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Prophylaxis in haemophilic children
1Department of Haematology, Great Ormond Street Childrens Hospital NHS Trust, London, UK.
Insights
Financial constraints limit access to optimal recombinant factor concentrate prophylaxis for children with severe hemophilia. Future efforts must focus on cost reduction and minimizing factor concentrate needs through methods like pharmacokinetic dosing or sustained-release pumps.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacoeconomics
Background:
- Recombinant factor concentrates are standard prophylaxis for severe hemophilia in children.
- Many children still lack access to this optimal therapy due to various reasons, primarily financial.
- There is a global need to reduce costs and factor concentrate requirements for hemophilia prophylaxis.
Purpose of the Study:
- To explore strategies for cost-effective hemophilia prophylaxis in children.
- To investigate methods for reducing overall factor concentrate usage.
- To assess the feasibility of advanced delivery systems for maintaining therapeutic factor levels.
Main Methods:
- Dosing prophylaxis based on pharmacokinetic principles.
- Utilizing a computerized pharmacokinetic model for cost-effective concentrate use.
- Evaluating an implantable sustained-release pump delivery system connected to a central venous access system.
Main Results:
- Pharmacokinetic dosing can lead to more cost-effective use of factor concentrates.
- An implantable pump system can maintain constant factor levels (e.g., 5%), reducing overall factor consumption.
- Estimated annual factor doses for the pump system range from 700-875 IU/kg for 2% plasma levels to 1700-2200 IU/kg for 5% levels.
Conclusions:
- Cost-effective strategies are crucial for expanding hemophilia prophylaxis access.
- Pharmacokinetic principles and advanced delivery systems like sustained-release pumps offer potential solutions for reducing factor concentrate requirements and costs.
- These innovations could improve the management of severe hemophilia in children globally.
Abstract:
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%.