Four research updates for World Hemophilia Day
Today marks World Hemophilia Day, intended to increase awareness of hemophilia and other bleeding disorders.
In conjunction with the observance — held annually on April 17 — HemOnc Today presents four updates on research and treatment advances in hemophilia.
• The rate of thrombotic events among patients treated with coagulation factor VIIa recombinant (NovoSeven RT, NovoNordisk) within its licensed indications continues to be low and has remained stable in postapproval surveillance. Read more.
• Patients with severe hemophilia A achieved factor VIII activity levels sufficient to reduce or prevent spontaneous hemorrhage after a single IV infusion of AAV8-HLP-hFVIII-V3. Researchers observed the factor VIII levels in this trial using lower doses of adeno-associated viral (AAV) vector than in previous gene therapy studies for hemophilia A. Read more.
• Patients with severe hemophilia B demonstrated stable therapeutic expression of the factor IX gene over an 8-year period without late toxicities after systemic administration of a gene therapy called scAAV2/8-LP1-hFIXco. Read more.
• Immune tolerance induction with standard or extended half-life recombinant factor VIII after initial treatment with emicizumab (Hemlibra, Genentech) appears safe and effective in pediatric patients with hemophilia A and active inhibitors. Read more.