Which medications in the drug class Plasma products are used in the treatment of Pediatric Von Willebrand Disease?

Updated: Feb 05, 2019
  • Author: Suchitra S Acharya, MD, MBBS; Chief Editor: Hassan M Yaish, MD  more...
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Answer

Plasma products

For patients with Type 1 von Willebrand disease who do not respond to desmopressin, and for individuals with the rare types 2A, 2B or Type 3 von Willebrand disease, plasma-derived factor VIII (FVIII) concentrates that contain von Willebrand factor in high molecular weight can be used. The product used must contain von Willebrand factor in the high–molecular weight form to be effective. However, most available FVIII concentrates do not contain sufficient von Willebrand factor to be used in von Willebrand disease. Cryoprecipitate contains multimeric von Willebrand factor; however, concerns about possible virus transmission have led many clinicians to choose FVIII products that contain multimeric von Willebrand factor and have undergone viral inactivation processes.

Only a minority of currently available FVIII products contain von Willebrand factor; the protein has been eliminated from the others. In general, the dosage of cryoprecipitate or von Willebrand factor to be used is calculated on the basis of ristocetin cofactor  units. Other blood products are rarely required for patients with von Willebrand disease. Rarely, platelet transfusion may benefit patients with type 3 von Willebrand disease or platelet-type von Willebrand disease who do not respond to von Willebrand factor–containing concentrates or cryoprecipitate.

Antihemophilic factor/von Willebrand Factor Complex, human (Alphanate, Humate-P, Wilate)

Some FVIII concentrates (eg, Humate-P, Alphanate, Wilate) also contain VWF in high molecular weight form. These concentrates are especially useful in types 2B and 3 vWD.

Alphanate is indicated to prevent excessive bleeding for surgical and invasive procedures in vWD in cases in which desmopressin is either ineffective or contraindicated. It is not indicated for patients with severe vWD (ie, Type 3) undergoing major surgery.

Humate-P is indicated for treatment and prevention of spontaneous and trauma-induced bleeding episodes for patients with mild-to-moderate or severe vWD. A recombinant VWF concentrate (Vonvendi) has been recently  licensed  for use in VWD for Type 2 and 3 disease

Aminocaproic acid (Amicar)

Inhibits fibrinolysis via inhibition of plasminogen activator substances and, to a lesser degree, through antiplasmin activity. Main problem is that the thrombi that forms during treatment are not lysed and effectiveness is uncertain. Has been used to prevent recurrence of subarachnoid hemorrhage (SAH). Useful in mucous membrane bleeding.


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