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. Those who indicated a willingness to be interviewed took part in a focus group or one-to-one interview to further explore their lived experiences. RESULTS: Two hundred and eighty women completed the survey (126 WHC, 96 WBD, 58 WITP) 13 women (seven WHC, three WBD, three WITP) participated in a focus group (11) or individual interview (2). The most frequently reported symptoms were heavy periods (81%) and bruising (81%). Compared to the group as a whole, more WBD took time off work or school due to symptoms, had discussed symptoms with a healthcare professional (HCP), and had seen a nurse or gynaecologist. WHC were least likely to be screened for a bleeding disorder or seen in a specialist setting. Themes discussed included difficulty obtaining a diagnosis, lack of awareness amongst and poor communication from HCPs, stress and anxiety, coping strategies and sources of support. CONCLUSION: Many of the experiences described by women with bleeding disorders 20 years ago remain prevalent. Healthcare provision needs to change to offer them better treatment and support.

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. Nurses already play a central role in patient and family education within the HTC. As such, they are well positioned to participate in the development of adaptations within the HTC infrastructure to provide clinical care and education specifically for WBD. The nursing experts in HTCs should play an active role in outreach as well as providing education to WBD. Despite this supposition, review of the growing body of literature surrounding the topic of WBD is notable for a paucity of literature highlighting the role of the HTC nurse and potential impact on this, relatively new, but steadily increasing, patient population.

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. RESULTS: A total of 11 patients undergoing CABG (n = 3), PCI (n = 5) or with AF (n = 3) and a diagnosis of haemophilia A (n = 8), haemophilia B (n = 1), factor XI deficiency (n = 1) and von Willebrand disease (n = 1) managed by a multidisciplinary team are reported. In patients undergoing CABG, factor levels were normalized for 7-10 days with trough levels of 70-80% with severe patients continuing high-dose factor prophylaxis (trough 20-30%) three weeks post-operatively with daily aspirin. In a patient with mild haemophilia A and an inhibitor, recombinant factor VIIa dosing was monitored with thromboelastometry. For PCI, a 3rd-generation drug-eluting stent with one month of dual antiplatelet therapy in addition to high-dose prophylaxis as needed was preferred. Patients with AF and severe haemophilia did not receive antithrombotic treatment, and a thrombin generation assay was used to guide heparin dosing in mild haemophilia. CONCLUSION: Our experience demonstrates the importance of interdisciplinary communication to identify strategies that decrease the risk of bleeding and thrombosis. The use of extended, increased intensity prophylaxis facilitated antiplatelet therapy. Global assays may help balance the intensity of haemostatic and antithrombotic treatment.

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. Relevant medical societies outside Europe were contacted for confirmation. RESULTS: We developed ten PoC for WGBD, stressing the importance and benefits of a centralized, multidisciplinary, comprehensive, family-centred approach to support and manage WGBD during all life stages. These PoC emphasise the right to equitable access and quality of care for all people with BDs, irrespective of gender. Multiple medical societies outside Europe also confirmed their support for endorsement. CONCLUSIONS: Ten PoC for WGBD evolved from an iterative process among stakeholders, supported by relevant medical societies worldwide. These PoC can serve as a benchmark for diagnosis and comprehensive multidisciplinary management of WGBD, and improve awareness of their unique challenges. They offer a framework to guide HTC/CCCs in providing equitable care for all WGBD, both in their own services and in other healthcare settings. Implementation of these principles aims to positively impact the health, wellbeing and quality of life for WGBD.

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. CONCLUSION: The proposed principles for holistic AHA care aim to ensure swift diagnosis and optimal patient management. Key to achieving this goal is training for healthcare personnel in non-specialist hospitals and collaboration between different specialists. We hope these principles will increase awareness of AHA in the wider medical community and catalyze efforts toward improving its practical, multidisciplinary management.

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. AIM: To summarize the evidence supporting the nurse's advanced role within haemophilia care and develop new competencies to deliver comprehensive care within a multidisciplinary team. METHODS: Systematic reviews were identified by PubMed literature search. The HNA conducted workshops to consult its membership, and the authors incorporated this input to update its competency framework within the structure outlined by Health Education England in multiprofessional framework for advanced clinical practice in England (2017). RESULTS: The proposed framework includes five domains (Clinical knowledge, Clinical/direct care, Communication and support, Collaborative practice and Research) supported by indicators for four levels of practice (beginner, competent, proficient and expert). The framework is a tool which nurses and their managers can use to assess skills and knowledge, and identify learning needs appropriate to personal development and improve patient care and outcomes. CONCLUSION: The HNA has developed a new competency framework to provide a strong foundation for haemophilia specialist nurses to continue improving services for people living with bleeding disorders and their families, as well as supporting personal development alongside new therapeutic options, models of care and follow-up.

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. Semi-structured audio-recorded interviews were undertaken over two time points; transcripts were analysed to systematically generate theory from data that contains both inductive and deductive thinking. RESULTS: Forty-three interviews were conducted with 25 participants. Most participants were positive about EHL treatment and intended to continue using them. Reduced frequency of infusions meant lives were less disrupted or dominated by haemophilia, and there was less perceived stress on overused veins. For those PwH who did not reduce infusion frequency, there were other perceived benefits from EHLs with respect to greater protection with higher trough levels and fewer bleeds. CONCLUSION: Patients switching to EHL treatments believe these products will result in fewer infusions and less disruption of everyday life, leaving them feeling more protected with fewer bleeds and increased activity levels, as well as enhanced well-being and mental health. Understanding patient expectation and experience around using products adds real-world data to clinical trial experience.

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. The second THE Meeting was held on 19-20 May in Frankfurt, Germany, and participants included doctors, nurses, physiotherapists, patient representatives and data management staff from 20 different countries. Topics covered the role of the multidisciplinary team in delivering the best haemophilia care, challenges in the management of haemophilia across Europe, available clotting factor treatments, future treatments and the use of genetics in advising carriers of haemophilia. This report is a summary of the key developments in haemophilia care presented by various investigators and healthcare professionals at THE Meeting 2016.

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. Strategies are urgently required to fulfil these needs. An immediate priority for information providers should be comprehensive education for people with haemophilia (PWH) and healthcare professionals (HCPs). Greater access to resources and training in psychological services will be required throughout the treatment pathway. More specific guidance is required to define the implementation model, criteria for accreditation, and responsibilities of care centres. Furthermore, PWH may revisit discussions with HCPs multiple times pre-infusion, thus the patient journey is unlikely to be linear. Consideration of these challenges, and of potential strategies to address them, will be integral to optimising the future success of this promising therapy.

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. References were hand searched. Abstracts of the 2019 and 2020 congresses on bleeding disorders were hand searched. Key journals were screened for relevant studies published after the search date until the analysis was completed on December 7, 2020. RESULTS: Of 635 potentially eligible publications, 27 published since 1998 were selected for review. Most studies were of moderate to high quality but meta-analysis of quantitative studies was not possible due to difference in outcomes and assessment.Women with a bleeding disorder experience obstacles to accessing care, difficulties living with their disorder, interference with schooling and work, and poor mental health. Diagnostic delay and lack of recognition of symptoms mean treatment and support may not be available. Where comparisons with controls were made, women's negative experiences were greater than those of men. CONCLUSIONS: Women with bleeding disorders experience major negative impacts of their disorders on daily life and mental well-being. Many of the challenges identified in earlier research are evident in more recent studies.

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. In both countries, nurses and physiotherapists play critical, multifaceted roles. Respondents agreed that MDTs are crucial for successful transitioning, which can be facilitated by close collaboration between pediatric and adult MDTs, even when they are not co-located. Physiotherapists are instrumental in providing non-pharmacological pain relief. Hematologists or physiotherapists typically make orthopedic referrals, with the nurse, physiotherapist and hematologist working together in patient preparation for (and follow-up after) surgery. MDT best practices include a non-hierarchical team approach, ensuring that all MDT members know all patients, and regular MDT meetings. Together, these real-life insights from the MDT perspective emphasize the value of the MDT approach in comprehensive hemophilia 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. Key variables were identified, and an algorithm proposed based on five variables: bleeding phenotype, musculoskeletal status, treatment adherence, venous access, and lifestyle. A complementary, patient-focused preference tool was also hypothesized, with the aim of exploring individual patients' priorities, preferences, and goals. It is hoped that the proposed algorithm and the hypothesized patient preference tool will assist in selecting a treatment for each patient that is as efficient as possible in preventing bleeds while also accounting for the patient's expectations and priorities.

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

Objective 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. Materials and Methods The study was performed at 30°C ( ± 2°C). A CI system with pump speed set at either 0.6 or 1.5 mL/h was used to evaluate the stability of three turoctocog alfa strengths (500, 1,000, and 3,000 IU), equating to doses of 1.1 to 16.1 IU/h per kilogram of body weight. The following parameters were evaluated at selected time points between 0 and 24 hours: appearance of solution, clarity, pH, potency, purity, content, total high molecular weight proteins (HMWPs), and oxidized rFVIII. Results The mean potency of turoctocog alfa was maintained within the predefined acceptance criteria during CI for both pump speeds with all three strengths at 6, 12, or 24 hours (500 IU: ≥484 IU/vial; 1,000 IU: ≥1,014 IU/vial; and 3,000 IU: ≥3,029 IU/vial). Furthermore, the appearance of solution, clarity, pH, purity, content of turoctocog alfa, total HMWP, and oxidized forms were also within the predefined limits, and comparable to the reference samples (time = 0 hours) for the pump speeds and product strengths assessed. Conclusion Physical and chemical stability of turoctocog alfa was maintained during CI over 24 hours. There was only minor degradation or changes in any of the parameters tested. Potency was within the prespecified acceptance limits throughout 24 hours of infusion. These findings confirm the suitability of turoctocog alfa for CI.

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. Additionally, hospital or community college programs employed the majority of educators. Demographic characteristics of radiologic technology educators varied according to the type of institution in which they were employed and by education level. CONCLUSION: Study findings show a potential loss of qualified educators in the near future and the need for increased efforts to prepare and recruit radiologic technologists into the education career path.

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. Our aim was to find the optimal dose of rFVIIa by in vitro spiking of patient samples and to correlate this with the response to rFVIIa in vivo . Prior to surgery, venous blood was collected into sodium citrate with corn trypsin inhibitor and spiked with 0.25-1.0 µg/ml rFVIIa in vitro , equivalent to a 15-70 µg/kg dose of rFVIIa in vivo . Analysis using thromboelastometry and thrombin generation assays, triggered with tissue factor, showed that the thrombin generation assay was insufficiently sensitive to the haemostatic defect in these patients. A concentration of 0.5 µg/ml was as effective as 1.0 µg/ml FVIIa in normalising thromboelastometry in vitro in all four patients. Therefore, patients received 15-30 µg/kg rFVIIa at 2-4 hourly intervals with tranexamic acid 1g every six hours. Post treatment samples were taken at 10-240 minutes and showed initial normalisation of thromboelastometry with gradual return to baseline after 2-4 hours. In conclusion, low-dose rFVIIa therapy was successfully used in four patients with severe FXI deficiency undergoing surgery to prevent bleeding and can be monitored using thromboelastometry.

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. PARTICIPANTS: Adolescents with inherited bleeding disorders who had registered at the center and were attending the multidisciplinary hemophilia and gynecology clinic for management of menorrhagia. INTERVENTIONS: Review of medical records and assessment of menstrual blood loss using the pictorial blood assessment chart and quality of life measurements during menstruation using a questionnaire. MAIN OUTCOME MEASURES: Scores on pictorial blood assessment charts and quality of life measurements before and after treatment. RESULTS: Of 153 girls aged 12 to 19 years who had registered at the center and had an inherited bleeding disorder, 42 (27%) attended the multidisciplinary clinic for management of menorrhagia. The majority (38/42; 90%) had experienced menorrhagia since menarche. Of the group, 5 (12%) required hospital admission for acute menorrhagia and severe anemia. Treatment options for menorrhagia included tranexamic acid, desmopressin, combined oral contraceptive pills, clotting factor concentrate, and the levonorgestrel intrauterine system. These treatment modalities, alone or in combination, were associated with a reduction in menstrual blood loss (median pre- and posttreatment pictorial blood assessment chart scores were 215 and 88, respectively) and improvement in quality of life scores (median pre- and posttreatment were 26 and 44, respectively). CONCLUSIONS: Menorrhagia is a common symptom in adolescents with inherited bleeding disorders. It can present acutely, and it adversely affects quality of life. Treatment options include hemostatic and/or hormonal therapies and can improve the quality of life of affected girls.