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.