Lyophilized cryoprecipitate for children with hemophilia A.

ABSTRACT

Cryoprecipitate is the most cost-effective treatment for bleeding events in hemophilia A children in Thailand. Lyophilized cryoprecipitate (LC), a freeze-dried heat-treated blood product from the National Blood Center, can be stored in a refrigerator. The objectives of this study were to assess the efficacy of LC in bleeding episodes of children with hemophilia A and to determine the pharmacokinetics of single dose LC infusion. Eight male children with congenital factor VIII deficiency, aged 1.2-13 years, with 12 active bleeding episodes were infused with reconsitituted LC (average factor VIII activity = 236 IU per bottle) at the dose of 1 bottle per 11 kg. All patients achieved clinical hemostasis by 12 hours post-infusion. The Factor VIII activity reached peak level within the first hour and declined slowly in the subsequent hours and retained in vivo Factor VIII activity at 27.3 +/- 5.6 per cent at 12 hours post-infusion. The incremental factor VIII activity was 2.37 +/- 1.05 per cent (mean +/- SD) per U infused per kilogram body weight. The half-life of infused factor VIII was 10.5 hours. The authors concluded that reconstituted LC infusion is efficacious in the treatment of bleeding episodes in children with hemophilia. Cost-effectiveness of this product and cryoprecipitate and other purified factor VIII should be evaluated.