Poster Presentations - 2019 - Haemophilia
Predictive Value Of Von Willebrand Factor For Adverse Clinical Outcome In Hypertensive Patients With Mild-to-moderate Aortic Regurgitation
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Vonvendi Gains Expanded Approval In Von Willebrand Disease
The Food and Drug Administration (FDA) has expanded the approval of Vonvendi® (von Willebrand factor [recombinant]) to include routine prophylaxis to reduce the frequency of bleeding episodes in adults with von Willebrand Disease (VWD), including those with Type 1 and Type 2 disease, and on-demand and perioperative management of bleeding in pediatric patients with VWD.
Vonvendi is a recombinant von Willebrand factor previously only approved for on-demand and perioperative use in adults with VWD and routine prophylactic use in adults with severe Type 3 VWD receiving on-demand therapy.
The expanded approval was based on data from multiple clinical trials in patients with VWD, including a phase 3 pediatric study (Study 3; ClinicalTrials.Gov Identifier: NCT02932618), a phase 3 adult study (Study 5; ClinicalTrials.Gov Identifier: NCT02973087), and a phase 3b continuation trial in adults and children (Study 6; ClinicalTrials.Gov Identifier: NCT03879135).
Study 3 was an open-label trial that evaluated the efficacy of Vonvendi with or without Advate for on-demand treatment of non-surgical bleeding episodes in 18 pediatric patients (median age, 11). The primary endpoint was the number of patients with treatment success, defined as a mean efficacy rating score of less than 2.5 for all bleeding episodes using the pre-specified 4-point efficacy rating scale.
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Children and adults living with VWD face unique challenges, including bleeds that can disrupt daily activities like school, work or spending time with loved ones, often leading to physical and emotional burdens.
Findings showed there were 104 nonsurgical bleeding episodes treated with Vonvendi, with or without Advate. Among the 98 evaluable bleeding episodes, the hemostatic efficacy rating count was "Excellent" (1 rating score) in 99% of bleeding episodes and "Good" (2 rating score) in 1%.
Additionally, Study 3 evaluated the efficacy of Vonvendi with or without Advate for the management of surgical bleeding from elective and emergency surgeries in 4 pediatric patients aged 6 to 12 years with severe VWD. Four surgeries (3 elective and 1 emergency) were treated with Vonvendi.
Results showed that all 4 surgeries had overall and intraoperative hemostatic efficacy ratings of "Excellent" (95% CI, 39.8-100). The blood loss rating was also "Excellent" for all surgeries.
Study 5 was a single-arm, open-label trial that evaluated the efficacy and safety of prophylactic treatment with Vonvendi in reducing the frequency of bleeding episodes in adults diagnosed with VWD. Patients who received on-demand treatment only (n=12; 1 with Type 1, 1 with Type 2, and 10 with Type 3 VWD) were enrolled into 1 treatment arm while those who had received previous prophylactic treatment with plasma-derived von Willebrand factor (n=10; 1 with Type 1, 1 with Type 2, and 8 with Type 3 VWD) were enrolled into a switch arm.
Findings showed in the prior on-demand treatment arm, 91.7% achieved annualized bleeding rate (ABR) reduction success, defined as at least 25% reduction in the ABR for spontaneous bleeding episodes requiring factor treatment from the patient's historical to the on-study period. In the Switch arm, 90% of patients achieved preservation success, defined as achieving an on-study ABR for spontaneous bleeding episodes requiring factor treatment that was no greater than the patient's historical ABR for treated bleeding episodes.
The most common adverse reactions reported with Vonvendi were headache, vomiting, nausea, dizziness, and generalized pruritus.
"Children and adults living with VWD face unique challenges, including bleeds that can disrupt daily activities like school, work or spending time with loved ones, often leading to physical and emotional burdens," said Cheryl Schwartz, Senior Vice President, US Rare Disease Business Unit Lead and US Commercial Operations at Takeda. "We are proud to have supported adults with VWD since Vonvendi's initial approval in 2015 and will now be able to help even more patients manage their condition."
This article originally appeared on MPR
Von Willebrand Factor And Its Propeptide In Children With Diabetes. Relation Between Endothelial Dysfunction And Microalbuminuria
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