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Factor VIII is released from endothelial cells as a two-chain molecule that is stabilized by the von Willebrand factor. In hemophilia patients, better stability might prolong factor VIII half-life and enhance its hemostatic effectiveness. To that end, a higher affinity for von Willebrand factor has recently been achieved with the synthesis of a truncated, single-chain, recombinant factor VIII (rVIII) molecule.
To evaluate the safety and effectiveness of this novel factor VIII, investigators conducted an industry-sponsored, open-label, nonrandomized, multicenter, phase I/III study of 173 patients with severe hemophilia A and prior exposure to factor VIII. Prophylactic rVIII was received by 146 patients (median dose, 31 IU/kg), and on-demand V…