Prophylaxis in haemophilic children.
Prophylaxis with recombinant factor concentrates is the standard for children with severe haemophilia at the end of the 1990s but there are still many who are not given this optimal therapy for a variety of reasons; the dominant one globally is almost certainly financial, and efforts over the next decade must concentrate on ways of reducing costs and reducing factor concentrate requirements. Dosing according to kinetic principles can give a more cost-effective use of concentrate and a computerized pharmacokinetic model can be used. Another possibility is the introduction of an implantable sustained-release pump delivery system connected to a central venous access system, which can deliver factor concentrate to maintain a constant level of around 5% and thus reduce the overall amount of factor used. The dose of factor required with this system has been estimated as 700-875 IU/kg per year to keep the plasma level at 2% and 1700-2200 IU/kg per year to maintain it at 5%.
Liesner
R J
RJ
Department of Haematology, Great Ormond Street Childrens Hospital NHS Trust, London, UK.
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005164:Factor IX / Q000008:administration & dosage / Q000627:therapeutic use*; D005169:Factor VIII / Q000008:administration & dosage / Q000627:therapeutic use*; D006467:Hemophilia A / Q000150:complications* / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D007592:Joint Diseases / Q000517:prevention & control; D011994:Recombinant Proteins / Q000627:therapeutic use; D013548:Sweden
D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX