Progress in the treatment of bleeding disorders

Abstract

The availability of plasma-derived and recombinant coagulation factors has transformed the management of patients with bleeding disorders, such as hemophilia and von Willebrand disease (VWD). However, several important clinical challenges remain that have become the focus of current research in coagulation therapy. Two prospective, non-interventional studies (HyQoL-Europe and HyQoL-Canada) are evaluating the impact of major transitional life events, such as changes in social, work and living situations, on the quality of life of adolescents and young adults with hemophilia A who are treated with the recombinant factor VIII (rFVIII) concentrate Helixate ®. A better understanding of the impact of these transitional life events on quality of life may help to develop improved interventions and counseling techniques that minimize the negative effects of these events on patients with bleeding disorders. A new clinical development program has been launched to evaluate the safety and efficacy of the low-volume, highly active, plasma-derived von Willebrand factor (VWF)/FVIII concentrate Biostate ®. The program, known as SWIFT (Studies with von Willebrand factor/factor VIII) includes four clinical trials involving adult and pediatric patients with hemophilia A or VWD. Lastly, fusion of human recombinant albumin to recombinant coagulation factor IX (rFIX) has created a new fusion protein (rIX-FP) that retains the biological activity of rFIX and has a more favorable pharmacokinetic profile due to the longer half-life. The use of this novel fusion protein may offer several advantages to patients with hemophilia B: less frequent administration, prolonged protection from bleeding and improved compliance – increasing the likelihood of a positive clinical outcome. These examples of current research endeavors are intended to enhance the treatment experience as well as provide new and improved therapies for patients with bleeding disorders.