The role of the specialist nurse in comprehensive care for bleeding disorders in Europe: An integrative review.

INTRODUCTION: Managing bleeding disorders (BDs) is complex, requiring a comprehensive approach coordinated by a multidisciplinary team (MDT). Haemophilia nurses (HNs) play a central role in the MDT, frequently coordinating care. As novel treatments bring change to the treatment landscape, ongoing education and development is key. However, understanding of the roles and tasks of HNs is lacking. AIM: The EAHAD Nurses Committee sought to identify and describe the roles and tasks of the European HN. METHODS: A five-step integrative review was undertaken, including problem identification, literature search, data evaluation, data synthesis and presentation. Relevant literature published from 2000 to 2022 was identified through database, hand and ancestry searching. Data were captured using extraction forms and thematically analysed. RESULTS: Seven hundred and seventy-seven articles were identified; 43 were included. Five main roles were identified, with varied and overlapping associated tasks: Educator, Coordinator, Supporter, Treater and Researcher. Tasks related to education, coordination and support were most frequently described. Patient education was often 'nurse-led', though education and coordination roles concerned both patients and health care practitioners (HCPs), within and beyond the MDT. The HN coordinates care and facilitates communication. Long-term patient care relationships place HNs in a unique position to provide support. Guidelines for HN core competencies have been developed in some countries, but autonomy and practice vary. CONCLUSION: As the treatment landscape changes, all five main HN roles will be impacted. Despite national variations, this review provides a baseline to anticipate educational needs to enable HNs to continue to fulfil their role.

Peri-operative desmopressin combined with pharmacokinetic-guided factor VIII concentrate in non-severe haemophilia A patients.

INTRODUCTION: Non-severe haemophilia A patient can be treated with desmopressin or factor VIII (FVIII) concentrate. Combining both may reduce factor consumption, but its feasibility and safety has never been investigated. AIM: We assessed the feasibility and safety of combination treatment in nonsevere haemophilia A patients. METHODS: Non-severe, desmopressin responsive, haemophilia A patients were included in one of two studies investigating peri-operative combination treatment. In the single-arm DAVID study intravenous desmopressin (0.3 µg/kg) once-a-day was, after sampling, immediately followed by PK-guided FVIII concentrate, for maximally three consecutive days. The Little DAVID study was a randomized trial in patients undergoing a minor medical procedure, whom received either PK-guided combination treatment (intervention arm) or PK-guided FVIII concentrate only (standard arm) up to 2 days. Dose predictions were considered accurate if the absolute difference between predicted and measured FVIII:C was ≤0.2 IU/mL. RESULTS: In total 32 patients (33 procedures) were included. In the DAVID study (n = 21), of the FVIII:C trough levels 73.7% (14/19) were predicted accurately on day 1 (D1), 76.5% (13/17) on D2. On D0, 61.9% (13/21) of peak FVIII:C levels predictions were accurate. In the Little DAVID study (n = 12), on D0 83.3% (5/6) FVIII:C peak levels for both study arms were predicted accurately. Combination treatment reduced preoperative FVIII concentrate use by 47% versus FVIII monotherapy. Desmopressin side effects were mild and transient. Two bleeds occurred, both despite FVIII:C > 1.00 IU/mL. CONCLUSION: Peri-operative combination treatment with desmopressin and PK-guided FVIII concentrate dosing in nonsevere haemophilia A is feasible, safe and reduces FVIII consumption.

Prevalence, burden and treatment effects of vaginal bleeding in women with (suspected) congenital platelet disorders throughout life: a cross-sectional study.

Congenital platelet disorders (CPDs) are rare bleeding disorders that are associated with mucocutaneous bleeds. However, data on vaginal bleeding in women with CPDs are scarce. A set of generic and bleeding-specific questionnaires were used to evaluate the prevalence of vaginal bleeding, its impact on quality of life (QoL) and sexual functioning and the consequences for pregnancy, miscarriage and delivery in a cohort of women who were referred for diagnostic evaluation for CPDs. A total of 78 women included in the study were either diagnosed with a CPD (n = 35) or were clinically suspected of a CPD (n = 43). Heavy menstrual bleeding (HMB) was reported by a large proportion of women, which mainly started at menarche. In all, 76% of women received any kind of HMB treatment, often leading to surgical prodecures. HMB was shown to have a high impact on QoL, which improved upon treatment. Even though women reported that vaginal bleeding affects sexuality, this topic is not frequently discussed with physicians. Heavy blood loss frequently occurred after miscarriage/delivery, often requiring treatment. Women with (suspected) CPDs frequently encounter HMB, negatively impacting daily life and sexual functioning. Together with peripartum bleeding, these data highlight the burden of vaginal bleeding in CPDs and importance of adequate treatment.

The impact of psychosocial determinants on caregivers' burden of children with haemophilia (results of the BBC study).

INTRODUCTION: Caring for a child with haemophilia is burdensome and impacting on caregivers' emotional and financial status. This paper assesses the impact of psychosocial determinants on caregivers' burden across European countries. METHODS: This non-interventional study enrolled caregiver/child dyads at haemophilia treatment centres (HTCs) using the "HEMOphilia associated CAregiver Burden scale" (HEMOCAB). Socio-demographic characteristics and clinical data were collected. RESULTS: A total of 144 dyads from Germany (n = 19), Italy (n = 19), Netherlands (n = 19), Turkey (n = 20), Sweden (n = 21), UK (n = 21) and Poland (n = 25) participated. Caregivers' mean age was 39.84 ± 7 (range 24-57); 81.3% were mothers, married (80.4%), living with a partner (86.6%), had a college/university degree (66.5%) and worked (74.2%). Around two thirds of caregivers (66.2%) reported that haemophilia affected their life; 26.8% reported an economic impact; 57.6% reported their child cannot do certain things because of his condition. Caregivers lost an average of 8.35 ± 14.5 days due to haemophilia. The highest burden was reported in the HEMOCAB domains "Perception of Child" (37.9 ± 24.7), "Emotional Stress" (37.4 ± 22.6) and "Medical Management" (33.1 ± 22.8). Significantly, higher burden was found in caregivers who reported that haemophilia "affects their life" (P < 0.0001), "has an economic impact" (P < 0.0001), "their child cannot do certain things" (P < 0.0001), "they spent ≥5 h/mo infusing" (P < 0.003) and "they needed ≥3 h/mo to reach the HTC" (P < 0.0001). CONCLUSION: This "snapshot" analysis of burden related to caring for a child with haemophilia across Europe revealed the greatest burdens are economic, including days lost from work, and things that a child cannot do, impacting on both child and caregiver.

The burden of bleeds and other clinical determinants on caregivers of children with haemophilia (the BBC Study).

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

The first Team Haemophilia Education meeting, 2015, Amsterdam, The Netherlands.

Haemophilia remains a complex disorder to diagnose and manage, requiring close cooperation between multidisciplinary healthcare professionals. There are still many unmet challenges in haemophilia care. The first Team Haemophilia Education (THE) meeting, held on 7-8 May 2015 in Amsterdam, The Netherlands, aimed to promote the optimal care of haemophilia patients through education of the multidisciplinary treatment team. This was achieved by reviewing the latest developments in haemophilia management, considering how these can be implemented in the clinic to improve patient care and providing a platform for networking and debate for all haemophilia treatment team members. Haemophilia treatment centres from several countries were asked to complete a premeeting online questionnaire to establish the biggest challenges that they face when managing patients. The concerns expressed were used to develop the agenda, which comprised a combination of formal presentations, case studies and informal workshops covering such topics as pharmacokinetics, laboratory assays and tailoring of treatment to individual patients. This report is a summary of the key developments in haemophilia care presented by various investigators and healthcare professionals at THE meeting 2015.