Advances in Hemophilia A Management.

Journal: Advances In Pediatrics
Treatment Used: Regular Replacement Therapy with Clotting Factor Concentrates, Bypassing Agents, Emicizumab, and Gene Therapy
Number of Patients: 0
Published:
MediFind Summary

Summary: This article discusses treatments for patients with hemophilia A.

Conclusion: Treatments for patients with hemophilia A include regular replacement therapy with clotting factor concentrates, bypassing agents, emicizumab, and gene therapy.

Abstract

Hemophilia A is an inherited insufficiency of Factor VIII (FVIII), one of the critical clotting factors. The gold standard for the management of moderate-to-severe hemophilia A is prophylaxis using regular replacement therapy with clotting factor concentrates. Compared with conventional treatment, extended half-life products reduce the burden of frequent factor replacement injections. Of note, up to 30% of patients with hemophilia A receiving prophylactic factor infusions develop "inhibitors," neutralizing anti-FVIII autoantibodies. Therapeutic options for patients with hemophilia A and inhibitors include the immune tolerance induction (ie, eradication of inhibitors) and the management of acute bleeds with bypassing agents and/or emicizumab. Emicizumab is a biphasic monoclonal antibody mimicking activated FVIII, approved for patients with hemophilia A with/without inhibitors. Gene therapy is an emerging therapy for hemophilia A, essentially curing patients with hemophilia A or transforming them to a milder phenotype by establishing continuous endogenous expression of FVIII after one-time treatment.

Authors
Sukjoo Cho, Ashley Perry, Anna Cheng, Carrie Wang, Juan Rico
Relevant Conditions

Blood Clots, Hemophilia A

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