Low Dose Prophylaxis in Hemophilia Care.

Abstract

Hemophilia is an inherited bleeding disorder which causes impaired blood clotting. The severity of disease depends on the type of Hemophilia, level of clotting factor concentrate (CFC), phenotypic heterogeneity and the development of inhibitors. The currently accepted standard of care of this disease is prophylaxis therapy (PT) with CFC. Prophylaxis therapy for Hemophilia is given in developed countries for the last few decades. On the contrary, episodic therapy (ET) is still the mode of treatment in middle and low income countries. ET is documented to have several potential risks such as increased bleeding rate, disability due to haemarthrosis, poor quality of life and increased chances of mortality. Several studies conducted in developed countries have confirmed the clinical efficacy of PT in Hemophilia treatment. Currently, there exist several challenges for implementation of this effective treatment in resource poor nations. Low dose prophylaxis (LDP) has been developed as a solution to minimize these challenges and to provide better care for subjects with Hemophilia from low resource countries. The impact of LDP was evaluated by several recent studies and the reported clinical outcomes seem to suggest an optimistic future for this line of therapy. Several themes related to Hemophilia care like inhibitor development, tolerance, pharmacokinetics of CFCs and cost-benefit analysis of different prophylaxis regimens are currently understood poorly. These distinct elements are highly relevant to assess the actual benefits of LDP regimen in a global scale.