LACHEN, Switzerland--()--Octapharma AG, a leading producer of human proteins for life saving therapies, proudly sponsored an octanate® symposium “Personalizing the Inhibitor Treatment and Prevention: New Pieces to an Old Puzzle” on July 10th, 2012 at the 30th World Federation of Haemophilia (WFH) Congress in Paris.
Over 520 attendees participated in the lunch symposium about inhibitor treatment and prevention in haemophilia A patients. Presented by renowned experts in the field, the symposium was chaired by Manuel Carcao, MD Doctor at the Hospital for Sick Children in Toronto, Canada, and co-chaired by Yves Gruel, Professor at the Trousseau University Hospital in Tours, France.
Haemophilia A is a bleeding disease characterized by coagulation factor VIII deficiency, an important protein factor in blood’s clotting process. Inhibitor development is today’s major complication in haemophilia A therapy with factor VIII (FVIII) concentrates, rendering the patient resistant to replacement treatment and thereby increasing the risk of unmanageable bleeds, arthropathy, and disability. Immune tolerance therapy with octanate® has shown to be highly successful in inhibitor elimination, even in patients with poor prognosis.
Wolfhart Kreuz, Associate Professor of the Goethe University Frankfurt, Germany, presented new successful interim data from an ongoing observational immune tolerance induction (ObsITI) study supporting the use of octanate® for ITI in the large cohort of hemophilia A patients with poor prognosis (n=48). In the study, inhibitor titers decreased below 0.6 BU in 79% of the patients after a median time of only four months. A significant decrease of the breakthrough bleeds and the use of bypassing agents were observed. After successful ITI with octanate® no relapses were monitored during the post-ITI observational period.
Dr Ana Pavlova, the Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Germany, presented the first analysis from the ObsITI study on the role of patient’s genetic parameters (F8 gene defect and polymorphism of immune response genes) as predictors of ITI outcome.
Dr Elena Santagostino, Hospital Maggiore Policlinico in Milan, Italy, presented the most recent prospective and retrospective clinical data obtained with different classes of FVIII concentrates in PUPs and compared them with the data for octanate®, with 5.1% inhibitor incidence, obtained in the ongoing prospective study with PUPs. Patients undergoing treatment with plasma-derived FVIII concentrates developed less inhibitors than those using currently marketed recombinant FVIII products.
From an economical and quality of life perspective it is more effective to prevent inhibitors instead of waiting for them to develop and then attempting ITI for eradication. In PUPs treated with octanate®, so far only 2 out of 39 (5.1%) developed clinically relevant inhibitors with a titre >5 BU over the course of the study. No inhibitors were observed in PUPs receiving prophylaxis with octanate®.
Dr Daniel Hart, The Royal London Hospital, UK, provided an overview of the challenges related to immunological responses. Computational biology may help to personalize inhibitor development and treatment by further investigating the anti-FVIII immune response as well as genetic variables.
Chairman Dr MD Manuel Carcao said “Inhibitor development is currently the most severe complication in haemophilia A patients. Eradicating inhibitors once they occur is critical. ITI with octanate® as done in the ObsITI study appears to be highly successful”.
About haemophilia A
Haemophila A is a factor VIII deficiency affecting coagulation process, preventing blood from clotting properly.
About octanate®
octanate® is a human plasma-derived FVIII concentrate of high-purity for intravenous administration. Coagulation FVIII present in octanate® is bound to its natural stabiliser, von Willebrand factor (VWF), in a VWF/FVIII ratio of approximately 0.4. Therefore, no additional stabilisers are required during manufacturing.
About the XXX Congress of the World Federation of Hemophilia
The XXX International Congress of the World Federation of Hemophilia provides the largest international scientific meeting place for the global bleeding disorders community and took place from July 8–12, 2012, in Paris, France.
About Octapharma
Octapharma, a Swiss-based independent fractionation specialist, is one of the largest plasma product manufacturers in the world. Our core business is the development, production and sale of premium human proteins from both human plasma and human cell-lines. Octapharma employs more than 4‘500 people in 28 countries. Patients in over 80 countries are treated with products in the following therapeutic areas:
- Haematology (coagulation disorders)
- Immunotherapy (immune disorders)
- Intensive Care and Emergency Medicine
Octapharma has dedicated resources to develop the first recombinant FVIII expressed in the human cell line. This unique approach is intended to reduce the immunogenic potential and sets Octapharma apart from other companies whose recombinant products are based on animal cells. Octapharma owns five state of the art production facilities in Austria, France, Germany, Sweden and Mexico. For more information visit www.octapharma.com