Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Experiment Videos

Prophylaxis in haemophilic children

R J Liesner1

  • 1Department of Haematology, Great Ormond Street Childrens Hospital NHS Trust, London, UK.

Blood Coagulation & Fibrinolysis : an International Journal in Haemostasis and Thrombosis
|August 1, 1997
PubMed
Summary

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.

Related Concept Videos

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Long-term tolerability, immunogenicity and efficacy of Nuwiq<sup>®</sup> (human-cl rhFVIII) in children with severe haemophilia A.

Haemophilia : the official journal of the World Federation of Hemophilia·2018
Same author

Immunogenicity, efficacy and safety of Nuwiq<sup>®</sup> (human-cl rhFVIII) in previously untreated patients with severe haemophilia A-Interim results from the NuProtect Study.

Haemophilia : the official journal of the World Federation of Hemophilia·2017
Same author

Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients.

Journal of thrombosis and haemostasis : JTH·2008
Same author

Does thrombophilia cause Perthes' disease in children?

The Journal of bone and joint surgery. British volume·1999
Same author

ABC of clinical haematology. Platelet disorders.

BMJ (Clinical research ed.)·1997
Same author

ABC of clinical haematology. The acute leukaemias.

BMJ (Clinical research ed.)·1997

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.

Related Experiment Videos

  • 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.