PARAMUS, N.J. (December 1, 2020) - Octapharma USA will present its most recent rare bleeding disorders research initiatives during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, a virtual medical congress to be held December 5 - 8.
Three scientific posters will be presented during the ASH meeting that focus on investigative treatment options utilizing NUWIQ®, Antihemophilic Factor (Recombinant) Lyophilized Powder for Solution for Intravenous Injection and WILATE®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection.
"Octapharma is committed to advancing clinical research that will help enhance the lives of people with bleeding disorders," said Octapharma USA President Flemming Nielsen. "We look forward to sharing the latest research developments with the medical community."
Ellis J. Neufeld, MD, PhD, of St. Jude Children's Research Hospital, Memphis, Tenn., will provide oral and poster abstracts for "Long-Term Prophylaxis with Simoctocog Alfa for the Management of Hemophilia A: Immunogenicity, Efficacy, and Safety Results from the NuProtect PUP Extension Study." The research is focused on evaluating the risk of factor VIII (FVIII) inhibitor development in pediatric patients during FVIII treatment. Prophylaxis with FVIII is the gold standard approach for management of hemophilia A, and long-term bleed prevention remains the key goal for patients and clinicians. The NuProtect extension study assessed the long-term immunogenicity, hemostatic efficacy and tolerability of NUWIQ® in 48 pediatric patients. No children with severe hemophilia A developed FVIII inhibitors during the study. Principal investigators: Ri Liesner, MD, Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children, London, United Kingdom, and Dr. Neufeld.
Another poster is entitled "Use of a Von Willebrand Factor/Coagulation Factor VIII Complex for Treatment of Refractory Inherited Platelet Disorders." Inherited platelet disorders are recognized as an important cause of mild to severe bleeding in both children and adults. Treatment of platelet disorders is mainly supportive. Normal hemostasis requires von Willebrand factor (VWF) and FVIII to support platelet adhesion and aggregation at sites of vascular injury. Four patients with qualitative platelet disorders, three with delta storage pool deficiencies and one with Bernard-Soulier syndrome achieved clinically relevant bleeding resolution with WILATE®. Notably, these patients were refractory to other therapies. Principal investigator: Amber Federizo, APRN, FNP-BC, Hemostasis and Thrombosis Center of Nevada, Las Vegas, Nev.
"Design of the Von Willebrand Factor in Pregnancy (VIP) Study" is a description of planned VWF in pregnancy research. This study will provide a better understanding of post-partum hemorrhage in women with von Willebrand disease (VWD) relative to VWD diagnosis, VWF levels, and other laboratory assessments of VWF associated parameters. The study evaluates the effectiveness of WILATE® in targeting VWF/FVIII during delivery and the immediate 72-hour postpartum period. Principal investigator: Jill M. Johnsen, MD, Bloodworks Research Institute and Department of Medicine, University of Washington, both in Seattle, Wash.
For more information on the ASH annual meeting, please visit hematology.org.