Long-Term Outcome after Joint Bleeds in Von Willebrand Disease Compared to Haemophilia A: A Post Hoc Analysis

Galen, Karin P M van; Timmer, Merel A.; Kleijn, P. de; Leebeek, Frank W.G.; Foppen, Wouter; Schutgens, Roger E.G.; Eikenboom, Jeroen; Meijer, Karina; Fijnvandraat, Karin; Gorkom, Britta Laros-van; Twisk, Jos; Mauser‐Bunschoten, Eveline P.; Fischer, Kathelijn

doi:10.1055/s-0038-1670704

Cited by 12 publications

(18 citation statements)

References 42 publications

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“…Although joint damage develops later in life in patients with VWD compared to those with haemophilia, prophylaxis may be equally as important for the reduction of joint haemorrhages and the subsequent development of arthropathy, particularly in patients with type 3 VWD; however, there are limited data reporting the existence and severity of arthropathy in VWD [12]. It is thought that joint bleeds occur in ~50% of patients with type 3 VWD and up to 10% in those with type 1 and type 2 VWD [13,14]. It is important to note that although FVIII levels are typically higher in patients with VWD than those with haemophilia, some patients with severe VWD have reduced FVIII levels, since VWF is a carrier for FVIII in circulation.…”

Section: The Role Of Prophylaxis In Hereditary Bleeding Disordersmentioning

confidence: 99%

Translating the success of prophylaxis in haemophilia to von Willebrand disease

Miesbach

Berntorp

2021

Thrombosis Research

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There is limited awareness of von Willebrand disease (VWD), leading to challenges in both diagnosis and defining the optimal treatment approach for these patients. Patients with VWD are typically treated ondemand, with short-term prophylaxis used during surgery. In contrast, early initiation, and long-term use of prophylaxis is the standard of care in patients with severe haemophilia and can be successfully used to prevent joint bleeding and reduce chronic arthropathy. Aim: To provide an understanding of the current evidence for the prophylactic treatment of patients with VWD and compare this to the management of patients with haemophilia. Methods: Review of published literature using a non-systematic search of PubMed and reference lists of sourced articles.Results: The successes seen with prophylaxis in haemophilia provide the rationale for long-term prophylaxis in patients with severe forms of VWD; preventing spontaneous, excessive and sometimes life-threatening bleeding, and reducing chronic joint disease. Currently, there are a few clinical trials assessing the long-term benefits of prophylaxis in VWD, and guidelines for the optimal prophylaxis treatment approach are lacking. Greater attempts to provide comprehensive, long-term care for patients with VWD are needed but still lacking within the community. This review highlights the success of prophylaxis in haemophilia and how this knowledge might be applied and translated to patients with VWD. Conclusions: Lessons can be learned from the use of prophylaxis in haemophilia and prophylaxis should be considered the standard of care for a subgroup of patients with severe VWD.

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Section: The Role Of Prophylaxis In Hereditary Bleeding Disordersmentioning

confidence: 99%

Translating the success of prophylaxis in haemophilia to von Willebrand disease

Miesbach

Berntorp

2021

Thrombosis Research

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“…Both are characterized by mucocutaneous bleeding. Type 3 VWD is the rarest and most severe form and causes severe mucocutaneous bleeding, as well as complications similar to those seen in moderate to severe haemophilia A, including hemarthrosis 5,6 . A study based on a survey of haematology departments in the United States, Canada and Europe found the incidence of ‘severe’ VWD in the United States to be 1.38 per million population 7…”

Section: Introductionmentioning

confidence: 99%

Occurrence rates of von Willebrand disease among people receiving care in specialized treatment centres in the United States

et al. 2021

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Introduction In the network of U.S. comprehensive haemophilia treatment centres (HTCs), von Willebrand disease (VWD) is the most common bleeding disorder other than haemophilia. Estimates of the size and characteristics of the VWD population receiving treatment are useful for healthcare planning. Aim Estimate the prevalence and incidence of VWD among males and females receiving care at U.S. HTCs (HTC‐treated prevalence and incidence). Methods During the period 2012–2019, de‐identified surveillance data were collected on all VWD patients who visited an HTC including year of birth, sex, race, Hispanic ethnicity, VWD type, and laboratory findings and used to calculate period HTC‐treated prevalence by VWD type and sex. Data from patients born 1995–1999 were used to estimate HTC‐treated incidence rates. Results During the period, 24,238 patients with a diagnosis of VWD attended HTCs; for 23,479 (96.9%), VWD type was reported or could be assigned. Age‐adjusted HTC‐treated prevalence was 8.6 cases/100,000 (7.2/100,000 for Type 1, 1.2/100,000 for Type 2 and 1.7/million for Type 3) and was twice as high in women as men (4.8 vs. 2.4 cases/100,000) for Type 1 and similar by sex for Type 2 and Type 3. HTC‐treated Type 1 incidence increased over the period, averaging nearly threefold higher for women than men (26.2 vs. 9.9/100,000 live births). Sex differences were less for Type 2 (2.2 vs. 1.4 cases/100,000 births) and slight in Type 3. Conclusion Prevalence and incidence of HTC‐treated VWD differ by sex and type and are likely strongly influenced by differences in rates of diagnosis.

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“…This was associated with more pain, functional limitations and less social participation compared to control VWD patients without joint bleeds. These patients had comparable outcomes to moderate severe haemophilia A patients (van Galen et al , ).…”

Section: Bleeding Phenotype Of Severe Vwdmentioning

confidence: 63%

How I manage severe von Willebrand disease

Leebeek¹,

Atiq²

2019

Br J Haematol

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Von Willebrand disease (VWD) is the most common inherited bleeding disorder. Most patients with mild and moderate VWD can be treated effectively with desmopressin. The management of severe VWD patients, mostly affected by type 2 and type 3 disease, can be challenging. In this article we review the current diagnosis and treatment of severe VWD patients. We will also discuss the management of severe VWD patients in specific situations, such as pregnancy, delivery, patients developing alloantibodies against von Willebrand factor and VWD patients with recurrent gastrointestinal bleeding. Moreover, we review emerging treatments that may be applied in future management of patients with severe VWD.

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Long-Term Outcome after Joint Bleeds in Von Willebrand Disease Compared to Haemophilia A: A Post Hoc Analysis

Cited by 12 publications

References 42 publications

Translating the success of prophylaxis in haemophilia to von Willebrand disease

Translating the success of prophylaxis in haemophilia to von Willebrand disease

Occurrence rates of von Willebrand disease among people receiving care in specialized treatment centres in the United States

How I manage severe von Willebrand disease

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