Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies
This manuscript summarizes the current status of prophylaxis and novel potential therapies to prevent bleeding in patients with von Willebrand disease (VWD). VWD is the most common inherited bleeding disorder, which is associated mainly with mucocutaneous bleeding and bleeding during surgical and de...
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| Format: | Article |
| Language: | English |
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Elsevier
2024-11-01
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| Series: | Research and Practice in Thrombosis and Haemostasis |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2475037924002942 |
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| author | Calvin B. van Kwawegen Frank W.G. Leebeek |
| author_facet | Calvin B. van Kwawegen Frank W.G. Leebeek |
| author_sort | Calvin B. van Kwawegen |
| collection | DOAJ |
| description | This manuscript summarizes the current status of prophylaxis and novel potential therapies to prevent bleeding in patients with von Willebrand disease (VWD). VWD is the most common inherited bleeding disorder, which is associated mainly with mucocutaneous bleeding and bleeding during surgical and dental interventions. More severely affected VWD patients, mostly those with type 2 and type 3, can also suffer from joint, muscle, and gastrointestinal bleeds. Most patients with mild and moderate VWD are treated with desmopressin. The majority of patients with type 2 and 3 are treated with von Willebrand factor concentrates, with or without factor VIII. These patients suffer from severe and frequent bleeds and may require regular infusions of von Willebrand factor concentrate to prevent bleeding, so-called prophylaxis, 1 to 3 times per week. In this article, we review the current status of prophylaxis in VWD. We will also discuss emerging treatments that may be used as long-term prophylaxis in patients with severe VWD. We include relevant new data on this topic that were presented during the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress. |
| format | Article |
| id | doaj-art-50afd696152e4585a4f8145c37f6ebdf |
| institution | OA Journals |
| issn | 2475-0379 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Research and Practice in Thrombosis and Haemostasis |
| spelling | doaj-art-50afd696152e4585a4f8145c37f6ebdf2025-08-20T01:56:57ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792024-11-018810259910.1016/j.rpth.2024.102599Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapiesCalvin B. van Kwawegen0Frank W.G. Leebeek1Department of Hematology, Erasmus University Medical Center, Rotterdam, The NetherlandsCorrespondence Frank W. G. Leebeek, Department of Hematology, Erasmus University Medical Center, PO Box 2040, 3000 CA Rotterdam, The Netherlands.; Department of Hematology, Erasmus University Medical Center, Rotterdam, The NetherlandsThis manuscript summarizes the current status of prophylaxis and novel potential therapies to prevent bleeding in patients with von Willebrand disease (VWD). VWD is the most common inherited bleeding disorder, which is associated mainly with mucocutaneous bleeding and bleeding during surgical and dental interventions. More severely affected VWD patients, mostly those with type 2 and type 3, can also suffer from joint, muscle, and gastrointestinal bleeds. Most patients with mild and moderate VWD are treated with desmopressin. The majority of patients with type 2 and 3 are treated with von Willebrand factor concentrates, with or without factor VIII. These patients suffer from severe and frequent bleeds and may require regular infusions of von Willebrand factor concentrate to prevent bleeding, so-called prophylaxis, 1 to 3 times per week. In this article, we review the current status of prophylaxis in VWD. We will also discuss emerging treatments that may be used as long-term prophylaxis in patients with severe VWD. We include relevant new data on this topic that were presented during the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress.http://www.sciencedirect.com/science/article/pii/S2475037924002942bleedingnonfactor therapiesprophylaxistreatment strategyvon Willebrand disease |
| spellingShingle | Calvin B. van Kwawegen Frank W.G. Leebeek Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies Research and Practice in Thrombosis and Haemostasis bleeding nonfactor therapies prophylaxis treatment strategy von Willebrand disease |
| title | Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies |
| title_full | Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies |
| title_fullStr | Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies |
| title_full_unstemmed | Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies |
| title_short | Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies |
| title_sort | prophylaxis in von willebrand disease with von willebrand factor concentrate and nonfactor therapies |
| topic | bleeding nonfactor therapies prophylaxis treatment strategy von Willebrand disease |
| url | http://www.sciencedirect.com/science/article/pii/S2475037924002942 |
| work_keys_str_mv | AT calvinbvankwawegen prophylaxisinvonwillebranddiseasewithvonwillebrandfactorconcentrateandnonfactortherapies AT frankwgleebeek prophylaxisinvonwillebranddiseasewithvonwillebrandfactorconcentrateandnonfactortherapies |