Abstract
Long-term (or continuous) prophylaxis is generally accepted as the best form of treatment
for patients with severe hemophilia A and B. Results of recent prospective, randomized
clinical trials, as well as observational studies performed in the last decades, have
provided strong and convincing evidence that continuous prophylaxis leads to reduction
in the number of bleeding episodes, better joint status and improved health-related
quality of life as compared with on-demand (or episodic) treatment. Nevertheless,
many questions regarding long-term prophylaxis still remain open, for instance: when
should prophylaxis be started (before or after the first joint bleeding), what is
the optimal dosage to replace the missing factor, when should discontinuation of long-term
prophylaxis be considered, what is the best way to measure the outcome of prophylaxis,
etc. Moreover, there are numerous obstacles to widespread use of prophylactic therapy.
The most challenging seem to be adequate venous access (particularly in younger patients)
and patients' adherence. The crucial barrier to long-term prophylaxis is, however,
the remarkably high cost of clotting factor concentrates. For most countries high-dose
or intermediate-dose prophylaxis regimens are not affordable due to lack of economic
resources. So, is continuous prophylaxis reserved exclusively for wealthy societies?
Fortunately, there is an increasing body of evidence to suggest that low-dose prophylaxis
offers significant benefits over on-demand treatment with comparable amounts of factor
concentrate (and much lower amounts if compared with intermediate or high-dose prophylaxis
regimens). The aim of this article is to discuss the clinical and economical aspects
of continuous prophylaxis in hemophilia with emphasis on the low-dose regimens.
Keywords
hemophilia - low-dose prophylaxis - on-demand therapy - costs
