Evidence for the benefits of prophylaxis in the management of hemophilia A

Journal:Thrombosis and Haemostasis
ISSN:0340-6245
DOI:http://dx.doi.org/10.1160/TH06-02-0125
Issue:2006: 96/4 (Oct) pp. 391-543
Pages:433-440

Evidence for the benefits of prophylaxis in the management of hemophilia A

W. Keith Hoots1,2, Diane J. Nugent3,4
1Department of Pediatrics and Internal Medicine, University of Texas Medical School, Houston, Texas, USA; 2Department of Pediatrics, University of Texas – M.D. Anderson Cancer Center, Houston, Texas, USA; 3Department of Hematology, Children’s Hospital

Summary

The optimal treatment of hemophilia has been evolving since the advent of factor VIII concentrates, continues to vary geographically, and remains a source of debate.There now exists an extensive clinical literature that demonstrates clear benefits of prophylaxis for patients with hemophiliaA compared to on-demand treatment, including a reduction in the number of bleeding episodes, improved joint function, and greater patient well-being. However, the value of these benefits must be weighed against the heavier economic burden of increased factor use.This paper reviews the literature that compares the benefits of prophylaxis with that of on-demand treatment, compares varying prophylaxis dose and administration frequency protocols, and considers the long-term cost-benefit of prophylactic therapy.

Keywords

cost-benefit, factor VIII, prophylaxis, hemophilia, on-demand, arthropathy

DOI

http://dx.doi.org/10.1160/TH06-02-0125

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