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Blood, 15 January 2005, Vol. 105, No. 2, pp. 518-525. Prepublished online as a Blood First Edition Paper on September 21, 2004; DOI 10.1182/blood-2004-06-2283. This Article Full Text (PDF) All Versions of this Article: 2004-06-2283v1 105/2/518    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Shapiro, A. D Articles by Roth, D. A Search for Related Content PubMed PubMed Citation Articles by Shapiro, A. D Articles by Roth, D. A Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted June 22, 2004 Accepted August 31, 2004 The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B Amy D Shapiro*, Jorge Di Paola, Alice Cohen, John Pasi, Margaret A Heisel, Victor S Blanchette, Thomas C Abshire, W K Hoots, Jeanne M Lusher, Claude Negrier, Chantal Rothschild, and David A Roth Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA Pediatrics, University of Iowa Hospitals and Clinics, Iowa City, IA, USA Newark Beth Israel Medical Center, Newark, NJ, USA Royal Free Hampstead NHS Trust, Royal Free Hospital, London, UK Minneapolis Children's Medical Center, Minneapolis, MN, USA Department of Pediatrics, University of Toronto, Toronto, Canada Emory University, AFLAC Cancer Center and Blood Disorders Service, Atlanta, GA, USA Gulf States Hemophilia Diagnostic HPB and Treatment Center, University of Texas-Houston Medical School, Houston, TX, USA Division of Hematology, Children's Hospital of Michigan, Detroit, MI, USA Centre Regional de Traitement de l'Hemophilie, Hopital Edouard Herriot, Lyon, France Centre de Traitements de l'Hemophilie, Groupe Hopitalier Necker-Enfants Malades, Paris, France Wyeth Research, Cambridge, MA, USA * Corresponding author; email: ashapiro{at}ihtc.org. This international clinical trial evaluated the safety and efficacy of recombinant factor IX (rFIX) in previously untreated patients (PUPs) with severe or moderately severe hemophilia B(FIX activity 3 IU/dL). Sixty-three PUPs aged <1 month to 14 years received rFIX (median treatment duration: 37 months; range: 464 months). Mean rFIX recovery (0.68±0.27 IU/dL per IU/kg) remained constant over 5 years and was similar in infants (1 month to <2 years) and children (2 to <12 years). Fifty-four PUPs used rFIX (median dose: 62.7 IU/kg per infusion; range: 8.2-292 IU/kg) to treat 997 hemorrhages. Bleeding was well controlled, with 75% of hemorrhages requiring only one rFIX infusion. Response to rFIX was 'excellent' or 'good' in 94% of cases. Effective hemostasis was achieved in 32 PUPs receiving rFIX for routine prophylaxis, with 91% of prophylaxis responses rated 'excellent'. rFIX administered for 30 surgical procedures in 23 PUPs achieved hemostasis for all rated procedures. Five patients experienced allergic-type manifestations, including 2 (3%) patients who developed FIX inhibitors (both >5 BU/dL). rFIX was well tolerated, with no associated thrombotic events or evidence of viral transmission. These data indicate that rFIX is a safe and effective treatment for PUPs with hemophilia B. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this?

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