Volume 7 (2020): Issue 1 (January 2020)

Guidance from the European Association for Haemophilia and Allied Disorders (EAHAD) sets out the educational milestones haemophilia nurses should aim to achieve. However, little is known about the resources nurses use for education and current awareness.

To assess the current educational level of haemophilia nurses, how and where they access ongoing education, where they feel they need extra support, and how best this teaching could be delivered.

Haemophilia nurses in the Haemnet Horizons group devised and piloted a questionnaire. This was distributed in hard copy to nurses attending the 2019 EAHAD Congress and promoted as an online survey hosted by Survey Monkey.

Seventy-five replies were received from nurses in Europe (46 in the UK), and two from nurses in Chile and the Philippines. Most described their role as ‘specialist nurse’, with the majority having worked in haemophilia care for up to ten years. Half had a nursing degree and one quarter had a nursing diploma. Three quarters had attended at least one course specifically related to haemophilia nursing. Almost all used academic sources, study days and the websites of health profession organisations as information sources. Most also used Google or Wikipedia, but fewer used Twitter. Patient association websites were more popular among non-UK nurses. About half attended sponsored professional meetings and three quarters reported that educational meetings were available in their workplace. A clear majority preferred interactive and face-to-face activities using patient-focused content.

The study shows that nurses, predominantly in Western Europe, access a range of educational resources, most of which are ‘traditional’. Use of online sources is high, but social media are less popular than Google or Wikipedia. Further research is needed to explore the potential of new media for haemophilia nurse education, and whether the current educational levels and needs highlighted in the survey remains the same across the whole of Europe.

Pain associated with bleeding disorders has been demonstrated to have an impact on patients’ and families’ quality of life. Both acute and chronic pain are common experiences and require attention by professionals working in haemophilia treatment centres (HTCs). The benefits of psychological pain management strategies such as cognitive behaviour therapy and self-management skills training are well documented; however, it is not well understood how Canadian social workers involved in haemophilia care perceive and provide pain management support to patients.

To explore the current understanding of pain management and practice as well as the education needs of members of Canadian Social Workers in Hemophilia Care (CSWHC).

Twelve semi-structured qualitative interviews were conducted with members of CSWHC. Transcribed interviews were coded with NVivo software and thematically analysed.

The four key themes reflecting the experiences of social workers are: 1) Limited comprehension of key issues related to pain; 2) Conditioning to push through pain; 3) Expanding pain knowledge to enhance practice; 4) How we practice social work and choose to step in.

The current practice of CSWHC members aligns with literature in three main areas including assessment, instrumental services, and counselling. Social workers support the development of pain education and practical resources for patients with haemophilia who experience pain. While formal education, advocacy, and policy development of pain assessment and management are recognised, these areas require further research and development.

Chronic pain is common in people with bleeding disorders and can complicate clinical management, impair quality of life, and contribute to disability. People living with bleeding disorders often seek advice on pain management from the bleeding disorder treatment team; however, lack of condition-specific assessment tools to guide clinical communication about pain are a barrier to care.

To develop and examine the clinical feasibility of a patient-reported outcome (PRO) tool designed to facilitate pain assessment and support clinical communication about pain for adults attending outpatient bleeding disorder clinics.

Tool development involved patient cognitive interviews and item refinement by a multidisciplinary clinician and patient working group. Clinical feasibility of the tool was evaluated with a survey of a small clinical sample in an outpatient bleeding disorder clinic. The Pain Treatment Planning Questionnaire (PTPQ) contains 28 items on the pain experience and treatments used to manage or prevent pain.

Participants completing the feasibility testing (n=42, 62% male) reported mild mean pain scores (usual pain μ=2.4, SD=2.0) with the majority (57.1%) reporting persistent pain in the past 30 days. Median PTPQ completion time was five to seven minutes and mean item response rate was 95.2%. The majority (95.2%) of participants found the questionnaire easy to understand, reported no difficulty understanding items, and recommended no changes to the questionnaire.

Preliminary testing among a small sample in a clinical setting suggests that the PTPQ is a clinically feasible, acceptable, condition-specific PRO pain assessment tool for adult patients with bleeding disorders. Further testing is required to determine if the PTPQ affects treatment decision-making and patient outcomes.

The ankle joint is the most common site of bleeding for people with haemophilia (PWH) in the developed world. Recent surveys suggest that PWH do not always have access to non-surgical musculoskeletal interventions and that when provided; there is considerable heterogeneity in clinical practice.

To determine patient perceptions and the potential benefits of a new combined multidisciplinary physiotherapy-podiatry haemophilia clinic, and to observe the effect on frequency of bleeds and ankle joint Haemophilia Joint Health Scores (HJHS).

PWH with a history of ankle bleeds, pain, foot and/or ankle deformities from a single UK haemophilia centre were referred to the clinic from December 2017 to December 2018. Pre- and post-intervention ankle joint HJHS data and ankle annualised joint bleed rate (AJBR) were collected together with a satisfaction questionnaire asking patients their views on the clinic's value, usefulness and their satisfaction after the initial appointment.

Twenty-seven PWH (16 children and 11 adults) attended the clinic. All patients agreed or strongly agreed that they were satisfied with the new clinic. The combined multidisciplinary nature of the clinic meant that patients only needed to attend one appointment with the expertise of two professionals, rather than attending two separate appointments. All patients reported it “more useful to see the physiotherapist and podiatrist together”. There were no statistically significant differences in ankle AJBR or HJHS scores post-intervention compared to pre-intervention.

Establishing a multidisciplinary physiotherapy-podiatry clinic for PWH with a history of ankle bleeds, pain, foot and/or ankle deformities appears to increase patient satisfaction. We did not observe a significant change in ankle AJBR or ankle HJHS scores, suggesting they might not be sufficient to evaluate potential benefits to patients. A larger study incorporating validated tools, focusing on patient-reported foot function, pain, activity and quality of life is needed to confirm if there is any effect of a combined physiotherapy-podiatry intervention on ankle joint AJBR and function.

The aims were to evaluate the driving forces for prescribing factor products, to evaluate current attitudes and knowledge toward factor product and industry, and to discuss the implications for health care providers in practice.

An anonymous electronic survey was distributed to 744 HTC health care providers (HCPs); 118 responses were analysed.

The most common driving force for HCPs to change a patient's factor product was poor response to current therapy, while the most common perception of patients’ motivation to switch products was the potential for fewer infusions. HCPs with strong influence over the prescribed therapy identified inadequate pharmacokinetic (PK) studies as an important driving force; patients/caregivers perceived as having a strong influence over which therapy is prescribed selected less frequent dosing as an important motivator. HCPs who allow patients/caregivers to have a strong influence over which factor is prescribed were more likely to cite patient/caregiver request as a significant driving force for change in therapy.

The haemophilia treatment landscape continues to evolve and is becoming increasingly complex. The multitude of treatment options available now offer choices, presenting a need to focus on patient-centric prophylaxis.

Switching between clotting factor products is becoming increasingly common as product choice increases and financial pressure grows to choose the most cost-effective options. Guidance on carrying out the switch recommends a complex and long process that may benefit from being defined in a protocol. Haemophilia nurses may be responsible for managing product switches; anecdotal evidence suggests that clinical practice is variable.

To explore the role of specialist nurses in switching between clotting factor products and their use of a protocol.

Nurses attending the 2018 World Federation of Hemophilia Congress were surveyed about clinical practice at their treatment centre and use of a protocol for switching clotting factor products.

Of 192 nurses attending the conference, 49 nurses returned completed questionnaires, 45 of which were included in the study after exclusions. Responses were exclusively from economically developed countries. Almost all respondents (96%) had direct experience of switching. Half of those who responded to a question about protocol-based switching reported that switches were based on a protocol. When authorship was reported, the protocol was written by haemophilia nurses in about half of cases. Practice about blood testing to determine individual pharmacokinetic parameters prior to the switch was variable, but most nurses (86%) reported screening for inhibitors prior to switching. Respondents agreed to share their protocols among their peers, although only four were received by the research team.

Clinical practice in switching between clotting factor products is variable. Some nurses are switching treatments for patients without the supported of a written protocol, whereas others are involved in writing and implementing protocols. Sharing protocols is a first step in helping to establish best practice.

Haemophilia is a rare hereditary haemorrhagic disease caused by coagulation factor VIII (haemophilia A) or IX (haemophilia B) deficiency. Very few data exist on this disease in Congo. This survey aims to describe the epidemiological and clinical aspects of the children affected.

A descriptive cross-sectional study was carried out in the haematology department of the Brazzaville University Hospital over a period of two years. Children (under 18 years of age) with haemophilia and with a factor VIII or IX level less than or equal to 30% were identified. The parameters analysed included age, diagnostic delay, type and severity of haemophilia, type and frequency of bleeding manifestations, complications and history of transfusion.

Nineteen patients were identified with an average age at diagnosis of four years. The average time to diagnosis was six years, and the most frequent first known bleeding episode was haemorrhage during circumcision. Family history was found in 14 cases. There were 13 cases of haemophilia A and six cases of haemophilia B. Fourteen cases were severe haemophilia; no mild cases were identified. Haemorrhagic manifestations included haemarthrosis, haematomas and mucocutaneous haemorrhages. The average number of haemorrhagic episodes per year was 12. Haemophilic arthropathy was present at diagnosis in seven cases, with the main location being the knee. The average number of hospitalisations before diagnosis was two. Sixteen patients had been transfused at least once.

Although circumcision is the most frequent first known haemorrhagic manifestation of haemophilia in Congo, patients are often diagnosed late, sometimes with severe osteoarticular complications. Further measures are needed to help ensure early diagnosis and improve care.

Haemophilia is an X-linked congenital bleeding disorder due to deficiency of coagulation factor VIII (in haemophilia A) or factor IX (in haemophilia B) caused by mutations of the respective clotting factor genes. Treatment involves the administration of an appropriate dose of factor concentrate, as soon as possible, in the event of any bleeding episode. In low-resource settings, such as Northeast India, where factor concentrates are not widely available, people with haemophilia (PwH) may bleed profusely even from trivial external injuries, warranting transfusion of blood or blood products. We previously reported on the use of a low cost, foam-based haemostatic patch to treat an external bleed in a single patient. In this study, we investigated its use to treat a range of external injuries in PwH presenting at Assam Medical College and Hospital.

Over 24 months, eligible PwH with external injuries attending our haemophilia clinic were treated with a topical haemostatic patch (VELSEAL-T) at the target bleeding site. The time to cessation of bleeding was recorded and the wound sites evaluated after haemostasis to monitor efficacy and safety.

Out of 72 individuals with bleeding disorders who volunteered to participate, 59 cases of external bleeding in 48 PwH were eligible for inclusion in the study. Nine (15.3%) had aberration wounds, 24 (40.7%) cut wounds, 21 (35.6%) tooth and/or gum bleeding and five (8.4%) bleeding from puncture wounds. The average time required for achievement of haemostasis was 9.9 (±4.7) minutes. Aberration wounds required the least amount of time for haemostasis at 7.3 (±4.4) minutes. Cut wounds required a mean time of 8.5 (±2.9) minutes; puncture wounds required 9.0 (±3.1) minutes; gum bleeding required the longest time to achieve haemostasis with a mean of 12.7 (±5.6) minutes.

The use of this topical haemostatic patch has been shown to be beneficial in the treatment of external injuries in PwH, and provides a good treatment option in resource-constrained areas. A larger controlled study would be helpful to further investigate its efficacy and safety.

Adherence to treatment recommendations in patients with chronic disease is complex and is influenced by numerous factors. Haemophilia is a chronic disease with reported levels of adherence ranging from 17–82%.

Based on the theoretical foundation of the World Health Organization Multidimensional Adherence Model, the objective of this study was to identify the best combination of the variables infusion frequency, annualised bleed rate, age, distance to haemophilia treatment centre (HTC) and Haemophilia Joint Health Score (HJHS), to predict adherence to treatment recommendations in patients with haemophilia A and B on home infusion prophylaxis in Canada.

A one-year retrospective cohort study investigated adherence to treatment recommendations using two measures: 1) subjective report via home infusion diaries, and 2) objective report of inventory ordered from Canadian Blood Services. Stepwise regression was performed for both measures.

Eighty-seven patients with haemophilia A and B, median age 21 years, were included. Adherence for both measures was 81% and 93% respectively. The sample consisted largely of patients performing an infusion frequency of every other day (34%). Median scores on the HJHS was 10.5; annualised bleed rate was two. Distance to the HTC was 51km. Analysis of the objective measure weakly supported greater infusion frequency as a treatment-related factor for the prediction of lower adherence, however the strength of this relationship was not clinically relevant (R2=0.048). For the subjective measure, none of the explanatory variables were significant.

Adherence is a multifaceted construct. Despite the use of theory, most of the variance in adherence to treatment recommendations in this sample of patients with haemophilia remains unknown. Further research on other potential predictors of adherence, and possible variables and relationships within factors of the MAM is required.

Some studies suggest that people with haemophilia (PwH) who use prophylaxis value low frequency of clotting factor administration more than a lower risk of bleeding. However, more frequent infusions offer the potential of reducing joint disease and pain, which in turn may improve functioning and quality of life.

To explore the impact on health-related quality of life (HRQoL) aspects of haemophilia associated with adherence and annual infusion rate in the context of factors influencing treatment that are important to patients, including prophylaxis, chronic pain, concomitant conditions and hospital admission.

HRQoL was assessed in participants with severe haemophilia in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study who were using prophylaxis. Patients using on-demand treatment were excluded. This multivariate analysis examined the interaction between factors potentially influencing treatment and HRQoL, and minor and major bleeds.

From the total CHESS population (n=1,285), 338 (26%) participants provided responses for major and minor bleeds and target joints, and 145 (11%) provided EQ-5D-3L responses. Major and minor bleeds were associated with pain. Patients with severe chronic pain reported a substantial negative impact on HRQoL; but this was significantly improved by increases in the annual infusion rate. This was not apparent in participants with mild or moderate pain.

Increasing the frequency of prophylaxis infusions is associated with improved quality of life in PwH who have severe chronic pain. However, increasing the number of infusions per week in those with mild or moderate chronic pain with the intention of improving prophylactic effect may not have the same effect.

Patients with haemophilia who are not adequately treated experience a lifetime burden of joint complications and loss of functional ability due to repeated bleeding episodes caused by low levels of clotting factor VIII or IX in the blood. These complications can significantly impact day-to-day life, including active participation in school and academic study in children with haemophilia (CwH). Treatment with factor replacement therapy can help to prevent this, but access to factor has been challenging in low-resource settings such as Northeast India. This study shows the impact of factor replacement therapy on bleeding episodes, joint complications and school absence among CwH in this setting.

A retrospective observational study was undertaken to examine the impact of receiving regular factor replacement therapy (prophylaxis or on demand) on school absences among CwH registered with the haemophilia treatment centre at Assam Medical College and Hospital. Annual bleed rate (ABR), Haemophilia Joint Health Score (HJHS) and Functional Independence Score in Haemophilia (FISH) were also assessed.

Thirty-eight CwH were eligible for the study; 26 (68.4%) were on prophylaxis therapy and 12 (31.5%) received on-demand therapy. In the year before starting regular treatment, the mean ABR was 37.8 (+20.0), HJHS was 31.1 (+18.1) and mean FISH score was 21.1 (+4.2). At the end of the study period the mean (+SD) ABR in prophylactic therapy was significantly lower at 5.8 (+4.6) (p<0.001) and the HJHS was significantly lower at 4.7 (+4.6). FISH score significantly improved to 27.9 (+3.3) (p<0.001). Prophylaxis showed better (but not significant) results in comparison to on-demand therapy.

Treatment with factor replacement significantly reduces school absence in CwH and correlates strongly with joint health and functional improvement, with the effect slightly better with prophylaxis than on-demand therapy. Low dose prophylaxis is a good treatment option in low-resource settings, but improvements are also needed in rates of diagnosis.

People with haemophilia (PwH) experience recurrent bleeds in weight-bearing joints. Optimal management for people with severe haemophilia involves prophylaxis with factor VIII or IX, which helps to reduce the risk of bleeds and joint damage. However, this is expensive and frequently not an option in economically developing countries, where on-demand treatment is more commonly used as bleeding occurs. PwH with moderate and mild haemophilia are also treated on demand. Pain from bleeds and arthropathy is common in PwH; it is recognised as a burden that impairs quality of life and can be challenging to manage.

This study aims to establish greater understanding of the experience of pain in PwH in different countries, the factors that influence this, and how pain is currently managed.

PwH attending haemophilia treatment centres (HTCs) completed an anonymous questionnaire about their experience of pain and pain-relief within the previous 28 days (up to 10 PwH per participating HTC).

209 PwH from 20 HTCs in 11 countries participated in the study. The median age was 36 (range 8–84); 181 (86.6%) had haemophilia A, 25 (12.0%) haemophilia B, and three (1.4%) did not know; 148 (70.8%) had severe haemophilia, 28 (13.4%) moderate, and 31 (14.8%) mild. Twenty-eight (13.4%) had an inhibitor. The majority (n=121; 57.9%) were on prophylaxis; 61 (29.2%) were treated on demand; 20 (9.6%) used a combination; 7 (3.3%) did not know. 154 PwH (73.9%) experienced a total of 1,945 days of pain with severity on a visual analogue scale reported as 4.5. The most commonly reported sites of pain were joints and muscles. There was no significant difference in pain incidence between countries. Children aged less than 16 years reported the lowest amount of pain, with reported pain increasing with age in older respondents. Simple analgesia such as paracetamol was used but participants reported that it did not relieve pain. Alternative pain-relief strategies including rest, physiotherapy, walking aids, alcohol or marijuana were also used with varying effect.

Pain is common among PwH and increases with age. Age and developmentally appropriate pain assessment should be a part of routine haemophilia care.

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.

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.

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.

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.

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.

Around 90% of all undiagnosed people with haemophilia (PWH) live in developing countries. In Mali, in sub-Saharan Africa, nearly 90% of potential PWH are not identified. We initiated a two-year study involving an integrated programme of training and awareness-raising with the aim of improving diagnosis and access to care for PWH, based on partnership with those who regularly interact with them.

Our training programme focused on four regions of Mali and the district of Bamako, and included three types of health professionals from different districts and hospitals: medical doctor, nurse and laboratory technician. We also targeted traditional healers, who continue to be strongly involved in local healthcare, and provided training sessions for patients and their families on the symptoms, diagnosis, treatment and complications of haemophilia. A complementary programme of awareness-raising, including the national media, ran alongside the training sessions.

Overall, the programme involved 495 participants: 213 health care professionals, 24 patients, 79 parents of patients, 126 traditional healers, and 53 media workers. A direct result was development of collaboration between these groups in identifying haemophilia, and the transfer of four patients from a traditional healer's office to hospital for diagnosis and treatment. The number of diagnosed PWH increased from 42 in 2016 to 126 in 2017.

The integrated haemophilia educational programme, which took into account the nature of the local environment and involved all relevant stakeholders, showed that taking a collaborative approach is a successful strategy for improving diagnosis and care for PWH in Mali. This approach could be relevant in other developing countries.

A growing body of evidence supports the recommendation of both physiotherapy and physical activity in people with haemophilia. Physical benefits include increasing strength and flexibility and reducing the risks of osteoporosis, arthropathy, and intramuscular and joint bleeds; social benefits have also been observed. However, anecdotal evidence suggests that people with haemophilia may still be averse to engaging with physical activity due to fears of causing bleeding, joint pain and joint damage.

Qualitative interviews were conducted with young men with haemophilia treated at comprehensive care centres in London, to explore and identify the reasons behind risk-averse behaviours towards exercise and physical activity. The interview questions were designed to prompt discussion and capture opinions relating to participants’ physical activity and gym membership/use, and the degree to which their haemophilia impacts on both.

Ten participants were interviewed. Preferred activities were variable, with five participants describing themselves as very physically active, three moderately active, and two reporting little physical activity; four described themselves as ‘not gym-confident’. Seven participants described themselves as highly or moderately motivated to undertake physical activity, with motivations including weight loss and getting fit for summer holidays. However, there was some anxiety around weight-bearing exercise due to the fear of pain or injury. All participants had been exposed to personal trainers (PTs) and recognised the importance of being properly introduced to training equipment but felt that PTs were too expensive for them. The majority of participants reported sports-related injuries and self-perceived limitations on activity due to their personal/individual experience of living with haemophlia. Physiotherapists were often the first point of contact for advice and support on safe physical activity. All participants recognised the benefits of physical activity and had been encouraged in this by their physiotherapists.

Young men with haemophilia are keen to use the gym as part of their personal fitness regimens. The ongoing safety concerns of health care professionals warrants further research.

Recent treatment option advances in haemophilia care have contributed to a discourse of ‘normality’ around the condition, in which people with haemophilia (PwH) are increasingly expected to live ‘normal’ lives unburdened by their condition.

The aim of this article is to explore notions of ‘normality’ in the experience of haemophilia across generations. This is one of the main themes identified in a large-scale ethnographic study of the everyday life of PwH, a broad qualitative investigation of beliefs and experiences related to condition, treatment, and personal ways of managing the condition.

The study used ethnographic research methods. Five haemophilia experts helped frame the research design by contributing historical and disease area context prior to the initiation of field research. PwH were recruited through patient organisations in five European countries (Italy, Germany, Spain, UK and Ireland). During field research, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of PwH interactions with friends, family, and health care professionals (HCPs). They also conducted on-site observation at haemophilia treatment centres (HTCs) and HCP interviews. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenges mapping, and clustering exercises. This article explores findings related to the discourse of ‘normality’ and is thus focused on a subset of the data from the study.

Fifty-one PwH, aged 1.5 to 82 years, were interviewed and followed in their daily lives. Six treatment centres were visited, and 18 HCPs were interviewed. The study found that a discourse of present day ‘normality’, as compared to a difficult past, is ingrained in the haemophilia community. As a result, unlike most older PwH (40+), younger PwH (under 18) are not always taught to acknowledge the severity of their condition or how to sense bleeds (disease-related embodied knowledge), and risk unknowingly doing long-term damage to their bodies. Twenty-seven per cent (n=7/26) of younger PwH (children, teenagers) in the study were observed or described as engaging in high-risk behaviours in the short term indicating a lack understanding of long-term consequences.

These findings suggest that the discourse of ‘normality’ presents a number of challenges that need to be addressed, namely the potential for younger PwH to be unaware of bleeds and the general underreporting of haemophilia-related complications and limitations. One way forward in realising the full potential of advanced treatment could be to teach young PwH, through evidence-based initiatives, how to develop an embodied sense of their bleeds. Furthermore, if the current state of life with haemophilia is accepted as finally ‘normal’, then progress in further improving care may be stalled. It is important that remaining and new challenges are recognised in order for them to be acted upon.

With the major advances in treatment of haemophilia in recent decades, people with haemophilia (PwH) are more protected in their daily lives than ever before. However, recent studies point to persisting or increasing patient experience of uncertainty.

The aim of this article is to further investigate findings related to how PwH understand and cope with uncertainty around their protection in their everyday life, one of the main themes identified in a large-scale ethnographic study of the everyday life of PwH, including beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition.

The study used ethnographic research methods. Five haemophilia experts provided historical and disease area context prior to the initiation of field research. During field research, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and health care professionals (HCPs). Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related to uncertainty and thus focuses on a subset of the data from the study.

Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives, and 18 HCPs were interviewed. Fifty-two per cent (n=26/50) of PwH in the study experience difficulties translating clinical understanding of protection into specific activities in everyday life. Many have developed their own mental models and care adaptations to navigate treatment uncertainy: these seldom match the medical community's view. These mental models of protection among PwH can cause distress and influence behaviour in a way that can limit possibilities, and/or increase risk. There is also a prevalent tension in the strategies PwH have for managing their protection in terms of day-to-day vs. long-term ambitions.

These findings on PwH's experience of treatment uncertainty suggest a need to develop tools and communication materials to help PwH better understand the protection provided by their treatment regimen and what that means practically for everyday life.

Recent improvements in approaches to treatment have opened a window of opportunity to redefine and expand the goals of treatment in haemophilia This article explores treatment culture in light of these improvements and its potential impact on the range of possibilitis in the lived experience of haemophilia.

The aim of this article is to further investigate findings related to how health care professionals (HCPs) approach haemophilia treatment and care, one of the main themes identified in an ethnographic study of the everyday life of people with haemophilia (PwH). This large-scale study investigated PwH's beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition.

The study used ethnographic research methods. Five haemophilia experts helped frame the research design by providing historical and disease area context prior to the initation of field research. In the field, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and HCPs. Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related specifically to how HCPs approach haemophilia treatment and care, and is thus focused on a subset of the data from the study.

Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives. Eighteen HCPs from seven HTCs were interviewed, and on-site observation was undertaken at six of the HTCs. Most haematologists in the study ‘treated for stability’, rather than to guide PwH to overcome limitations. ‘Treating for stability’ here refers to an approach to haemophilia care that focuses on measuring success in terms of annual bleed rate, instilling a focus on mitigating risk, rather than an approach that allows PwH to overcome the limitations they face due to their condition. However, some haematologists had moved beyond treating for stability to instead treat for possibilities, enabling a better quality of life for PwH.

These results suggest that a culture of ‘treating for stability’ could be limiting progress in expanding the goals of treatment in haemophilia. Expanded metrics of success, more flexible approaches to treatment, and higher ambitions on behalf of PwH may be needed in treatment and care, in order for PwH to fully benefit from treatment advances and to increase their quality of life.