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2023, Number 2

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Rev Hematol Mex 2023; 24 (2)

Emicizumab: Treatment of hemophilia A with and without high response of inhibitors

Tirado LM, Esparza MMR

Language: Spanish
References: 33
Page: 68-78
PDF size: 264.48 Kb.


ABSTRACT

Hemophilia A is considered within the group of rare and orphan diseases since they only affect a small number of people. This congenital hemorrhagic disease caused by coagulation factor VIII deficiency, due to its low prevalence and incidence, can take time to be diagnosed. Treatment of this disease has been established since the 1970s with the availability of coagulation factor concentrates of plasmatic origin. Then safe concentrates were developed, duly inactivated or of recombinant origin. Prophylactic treatment with recombinant factor VIII concentrate is currently the first-line treatment for patients with moderate to severe hemophilia A. But the main complication is that these patients generated inhibitors against prophylactic treatment. From this, the need has emerged of developing new treatments that aim to reduce the annual rate of muscle and joint bleeding, avoid the development of inhibitors and that the patient has sustained protection. There are alternatives for those patients with a high response to inhibitors that include eradication with immune tolerance induction and episodic or prophylactic treatment with bypass agents, such as activated recombinant factor VII or activated prothrombin complexes, but the main problem is that these treatments are dependent on adequate venous access, therefore, more effective and less onerous treatments are needed. Emicizumab is configured as a therapeutic alternative to cover these unsatisfied needs, since it is administered subcutaneously once a week or once every two weeks or even once every four weeks, in addition to facilitating home treatment.


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