Indications
VONVENDI [von Willebrand factor (Recombinant)] is a recombinant von Willebrand factor (rVWF) indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for on-demand treatment and control of bleeding episodes; perioperative management of bleeding; routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe Type 3 von Willebrand disease receiving on-demand therapy.1
100% bleed control with an “Excellent” or “Good” rating1
96% Of all bleeds had an “Excellent” bleed resolution1
3.1% Of all bleeds had a “Good” bleed resolution1
aFor one bleeding episode, a participant received VWF/FVIII complex; this episode was not included in the analysis.
A prospective, multicenter, open-label study assessed the efficacy and safety of on-demand use of VONVENDI, with and without rFVIII, in adult patients with severe VWD. 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.1,2
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
Primary endpoint
Secondary endpoints included:
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.
(157/192) OF BLEEDS TREATED WERE RESOLVED WITH 1
INFUSION (MEDIAN 1, RANGE 1-4)1
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
| Number of infusions per bleed | 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 |
MUCOSAL: MOUTH AND ORAL CAVITY
Median number of infusions: 1 (range 1-4)
Rated 100% “Excellent” (n=26)
MUCOSAL: NASOPHARYNGEAL
Median number of infusions: 1 (range 1-2)
Rated 97.6% “Excellent” and 2.4% “Good” (n=42)
MUCOSAL: GENITAL TRACT FEMALE
Median number of infusions: 1 (range 1-2)
Rated 96.9% “Excellent” and 3.1% “Good” (n=32)
GASTROINTESTINAL
Median number of infusions: 1 (range 1-2)
Rated 83.3% “Excellent” and 16.7% “Good” (n=6)
MUSCULOSKELETAL: JOINT
Median number of infusions: 1 (range 1-3)
Rated 96.6% “Excellent” and 3.4% “Good” (n=59)
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
Do not use in patients who have had life-threatening hypersensitivity reactions to VONVENDI or its components (tri-sodium citrate-dihydrate, glycine, mannitol, trehalose-dihydrate, polysorbate 80, and hamster or mouse proteins).
Thromboembolic reactions, including disseminated intravascular coagulation, venous thrombosis, pulmonary embolism, myocardial infarction, and stroke can occur, particularly in patients with known risk factors for thrombosis, including low ADAMTS13 levels. Monitor for early signs and symptoms of thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis, and syncope, and institute prophylaxis measures against thromboembolism based on current recommendations.
In patients requiring frequent doses of VONVENDI in combination with recombinant factor VIII, monitor plasma levels for FVIII:C activity because sustained excessive factor VIII plasma levels can increase the risk of thromboembolic events.
One out of 100 subjects treated with VONVENDI in clinical trials developed proximal deep vein thrombosis in perioperative period after total hip replacement surgery.
Hypersensitivity reactions have occurred with VONVENDI. These reactions can include anaphylactic shock, generalized urticaria, angioedema, chest tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia, pruritus, restlessness, blurred vision, wheezing and/ or acute respiratory distress. Discontinue VONVENDI if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Inhibitors to VWF and/or factor VIII can occur. If the expected plasma levels of VWF activity (VWF:RCo) are not attained, perform an appropriate assay to determine if anti-VWF or antifactor VIII inhibitors are present. Consider other therapeutic options and direct the patient to a physician with experience in the care of either VWD or hemophilia A.
In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI therapy may not be effective and infusion of this protein may lead to severe hypersensitivity reactions. Since inhibitor antibodies can occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.
In clinical trials, the most common adverse reactions observed in ≥2% of subjects (n=100) were headache, vomiting, nausea, dizziness, arthralgia, joint injury, vertigo, ALT increased and generalized pruritus.
One subject treated with VONVENDI in perioperative setting developed deep vein thrombosis after total hip replacement surgery.
Please see VONVENDI full Prescribing Information.VONVENDI [von Willebrand factor (Recombinant)] is a recombinant von Willebrand factor (rVWF) indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for:
Do not use in patients who have had life-threatening hypersensitivity reactions to VONVENDI or its components (tri-sodium citrate-dihydrate, glycine, mannitol, trehalose-dihydrate, polysorbate 80, and hamster or mouse proteins).
Thromboembolic reactions, including disseminated intravascular coagulation, venous thrombosis, pulmonary embolism, myocardial infarction, and stroke can occur, particularly in patients with known risk factors for thrombosis, including low ADAMTS13 levels. Monitor for early signs and symptoms of thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis, and syncope, and institute prophylaxis measures against thromboembolism based on current recommendations.
In patients requiring frequent doses of VONVENDI in combination with recombinant factor VIII, monitor plasma levels for FVIII:C activity because sustained excessive factor VIII plasma levels can increase the risk of thromboembolic events.
One out of 100 subjects treated with VONVENDI in clinical trials developed proximal deep vein thrombosis in perioperative period after total hip replacement surgery.
Hypersensitivity reactions have occurred with VONVENDI. These reactions can include anaphylactic shock, generalized urticaria, angioedema, chest tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia, pruritus, restlessness, blurred vision, wheezing and/or acute respiratory distress. Discontinue VONVENDI if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Inhibitors to VWF and/or factor VIII can occur. If the expected plasma levels of VWF activity (VWF:RCo) are not attained, perform an appropriate assay to determine if anti-VWF or antifactor VIII inhibitors are present. Consider other therapeutic options and direct the patient to a physician with experience in the care of either VWD or hemophilia A.
In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI therapy may not be effective and infusion of this protein may lead to severe hypersensitivity reactions. Since inhibitor antibodies can occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.
In clinical trials, the most common adverse reactions observed in ≥2% of subjects (n=100) were headache, vomiting, nausea, dizziness, arthralgia, joint injury, vertigo, ALT increased and generalized pruritus.
One subject treated with VONVENDI in perioperative setting developed deep vein thrombosis after total hip replacement surgery.
Please see VONVENDI full Prescribing Information.![VONVENDI® [von Willebrand factor (Recombinant)] logo.](/dist/images/vonvendi-logo-white.svg){kind=logo}