5. Prophylaxis in Children

  1. Emérito-Carlos Rodríguez-Merchán MD, PHD2,3 and
  2. Leonard A. Valentino MD4
  1. Marilyn J. Manco-Johnson MD

Published Online: 12 MAY 2011

DOI: 10.1002/9781119979401.ch5

Current and Future Issues in Hemophilia Care

Current and Future Issues in Hemophilia Care

How to Cite

Manco-Johnson, M. J. (2011) Prophylaxis in Children, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. doi: 10.1002/9781119979401.ch5

Editor Information

  1. 2

    Department of Orthopedic Surgery and Hemophilia Unit, La Paz University Hospital, Spain

  2. 3

    School of Medicine, Autonomous University, Madrid, Spain

  3. 4

    Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL, USA

Author Information

  1. University of Colorado Denver, Aurora, CO, USA

Publication History

  1. Published Online: 12 MAY 2011
  2. Published Print: 13 MAY 2011

ISBN Information

Print ISBN: 9780470670576

Online ISBN: 9781119979401

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Keywords:

  • prophylaxis;
  • children;
  • severe hemophilia;
  • arthropathy;
  • factor VIII deficiency;
  • factor IX deficiency;
  • prevention of complications;
  • pharmacokinetics;
  • dosing;
  • outcomes

Summary

Prophylaxis is a therapy for severe hemophilia designed to prevent joint and other hemorrhages as well as the consequences of bleeding events. In primary prophylaxis, which is preferred for the best maintenance of health and joint function, factor VIII or IX is replaced on a regular schedule, beginning in the first few years of life, at a dose and frequency sufficient to prevent spontaneous bleeding. While alternate day dosing for factor VIII, which is based on pharmacokinetic data, has been demonstrated in a randomized clinical trial to prevent arthropathy and life-threatening hemorrhages, other non-pharmacokinetic-based regimens appear to be clinically effective. There is less data available for severe factor IX deficiency,; however, prophylaxis two to three times weekly is similarly employed to prevent joint damage in hemophilia B. Limitations to prophylaxis include cost, factor availability, venous access and the stress of an intensive medical regimen; however, these challenges can all be successfully addressed with adequate support. Prophylaxis is currently accepted as standard of care treatment for all pediatric patients with severe hemophilia. The promise of new longer-acting recombinant factor VIII and factor IX proteins that will prevent spontaneous bleeding with weekly or less frequent infusions should dramatically increase the application of prophylaxis to patients with severe hemophilia.