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Treat bleeds as needed with on-demand treatment for VWD¹

Indications

VONVENDI [von Willebrand factor (recombinant)] is indicated in adult and pediatric patients with von Willebrand disease (VWD) for¹:

• On-demand treatment and control of bleeding episodes.
• Perioperative management of bleeding.

For adult patients only:

• Routine prophylaxis to reduce the frequency of bleeding episodes.

100% of adult patients using VONVENDI^® [von Willebrand factor (Recombinant)] experienced on-demand treatment success¹

• The primary efficacy assessment showed that the percentage of patients (n=18) with recombinant VWF treatment success was 100% (95% CI; 81.5 to 100)¹
• This assessment excluded patients with gastrointestinal bleeds (n=2) and patients for whom the number of infusions to control a bleeding episode was estimated retrospectively (n=2)¹

Over a period of 12 months, 22 adults with VWD and 192 bleeds were treated with VONVENDI with or without FVIII^1,2,a

*For one bleeding episode, a participant received VWF/FVIII complex; this episode was not included in the analysis.

Primary endpoint

• Number of study participants with treatment success for control of bleeding episodes¹
  • Treatment success was defined as a mean efficacy rating score of less than 2.5 for all bleeding episodes in a participant treated with VONVENDI¹

Secondary endpoints included:

• The number of treated bleeding episodes with an efficacy rating of “Excellent” or “Good”¹
• The number of infusions and number of units of VONVENDI administered with and without rFVIII per bleeding episode¹

The efficacy rating was assessed on a 4-point scale (Excellent=1, Good=2, Moderate=3, None=4), with the investigator comparing the prospectively estimated number of infusions needed to treat the bleeding episode to the actual number of infusions administered.¹

Excellent: Bleeds stopped with the estimated number of infusions or fewer.¹

Good: Minor bleeds stopped with only 1-2 more infusions than estimated. Major bleeds stopped with less than 1.5 times the estimated number of infusions.¹

A single infusion of VONVENDI resolved the majority of bleeds in adults

• Major/severe bleeds were resolved with a median of 2 infusions (range 1-3)²

The study protocol stipulated that the first dose of VONVENDI be administered together with recombinant factor VIII (rFVIII); however, according to dosing guidelines in the USPI, VONVENDI may be administered alone if an immediate rise in FVIII:C is not necessary, or if the baseline FVIII:C level is ≥40%.^1,2

1 INFUSION TREATED AND CONTROLLED MUCOSAL, GASTROINTESTINAL, AND JOINT BLEEDS IN THE TRIAL¹

n=number of bleeds assigned to this location.
Not all bleeds are included in this chart.

The study protocol stipulated that the first dose of VONVENDI be administered together with recombinant factor VIII (rFVIII); however, according to dosing guidelines, VONVENDI may be administered alone if an immediate rise in FVIII:C is not necessary, or if the baseline FVIII:C level is ≥40%.^1,2

Summary of on-demand safety information in adult patients ²

6.4% (8/125) of adverse events (AEs) observed during the trial were considered to have a causal relationship to VONVENDI; they subsequently resolved.

• 75% (6/8) of AEs in 4 subjects were not serious:
  • One patient experienced mild infusion-site paresthesia, moderate dysgeusia, and moderate tachycardia
  • One patient experienced mild electrocardiogram T wave inversion
  • One patient experienced mild generalized pruritus
  • One patient experienced mild hot flush

One patient experienced 2 simultaneous serious AEs (chest discomfort and increased heart rate); symptoms improved after 10 minutes of oxygen therapy with a full recovery within 3 hours.

References

1. VONVENDI [von Willebrand factor (Recombinant)] Prescribing Information.
2. Gill JC, Castaman G, Windyga J, et al. Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease. Blood. 2015;126(17):2038-2046.
3. Data on file, Takeda, Inc.