Octapharma AG is leading an international initiative focused on confronting the major risk associated with hemophilia A therapy - anti-factor VIII (FVIII) antibodies, also known as inhibitors. This initiative, combined with Octapharma's efforts to pursue the first recombinant FVIII therapy produced from a human cell line, could dramatically impact the treatment of an estimated one in every 5,000 to 10,000 men born with hemophilia A worldwide. Globally, 75% of the hemophilia cases go undiagnosed or untreated.
Octapharma AG, the third largest plasma products manufacturer in the world, recently brought together many of the most respected blood coagulation disorder researchers for two symposia on this important issue during the International Society on Thrombosis and Haemostasis (ISTH) Biennial Congress in Boston. Octapharma was one of only five Platinum Sponsors at the ISTH Congress, which attracted approximately 7,500 representatives of the medical and research community as well as many patients.
Clinical experts noted during the symposia that up to 40 percent of previously untreated patients (PUPs) with hemophilia A develop the most serious clinical complication of FVIII replacement therapy - inhibitory antibodies - which can result in uncontrolled hemorrhage, increased hospitalizations and joint damage, resulting in increased morbidity and mortality.
"We brought the international community together to confront this issue because our worldwide commitment to hemophilia A patients dates back to Octapharma's formation 25 years ago," said Octapharma AG Vice Chairman Kim Bjornstrup. "Our first therapies were developed for the hemophilia community and today most hemophilia patient advocates say that inhibitor development is the greatest obstacle to effective treatment. Octapharma is focused on introducing innovative development strategies that will help improve patient quality of life by finding an effective treatment and developing products to overcome this obstacle."
The Octapharma-sponsored symposium "Prevention and Eradication of FVIII Inhibitors: Bridging Lab and Field Research" was chaired by David Lillicrap, M.D., Professor of Pathology and Molecular Medicine at Queen's University in Ontario, Canada, and Georges E. Rivard, M.D., Professor of Pediatrics at Universite de Montreal. The symposium provided an opportunity for discussing recent data supporting the use of von Willebrand factor (VWF)/FVIII concentrates for Immune Tolerance Induction (ITI) in hemophilia A patients with poor prognosis for a successful ITI outcome.
Presenters reviewed a balanced mix of clinical, preclinical and basic data on the role of the presence of VWF in FVIII concentrates and the clinical experience on prevention and eradication of FVIII inhibitors. In addition, the most recent prospective and retrospective clinical data obtained with different classes of FVIII concentrates used for ITI treatment were presented along with an update on the progress of the ongoing prospective study designed to further investigate the risk of FVIII inhibitor development in previously untreated patients with hemophilia.
"A growing body of clinical experience suggests that VWF-containing pdFVIII concentrates increase the likelihood of successful tolerization, particularly in patients with poor prognostic factors," said symposium presenter Wolfhart Kreuz, M.D. of the Hemophilia Comprehensive Care Centre at Johann Wolfgang Goethe University Hospital in Frankfurt, Germany. "Successful ITI leads to normalization of the FVIII half-life, allows fully effective on-demand replacement therapy and prophylaxis, with consequent improvement in the patient's quality of life and a marked reduction in the cost of treatment."