VONVENDI IN PEDIATRIC PATIENTS

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Learn about VONVENDI for on-demand and surgical use in children with von Willebrand disease (VWD).

VONVENDI successfully treated bleeding on-demand in children with VWD

VONVENDI, given with or without additional recombinant factor VIII (rFVIII), was studied in a clinical trial to test how it would work to control bleeding episodes in pediatric patients.

100%

of people (18 of 18) had their bleeds successfully treated with VONVENDI

99%

of 104 bleed treatments were rated as “Excellent,” with the remaining 1% rated as “Good”*

82%

of 104 bleeds were cleared up after just 1 infusion (94% with 1-2 infusions)

*Of 104 treated episodes, 98 had a known efficacy rating.
6 bleeds were missing details.

On-demand study description

The clinical trial included 18 pediatric patients with VWD. In pediatric patients, an initial dose of rFVIII was administered with VONVENDI based on the patient’s FVIII level. Investigators rated bleed control according to a scale of “Excellent=1,” “Good=2,” “Moderate=3,” or “None=4.” Treatment success was defined as an average efficacy rating score of less than 2.5 for all bleeding episodes.

In the pediatric clinical study, a median of 1 infusion of VONVENDI (with or without rFVIII) (range 1-9) treated mild-to-moderate bleeding episodes. Children who experienced a severe bleed were treated with an average of 2 infusions (ranges 1-3). In 2 cases, >5 infusions were needed to control bleeding.

Your doctor will decide if you may use VONVENDI by itself, or if you also require rFVIII along with VONVENDI, based on your clinical situation.

Study safety

122 adverse events occurred in the on-demand study, with 1 case of moderate nausea considered related to VONVENDI treatment.

100% of children with VWD (4 of 4) had their surgical bleeding successfully managed with VONVENDI

VONVENDI, given with or without additional recombinant factor VIII (rFVIII), was studied in a clinical trial to test how it would work to prevent excessive bleeding during and after surgery in pediatric patients.

100% of pediatric surgeries (4 of 4) were managed successfully with a rating of "Excellent."
The blood loss rating (actual vs predicted blood loss) was “Excellent” for all 4 surgeries

Surgical study description

The clinical trial included a total of 4 pediatric patients, who underwent a total of 4 surgeries.

In this trial, patients received VONVENDI before and after surgery as determined by the care team. rFVIII was given as needed.§ Investigators rated bleed control on a 4-point scale where “Excellent”=1, “Good”=2, “Moderate”=3, and “None”=4. A rating of "Excellent" or "Good" was required to declare the outcome a success.

§Your doctor will decide if you may use VONVENDI by itself, or if you also require rFVIII along with VONVENDI, based on your clinical situation.

Study safety

2/4 patients experienced a total of 3 adverse events, with 1 report of mild dizziness considered related to the trial surgery.

Learn more about VONVENDI's safety profile

VONVENDI's safety profile was established in 6 clinical trials across adult and pediatric patients.

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