Von Willebrand disease medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Prince Tano Djan, BSc, MBChB [2]
Overview
Medical treatment of vWD involves normalizing the von Willebrand factor and factor VIII levels. Endogenous factor levels can be increased by the use of desmopressin or by infusing exogenous coagulation factors example high-purity or low-purity von Willebrand factor concentrate.[1][2]
Pharmacologic medical therapy is recommended in vWD patients.[3][4][5] Medical therapy depends on the type of vWD. Desmopressin is used for type 1 and 2 vWD. VWF-Factor VIII or vWF concentrate is used in some of type 2 vWD and all of type 2 vWD. Alternate or additional therapy involves the use of tranexamic acid, IL-11 [6][7]
or aminocaproic acid.
Medical Therapy
Medical treatment of vWD involves normalizing the von Willebrand factor and factor VIII levels. Endogenous factor levels can be increased by the use of desmopressin or by infusing exogenous coagulation factors example high-purity or low-purity von Willebrand factor concentrate.[1][2]
Pharmacologic medical therapy is recommended in vWD patients as shown below:[3][4][5]
| Type | Treatment | Additional/Alternative treatement |
|---|---|---|
| Low vWF | Desmopressin administered intravenously 0.3μg per kilogram body weight,
intranasally 300μg (150μg per nostril); in patients with body weight <50Kg, only one dose of 150μg or subcutaneously 0.3μg/kilogram |
Tranexamic acid 1g 3 to 4 times daily |
| 1 | Demospressin at same dose as above | Tranexamic acid 1g 3 to 4 times daily |
| 2 | Demospressin at same dose as above or vWF-Factor VIII or vWF concentrate | Tranexamic acid 1g 3 to 4 times daily |
| 3 | vWF-Factor VIII or vWF concentrate | Tranexamic acid 1g 3 to 4 times daily |
Desmopressin is contraindicated in patients with type 2B disease.
For women with heavy menstrual bleeding, the combined oral contraceptive pill may be effective in reducing bleeding or in reducing the length or frequency of periods. Prophylactic treatment is sometimes given for patients with vWD who are scheduled for surgery. They can be treated with human derived medium purity factor VIII concentrates complexed to vWF (antihemophilic factor, more commonly known as Humate-P) Mild cases of vWD can be trialled on desmopressin (1-desamino-8-D-arginine vasopressin, DDAVP) (desmopressin acetate, Stimate), which works by raising the patient's own plasma levels of vWF by inducing release of vWF stored in the Weibel-Palade bodies in the endothelial cells.
Daily subcutaneous administration of interleukin-11 have been used with success as an alternative modality which increases von Willebrand factor and factor VIII levels by a factor of 1.3 to 2. It is presumabed to increase von Willebrand factor messenger RNA levels in patients with type 1 von Willebrand’s disease that is unresponsive to treatment with desmopressin.[6] Interleukin-11 reduced the severity of bleeding in women with menorrhagia.[7]
References
- ↑ 1.0 1.1 Borel-Derlon A, Federici AB, Roussel-Robert V, Goudemand J, Lee CA, Scharrer I; et al. (2007). "Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients". J Thromb Haemost. 5 (6): 1115–24. doi:10.1111/j.1538-7836.2007.02562.x. PMID 17403090.
- ↑ 2.0 2.1 Lethagen S, Carlson M, Hillarp A (2004). "A comparative in vitro evaluation of six von Willebrand factor concentrates". Haemophilia. 10 (3): 243–9. doi:10.1111/j.1365-2516.2004.00893.x. PMID 15086321.
- ↑ 3.0 3.1 Leissinger C, Carcao M, Gill JC, Journeycake J, Singleton T, Valentino L (2014). "Desmopressin (DDAVP) in the management of patients with congenital bleeding disorders". Haemophilia. 20 (2): 158–67. doi:10.1111/hae.12254. PMID 23937614.
- ↑ 4.0 4.1 Lavin M, O'Donnell JS (2016). "New treatment approaches to von Willebrand disease". Hematology Am Soc Hematol Educ Program. 2016 (1): 683–689. doi:10.1182/asheducation-2016.1.683. PMID 27913547.
- ↑ 5.0 5.1 Castaman G, Goodeve A, Eikenboom J, European Group on von Willebrand Disease (2013). "Principles of care for the diagnosis and treatment of von Willebrand disease". Haematologica. 98 (5): 667–74. doi:10.3324/haematol.2012.077263. PMC 3640108. PMID 23633542.
- ↑ 6.0 6.1 Ragni MV, Novelli EM, Murshed A, Merricks EP, Kloos MT, Nichols TC (2013). "Phase II prospective open-label trial of recombinant interleukin-11 in desmopressin-unresponsive von Willebrand disease and mild or moderate haemophilia A." Thromb Haemost. 109 (2): 248–54. doi:10.1160/TH12-06-0447. PMC 3689588. PMID 23238591.
- ↑ 7.0 7.1 Ragni MV, Jankowitz RC, Jaworski K, Merricks EP, Kloos MT, Nichols TC (2011). "Phase II prospective open-label trial of recombinant interleukin-11 in women with mild von Willebrand disease and refractory menorrhagia". Thromb Haemost. 106 (4): 641–5. doi:10.1160/TH11-04-0274. PMC 3947632. PMID 21833452.