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Thromb Haemost 2003; 89(03): 480-485
DOI: 10.1055/s-0037-1613377
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

A quantitative measure of the efficacy of factor VIII in hemophilia A mice

Ernest T. Parker
1   Winship Cancer Institute, Emory University, Atlanta, Georgia, USA
,
Pete Lollar
1   Winship Cancer Institute, Emory University, Atlanta, Georgia, USA
› Author Affiliations
Further Information

Publication History

Received 10 September 2002

Accepted after revision 07 January 2003

Publication Date:
09 December 2017 (online)

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Summary

We developed a quantitative method to study the efficacy of intravenously delivered human factor VIII in hemophilia A mice. Mortality was assessed after tail transection under conditions in which there were no survivors in untreated hemophilia A mice. Blood loss was significantly greater in untreated hemophilia A mice compared to normal C57BL/6 mice, and in hemophilia A non-survivors that were treated with subtherapeutic doses of factor VIII. The up-and-down method for small samples yielded an estimated dose of factor VIII producing survival in 50% of the mice (ED50) of 58 units/kg (95% confidence interval: 42.4 - 78.5 units/kg). This method should be useful in the evaluation and comparison of novel factor VIII products delivered either parenterally or in a gene therapy setting.

Keywords

Hemophilia A - factor VIII - ED50 - animal model
 

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