New clinical data from Octapharma's haematology portfolio will be presented during the scientific program at the 65th American Society of Hematology (ASH) Meeting and Exposition, which is being held on December 9–12, 2023, in San Diego, California. New data from the prospective clinical trial using wilate® for prophylaxis in patients with von Willebrand disease (VWD) will be presented. Furthermore, the design of a new phase IV clinical trial of bone and joint health in people with haemophilia A will be presented, in addition to updates from research collaborations between Octapharma and academic institutions.
"We are excited to present new data at ASH 2023 highlighting our ongoing commitment to understand the mechanisms underlying bleeding disorders and to improve the standards of care for patients. We are delighted to support clinical and scientific research that advances the care of people with bleeding disorders." – Olaf Walter, Board Member and Head of International Business Units at Octapharma.
The WIL-31 study investigated the efficacy and safety of prophylaxis with wilate® in adults and children of any gender with all types of VWD. WIL-31 is the largest prospective prophylaxis study in VWD and the only one with a prospective on-demand run-in study as an intra-individual comparator. The results of the study showed that wilate® prophylaxis is highly effective at reducing the number of bleeds in children and adults and provides compelling evidence to support the use of regular prophylaxis in patients with severe VWD.
Data from sub-analyses of the study will be presented at ASH 2023, focusing on the effectiveness of prophylaxis in patients with different types of bleeding and in different subsets of patients. Women with bleeding disorders such as VWD are at an increased risk for heavy menstrual bleeding, but they are underdiagnosed and undertreated. A sub-analysis of WIL-31 will be presented, demonstrating that wilate® prophylaxis was effective in reducing the incidence of heavy menstrual bleeding. An additional sub-analysis focused on the efficacy and safety of wilate® prophylaxis in children and adolescents will also be presented.
People with haemophilia A often suffer from joint health issues and reduced bone strength. Prophylaxis with factor VIII replacement therapy has been associated with reductions in joint bleeding rates, joint deterioration, and degenerative joint disease. In contrast, there is a shortage of data on the long-term effects of prophylaxis with the non-factor therapy emicizumab. The new phase IV clinical trial PROVE aims to assess the long-term effects of prophylaxis with Nuwiq® or emicizumab on joint and bone health. The study design will be presented for the first time at ASH 2023.
In addition to clinical studies, Octapharma is proud to support scientific investigations with academic research partners that further our understanding of different aspects of bleeding disorders. Results from various avenues of scientific investigation will be presented at ASH 2023.
In PUPs with haemophilia A, the development of inhibitors to FVIII is the most significant treatment complication, and there is a need to better understand the mechanisms underlying inhibitor development. The NuProtect study with Nuwiq® was the largest prospective study of a single FVIII product in PUPs. Researchers from University College London, UK, will present new data from a sub-study of the NuProtect study, revealing differences in gene expression profiles between PUPs who developed inhibitors and those who did not.
In collaboration with the ETH Zurich and Balgrist Campus Zurich, Switzerland, the effect of FVIII on bone remodelling has been evaluated in vivo, with the results indicating that FVIII and/or von Willebrand can further inhibit the formation of osteoclasts, cells that play a key role in bone remodelling.
Platelets and endothelial cells play key roles in haemostasis. Research performed in collaborations with the ETH Zurich, Switzerland, and the University of Eastern Piedmont, Italy, revealed significant differences in the binding of different recombinant FVIII products to platelets and endothelial cells.
"We are excited to present additional data from the WIL-31 study, the largest prospective study in VWD, that further supports the use of prophylaxis for children, adolescents and women with von Willebrand disease. We are also proud to introduce the upcoming PROVE study, which will help us understand how best to protect people with haemophilia A from debilitating joint damage." – Larisa Belyanskaya, Head of IBU Haematology.
The posters will be presented at the following times:
Saturday December 9, 17:30-19:30 (PST)
Poster 1255: Design of a Comparative Study (PROVE) To Assess the Long-Term Effects of Prophylaxis With Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia A Patients. Presenting author: Robert Klamroth
Poster 1227: Transcriptomic profiling to understand inhibitor development in previously untreated patients with severe hemophilia A. Presenting author: Paul Batty
Poster 1244: Impact of recombinant factor VIII and platelet interaction on platelet functionality and hemophilia A treatment. Presenting author: Fabrizio A. Pennacchio
Monday December 11, 18:00-20:00 (PST)
Poster 3977: Factor VIII is an endothelial factor that promotes vessel stability. Presenting author: Antonia Follenzi
Poster 3979: Inter-donor variability in Osteoclastogenesis and responses to Osteoprotegerin (OPG), von Willebrand Factor (vWF), and Factor VIII (FVIII). Presenting author: Joyce Kimenai
Poster 3992: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Children and Adolescents with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F. Sidonio, Jr
Poster 3997: Efficacy of Regular Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Reducing Heavy Menstrual Bleeding in Females with von Willebrand Disease. Presenting author: Robert F. Sidonio, Jr
