SANOFI Rare Blood Disorders Medical

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Brief Description

Stable clot formation is the result of the complex interplay between platelets, red blood cells, and coagulation factors. This process is negatively regulated by the natural anticoagulation system.(1) Referring to the second phase of this process, hemostasis is critical for survival, which is why vertebrate evolution developed a system for the cessation of bleeding, the coagulation cascade. Individuals who have dysfunction in procoagulants are at increased risk of death due to hemorrhage, and the most common deficiencies in secondary hemostasis are in factors VIII (FVIII) and (FIX), resulting in hemophilia A and B, respectively.(2) Current treatment options – such as factor replacement, bypass agents, gene therapy, and factor mimetics - have challenges and impact on health burden of hemophilia care, which results in the need for novel treatment approaches(3). Since hemophilia patients have a deficiency in procoagulant factors, tipping the scale toward bleeding, inhibition of natural anticoagulant has been utilized to restore hemostatic balance. (4, 5) Nevertheless, low levels of natural anticoagulants, even in case of hemorrhagic diseases such as hemophilia, are associated with an increased risk of thrombosis.(6) A plausible explanation for these events may be our limited understanding of the physiology of natural anticoagulants and their interaction with other physiological pathways.(7) It is thus becoming increasingly important for hematologists and other health care professionals caring for patients with hemophilia to deepen their understanding of the intrinsic mechanisms of this disease and the importance of rebalancing hemostasis. 8-12 In the near future, one of the recurrent challenges for hemophilia management will be to define the most appropriate treatment according to the needs of each person and local treatment availability. (3).