Vonicog alfa

Treatment with recombinant von Willebrand factor stabilizes endogenous FVIII:C.

A single infusion of a new recombinant von Willebrand factor (rVWF) effectively controlled over 80% of bleeding episodes in patients with severe von Willebrand disease (VWD).

Out of a total of 192 bleeding episodes in 22 patients treated with the rVWF known as vonicog alfa, all bleeds were treated successfully, with an overall treatment success rate on a per subject level of 100%.

rVWF was initially given together with rFVIII and then subsequently alone as long as hemostatic FVIII:C levels were maintained.

rVWF is safe and effective in treatment bleeds in VWD patients and it stabilizes endogenous FVIII:C, which may eliminate the need for rFVIII after the first infusion.

One infusion is adequate to treat > 80% of bleeds, with a range of one to four infusions.

A median of two infusions were required to control major bleeds.

Infusions not associated with thrombotic events or severe allergic reactions.

No patients in the study developed anti-VWF binding or neutralizing antibodies to VWF.

Most bleeding episodes are mucosal, epistaxis, menorrhagia, mouth/oral cavity, followed by joint bleeds.

FVIII:C levels increase rapidly after rVWF, with hemostatic levels achieved within 6 hours and sustained through 72 hours post-infusion.

Desmopressin (1-desamino-8-D-arginine-vasopressin, DDAVP) is used to transiently correct FVIII/VWF levels by inducing the release of VWF from endothelial cellular compartments.

In patients with type 3 VWD and in severe forms of types 1 and 2 VWD, DDAVP may not be effective.

Lasts longer than currently available plasma-derived products.

Offers minimization of any potential risk for transmission of adventitious agents and other blood-borne pathogens.

Leave a Reply

Your email address will not be published. Required fields are marked *