UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.

Introduction: 2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and severe to moderately severe haemophilia B and a third in 2024. Gene therapy is a transformative, irreversible treatment with long-lasting effects, necessitating development of new clinical pathways to ensure optimal outcomes.

Aim: To develop a consensus framework and service specification for delivery of AAV gene therapy for haemophilia in adults within the UK using the hub-and-spoke model proposed by the European Association of Haemophilia and Allied Disorders and the European Haemophilia Consortium.

Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK.

With the first gene therapies for haemophilia approved by the European Commission, the US Food and Drug Administration, and the Medicines and Healthcare products Regulatory Agency, it is important to consider the remaining unmet needs and challenges that may arise throughout patients' treatment journeys. We discuss existing unmet needs and important considerations prior to, during, and following haemophilia gene therapy treatment in the UK, and propose potential next steps. Key areas for attention are education, psychological support, and guidance on implementation.

The views of women with bleeding disorders: Results from the Cinderella study.

Introduction: Despite therapeutic advances in bleeding disorder treatment, over the past 20 years women with bleeding disorders have reported delayed diagnosis, impaired quality of life, dismissive attitudes from health professionals and inappropriate care.

Aim: To explore the lived experience of women with a diagnosed bleeding disorder and to improve understanding of their unmet needs.

Methods: Women haemophilia carriers (WHC), women with a diagnosed bleeding disorder (WBD) and women with immune thrombocytopenia (WITP) were invited to complete an online survey.

The lived experience of women with a bleeding disorder: A systematic review.

Background: Research studies have described the morbidity associated with inherited bleeding disorders such as hemophilia and von Willebrand disease in women, but their effect on daily living has long been underrecognized. This systematic review sought to document the lived experience of women with a bleeding disorder by assessing research findings on quality of health care, socioeconomic factors, and mental health.

Methods: A systematic search was carried out in Web of Science, the Cumulative Index to Nursing and Allied Health Literature, and PubMed on July 31, 2020.

Multidisciplinary Team Care of Patients with Hemophilic Arthropathy: A Qualitative Assessment of Contemporary Practice in the UK and Canada : Canada/UK: MDT Practices for Hemophilia.

We used a structured interview to explore approaches to comprehensive hemophilia and arthropathy care among 24 healthcare professionals (HCPs) from multidisciplinary teams (MDTs) in Canada and the UK. Represented MDTs typically comprise a hematologist, nurse, physiotherapist, and sometimes an orthopedic surgeon; pediatric (and some adult) MDTs also include a social worker/psychologist. HCPs emphasized the centrality of a team approach, facilitated through MDT meetings and involvement of all MDT members in patient care.

Hemophilia treatment in 2021: Choosing the"optimal" treatment using an integrative, patient-oriented approach to shared decision-making between patients and clinicians.

The mainstay of hemophilia treatment is to prevent bleeding through regular long-term prophylaxis and to control acute breakthrough bleeds. Various treatment options are currently available for prophylaxis, and treatment decision-making is a challenging and multifaceted process of identifying the most appropriate option for each patient. A multidisciplinary expert panel convened to develop a practical, patient-oriented algorithm to facilitate shared treatment decision-making between clinicians and patients.

European principles of care for women and girls with inherited bleeding disorders.

Introduction: Despite increasing awareness of issues faced by women and girls with inherited BDs (WGBD), standards of care are lacking, with disparities in diagnosis and treatment for WGBD across Europe. We aimed to develop practical principles of care (PoC) to promote standardization of care for WGBD within European Haemophilia Treatment and Comprehensive Care Centres (HTC/CCCs).

Methods: The co-creation process, supported by the European Association for Haemophilia and Allied Disorders, consisted of four multidisciplinary meetings with health care providers (HCPs) experienced in WGBD care, and European Haemophilia Consortium representatives, combined with broad patient and HCP consultations in the European haemophilia community.

Challenges of antithrombotic therapy in the management of cardiovascular disease in patients with inherited bleeding disorders: A single-centre experience.

Introduction: Cardiovascular events in patients with inherited bleeding disorders are challenging to manage. The risk of bleeding secondary to antithrombotic treatment must be balanced against the risk of thrombosis secondary to haemostatic therapy.

Methods: Patients with inherited bleeding disorders with coronary artery bypass grafting (CABG), percutaneous coronary intervention (PCI) or atrial fibrillation (AF) from a single centre (2010-2018) are included.

Principles of care for acquired hemophilia.

Objective: To establish clear priorities for the care of patients with acquired hemophilia A (AHA) by proposing 10 key principles of practical, holistic AHA management.

Method: These principles were developed by the Zürich Haemophilia Forum, an expert panel of European hemophilia specialists comprising physicians and nursing and laboratory specialists.

Results: The 10 proposed principles for AHA care are as follows: (a) Improving initial diagnosis of AHA; (b) Differential diagnosis of AHA: laboratory assessment of patients with unusual bleeding; (c) Effective communication between laboratories, physicians, and specialists; (d) Improving clinical care: networking between healthcare professionals in the treating hospital and specialist hemophilia centers; (e) Comprehensive assessment of bleeding; (f) Appropriate use of bypassing agents; (g) Long-term follow-up and monitoring for efficacy and safety of immunosuppressive treatment; (h) Inpatient/outpatient settings; (i) Access to innovative and disruptive treatments; (j) Promotion of international collaborative research.

Stability of Turoctocog Alfa, a Recombinant Factor VIII Product, during Continuous Infusion In Vitro.

 Turoctocog alfa is a recombinant factor VIII (rFVIII) for the prevention and treatment of bleeding in patients with hemophilia A, including those undergoing surgery and invasive medical procedures. This in vitro study evaluated the physical and chemical stability of turoctocog alfa during continuous infusion (CI) over 24 hours at 30°C.  The study was performed at 30°C ( ± 2°C).

Women and girls with heavy menstrual bleeding and inherited bleeding disorders: A call to action for the Haemophilia Treatment Centre Nurse.

Over the last several decades, the increasing focus on women with inherited bleeding disorders (WBD) has brought more patients into Haemophilia Treatment Centres (HTC) around the world. These women present with unique challenges including a significant risk of heavy menstrual bleeding (HMB). The influx of a new patient group has necessitated expansion of the services provided by the multi-disciplinary team in HTCs.

The UK haemophilia specialist nurse: Competencies fit for practice in the 21st century.

Introduction: Nurses play a central co-ordinating role in delivering comprehensive care for people with haemophilia and allied bleeding disorders, for which they need a broad range of competencies. The UK Haemophilia Nurses Association (HNA) published a role description in 1994 which was developed into a competency framework in 2014. This has now been updated to reflect current educational and clinical practice.

HOw Patients view Extended half-life products: Impressions from real-world experience (The HOPE study).

Introduction: Extended half-life (EHL) clotting factors have been shown to offer people with haemophilia (PwH) protection from bleeding with fewer infusions, which might reduce treatment burden.

Aim: The HOw Patients view Extended half-life products (HOPE) study aimed to explore, understand and describe patient expectations around the prophylactic use of EHL products and to establish whether these expectations were met through individual follow-up analysis.

Methods: The HOPE study was a prospective, qualitative cohort study conducted among PwH who had switched to Fc fusion protein EHL products in routine clinical care and who had not been recruited to clinical trials of these products.

The second Team Haemophilia Education Meeting, 2016, Frankfurt, Germany.

The first Team Haemophilia Education (THE) Meeting was held on 7-8 May 2015 in Amsterdam, The Netherlands. It aimed to promote the optimal care of patients with haemophilia through education of the multidisciplinary treatment team. This was achieved by reviewing the latest developments in haemophilia management, considering how these can be implemented in the clinic to improve patient care and providing a platform for networking and debate for all haemophilia treatment team members.

Monitoring low dose recombinant factor VIIa therapy in patients with severe factor XI deficiency undergoing surgery.

Although factor XI (FXI) concentrate is an effective replacement therapy in severe FXI deficiency without inhibitors, some patients are unwilling to receive it because it is plasma-derived. We report on the use and monitoring of low dose, recombinant factor VIIa (rFVIIa, NovoSeven®), to cover surgery (caesarean section, cholecystectomy and abdominoplasty) in four female patients (FXI:C 2-4 IU/dl, aged 32-51 years) who wished to avoid exposure to plasma. None of our patients had inhibitors to FXI.

Menorrhagia in adolescents with inherited bleeding disorders.

Study Objectives: We reviewed the management and treatment outcomes of menorrhagia in adolescents with inherited bleeding disorders and assessed the impact of menorrhagia on their quality of life.

Design: Retrospective review of case notes and a questionnaire study.

Setting: Comprehensive-care hemophilia treatment center.

Professional profile of radiologic technology educators.

Objective: Full-time radiologic technology educators (n = 565) were surveyed to determine their demographic characteristics and professional profile.

Results: Overall, the majority of radiologic technology educators surveyed were women between the ages of 40 and 59, had a bachelor's or master's degree, were certified in radiography and reported annual incomes from 40,001 dollars to 60,000 dollars. Most educators spent between 1 hour and 8 hours per week on classroom instruction/laboratory and in the clinical setting.