Von Willebrand disease medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2] Associate Editor(s)-in-Chief: Prince Tano Djan, BSc, MBChB [3] Nazia Fuad M.D.
Overview
The mainstay of management of VWD is medical therapy. Medical therapy of von Willebrand's disease ( 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. Medical therapy depends on the type of von Willebrand's disease. Desmopressin is used for type 1 and 2 von Willebrand's disease. von Willebrand factor-factor VIII or von Willebrand factor concentrate is used in some of type 2 von Willebrand's disease and all of type 3 von Willebrand's disease. Alternate or additional therapy involves the use of tranexamic acid or aminocaproic acid.
Medical Therapy
Pharmacologic medical therapies for VWD include desmopressin (DDAVP), recombinant VWF , von Willebrand factor/factor VIII (vWF/FVIII) concentrates and antifibrinolytic agents.[1][2][3][4][5]
Desmopressin
- Desmopressin works by raising the patient's own plasma levels of von Willebrand factor by inducing release of von Willebrand factor stored in the Weibel-Palade bodies in the endothelial cells.
- It is used to treat patients with mild to moderately severe type 1 and some cases of type 2A vWD
- Desmopressin is usually not effective in type 2B, 2N, and 3 disease.
- Desmopressin is contraindicated in patients with type 2B disease.
- A test dose is given by nasal spray (1.5 mg/mL) or intravenously or subcutaneously (0.3 µg/kg).
- Fluids are to be restricted for 24 hours following the dose to avoid hyponatremia.
- Preferred regimen (1) IV: 0.3 mcg/kg in 50 mL saline over 20 minutes
- Preferred regimen (2) Nasal spray: weight >50 kg: 300 mcg (1 spray in each nostril); <50 kg: 150 mcg (1 spray in one nostril)
- May repeat dose after 12 hours and 24 hours
- DDAVP should not be used along antifibrinolytic agents such as tranexamic acid or aminocaproic acid.
VWF concentrates containing all VWF multimers
- Patients with type 3 VWD, more severe type 1, and those with types 2A, 2B, and 2M disease will need replacement therapy with a VWF-containing product,.
- Patients with more serious bleeding when other measures have failed.
- In those patients who require more prolonged treatment like post-surgery
- Major bleeding or surgery: Preferred regimen : 40 to 60 ristocetin cofactor units/kg followed by 20 to 40 ristocetin cofactor units/kg every 12 to 24 hours to keep VWF level 50 to 100 international units/dL for 7 to 14 days,
- Minor bleeding or surgery: Preferred regimen: 30 to 60 ristocetin cofactor units/kg followed by 20 to 40 ristocetin cofactor units/kg every 12 to 48 hours to keep VWF level >30 international units/dL for 3 to 5 days,
- Cryoprecipitate can also be used to treat vWD, since cryoprecipitate contains factor I, factor VIII, and vWF.
Antifibrinolytic agents: Aminocaproic acid,Tranexamic acid
- Used alone or in conjunction with other therapy except DDAVP.
- Useful for mucosal bleeding specially for dental procedures.
- Use after trial of DDAVP or other measures in patients with acquired von Willebrand syndrome (aVWS), particularly when associated with autoimmune diseases.
- May be used in conjunction with VWF concentrates to increase the half-life of VWF.
- Preferred regimen (1) : Aminocaproic acid, 25 to 50 mg/kg PO, QID (maximum 5 g dose)
- Preferred regimen (2) : Tranexamic acid, 10 mg/kg, I/V TDS.
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.
References
- ↑ 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.
- ↑ 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.
- ↑ 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.
- ↑ 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.
- ↑ 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.