<abstract><title style='display:none'>Abstract</title> The terms ‘nurse-led service’ and ‘nurse-led care’ are often used, but are frequently not well defined. As health care delivery evolves across the world and struggles to cope with changes in medical care and with the rising number of people living longer with long term conditions, it is frequently suggested that more care should be delivered and coordinated by nurses who have the most day-to-day contact with affected individuals and their families. This paper addresses the notion of modern ‘nurse-led’ care in the 21st century, with a focus on haemophilia. Haemophilia is one specialty in which totally nurse-led and delivered care could become a reality, there is already evidence from both developing and developed countries of nurses providing excellent and innovative haemophilia care.</abstract>

What is a nurse-led service? A discussion paper

<abstract><title style='display:none'>Abstract</title> Tranexamic acid inhibits fibrinolysis by competitively blocking the lysine binding sites of plasminogen, inhibiting binding between fibrin and plasminogen, and activation of plasminogen. It also competitively inhibits tissue plasminogen activator and inhibits plasmininduced platelet activation. The synthesis of tranexamic acid was first reported in 1962. It is used to treat or prevent excessive blood loss from trauma, surgery, and in various medical conditions including haemophilia and heavy menstrual bleeding. As a medicine affecting coagulation, it is listed in the World Health Organization’s List of Essential Medicines. Tranexamic acid remains a versatile and inexpensive agent with potential benefit in health services from the lowest to highest income countries. This pharmacy review focuses on the evidence base to support the use of tranexamic acid in acquired and inherited bleeding disorders to reduce bleeding complications.</abstract>

The use of tranexamic acid in reducing bleeding complications

<abstract><title style='display:none'>Abstract</title> Wider access to modern treatment of haemophilia has led to a growing interest in the family’s role in management. An increasing amount of research has sought to understand the psychosocial impact of living with a child with haemophilia. Understanding how such demands affect parents and families who live with the daily threat of bleeding can help health professionals to provide effective support. A literature review was undertaken with the aim of summarising the key findings from studies published since 2000. The literature review describes many common themes from observational studies that were generally consistent with those emerging from interviews of parents of children with haemophilia. Few intervention studies were identified. Overall, this evidence shows that raising a child with haemophilia can be challenging for parents and the family. Quality of life is impaired in the parents of a child with haemophilia and that many aspects of life are affected. However, providing care can also be rewarding and programmes of support, education and appropriate treatment evidently improve the well-being of parents and families.</abstract>

The impact on parents of having a child with haemophilia

<abstract><title style='display:none'>Abstract</title>These guidelines have been developed by an expert panel of haemophilia treaters to support the appropriate management of people with haemophilia in Egypt. Although the guidelines are based primarily on the World Federation of Hemophilia (WFH) Guidelines for the Management of Hemophilia, they aim to address unmet needs and local requirements in the Egyptian setting.</abstract>

Guidelines for the management of haemophilia in Egypt

<abstract><title style='display:none'>Abstract</title> Introduction: Von Willebrand disease (VWD) is the most common hereditary coagulation abnormality. Individuals with VWD experience increased mucosal bleeding as well as gingival bleeding. Some evidence suggests that gingival bleeding is related to poor oral hygiene for fear of plaque removal methods that may cause bleeding to the gingival tissue. No studies have shown a correlation between VWD and gingival bleeding; however, these studies have not adjusted for possible confounding factors, such as presence of dental plaque, dental care utilisation, and oral hygiene habits. Aims: To determine the relationship between von Willebrand factor (VWF) levels and the amount of gingival bleeding, when controlling for possible confounding variables. Methods: This multi-site study included 44 women with VWD who completed a questionnaire to evaluate demographics, oral hygiene habits, perception of own oral health, oral health quality of life, and dental care utilisation. Clinical dental examinations were conducted to determine the presence of plaque and gingival bleeding on six teeth in each individual. Von Willebrand disease type and severity were determined through a medical chart review. Institutional review board (IRB) approval was obtained prior to data collection. Results: Paired sample t-tests revealed that VWF levels did not significantly affect the amount of gingival bleeding noted (p&gt;0.05) when controlling for presence of plaque. Multiple linear regression models revealed that other factors, such as age and last dental visit (p=0.044), had a larger effect on the amount of gingival bleeding. Conclusion: In women with VWD, the presence of plaque and dental care utilisation determines gingival bleeding more than the severity of VWD.</abstract>

Gingival bleeding and oral hygiene in women with von Willebrand Disease (VWD): a pilot study

<abstract><title style='display:none'>Abstract</title> An integrated model of specialised-delivered care is widely accepted as the standard of care for people with haemophilia in the UK. Assessment of available evidence on patient outcomes confirms this approach. But leading the specialist care for this group of patients does not require a medical qualification. Specialist nursing is well established within the haemophilia service and offers perhaps the greatest resource as health services cope with cost constraints on the specialist provision of services.</abstract>

Who should care for people with bleeding disorders?

<abstract>
<title style='display:none'>Abstract</title>
With over three decades of nursing experience, Simon reflects on his experience as a research nurse at the Oxford Haemophilia and Thrombosis Centre at a time of huge change for haemophilia care and treatment. He recalls the impact of conversations with people with haemophilia about gene therapy, how these challenged his assumptions about living with haemophilia, and how this prompted him to pursue a PhD. Highlighting the importance of listening to individual patient stories, Simon discusses how qualitative research contributes to a deeper understanding of what it is to live with a bleeding disorder. He considers the role of nurses in research and the importance of collaboration between research and clinical nursing teams as new treatments for haemophilia and other bleeding disorders emerge.
</abstract>

Living, Caring, Learning – The power of qualitative research in bleeding disorders care: One voice, from soloist to choir

<abstract>
<title style='display:none'>Abstract</title>
Michelle brought experience of working with people with chronic pain to her role as a bleeding disorders nurse, before going on to develop a research department at the National Hemophilia Foundation (now the National Bleeding Disorders Foundation). Reflecting on the pain experienced by various individuals she cared for, she highlights the importance of listening carefully to what patients have to say and the value of patient-reported information. Michelle explains how understanding this led her into patient advocacy, and how nurses acting as advocates for their patients supports their care and thereby enable people with bleeding disorders to live the best life they can.
</abstract>

Living, Caring, Learning – Listening to patients with bleeding disorders and not being afraid to advocate

Editorial – Living, Caring, Learning: The nurse-patient relationship in bleeding disorders care

<abstract>
<title style='display:none'>Abstract</title>
Cyrus, a haemophilia nurse in Kenya, reflects on his experience of treating a patient with a rare complication of haemophilia in a setting with limited access to treatment. The patient developed a debilitating abdominal pseudotumor following an inadequately treated traumatic bleed. After consulting with experts in other countries, surgery was deemed too great a risk. Initiating FVIII prophylaxis with the aim of shrinking the pseudotumour was identified as the most appropriate approach to treatment, however supply issues made it difficult to maintain a prophylactic regimen. When the pseudotumour continued to grow, posing a risk to adjacent organs, an international partnership enabled the initiation of emicizumab prophylaxis through a clinical trial. It was hoped that emicizumab would prevent further bleeding into the pseudotumour and allow it to shrink over time. The patient continued to experience breakthrough bleeding and was withdrawn from the trial after 2.5 years. By this time the supply of FVIII to Kenya through the through the WFH Humanitarian Aid Program had increased and the patient was able to resume FVIII prophylaxis. The pseudotumour is now stable and he continues to be cared for by the same nursing team. Reflecting on this case, Cyrus highlights the value of sharing expertise across borders and working in partnership to try to ensure the best treatment outcome for a patient with a rare complication in a resource-constrained setting. He also reflects on being compassionate in his practice and the importance of a holistic approach to patient support.
</abstract>

Living, Caring, Learning – Collaborating across borders to support a haemophilia patient with a rare complication

<abstract>
<title style='display:none'>Abstract</title>
Cathy, a haemophilia nurse specialist in the UK, reflects on a challenging patient with a rare inherited platelet disorder. The patient did not attend regular clinic appointments, and when presenting at clinic due to severe bleeding or feeling unwell was often aggressive and abusive towards staff. The care team were concerned that he was putting his health at risk and followed protocols for vulnerable adults to try and ensure he was accessing the care he needed. This involved bringing in expertise outside of the multidisciplinary team (MDT), including non-medical services. Steps were also taken to ensure staff safety and wellbeing through in-house psychological support and the agreement of strategies for dealing with difficult behaviour. Ultimately, it was necessary to find an alternative care pathway for the patient. While acknowledging her disappointment in this outcome, Cathy reflects on how the processes followed have reinforced the development of individualised care plans for all patients with complex needs, and the importance of access to specialist services beyond the MDT. She also highlights considerations around the safety and wellbeing of the care team, the role of discussion and ‘debriefing’, and the value of staff access to psychological support.
</abstract>

Living, Caring, Learning – Setting boundaries for the wellbeing of everyone in bleeding disorders care

<abstract>
<title style='display:none'>Abstract</title>
<sec>
<title style='display:none'>Background</title>
The burden of severe haemophilia A (HA) has been studied extensively owing to the higher bleeding frequency and associated treatment requirements, leaving a clear unmet need for research focused on the burden of mild and moderate HA.
</sec>
<sec>
<title style='display:none'>Aims</title>
This study sought to characterise the clinical and psychosocial burden of mild and moderate HA in the Italian cohort of the CHESS II study.
</sec>
<sec>
<title style='display:none'>Methods</title>
This was a retrospective analysis of clinical and psychosocial outcomes in a cohort of male adults (≥18 years old) with mild or moderate HA who participated in the cross-sectional CHESS II study (October 2019-November 2020). Treatment patterns, acute and chronic clinical outcomes and mental health indicators were collected via physician-completed forms. Psychosocial outcomes related to impact of HA on social activities, exercise, opportunities, and lifestyle were collected via a participant self-complete questionnaire. All results were reported descriptively.
</sec>
<sec>
<title style='display:none'>Results</title>
A total of 113 people with haemophilia A (PwHA) were included, 79 (70%) with moderate HA and 34 (30%) with mild HA, with mean age of 41.4 and 36.6 years, respectively. No one in the sample was receiving a prophylaxis at the time of data capture, with factor VIII use in the 12 months prior reported in 30% and 29% of moderate and mild PwHA, respectively. Ninety-one PwHA (81%) experienced ≥1 bleeding event in the preceding 12 months. People with moderate HA had higher mean annual bleed rate (2.9 vs. 1.1, respectively) and higher prevalence of chronic pain (74% vs. 35%), anxiety (20% vs. 12%), and/or depression (15% vs. 3%). Target joints were reported in 22% and 12% of moderate and mild PwHA, and problem joints in 51% and 12%, respectively. Of 113 participants, 44 (39%) completed the self-complete form (moderate HA, 57%; mild HA, 43%). Overall, 40% vs. 10% of those with moderate vs mild HA reported reducing or giving up social activities, 44% vs. 21% reducing or giving up exercise, 36% vs. 26% missing out on opportunities, and 48% vs. 26% reported HA impacted their lifestyle.
</sec>
<sec>
<title style='display:none'>Conclusion</title>
Moderate PwHA from the Italian CHESS II cohort appeared to have greater clinical morbidity and lifestyle impact than mild PwHA. Psychosocial outcomes were also worse among moderate PwHA, but significant burden was also observed among mild PwHA. These findings, and the absence of prophylactic treatment in the sample examined, highlight that improving management for potentially undertreated mild/moderate PwHA may aid the avoidance long-term clinical morbidity and negative psychosocial impact.
</sec>
</abstract>

Real-world clinical and psychosocial outcomes among people with mild or moderate haemophilia A treated on-demand in the Italian CHESS II cohort: a real-world data analysis

<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Background</title>Haemophilia treatment has improved life expectancy as well as lowered annual bleed rates and treatment burden for recent generations of people with haemophilia (PwH). However, PwH still face significant disease-related challenges on a day-to-day basis.</sec><sec><title style='display:none'>Aims</title>The aim of the present study was to explore the everyday life of PwH, including their beliefs and experiences related to their condition, their treatment, the challenges they face, and their ways of managing their condition.</sec><sec><title style='display:none'>Materials and methods</title>PwH were recruited through patient organisations in five European countries (Italy, Germany, Spain, UK and Ireland). Between 8–12 hours were spent with each person with haemophilia and/or their family, to follow them in their daily lives. Patient consultations with health care professionals (HCPs) were also observed when it was agreed in advance by both parties. In addition, HCPs were interviewed and haemophilia treatment centres (HTCs) were visited. Data were collected through semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and HCPs.</sec><sec><title style='display:none'>Results</title>Research was conducted with 42 people with haemophilia A and 9 people with haemophilia B, between 1.5 and 82 years of age. Interviews often included the wider social ecology of each PwH, including friends, family, and caregivers. In addition, 18 HCPs from seven HTCs were interviewed (on-site observation was carried out at six HTCs). The study found that despite treatment adherence and advances in treatment approaches, many PwH may be suboptimally protected and still experienced regular bleeds, pain and restrictions in their daily life. Moreover, many had built a narrative of normalcy around this way of living with haemophilia, and as such these issues are an expected and neglected reality of living with haemophilia today.</sec><sec><title style='display:none'>Conclusions</title>The results of this research indicate the need for more personalised and optimised treatment approaches which are better adapted to personal and life-stage specific challenges of PwH. Such an approach could help reduce challenges for PwH, their families, and the health care system, and further research into such approaches would be valuable.</sec></abstract>

“What more can we ask for?”: an ethnographic study of challenges and possibilities for people living with haemophilia

<abstract>
<title style='display:none'>Abstract</title>
The current standard of care for treating people with haemophilia (PWH) in the developed world is prophylaxis with regular infusions of clotting factor concentrates. Gene therapy is being investigated as a new treatment paradigm for haemophilia and if approved would potentially eliminate the need for chronic, burdensome infusions. In recent years, shared decision making (SDM) has become increasingly common in patient care settings. SDM is a stepwise process that relies on reciprocal information sharing between the practitioner and patient, resulting in health care decisions stemming from the informed preferences of both parties. SDM represents a departure from the traditional, paternalistic clinical model where the practitioner drives the treatment decision and the patient passively defers to this decision. As the potential introduction of gene therapy in haemophilia may transform the current standard of care, and impact disease management and goals in unique ways, both practitioners and PWH may find their knowledge tested when considering the appropriate use of a novel technology. Therefore, it is incumbent upon haemophilia practitioners to foster an open, trusting, and supportive relationship with their patients, while PWH and their caregivers must be knowledgeable and feel empowered to participate in the decision making process to achieve truly shared treatment decisions.
</abstract>

Personalising haemophilia management with shared decision making

<abstract><title style='display:none'>Abstract</title> Background: A broad scoping exercise was undertaken to assess and quantify haemophilia nursing care in Europe. Methods: A web-based survey in English was sent to known networks of haemophilia nurses working in Europe. This survey included questions concerning the haemophilia treatment centre, educational level, work activities, gaining knowledge/ expertise and development in the future. Results: In total, 94 nurses in 14 countries in Europe completed the survey. Overall, the majority (62%) of the nurses had over 20 years’ nursing experience, with 44% having more than 10 years’ experience in haemophilia. The educational level varied; with highest educational level of attainment being 41% at nondegree level nursing entry qualification, 35% BSc and 24% MSc. 21% worked in a centre where they treat only children, 26% only adults and 53% both. All had good access to treatment. The core activities (rated &gt;80%) of a haemophilia nurse were: prepare and administer medication, venepuncture and CVADuse (except cannulation), providing education and telephone advice, coordination of (multidisciplinary) care and assistance with clinical trials. Furthermore, 35% stated that they initiated and performed nurse-led research. In the future, almost all nurses would like to develop their expertise and knowledge base by studying at Masters level and above, to have more responsibility, and to conduct research. Conclusion: This scoping exercise provides a baseline assessment of haemophilia nursing across Europe. The results may provide a basis for a more thorough investigation of the current role and the principles of haemophilia nursing care; future opportunities, and the training requirements to develop the specialty.</abstract>

The role of the European haemophilia nurse

<abstract><title style='display:none'>Abstract</title> The role of the nurse continues to develop, probably at greater speed than ever before. Would Florence Nightingale ever have dreamt of nurses who could manage whole episodes of patient care from diagnosis, through admission, requesting investigations, prescribing treatments and evaluating outcomes? She probably did - when she instigated the first nursing outcome measures looking at infection control. Nurses can and do, do most things. What is important is that they are appropriately trained and continue to develop skills, that are relevant both to them and the patients for whom they care. Assessing this ability, or competence, requires knowledge and skills in itself. In this paper we describe the process of defining competence in haemophilia nursing. Some of these competencies are transferable from other areas of nursing, others are haemophilia-specific. Together they provide a personal development framework for nurses who work within haemophilia as part or all of their role.</abstract>

A core competency framework for haemophilia nurses in the UK

<abstract><title style='display:none'>Abstract</title> Haemophilia caregivers face limitations in their life leading to perceived burden. This single-centre study investigates the impact of burden on caregivers’ health-related quality of life (HRQoL). Methods: Questionnaires for caregivers comprised demographic data, HRQoL (EQ-5D, SF-36) and caregiver burden (IOF: Impact on Family Scale). Children were also asked about their HRQoL (EQ-5D, Haemo-QoL Short Form). Results: 20 consecutive parent/child dyads participated. 80% were mothers (mean age of 39.80±6.2 years) with 1-3 haemophilic children aged 8-17 years and 2.5±1.2 children &lt;18 years living in the household. Mothers did most childcare (80%), 50% worked part-time, and 55% reported that haemophilia had an economic impact on their family. 80% of boys had haemophilia A; 60% were severely affected. Most received home treatment (85%) and prophylaxis (80%). Caregivers’ and boys’ HRQoL was similarly good (EQ-5Dparents M=0.90±0.1 vs. EQ-5Dchild M=0.81±0.3); by contrast, boys reported better values in the EQ-VAS (Mchild=90.25±10.0 vs. Mparents=82.16±14.8). Caregivers reported highest impairments in the dimensions ‘vitality’ (M=60.00±20.5) and ‘emotional role’ (M=70.37±42.6) of the SF-36. In the IOF, caregivers reported highest impairments in the dimension ‘negative impact’ (M=60.08±20.7). Caregivers reporting high burden had significantly worse HRQoL in the domains ‘bodily pain’ (p&lt;.028) and ‘social functioning’ (p&lt;0.024) of the SF-36. Caregivers who reported that haemophilia had an economic impact on their family and those with a chronic disease showed significant higher impairments in caregiver burden and their HRQoL. Conclusions: The perceived burden of haemophilia has a direct impact on caregivers’ HRQoL. Further studies with haemophilia-specific instruments are needed to verify these findings.</abstract>

Caregiver burden in haemophilia: results from a single UK centre

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The Journal of Haemophilia Practice

 The Journal of Haemophilia Practice is an international, open-access, peer-reviewed journal that aims to provide a forum for all members of the...

 The Journal of Haemophilia Practice is an international, open-access, peer-reviewed journal that aims to provide...