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Blood, 1 July 2006, Vol. 108, No. 1, pp. 57-62. Prepublished online as a Blood First Edition Paper on March 23, 2006; DOI 10.1182/blood-2005-02-0788. This Article Full Text Full Text (PDF) All Versions of this Article: 2005-02-0788v1 108/1/57    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map 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 HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Lovejoy, A. E. Articles by Nugent, D. J. Search for Related Content PubMed PubMed Citation Articles by Lovejoy, A. E. Articles by Nugent, D. J. Related Collections Hemostasis, Thrombosis, and Vascular Biology Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  CLINICAL TRIALS AND OBSERVATIONS Safety and pharmacokinetics of recombinant factor XIII-A2 administration in patients with congenital factor XIII deficiency Amy E. Lovejoy, Tom C. Reynolds, Jennifer E. Visich, Michael D. Butine, Guy Young, Melissa A. Belvedere, Rachelle C. Blain, Susan M. Pederson, Laura M. Ishak, and Diane J. Nugent From the Department of Hematology, Children's Hospital of Orange County, Orange, CA; and ZymoGenetics, Seattle, WA. Congenital factor XIII (FXIII) deficiency is associated with a tendency for severe bleeding, a risk for spontaneous abortion, and a high rate of spontaneous intracranial hemorrhage. This phase 1 escalating-dose study was developed to evaluate the safety and pharmacokinetics of a single administration of human recombinant FXIII-A2 (rFXIII-A2) homodimer in adults with congenital FXIII deficiency. Pharmacokinetics and activity of rXIII and changes in endogenous B subunit levels were assessed. Recombinant FXIII-A2 homodimer were complexed with endogenous FXIII-B subunits to form an FXIII-A2B2 heterotetramer with a half-life of 8.5 days, similar to that of endogenous FXIII. The median dose response was a 2.4% increase in FXIII activity based on unit per kilogram rFXIII administered. After the administration of rFXIII-A2, clot solubility normalized as measured by clot lysis in urea. Clot strength and resistance to fibrinolysis, as assessed by thromboelastography, also improved. Safety reviews were conducted before each dose escalation; no serious adverse events, including bleeding or thrombosis, were noted during the study. In addition, there was no evidence of the generation of specific antibodies to rFXIII or yeast proteins. Recombinant FXIII appears to be a safe and potentially effective alternative for FXIII replacement in patients with FXIII deficiency. (Blood. 2006;108:57-62) CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: J.-P. Fermand Initial Therapy for Multiple Myeloma: Role of Stem Cell Transplantation ASCO Educational Book, January 1, 2008; 2008(1): 375 - 379. [Abstract] [Full Text] [PDF] M. Attal, P. Moreau, H. Avet-Loiseau, and J.-L. Harousseau Stem Cell Transplantation in Multiple Myeloma Hematology, January 1, 2007; 2007(1): 311 - 316. [Abstract] [Full Text] [PDF]

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