rss_2.0The Journal of Haemophilia Practice FeedSciendo RSS Feed for The Journal of Haemophilia Practicehttps://sciendo.com/journal/JHPhttps://www.sciendo.comThe Journal of Haemophilia Practice Feedhttps://sciendo-parsed.s3.eu-central-1.amazonaws.com/6472179a215d2f6c89dbbabe/cover-image.jpghttps://sciendo.com/journal/JHP140216Living, Caring, Learning – Setting boundaries for the wellbeing of everyone in bleeding disorders carehttps://sciendo.com/article/10.2478/jhp-2024-0011<abstract> <title style='display:none'>Abstract</title> <p>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.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00112024-05-07T00:00:00.000+00:00Living, Caring, Learning – Collaborating across borders to support a haemophilia patient with a rare complicationhttps://sciendo.com/article/10.2478/jhp-2024-0010<abstract> <title style='display:none'>Abstract</title> <p>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.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00102024-05-07T00:00:00.000+00:00Editorial – Living, Caring, Learning: The nurse-patient relationship in bleeding disorders carehttps://sciendo.com/article/10.2478/jhp-2024-0013ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00132024-05-07T00:00:00.000+00:00Living, Caring, Learning – Listening to patients with bleeding disorders and not being afraid to advocatehttps://sciendo.com/article/10.2478/jhp-2024-0009<abstract> <title style='display:none'>Abstract</title> <p>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.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00092024-05-07T00:00:00.000+00:00Living, Caring, Learning – The power of qualitative research in bleeding disorders care: One voice, from soloist to choirhttps://sciendo.com/article/10.2478/jhp-2024-0012<abstract> <title style='display:none'>Abstract</title> <p>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.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00122024-05-07T00:00:00.000+00:00Real-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 analysishttps://sciendo.com/article/10.2478/jhp-2024-0008<abstract> <title style='display:none'>Abstract</title> <sec> <title style='display:none'>Background</title> <p>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.</p> </sec> <sec> <title style='display:none'>Aims</title> <p>This study sought to characterise the clinical and psychosocial burden of mild and moderate HA in the Italian cohort of the CHESS II study.</p> </sec> <sec> <title style='display:none'>Methods</title> <p>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.</p> </sec> <sec> <title style='display:none'>Results</title> <p>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.</p> </sec> <sec> <title style='display:none'>Conclusion</title> <p>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.</p> </sec> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00082024-04-22T00:00:00.000+00:00A multi-stakeholder collaborative approach to awareness, education and support needed in the haemophilia gene therapy patient journey – a report on insights from a European patient advisory boardhttps://sciendo.com/article/10.2478/jhp-2024-0007<abstract> <title style='display:none'>Abstract</title> <p>Gene therapy presents a potentially transformational approach to haemophilia management. The patient journey for gene therapy is unlike that for other haemophilia treatments, and its one-time, irreversible nature has led to broad agreement that informed shared decision-making is essential. Understanding the educational and support needs of people with haemophilia (PwH) who may consider or undergo gene therapy in the future is key to enabling this. A one-day patient advisory board was conducted with the aim of gaining insights on what education and support would enable PwH to have appropriate conversations with health care practitioners (HCPs) about gene therapy and participate in informed shared decision-making. The participants were 11 age-diverse men with severe haemophilia A or B, including six who had received gene therapy in phase 1-3 clinical trials. Two were members of a national patient organisation or a patient organisation leader. Participants agreed that informed shared decision-making was essential for gene therapy, but most felt the choice to have gene therapy would be limited by healthcare budgets and access arrangements. They identified key education needed by PwH to enable true informed decision-making, best delivered through a collaborative approach by patient organisations and HCPs. Eligibility criteria should be more widely shared within the community as part of general education around gene therapy. Gene therapy should be presented as one of a range of treatment options; understanding its risks, uncertainties and benefits is key to managing expectations and minimising treatment remorse. Awareness of how different factor levels impact bleeding tendency and frequency would be helpful, as success of gene therapy cannot be determined by factor activity alone. Other key educational needs included follow-up commitments, the role and potential side effects of corticosteroids or other immunosuppressants, impact on sense of identity, mental health, family planning, and managing bleeds after gene therapy. Teach-back could help ensure appropriate levels of understanding; psychological support could aid managing expectations. Peer-to-peer learning delivered across a variety of platforms, to share experiences of gene therapy and contextualise more ‘formal’ gene therapy education, was considered invaluable.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00072024-04-18T00:00:00.000+00:00Exploring patient and clinician perspectives on the benefits and risks of emerging therapies for the treatment of haemophilia: a qualitative studyhttps://sciendo.com/article/10.2478/jhp-2024-0006<abstract> <title style='display:none'>Abstract</title> <sec> <title style='display:none'>Background</title> <p>Enhanced horizon scanning for emerging treatments has identified that both haemophilia A and haemophilia B pathways will be enriched with a range of new medicines with varying benefit-risk profiles. Patient and clinician views on the balance of the benefits and risks associated with emerging therapies will affect their use for the treatment of haemophilia, while also introducing the need for effective communication strategies to enable informed patient-clinician decision-making.</p> </sec> <sec> <title style='display:none'>Aim</title> <p>This study aimed to explore patient and clinician views on the perceived benefits and risks of emerging therapies for the treatment of haemophilia. The study also aimed to gain insight into clinician-patient communication on benefit and risk and how this shapes decision-making on new therapeutic options.</p> </sec> <sec> <title style='display:none'>Methods</title> <p>Qualitative methods, using online focus groups and one-to-one interviews, were guided by a defined set of questions. Data were collected in 2022 and 2023 from a sample of adult patients identified through the national patient group (The Haemophilia Society) and clinicians identified through the NHS England Clinical Reference Group (CRG) and HAEM-NET. Data were analysed thematically.</p> </sec> <sec> <title style='display:none'>Findings</title> <p>Seven patients and ten clinicians (seven consultant haematologists; three advanced nurse practitioners) participated. Five summary themes were identified, three of which related to clinician-patient communication: (i) active vs. passive patients; (ii) health literacy; (iii) external factors. One theme related to gene therapy perspectives on benefit-risk, and one theme concerned the legacy of the infected blood scandal.</p> </sec> <sec> <title style='display:none'>Conclusion</title> <p>There remains scepticism about gene therapy across all research participant groups, which suggests that uptake is likely to be relatively slow, with divergence anticipated between haemophilia A and B. Treatment decision-making and benefit-risk discussions are complex and multifaceted issues which in haemophilia are heavily influenced by the infected blood scandal. Clinicians frame treatment decision-making which necessitates the requirement for benefit-risk training and high-quality tailored patient gene therapy information materials.</p> </sec> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00062024-04-08T00:00:00.000+00:00CATCH 2023 Meeting Summary: Collaborate & Address Treatment Challenges in Haemophiliahttps://sciendo.com/article/10.2478/jhp-2024-0003<abstract> <title style='display:none'>Abstract</title> <p>Access to treatment and healthcare services for people with haemophilia in the United Kingdom (UK) and Republic of Ireland ranks highly by international standards for contemporary haemophilia management. Collaborate &amp; Address Treatment Challenges in Haemophilia (CATCH) is an annual Sobi™ medical education meeting which brings together multidisciplinary haemophilia treaters throughout the UK and Ireland to discuss all aspects of haemophilia management, including associated challenges and unmet need. This report summarises key issues explored and discussed during CATCH 2023, including ‘raising the bar’ in haemophilia care; haemophilia care for women and girls; changing haemophilia treatment paradigms to consider disease impact as well disease severity; bone health and haemophilia; and shared decision-making.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00032024-04-08T00:00:00.000+00:00Living, Caring, Learning - Thinking outside the box to solve care challenges in a rare blood disorderhttps://sciendo.com/article/10.2478/jhp-2024-0001<abstract> <title style='display:none'>Abstract</title> <p>A specialist nurse for over 20 years, Sandra reflects on her experience of finding ways to deliver effective care for a young girl with a very rare clotting disorder. Symptoms of the girl’s disorder were evident from birth and through an emergency use request Sandra and the care team enabled her family to access a treatment that at the time was in clinical trial. Poor venous access meant there was a need to adapt how treatment was administered and she worked with the girl’s parents to ensure that she was treated effectively. Alongside educating the parents, Sandra highlights the importance of her role in educating co-workers and other hospital staff likely to come into contact with the girl, to ensure that she always had access to timely and appropriate care. She also reflects on other instances where thinking creatively enabled patients in her care access to treatments that may not otherwise have been accessible. Now retired, Sandra continues to be involved in advocacy for people with bleeding disorders.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00012024-02-26T00:00:00.000+00:00System change in practice: A report from the EHC Think Tank workstreams on Registries and Patient Agencyhttps://sciendo.com/article/10.2478/jhp-2024-0005<abstract> <title style='display:none'>Abstract</title> <sec> <title style='display:none'>Introduction</title> <p>System change addresses the fundamental causes of persistent, complex problems to achieve large-scale, sustainable solutions at multiple levels. Recognising the need for system change to ensure equitable access to healthcare for people with bleeding disorders and other rare diseases, the European Haemophilia Consortium (EHC) established a Think Tank to work alongside its traditional advocacy initiatives. The Think Tank has mobilised a broad range of healthcare stakeholders to identify challenges and co-create potential solutions through a series of thematic workstreams exploring specific aspects of the healthcare system. This paper reports on outcomes and learnings from the Registries and Patient Agency workstreams.</p> </sec> <sec> <title style='display:none'>Methodology/Process</title> <p>During a series of online meetings and face-to-face discussions, workstream stakeholders contributed to a three-phase process: 1. Discovery; 2. Strategy; 3. Innovation. Having identified key challenges to system change for Registries and Patient Agency, stakeholders mapped the system in which they were working to refine the challenges, recognise enablers and constraints to progress, and use leverage points to co-create strategies for change.</p> </sec> <sec> <title style='display:none'>Results</title> <p>The Registries workstream prioritised actions to address challenges around lack of common purpose and data quality, and agreed to move forward with developing a consensus statement to facilitate buy-in from key stakeholders, working on good governance for registries, hosting a network for registry owners, and creating a model for patient data input and feedback. A Registries Roadmap was completed for 2025 and 2030, and a project initiated to align registries in Europe. The Patient Agency workstream agreed actions should focus on challenges related to the role of the patient, recognising the need to elevate patient influence in all aspects of the healthcare system. Actions aimed to address the current stakeholder hierarchy and gaps in patient health literacy, and to optimise the potential of digital tools to enable patient contributions to patient-reported outcome and experience measures (PROMs and PREMs). Projects include developing a patient agency guidebook and a patient experience data (PED) dossier on von Willebrand disease, to provide a one-stop repository for regulators, researchers, clinicians and patients.</p> </sec> <sec> <title style='display:none'>Conclusions</title> <p>There is a clear need for system change to ensure equitable access to healthcare for people with rare diseases such as bleeding disorders. Bringing together multiple stakeholders with different and complementary knowledge and approaches has facilitated the development of innovative strategies for system change in relation to Registries and Patient Agency. Work has started on pilot projects to move these strategies forward.</p> </sec> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00052024-02-26T00:00:00.000+00:00Living, Caring, Learning - Building family relationships in haemophilia carehttps://sciendo.com/article/10.2478/jhp-2024-0002<abstract> <title style='display:none'>Abstract</title> <p>With over 35 years’ experience of paediatric nursing, almost half of which she has spent in haemophilia care, Robyn reflects on the importance of taking a family-focused approach and engaging with parents and caregivers. She describes her experience of providing care for a family with two boys with severe haemophilia A and inhibitors, and how listening to and working closely with the parents enabled good outcomes. Robyn points to the central role of nursepatient relationship in haemophilia care but highlights the importance of ensuring that this close therapeutic relationship remains professional.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00022024-02-26T00:00:00.000+00:00Living, Caring, Learning - Changing mindsets and enabling goals for people with haemophiliahttps://sciendo.com/article/10.2478/jhp-2024-0004<abstract> <title style='display:none'>Abstract</title> <p>In a nursing career spanning almost five decades, Anne Louise has spent over 30 years caring for people with haemophilia in South Africa. She reflects on the importance of prophylaxis and how tailoring treatment to meet individual needs not only helps to prevent bleeds but can make goals and aspirations attainable. Describing her experience of how she supported a young man with severe haemophilia in achieving his ambition to become a paramedic, Anne-Louise demonstrates the importance of the nurse-patient relationship and the role of the nurse in providing individualised care. She discusses advocating for prophylaxis and the need to change mindsets, and highlights the need for further change in access to treatments for haemophilia in South Africa. The patient also describes his journey to achieving the level of fitness he needed to undertake the entrance exam to train as paramedic.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2024-00042024-02-26T00:00:00.000+00:00Short- and longer-term goals for change – A report from the 2nd workshops of the EHC Think Tank Workstreams on Access Equity and Future Care Pathwayshttps://sciendo.com/article/10.2478/jhp-2023-0023<abstract> <title style='display:none'>Abstract</title> <sec> <title style='display:none'>Introduction</title> <p>The second series of workshops for the EHC Think Tank Workstreams on Access Equity and Future Care Pathways involved working towards consensus on addressing challenges to progress around achieving equitable access to care and shaping rare disease care pathways that meet patient needs while remaining practicable and affordable to healthcare providers. This report summarises workshop outcomes from these two workstreams, in which stakeholder participants identified a ‘guiding star’ determining the direction of ongoing focus, defined achievable ‘near star’ milestones, and enablers and constraints to achieving these.</p> </sec> <sec> <title style='display:none'>Guiding Stars</title> <p>The Access Equity Workstream proposed focusing on developing a healthcare system that enables patients to benefit from care and treatment fairly and impartially. The Future Care Pathways Workstream agreed that their focus would be on developing care pathways that provide the right intervention at the right time by the right healthcare professional in the right formats with a variety of delivery methods to suit the person.</p> </sec> <sec> <title style='display:none'>Near Stars</title> <p>For the Access Equity Workstream, changes in narrative and behaviour were the achievable milestones that the group agreed to prioritise. Greater stakeholder collaboration and consistent data collection and use will be important enablers for change. Participants proposed greater emphasis on investment and value (instead of cost and return) and a change of mindset from ‘fixing the patient’ to achieving a healthy life through early intervention and preventing comorbidities. However, lack of clarity over what constitutes ‘value’ and around stakeholders' responsibilities, limited outcome measures and resistance to change may constrain progress. A near star for the Future Care Pathways workstream was the development of seamless, personalised care pathways with integrated digital and AI-based technologies to enable real-time measurement of pathway effectiveness. Participants felt that understanding and respecting patient behaviour and the nudges and incentives needed to promote pathway acceptance will be important. As in the Access Equity Workstream, they recognised the role of routine, standardised data collection for measuring outcomes, sharing information and informing decision-making. They predicted that building trust between stakeholder groups (including patients, healthcare providers, academic and life science companies) and using patient networks and advocates effectively would enable collaboration and ensure that patient needs and insights are acted upon. However, financial and legal aspects, inadequate implementation of technological infrastructure, limited systems integration, and lack of stakeholder time, effort and energy are all potential constraints that will need to be addressed.</p> </sec> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00232023-12-29T00:00:00.000+00:00A multidisciplinary approach to optimising the virtual management of haemophilia: a roundtable meeting of UK expertshttps://sciendo.com/article/10.2478/jhp-2023-0022<abstract> <title style='display:none'>Abstract</title> <p>The necessity of a multidisciplinary team (MDT) approach in haemophilia care is well recognised globally, with international guidelines advocating this. Prior to the coronavirus disease 2019 pandemic, virtual MDT haemophilia care was gaining support worldwide. However, the pandemic necessitated the rapid implementation of innovative virtual solutions to ensure continued access to multidisciplinary care. A multidisciplinary panel of healthcare professionals who specialise in haemophilia care in the United Kingdom gathered to discuss the following: the current landscape of haemophilia MDT care and best practices, the benefits, challenges, and opportunities for virtual MDT care, managing bleeds remotely, virtual paediatric care, and the future of virtual MDT care. The consensus was that virtual MDT care is widely used, however formats vary depending on the healthcare setting, available resources, MDT preferences, and local policy. Advisors agreed that virtual MDT care has several benefits, such as improved convenience/choice for their patients and wider patient reach. However, many patient-specific and logistical challenges exist. Hybrid care models may provide an opportunity to overcome these challenges. The decision on how bleeds are managed (virtually versus face-to-face) depends on provider preference, the patient-provider relationship, and the patient’s disease severity, history, and ability to self-manage. As such, this should be assessed on a case-by-case basis. Virtual tracking tools cannot be solely relied upon for MDT decisionmaking as patient accuracy cannot be ascertained. The MDT composition for paediatric care should be tailored to the patients’ and their parents’/caregivers’ needs. Lastly, hybridised care will likely be adopted for future haemophilia management and will facilitate the advancement of MDT care.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00222023-12-29T00:00:00.000+00:00Recognising the importance of the research nurse and study coordinators in enhancing retention of people with haemophilia in clinical trialshttps://sciendo.com/article/10.2478/jhp-2023-0017<abstract> <title style='display:none'>Abstract</title> <sec> <title style='display:none'>Introduction</title> <p>For retention in clinical trials of investigational medical products (CTIMPS), the strategy and tactics to keep enrolled participants from discontinuing participation (dropouts) are important. Haemophilia trials often have extended follow-up and require motivated participants who commit for the duration of the study, which may be underestimated at initiation. Study discontinuation may lead to inconclusive results and prolonged trials. Research nurses (RNs) and study coordinators (SCs) play an important role in clinical trials and are considered the link between principal investigator and study participants. We discuss the importance of the RNs and SCs in retention of participants, the barriers and challenges to retention, and the interventions utilised to support it.</p> </sec> <sec> <title style='display:none'>Methods</title> <p>We conducted a 12-question online survey at registration for a virtual research study update summit. The survey investigated the thoughts of RNs/SCs about retention in CTIMPS, their experiences and challenges, and interventions to prevent attrition.</p> </sec> <sec> <title style='display:none'>Results</title> <p>Eighty-seven RNs and SCs from 24 countries (Asia, Australia, Europe, the Middle East and North America) participated. The majority (62/75 responses) reported having 1-5 or 6-10 clinical trial participants (46.6% and 36% respectively); the remainder reported 11-40 trial participants. The majority reported no or few participants dropping out of haemophilia clinical trials in the preceding three years (42.8% and 33.7% respectively). RNs/SCs believed participants dropped out because of ‘loss of interest’ (50%), ‘study going on too long’ (39%), ‘too many visits’ (virtual or at centre) (37.5%), ‘visits are too time-consuming’ (34%) and a ‘lack of time’ (32.8%). Over half of RNs/SCs believed ‘dropouts’ would be higher if they did not make extra efforts in retention. The top interventions to retain participants were: keeping an open dialogue, organising visits to fit participant schedules, discussing adverse events, understanding the protocol, and allowing participants time to ask questions. Retention is achieved through flexibility, timeliness, combining trial activities with routine care, shared decision making and effective communication, including via contemporary technology.</p> </sec> <sec> <title style='display:none'>Conclusion</title> <p>This study investigating the retention role of RNs/SCs revealed low dropout rates in haemophilia trials. RNs/SCs are able to offer flexibility to trial procedures by, for example, scheduling visits within trial timing ‘windows’ to support and facilitate individualised follow-up. Patient-centred care and attention, including trust, attitude and expectations, aid successful retention and trial outcomes. This often unrecognised role is important in supporting people with haemophilia in clinical trials to promote good study outcomes.</p> </sec> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00172023-12-20T00:00:00.000+00:00Living, Caring, Learning – Addressing challenges in the transition to adult carehttps://sciendo.com/article/10.2478/jhp-2023-0020<abstract> <title style='display:none'>Abstract</title> <p>Greta, a specialist nurse at one of the largest haemophilia treatment centres in the Netherlands, reflects on the role one particular patient played in helping to reshape her centre’s approach to the process of transition from paediatric to adult care. The patient, a young man with severe haemophilia B and an inhibitor, experienced frequent bleeds and venepuncture was sometimes difficult. He continued to be reliant on his mother to administer injections and order his treatment. Greta considers the importance of ensuring young people transitioning to adult care have the knowledge and skills they need to manage their haemophilia while balancing this with the reality of an individual’s circumstances, often including the ongoing role of parents in their lives as supporters and caregivers. She suggests that transition is best approached as a gradual process rather than an event, reflecting on how this helps to build trust between the new health care team and both the patient and their parents. She also points to the importance of supporting parents through the transition process as needed. Greta is clear that an approach to care that seeks to understand individuals and acknowledges the feelings of everyone involved can make a difference in enabling successful transition to adult care and in patient care more generally.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00202023-12-17T00:00:00.000+00:00Living, Caring, Learning – Trust and teamwork in patient carehttps://sciendo.com/article/10.2478/jhp-2023-0021<abstract> <title style='display:none'>Abstract</title> <p>Marcela, a nurse specialist in Brazil, reflects on the importance of trust in the therapeutic relationship in achieving the best patient outcomes. She describes her experience with a patient with severe haemophilia A and an inhibitor. The patient was non-adherent to his treatment regimen and immune tolerance induction (ITI) failed. At a time when he was experiencing increasing joint problems and pain due to bleeding, the treatment centre was approached regarding a clinical trial of a novel non-factor replacement therapy. Following discussion among members of the care team and with the patient, a decision was made to put him forward for the trial, despite concerns about his ability to adhere to the trial treatment regimen. The patient completed the trial, his quality of life improved and he continues to have prophylaxis with emicizumab. Marcela goes on to reflect on the role this new treatment likely played when the patient was involved in a serious accident and the surgeries that followed. She highlights the importance of not giving up on patients who present a challenge and working closely together with treatment centre colleagues to achieve the best care for individual patients.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00212023-12-17T00:00:00.000+00:00Factors associated with delays in seeking treatment for acute bleeding among persons living with haemophilia in Uganda: a cross-sectional mixed methods studyhttps://sciendo.com/article/10.2478/jhp-2023-0018<abstract> <title style='display:none'>Abstract</title> <sec> <title style='display:none'>Introduction</title> <p>Haemophilia is associated with a high tendency of acute bleeding episodes which can lead to severe morbidity and mortality in the absence of prompt care. Although delay in seeking care can lead to adverse outcomes, many people with haemophilia (PwH) in Uganda do not report to haemophilia treatment centres (HTCs) for treatment in the event of acute bleeding within the two hours recommended by the World Federation of Hemophilia (WFH). Information on factors associated with delays in seeking care is limited.</p> <fig id="j_jhp-2023-0018_fig_005" position="float" fig-type="figure"> <caption> <p>A study among people with haemophilia presenting at five regional treatment centres across Uganda found transport costs is a major issue impacting treatment seeking for acute bleeds</p> </caption> <graphic xmlns:xlink="http://www.w3.org/1999/xlink" xlink:href="graphic/j_jhp-2023-0018_fig_005.jpg"/> </fig> </sec> <sec> <title style='display:none'>Aims</title> <p>This study aims to determine the extent and factors associated with delays in seeking treatment for acute bleeding among people with haemophilia (PwH) in Uganda to inform appropriate mitigating stratregies and hence improve health outcomes and quality and life.</p> </sec> <sec> <title style='display:none'>Methods</title> <p>The study was conducted in five haemophilia treatment centres (HTCs) across Uganda and used a cross-sectional design with mixed methods of data collection. PwH seeking care at the HTCs for acute bleeding episodes were consecutively sampled. A pre-tested questionnaire was used to collect data. Alongside this, qualitative interviews explored health workers’ perceptions of factors associated with delays in seeking treatment among patients. Health workers were purposively selected. The quantitative data were analysed using STATA version 16; qualitative data were analysed manually using inductive thematic analysis to generate codes, categories, sub-themes and themes. Multivariate modified Poisson regression analysis was used to determine factors associated with delay to seeking treatment at HTCs.</p> </sec> <sec> <title style='display:none'>Results</title> <p>224 male PwH, aged 1 to 67 years (median 11 years) were included in the analysis. The time taken following an acute bleeding symptom to attending the HTC ranged between 1 and 65 hours, with a median of 9 hours. PwH were more likely to delay seeking treatment for acute bleeding episodes if they used public means of transport to the HTC (aPR 1.39; 95% CI 1.22-1.59). PwH who took immediate other actions on recognition of a sign of an acute bleeding episode were less likely to delay seeking treatment at the HTC (aPR 0.75; 95% CI 0.66-0.84).</p> </sec> <sec> <title style='display:none'>Conclusion</title> <p>Delays in seeking treatment among PwH in Uganda are the result of an interplay between individual and health system factors. Continued information and education programmes are needed to ensure PwH and caregivers understand the benefits of early treatment-seeking, alongside expanding HCP knowledge and capacity building. Mobile clinics and home therapy could reduce travel and waiting times for those needing to access treatment. Addressing these issues could help to provide a level of care for PwH that helps to ensure improved quality of life and better health outcomes.</p> </sec> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00182023-12-17T00:00:00.000+00:00Factor X deficiency: a comment on two recent case studieshttps://sciendo.com/article/10.2478/jhp-2023-0019<abstract> <title style='display:none'>Abstract</title> <p>Hereditary factor X deficiency (HFXD) is a rare genetic bleeding disorder, affecting approximately 1 in 1,000,000 individuals globally, and resulting in abnormally low activity of clotting factor X (FX). Diagnosis may occur early in life, particularly in patients with a family history of the disorder or severe deficiency, or later in life for those with mild-moderate deficiency. Regardless of age at diagnosis, this disorder can significantly impact the lives of patients, as well as those of caregivers and/or family members. Alongside a wide historical array of HFXD treatments, including supportive care with blood transfusions, FX replacement with fresh frozen plasma or prothrombin complex concentrates, specific replacement has been made possible with the isolation and concentration of human plasma-derived FX for therapeutic use, offering the potential for a streamlined prophylactic regimen and improved quality of life and wellbeing. Two recent case studies describe the diagnosis and management of children with HFXD in India and the Philippines, both of whom were diagnosed in infancy following severe bleeding episodes. They share important similarities but highlight key differences in the experience of diagnosis and management of HFXD. Together they illustrate the lack of a specific target population subgroup for this rare but serious bleeding disorders, and the importance of checking FX levels (among other clotting factors) when prothrombin time (PT) and partial thromboplastin time (PTT) are prolonged.</p> </abstract>ARTICLEtruehttps://sciendo.com/article/10.2478/jhp-2023-00192023-12-17T00:00:00.000+00:00en-us-1