Von Willebrand disease medical therapy: Difference between revisions
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==Overview== | ==Overview== | ||
The mainstay of management 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 | 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]]. | ||
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 | |||
==Medical Therapy== | ==Medical Therapy== | ||
Pharmacologic medical therapies for VWD include [[Desmopressin|desmopressin (DDAVP]]), recombinant VWF , von Willebrand factor/factor VIII (vWF/FVIII) concentrates and [[Antifibrinolytic Agent|antifibrinolytic agents.]]<ref name="pmid17403090">{{cite journal| author=Borel-Derlon A, Federici AB, Roussel-Robert V, Goudemand J, Lee CA, Scharrer I et al.| title=Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients. | journal=J Thromb Haemost | year= 2007 | volume= 5 | issue= 6 | pages= 1115-24 | pmid=17403090 | doi=10.1111/j.1538-7836.2007.02562.x | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=17403090 }} </ref><ref name="pmid15086321">{{cite journal| author=Lethagen S, Carlson M, Hillarp A| title=A comparative in vitro evaluation of six von Willebrand factor concentrates. | journal=Haemophilia | year= 2004 | volume= 10 | issue= 3 | pages= 243-9 | pmid=15086321 | doi=10.1111/j.1365-2516.2004.00893.x | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=15086321 }} </ref><ref name="pmid23937614">{{cite journal| author=Leissinger C, Carcao M, Gill JC, Journeycake J, Singleton T, Valentino L| title=Desmopressin (DDAVP) in the management of patients with congenital bleeding disorders. | journal=Haemophilia | year= 2014 | volume= 20 | issue= 2 | pages= 158-67 | pmid=23937614 | doi=10.1111/hae.12254 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=23937614 }} </ref><ref name="pmid27913547">{{cite journal| author=Lavin M, O'Donnell JS| title=New treatment approaches to von Willebrand disease. | journal=Hematology Am Soc Hematol Educ Program | year= 2016 | volume= 2016 | issue= 1 | pages= 683-689 | pmid=27913547 | doi=10.1182/asheducation-2016.1.683 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=27913547 }} </ref><ref name="pmid23633542">{{cite journal| author=Castaman G, Goodeve A, Eikenboom J, European Group on von Willebrand Disease| title=Principles of care for the diagnosis and treatment of von Willebrand disease. | journal=Haematologica | year= 2013 | volume= 98 | issue= 5 | pages= 667-74 | pmid=23633542 | doi=10.3324/haematol.2012.077263 | pmc=3640108 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=23633542 }} </ref> | |||
'''Desmopressin''' | |||
* [[Desmopressin]] works by raising the patient's own [[Blood plasma|plasma]] levels of [[von Willebrand factor]] by inducing release of [[von Willebrand factor]] stored in the [[Weibel-Palade body|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 [[Subcutaneous|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 | |||
in | * 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 Agent|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:''' Preffered 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, | |||
'''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. | |||
* Preffered regimen (1) : Aminocaproic acid, 25 to 50 mg/kg PO, QID (maximum 5 g dose) | |||
* Preffered 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== | ==References== | ||
Revision as of 16:30, 4 September 2018
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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
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: Preffered 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,
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.
- Preffered regimen (1) : Aminocaproic acid, 25 to 50 mg/kg PO, QID (maximum 5 g dose)
- Preffered 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.