INTRODUCTION:Joint bleeds in haemophilia cause destruction of articular structures, impaired function and pain. Up to 70% of people with haemophilia (PWH) report chronic pain. Little is known about the pain experiences in PWH in the UK.AIM:To identify prevalence and perceptions of pain among PWH living in the UK.METHODS:A cross-sectional, non-interventional survey study conducted among PWH (all severities). The survey incorporated elements from validated tools (EQ-5D; EQ-VAS) and was distributed via participating treatment centres.RESULTS:Five hundred and ninety-nine PWH responded, 91% aged > 18. 81% used factor prophylactically or on demand. More pain was reported by those treated on demand versus prophylaxis particularly in those who reported daily pain. 65% reported 'problem joints' based on individual impact rather than medically defined 'target joints', 2/3 reported multiple joint issues. The ankle was most commonly affected. 59% reported frequent pain, with 56% aware of pain constantly or most of the time and were more likely to report less favourable EQ-5D or EQ-VAS scores (p < .001). Pain frequency/awareness was consistent across all severities. Most discussed pain with care teams, 31% only when asked; 25% did not discuss it. Pain discussions resulted in physiotherapy referral (63%) analgesia prescription (48%), and a minority specialist pain referral (9%). Most felt well supported with regard to their pain, but 70% reported learning to live with it.CONCLUSION:Pain affects PWH of all ages and severities even in a well-resourced country significantly impacting quality of life. Clinicians must be more aware of chronic pain in PWH. Biopsychosocial approaches to pain assessment and management are recommended.

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.

Introduction Gene therapy has the potential to change the life experience of people with haemophilia and their families. A growing number of studies have examined the experience for those who have had gene therapy. A few studies have examined the process with other gene therapy among a wider cross-section of the haemophilia community. Exigency is a nested group of studies investigating the experience and understanding of the haemophilia community to identify what place gene therapy is likely to have in haemophilia care. Five groups have been identified: those who have already undergone gene therapy, those who do not want it, those who wanted to have it but withdrew or were withdrawn before dosing, those who have not yet been offered it and parents of children with haemophilia. Methods A qualitative, mixed-methods process will identify what each group understands about gene therapy and what it might mean for the haemophilia community in the future. Analysis All of the transcripts will be analysed by the lead and coinvestigator using a grounded theory approach. The texts will be coded into themes for further analysis. The data will be summarised and synthesised, and the views expressed will be represented descriptively. Ethics and dissemination Written consent will be required, and participants will be anonymised. All elements of the study will be reviewed by UK statutory bodies. The study findings will be submitted for publication in peer-reviewed journals, and at haemophilia conferences and symposia. The study results will also be disseminated directly to study participants. Each participant will receive a copy of any publication and a summary report at the end of the study. Trial registration number NCT04723680

Abstract Introduction Pain is recognised as a subjective phenomenon, often defined as ‘whatever the experiencing person says it is, existing whenever the experiencing person says it does’. Pain is a critical aspect of life for many people with haemophilia (PWH) but is under-recognised and inconsistently managed by clinicians. As haemophilia management moves towards non-factor-based treatments which may normalise life experience, it is unclear how this will impact on the experience and management of pain. Aims The Perceptions of Pain in Haemophilia study aimed to identify the impact of pain on men with haemophilia in the UK. Methods The study used mixed qualitative research methods (paper-based questionnaires and focus group interviews). Eligible PWH aged >18 years were invited to participate in a focus group to discuss pain, assessment and management. Each focus group discussion was recorded, transcribed and analysed thematically. Results Eighteen participants (13 haemophilia A (12 severe) and 5 severe haemophilia B) age range 18–58 years (median 32.5 years) joined focus groups conducted using an online video platform. The majority (95%) were treated with prophylaxis and reported few recent bleeds. Three main themes emerged: the impact of pain, managing pain, and factors influencing the experience of pain. Participants connected their earliest experiences of pain with childhood; it impacted their mental health and wellbeing, daily habits, routines, sports, hobbies, social life, work and education. Participants recognised the difference between the pain of acute bleeds and arthritic pain. Many did not like taking strong analgesics due to side-effects and concerns around addiction. Participants doubted the value of pain scales and noted a lack of empathy and understanding among health care professionals (HCPs), but valued physiotherapists. Participants recognised the value of talking about the negative impact of their pain experiences; however, they reported that family members, who often provided the most support, could not always truly understand their pain. Conclusion Pain is ‘normal’ for PWH, who adopt it into part of their everyday life experience. HCPs are ideally placed to impact this experience but seem to lack insight as to the extent of pain and how to manage it beyond prescribing stronger analgesia. The social and psychological implications of chronic pain should be better addressed by HCPs. This includes being cognisant that new therapeutic options will not resolve old pain.

Introduction: Many young men with hemophilia engage in physical activity and sport but face challenges to participation because of their hemophilia. Project GYM aimed to investigate the feasibility of a hemophilia-specific fitness program led by a personal trainer (PT) and its impact on gym activity, motivation, and adherence to exercise. Methods: This was a nonblinded, randomized feasibility study, recruiting participants aged 18 to 25 years with hemophilia A or B (all severities, +/- inhibitor) from three London hemophilia centers. All participants were given an activity tracker and free gym membership. Participants were randomized to a "gym only" or "gym and PT" arm. Participants completed questionnaires evaluating motivation to exercise, quality of life, physical activity levels, self-efficacy, and self-esteem at study start and study end. Results: Of 142 eligible individuals, 19 agreed to participate. Participants were healthy, with mean body mass index and adiposity slightly lower than the UK average. They reported low bleed numbers and had good joint health (median Hemophilia Joint Health Score [HJHS], 0; range, 0-13). The gym and PT group had more gym attendance than the gym-only group. Seven participants increased their activity levels and nine stayed the same, with no statistical difference between groups. HJHS scores improved in 3 participants and were unchanged in 12. There was no bleeding associated with gym activity. Conclusion: Project GYM has demonstrated the safety and feasibility of a tailored physical training program in young men with hemophilia. Increased gym attendance, with and without support from a PT, is associated with increased physical activity.

Contemporary hemophilia care supports physical activity, its benefits being well recognized. Despite recognition of the psychological challenges encountered by people with hemophilia, little is known about the psychological impact of physical fitness in this population.

Abstract Haemophilia nurses in the UK are instrumental in supporting people with haemophilia in self-management, including managing treatment options, recording treatment use and understanding the budgetary impact of prescribing practice. The widespread use of prophylaxis identified haemophilia as a high cost disorder to treat, resulting in a financially successful national tendering process with increased scrutiny of clotting factor use at both individual and haemophilia treatment centre level. The UK tenders, undertaken at a national level every three years, have ensured access to current and new therapies at the most cost-effective price through economies of scale in committing to purchase large volumes from suppliers. In the 2018 tendering round, NovoEight® (NovoNordisk) was added to the prescribing list and other recombinant factors were withdrawn, resulting in changes in prescribing for individual people with haemophilia. This ‘switching’ process is not uncommon in the UK, where national tenders have been in place since 2004. However, the unseen additional workload for nurses, driven by the demands of timely switching to meet product volumes and contracts, has never been captured. During the 2018 switch we interviewed 11 nurses and one operational manager from haemophilia centres across the UK to identify the barriers and facilitators to instigating this change. Ultimately the switching was completed in a timely manner, demonstrating significant cost reductions for factor concentrates. The unseen workload of the nurse – identifying which patients should have their product switched, discussion with and education of patients/families, adjusting prescriptions for home delivery of clotting factor concentrates and stock management and control to avoid waste, and organising the necessary additional clinic visits – was identified and costed based on salary per hour. Nurses remained positive that they were able to undertake this additional role but recognised that, with no specific national guidance regarding product choice, there may have inevitably been differences in approach between treatment centres.

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.

Abstract Management of haemophilia A requires administration of factor VIII therapy which, for those with severe haemophilia A in the UK, is predominantly self-administered at home in a prophylactic regimen to prevent or minimise bleeding. The UK undertakes a national tendering process every three years to ensure access to current and new therapies at a cost-effective price, through contracting for large volumes from individual suppliers. This means that some products may no longer be available and that new products can enter the UK market at any tendering stage. In the latest tendering round, in 2018, more than one product was withdrawn from the UK market and a new product (NovoEight®; Novo Nordisk) was added to the prescribing list. This meant people with haemophilia having to change products. The experience of 77 people with haemophilia or their carers who changed treatment products during this process was captured by questionnaires administered by haemophilia nurses from 12 treatment centres. Overall, although people with haemophilia felt that they had little influence in decision making about changing to NovoEight, they were confident with their new treatment including packaging and accessories for administration. This was seen more in those who switched from plasma-derived products where ease of infusion was rated highly. Users’ views of haemophilia treatment should be collected at times of change to identify facilitators and barriers experienced in self-management

IntroductionTreatment burden for the people with haemophilia has been documented, as has the burden of caring for a child with a common chronic disease such as asthma or diabetes. However, there remains a paucity of data about caregiver burden in haemophilia.AimsThe aim of this study was to evaluate the impact of bleeding on caregivers of children with haemophilia. Caregiver burden was stratified by the clinical status of their child.MethodsA multinational, non‐interventional study of caregivers of children with severe or moderate haemophilia, using the HEMOCABquestionnaire to evaluate caregiver burden.ResultsA total of 144 caregivers from seven EU countries participated in the study. Differences in caregiver burden were identified based on the clinical situation of the child. Greater burden was seen in caregivers of children who experienced joint bleeding in the preceding 12 months, or had target joints or reduced range of motion in most domains of the HEMOCAB. Caring for a child with a current inhibitor also caused significantly higher burden for caregivers when compared to caring for a child with tolerized inhibitor or without inhibitor. Caregivers of children with chronic pain reported significantly higher burden in all domains of the HEMOCAB except for “interaction with the father.”ConclusionCaregiver burden can be affected by the child's haemophilia status, particularly if joint health is impacted (eg bleeds, decreased mobility) or if the child suffers from chronic pain which was moderately correlated with joint bleeds.

Abstract Long-standing inhibitors present many day-today difficulties for the affected individual; the unpredictability of bleeds, bleed management, pain and treatment efficacy all affect quality of life. This study explored these issues through focus groups of affected individuals aged 16-25 in the UK. The data from the focus groups was analysed for recurring themes, which were coded under three umbrella headings: ‘daily impact’, ‘education and future’ and ‘resilience and support’. Participants felt isolated through geography and being extra ‘rare’ within the bleeding disorders community; used pain as a gauge of bleed resolution, often without use of analgesia; described transition to adult care as particularly worrying; and explained the potential impact of living with an inhibitor on future career options. Peer-to-peer networking could provide emotional support for these young adults, who could also be role models for the future. Despite the burden of living with an inhibitor and its treatment, participants described a good quality of life from their own perspectives. With new therapeutic options for these individuals on the horizon, they look forward to a future with fewer bleeds and less pain.

Abstract Transition is the term used to describe the process of approaching and crossing the chronological boundary between paediatric and adult care services. Transfer of care describes the administrative arrangements associated with moving from a paediatric to an adult service across this boundary. Transforming Transition was a nurse-led initiative designed to facilitate the sharing of knowledge and experience to enhance transition practice in the UK. The aims of the programme were to secure changes in working practices that enabled nurses to drive improved transition outcomes, enhance self-management by people with haemophilia and develop clinical leadership. From the outset of the programme it was clear that there were differences in practice and that sharing practice would be one route to addressing some of these variations. As a result of the knowledge and experience shared through the programme, resources described in this paper are available to all haemophilia centres. Sharing between people with haemophilia, their families and carers, and the haemophilia clinical team have resulted in the development of practical resources that enhance practice within the haemophilia centre. Sharing of practice between nurses and young people with haemophilia at the programme workshops provided a focus for development of local and regional action plans, a framework for reviewing progress, the introduction of transition clinics and improved transition documentation.

Abstract Services for people with haemophilia in Uganda are poor, resulting in a very low number of confirmed diagnoses. Over the past year, the Haemophilia Foundation of Uganda (HFU) has engaged in a concerted and coordinated media campaign focusing on radio, television and social media promoting the message that haemophilia results in painful shortened lives but is a treatable condition if appropriate services are in place. At the end of 2015, this awareness campaign culminated in a patient-screening day, along with a patient information camp. In advance of the patient testing day, a team from London’s Great Ormond Street Hospital ran an educational workshop for staff from Mulago Hospital. The team also attended the testing day, taking clinical histories from those attending, and ran workshop sessions at the patient meeting. The physician-training day attracted obstetricians, paediatricians, nurses, dentists, physiotherapists, midwives and pharmacists. Delegates understood that to develop a haemophilia service in Uganda required capacity building, and awareness raising, initially at a central level but gradually moving out to the regions. Physicians in Uganda have now embarked on a journey to providing haemophilia care. The next steps are to build political engagement and to continue raising awareness among the population.

The ubiquity of mobile technology offers the potential for instantaneous and two-way transfer of information, as well as the potential for improving medical care delivery and extending it to those in countries and regions with a less developed medical infrastructure. This review considers the role of mobile health (mHealth) technology in managing hemophilia. This is a disease area in which good record-keeping is an essential component of home-based hemophilia care. Health care professionals contribute patients' data to national registries and databases and frequently interrogate those data for commercial and academic purposes. Only rarely are the data used to directly improve the care of the individual patient. Patient-focused apps designed to respond to an individual's personal data may offer the potential to empower patients to live healthier lifestyles, reinforcing the care provided by health care professionals and offering a mechanism for peer-support and promoting adherence to an individualized management plan. Keywords: mHealth, hemophilia, peer-support, adherence