![VONVENDI [von Willebrand factor (Recombinant)]](/Content/images/structure/logo-vonvendi.svg){kind=logo}
n=number of bleeds assigned to this location. Not all bleeds are included in this chart.
may be all you need to treat and control bleeding episodes1
Dosing of VONVENDI® [von Willebrand factor (Recombinant)], with or without recombinant factor VIII (rFVIII), should be based on patient need as determined by monitoring levels and clinical judgment.
On-demand clinical trial design and results1,2
A phase 3 prospective, multicenter, open-label study assessed the efficacy and safety of on-demand use of VONVENDI [von Willebrand factor (Recombinant)], with and without rFVIII, in adult patients with severe von Willebrand disease (VWD) over a period of 12 months.
The first dose of VONVENDI was administered together with rFVIII. Bleeding episodes were to be treated with an initial infusion of 40 to 60 IU/kg VWF:RCo rVWF for minor to moderate bleeds and up to 80 IU/kg VWF:RCo for major bleeds.
A total of 193 bleeding episodes were reported in 22 subjects exposed to VONVENDI. Treatment success (primary efficacy endpoint) was defined as a mean efficacy rating score of less than 2.5 for all bleeding episodes in a subject treated with VONVENDI (with or without rFVIII) using a pre-specified, 4-point rating (Excellent=1, Good=2, Moderate=3, None=4). 100% of patients experienced treatment success (18/18; 95% CI, 81.5-100).
100% of patients experienced treatment success (18/18; 95% CI, 81.5-100)
Of the 22 participants, those with gastrointestinal bleeds (n=2) and those in whom the number of infusions to control a bleeding episode was estimated retrospectively (n=2) were excluded from the above primary analysis.1
100% bleed control with an "Excellent" or "Good" rating1
During the pivotal trial, all bleeding episodes (N=192) were controlled with an efficacy rating of excellent (96.9%) or good (3.1%).
Efficacy Rating Scale
The efficacy rating was assessed on a 4-point scale (Excellent=1, Good=2, Moderate=3, None=4), with the investigation comparing the prospectively estimated number of infusions to treat the bleeding episode to the actual number of infusions administered. The efficacy outcome included the number of treated bleeding episodes with a hemostatic efficacy rating of excellent or good.
Bleeds stopped with the estimated number of infusions or fewer.
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.
Patients treated with VONVENDI over a period of 12 months1,2
22 Participants
193 Bleeding episodes
For one bleeding episode, a participant received VWF/FVIII complex and that episode was therefore not included in the analysis.1
Bleeding episodes were treated with an initial infusion of 40 to 60 IU/kg of VONVENDI for minor to moderate bleeds and up to 80 IU/kg of VONVENDI for major bleeds. 2
The study protocol stipulated that the first dose of VONVENDI be administered together with 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
Bleed resolution by efficacy rating1
96% excellent 186 of 192 bleed resolutions were rated excellent
3.1% good 6 of 192 bleed resolutions were rated good
- All 192 bleed treatments in the study (95% CI:98.1%-100.0%) were rated as either “Excellent” (96.9%, 186/192) or “Good” (3.1%, 6/192)1
- Bleed control was defined as having an “Excellent” or “Good” rating
- Bleeding episodes were treated with an initial infusion of 40-60 IU/kg of VONVENDI for minor to moderate bleeds and up to 80 IU/kg of VONVENDI for major bleeds2
- A total of 8/125 (6.4%) of the adverse events (AEs) observed during the study were considered to have a causal relationship to VONVENDI and were subsequently resolved2
Ratings of “Excellent” or “Good” required that no additional von Willebrand factor (VWF) coagulation factor-containing product was needed. Sensitivity analysis of treatment success for bleeding episodes confirmed the primary analysis with 100% treatment success for each scenario.
1
infusion resolved the majority of bleeds, regardless of bleed location or severity1
The study protocol stipulated that the first dose of VONVENDI be administered together with 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
- 81.8% (157/192) of bleeds treated were resolved with 1 infusion (median 1, range 1-4)1
- Major/Severe bleeds were resolved with a median of 2 infusions (range 1-3)1
Number of infusions by severity of bleeding episode1
| Number of infusions | Minor n (%) (n=122) | Moderate n (%) (n=61) | Major/Severe n (%) (n=7) | Unknown n (%) (n=2) | All n (%) (n=192) |
|---|---|---|---|---|---|
| 1 | 113 (92.6%) | 41 (67.2%) | 1 (14.3%) | 2 (100%) | 157 (81.8%) |
| 2 | 8 (6.6%) | 13 (21.3%) | 4 (57.1%) | 0 (0.0) | 25 (13.0%) |
| 3 | 1 (0.8%) | 6 (9.8%) | 2 (28.6%) | 0 (0.0) | 9 (4.7%) |
| 4 | 0 (0.0) | 1 (1.6%) | 0 (0.0) | 0 (0.0) | 1 (0.5%) |
| Median | 1 | 1 | 2 | 1 | 1 |
| Range | 1-3 | 1-4 | 1-3 | 1-1 | 1-4 |
1
infusion treated and controlled mucosal, gastrointestinal, and joint bleeds in the pivotal study1
The study protocol stipulated that the first dose of VONVENDI be administered together with 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
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Reference: 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.
