Clinical management of woman with bleeding disorders: a survey among European haemophilia treatment centres

Introduction: The impact of bleeding for women with bleeding disorders (WBD) is of increasing focus and importance. Despite this, optimal management strategies are unclear and knowledge gaps persist. Aim: To examine practices and define research priorities on diagnosis and management of WBD in Europe. Methods: An electronic survey on clinical management of WBD was sent to 136 European haemophilia treatment centres (HTCs), including open questions on knowledge gaps and research priorities. Results: Fifty-nine HTCs from 12 Western (WE) and 13 Central/Eastern European (CEE) countries completed the survey. Less than half runs a joint clinic (24 HTCs, 42%). Most centres without a joint clinic have a named obstetrician (81%) and/or gynaecologist (75%) available for collaboration. Overall 18/54 (33%) European HTCs do not offer preimplantation genetic diagnosis. Third trimester amniocentesis to guide obstetric management is available 28/54 HTCs (52%), less frequent in CEE compared to WE countries (5/17 vs 23/37, P = .03). 53% of HTCs (28/53) reported that only 0%-25% of WBD seek medical advice for heavy menstrual bleeding (HMB). An algorithm managing acute HMB in WBD is lacking in 22/53 (42%) HTCs. The main reported knowledge and research gaps are lack of awareness & education on WBD among patients and caregivers, optimal diagnostic strategies and effective multidisciplinary management of pregnancy & HMB. Conclusion: Joint clinics, prenatal diagnostics and algorithms for managing acute HMB are lacking in many European HTCs. HMB may be an underestimated issue. This survey highlights the need to prioritize improvement of knowledge and patient care for WBD across Europe.</p

Examining international practices in the management of pregnant women with von Willebrand disease

Background: The management of pregnant women with von Willebrand disease (VWD) is complex as physiological pregnancy-induced increases in plasma von Willebrand factor (VWF) may be blunted or absent. Women with VWD experience a heightened risk of postpartum hemorrhage (PPH) and special consideration must be given regarding neuraxial anesthesia (NA) and the need for prophylaxis at time of delivery. These challenges are compounded by a lack of robust evidence to guide clinical decision-making. Objectives and methods: To determine the current international clinical practices in the management of pregnancy for women with VWD, the International Society on Thrombosis and Haemostasis (ISTH) conducted an international survey of health-care providers (HCP). Results: One hundred thirty-two respondents from 39 countries were included in the final analysis. Variations in clinical practice were identified in antenatal (monitoring of plasma VWF and ferritin levels), peripartum (optimal plasma VWF target at delivery) and postpartum management (definitions used for PPH and postpartum monitoring). A key area of divergence was suitability for NA for women with type 2 and type 3 VWD, with many respondents advising against the use of NA even with VWF supplementation (29% type 2 VWD, 37% type 3 VWD) but others advising use once plasma VWF activity was >50 IU/dL (57% type 2 VWD; 50% type 3 VWD). Conclusions: This survey highlighted areas of uncertainty surrounding common management issues for pregnant women with VWD. These data underscore the need for international collaborative research efforts focused on peripartum management to improve care for pregnant women with VWD.</div

European principles of care for women and girls with inherited bleeding disorders

Introduction: Despite increasing awareness of issues faced by women and girls with inherited BDs (WGBD), standards of care are lacking, with disparities in diagnosis and treatment for WGBD across Europe. We aimed to develop practical principles of care (PoC) to promote standardization of care for WGBD within European Haemophilia Treatment and Comprehensive Care Centres (HTC/CCCs). Methods: The co-creation process, supported by the European Association for Haemophilia and Allied Disorders, consisted of four multidisciplinary meetings with health care providers (HCPs) experienced in WGBD care, and European Haemophilia Consortium representatives, combined with broad patient and HCP consultations in the European haemophilia community. Relevant medical societies outside Europe were contacted for confirmation. Results: We developed ten PoC for WGBD, stressing the importance and benefits of a centralized, multidisciplinary, comprehensive, family-centred approach to support and manage WGBD during all life stages. These PoC emphasise the right to equitable access and quality of care for all people with BDs, irrespective of gender. Multiple medical societies outside Europe also confirmed their support for endorsement. Conclusions: Ten PoC for WGBD evolved from an iterative process among stakeholders, supported by relevant medical societies worldwide. These PoC can serve as a benchmark for diagnosis and comprehensive multidisciplinary management of WGBD, and improve awareness of their unique challenges. They offer a framework to guide HTC/CCCs in providing equitable care for all WGBD, both in their own services and in other healthcare settings. Implementation of these principles aims to positively impact the health, wellbeing and quality of life for WGBD.</p

von Willebrand disease: proposing definitions for future research

von Willebrand disease (VWD) is a common bleeding disorder, which affects 1 in 100 individuals based on laboratory testing and at least 1 in 1000 individuals based on presence of abnormal bleeding symptoms.1,2 VWD was first described almost 100 years ago, and since the initial report, major advances in both diagnostic testing and treatment options have improved outcomes for patients living with VWD; however, many patients still experience significant complications and barriers to treatment. An underlying problem is the lack of consistent unified definitions. In recent work developing evidence-based guidelines for VWD,3,4 it was noted that studies on VWD often used varying definitions. For example, studies of von Willebrand factor (VWF) concentrates did not have consistent definitions for major bleeding, studies on VWF prophylaxis did not use consistent definitions of what constituted a prophylaxis regimen, and studies on desmopressin did not use consistent definitions of desmopressin responsiveness. In addition, common bleeding conditions, such as heavy menstrual bleeding (HMB) and postpartum hemorrhage are variably defined. Such inconsistencies in describing study regimens and endpoints hinder the ability to compare study outcomes and to advance treatment of patients with VWD. We propose definitions for future use in VWD research to facilitate comparison of treatment options. These definitions are based on the most common usage in the literature and endeavor to encompass the most common situations in VWD. The proposed definitions were derived from existing literature and discussed at the first in-person meetings of the guideline panels. Group members made amendments, and the consensus document was circulated to the group. All authors approved the final document