Pharmacokinetics, Safety, and Efficacy of Recombinant Factor VIII Fc Fusion Protein: A Practical Review.

Prophylaxis for hemophilia A with conventional factor VIII (FVIII) products requires frequent intravenous dosing, which may reduce adherence. Recombinant factor VIII Fc fusion protein (rFVIIIFc) has a prolonged half-life compared with conventional rFVIII, and has demonstrated safety and efficacy for the prevention and treatment of bleeding episodes in phase 3 studies of patients with severe hemophilia A. Most subjects experienced reduced prophylactic dosing frequency with rFVIIIFc compared with prestudy FVIII; the median total weekly prophylactic consumption was comparable. No subjects developed inhibitors. These results suggest that prophylaxis with rFVIIIFc in patients with hemophilia A may allow less frequent prophylactic dosing while maintaining efficacy, with comparable prophylactic consumption.

The universal data collection surveillance system for rare bleeding disorders.

Since 1998, the CDC has coordinated a national public health surveillance project-the Universal Data Collection (UDC) program-on chronic, rare, inherited bleeding disorders. In this program, uniform data are gathered through a network of 130 hemophilia treatment centers (HTCs) throughout the U.S. and its territories. Initially, the program was designed to address two primary goals: (1) establishment of a blood-safety monitoring system among people with bleeding disorders, and (2) collection of a uniform set of clinical outcomes data that could be used to monitor trends in the prevalence of infectious diseases and joint complications among this population. To this end, the program has been acquiring useful longitudinal data to monitor complications of bleeding disorders. For example, with the establishment of range-of-motion measurements for joints as required data elements, a large database has been developed for studies examining risk factors for joint-disease progression. The UDC program data have been used to provide evidence for a national prevention campaign to promote the need for patients with hemophilia to establish or maintain a healthy weight to help prevent joint disease. Risk factors leading to complications such as joint infection have also been identified. The application of geographic information systems technology to UDC program data has helped identify needs for outreach and availability of blood products and sources of care. Future analyses of data collected on babies, women, and individuals with rarer bleeding disorders than hemophilia will provide further information, leading to improved public health prevention strategies.