The hope and reality of long-acting hemophilia products

Authors

  • Steven W. Pipe

    Corresponding author
    1. Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, Michigan
    2. Department of Pathology, University of Michigan Medical Center, Ann Arbor, Michigan
    • Associate Professor of Pediatrics and Communicable Diseases, Associate Professor of Pathology, University of Michigan, MPB D4202, 1500 E. Medical Center Drive, Ann Arbor, MI 48109
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  • Conflict of interest: Consultancy for Baxter Bioscience, Novo Nordisk, Biogen Idec and CSL Behring

Abstract

Recombinant DNA technology and protein engineering are creating hope that we can address ongoing challenges in hemophilia care such as reducing the costs of therapy, increasing the availability to the developing world, and improving the functional properties of these proteins. Technological advances to improve the half-life of recombinant clotting factors have brought long-acting clotting factors for hemophilia replacement therapy closer to reality. Preclinical and clinical trial results are reviewed as well as the potential benefits and risks of these novel therapies. Am. J. Hematol. 2012. © 2012 Wiley Periodicals, Inc.

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