strategic priorities of the world federation of haemophilia.

The World Federation of Haemophilia (WFH) is a global network of national member organizations (NMOs) that advocate, collectively and individually, to improve lives of people with inherited bleeding disorders. The WFH vision of "Treatment for All" speaks to a future in which all people with an inherited bleeding disorder will have access to care, regardless of their gender or where they live. Over the last several years, initiatives including the WFH Humanitarian Aid program, the World Bleeding Disorders Registry, and Guidelines for the Management of Haemophilia and von Willebrand disease have significantly changed how the WFH and its partners work to improve and sustain care for people with bleeding disorders.

Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set.

Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.

Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables.

Fostering Inter-Professional Education through Service Learning: The Belize Experience.

Service-learning and interprofessional education are forms of experiential learning that instill confidence in students. This pilot study used a post-service learning survey to explore the benefits of interprofessional education in a service-learning experience with students. Findings indicated that this service-learning experience contributed to the development of cultural awareness, and professional skills, as well as increased team attitudes and perceptions related to the respective roles of occupational and physical therapy students.

Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.

Background: Gene therapy for people with hemophilia (PWH) will soon become available outside current clinical trials. The World Federation of Hemophilia (WFH), in collaboration with International Society of Thrombosis and Hemostasis Scientific and Standardization Committee (ISTH SSC), the European Haemophilia Consortium (EHC), the US National Hemophilia Foundation (NHF), the American Thrombosis and Hemostasis Network (ATHN), industry gene therapy development partners and Regulatory liaisons have developed the Gene Therapy Registry (GTR), designed to collect long-term data on all PWH who receive hemophilia gene therapy.

Objective: The objectives of the GTR are to record the long-term safety and efficacy data post gene therapy infusion and to assess the changes in quality of life and burden of disease post-gene-therapy infusion.

Reimbursing the value of gene therapy care in an era of uncertainty.

Early-stage gene therapy (GT) clinical trials are demonstrating exciting results for persons with haemophilia (PWH), with the first products possibly licenced over the next few years for haemophilia A and B. These new treatments offer the possibility of a one-off approach to the treatment of haemophilia, with demonstrated increases in factor level expression and substantial reductions in both bleeds and factor utilization. However, clinical trial participants have demonstrated variable expression in factor levels, including decreases, over time, suggesting in some cases the effect may not last.

The World Federation of Hemophilia Annual Global Survey 1999-2018.

Introduction: The World Federation of Hemophilia (WFH) strives to achieve care for all patients with inherited bleeding disorders through research, advocacy, capacity building and education. The WFH developed and implemented the Annual Global Survey (AGS), through which comprehensive demographic and treatment data on bleeding disorders are collected each year from its constituent non-governmental national organizations.

Aim: To describe the development, methodology and achievements of the WFH AGS over the past 20 years.

Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table.

Introduction: With approval of gene therapy for haemophilia likely in the near future, policy frameworks are needed to guide the path forward for this disruptive and novel therapeutic advance.

Aim: The WFH has initiated a series of multi-stakeholder Gene Therapy Round Tables (GTRT) to better understand where guidance is needed and develop initial consensus statements to inform policy.

Methods: The first day of the 2nd GTRT was devoted to didactic presentations on models of access to gene therapy, payment and health technology assessment considerations, regulatory issues and the generation of evidence on safety and durable efficacy of gene therapy products.

Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries.

Background: The large observed variability in hemophilia prevalence prevents robust estimation of burden of disease.

Objective: To estimate the prevalence and prevalence at birth of hemophilia and the associated life expectancy disadvantage.

Design: Random-effects meta-analysis of registry data.

The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world.

In this first in a series of round table meetings, the 1st World Federation of Hemophilia Gene Therapy Round Table was convened to initiate a global dialogue on the expected challenges and opportunities that a disruptive therapy, such as gene therapy, will bring to the haemophilia community. Perspectives from key stakeholder groups, including healthcare professionals, regulators, payors, people with hemophilia and pharmaceutical industry representatives, were sought in the identification of the key issues we expect to face. Didactic presentations and open discussion covered the clinical development of gene therapy in haemophilia; regulatory perspectives of gene therapy; making informed decisions; accessibility, affordability and pricing of gene therapy; and ethical issues of gene therapy clinical trials.

Open-Label Study of Sleep Disturbances in Patients with Parkinson's Disease Treated with Rasagiline.

Background: The prevalence of sleep disturbances among patients with Parkinson's disease (PD) is estimated to occur in 37% to 98% of patients. Sleep disturbances have been associated with a reduced quality of life for patients with PD. The objective of this study was to assess the impact of rasagiline treatment on the severity of sleep disturbances among patients with idiopathic PD.

Systematic literature review of the risk factors, comorbidities, and consequences of hypogonadism in men.

The objective of this review was to summarize the literature on the risk factors, comorbidities, and consequences of male hypogonadism, which is defined as a syndrome complex that includes biochemical confirmation of low testosterone (T) and the consistent symptoms and signs associated with low T. A systematic literature search was performed in PubMed/MEDLINE, EMBASE, Cochrane Library for articles published in the last 10 years on risk factors, comorbidities, and consequences of male hypogonadism. Of the 53 relevant studies identified, nine examined potential risk factors, 14 examined potential comorbidities, and 30 examined potential consequences of male hypogonadism.

Incidence of difficult bag-mask ventilation in children: a prospective observational study.

Background: Difficult airway (DA), including difficult bag-mask ventilation (DBMV), and difficult intubation (DI) is an important challenge for the pediatric anesthesiologist. While expected DBMV can be successfully managed with appropriate equipment and personnel, unexpected DBMV relies on the resources available and the experience of the anesthesiologist at the time of the emergency. The incidence and risk factors of unexpected DA in otherwise healthy children, including DBMV among pediatric patients are not known.

Effectiveness of a low-cost virtual reality system for children with developmental delay: a preliminary randomised single-blind controlled trial.

Objectives: Physical and occupational therapists have started to use the Nintendo Wii™ gaming system with adults and children as part of their regular treatment. Despite the growing use of the Wii and trend towards evidence-based practice, limited evidence is available on the effectiveness of virtual reality using the Wii for children with developmental delay. The purpose of this study was to determine the feasibility and preliminary effectiveness of a low-cost gaming system for young children with developmental delay.

FGF-2 expands murine hematopoietic stem and progenitor cells via proliferation of stromal cells, c-Kit activation, and CXCL12 down-regulation.

Cytokine-induced expansion of hematopoietic stem and progenitor cells (HSPCs) is not fully understood. In the present study, we show that whereas steady-state hematopoiesis is normal in basic fibroblast growth factor (FGF-2)-knockout mice, parathyroid hormone stimulation and myeloablative treatments failed to induce normal HSPC proliferation and recovery. In vivo FGF-2 treatment expanded stromal cells, including perivascular Nestin(+) supportive stromal cells, which may facilitate HSPC expansion by increasing SCF and reducing CXCL12 via mir-31 up-regulation.