Evidence for the benefits of prophylaxis in the management of hemophilia A.
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.
Hoots
W Keith
WK
Department of Pediatrics and Internal Medicine, University of Texas Medical School, Houston, Texas, USA. keith.hoots@uth.tmc.edu
Nugent
Diane J
DJ
D000367:Age Factors; D003029:Coagulants / Q000008:administration & dosage / Q000191:economics / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D004334:Drug Administration Schedule; D005169:Factor VIII / Q000008:administration & dosage / Q000191:economics / Q000627:therapeutic use*; D006297:Health Services Accessibility; D006395:Hemarthrosis / Q000191:economics / Q000209:etiology / Q000517:prevention & control*; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000191:economics; D006801:Humans; D017410:Practice Guidelines as Topic; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic
D003029:Coagulants; D005169:Factor VIII