Prophylaxis in von Willebrand Disease: Coming of Age?

Although in most cases von Willebrand disease (VWD) is a mild disorder, a subgroup of patients experience frequent bleeding. In contrast to severe hemophilia in which prophylaxis is the accepted standard of care, this is less frequently used in VWD. Most type 1 VWD patients can be adequately managed with episodic desmopressin and tranexamic acid. In patients with more severe disease, especially those with type 3 VWD, joint bleeds, epistaxis, menorrhagia, and gastrointestinal bleeding are problematic and usually require treatment with von Willebrand factor/factor VIII (VWF/FVIII) concentrate. While in the past these patients were managed with on-demand VWF/FVIII concentrate, several recent reports have demonstrated the value of prophylactic treatment. Despite some uncertainties about the economic impact of treatment of severe VWD, prophylaxis with VWF concentrate should now be considered as the standard of care for the more severe end of the spectrum of affected individuals. The recent introduction of recombinant VWF concentrate is likely to improve the acceptability of prophylaxis in VWD.

[Haemophilia A, B or von Willebrand disease type 3. Census of patients in the eastern part of Germany]. / Patienten mit Hämophilie A, B oder von-Willebrand-Syndrom Typ 3. Systematische Erfassung im Osten Deutschlands.

UNLABELLED: Diagnostics and treatment of severe forms of haemorrhagic diatheses (HD), such as haemophilia A, B or type 3 von Willebrand disease (VWD) need high standards in haemophilia treatment centers (HTCs). Due to their generally low incidence, a close cooperation of haemophilia treaters is needed to optimize treatment strategies. For this purpose, the Kompetenznetz Hämorrhagische Diathesen Ost was founded. The first project was the conduction of a survey of epidemiological data of patients with HA, HB and type 3 VWD in all HTCs in the eastern part of Germany. METHOD: The study assessed the data regarding numbers of patients treated with HA, HB or type 3 VWD, disease severity, regime of coagulation factor replacement (CFR) and the frequency of factor VIII and IX inhibitors. RESULTS: Up to now, data of 838 patients from 27 HTCs were evaluated (5-151 per HTCs). Among the included patients, 201 were children or adolescents (<18 years). 81 children suffered from severe HA, 20 from severe HB and 10 from type 3 VWD. In 637 adults we found 246 with severe HA, 51 severe HB and 28 VWD of type 3. The most commonly used treatment of CFR in patients with severe disease was prophylactic modality. 90% of the children received prophylaxis, but also in 64% of the adults an intermittent or long-term secondary prophylaxis was the preferred treatment. At the time of survey conduction, a factor VIII or IX inhibitor was present in 1.9% of the children and in 1.6% of the adults with haemophilia. CONCLUSION: Our data allow an overview of the number of patients and treatment strategies in the eastern part of Germany.