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Thromb Haemost 2003; 89(03): 480-485
DOI: 10.1055/s-0037-1613377
DOI: 10.1055/s-0037-1613377
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
A quantitative measure of the efficacy of factor VIII in hemophilia A mice
Weitere Informationen
Publikationsverlauf
Received
10. September 2002
Accepted after revision
07. Januar 2003
Publikationsdatum:
09. Dezember 2017 (online)
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.
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