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Updated: Aug 31, 2025
08:13
Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
Published on: September 30, 2021
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Advances in Hemophilia A Management.
Sukjoo Cho1, Ashley M Perry1, Anna M Cheng2
1Division of Hematology/Oncology, Department of Pediatrics, University of South Florida Morsani College of Medicine, 2 Tampa General Circle, 5th Floor, Tampa, FL 33606, USA.
Advances in Pediatrics
|August 19, 2022
Summary
Hemophilia A treatment advances include extended half-life products and emicizumab, offering alternatives to frequent infusions. Gene therapy presents a potential one-time cure for hemophilia A by establishing continuous Factor VIII expression.
Area of Science:
- Hematology
- Genetics
- Pharmacology
Background:
- Hemophilia A is an inherited bleeding disorder caused by Factor VIII (FVIII) deficiency.
- Prophylaxis with FVIII concentrates is standard for moderate-to-severe hemophilia A.
- Inhibitor development affects up to 30% of patients on prophylaxis.
Purpose of the Study:
- To review current and emerging therapies for hemophilia A.
- To discuss management strategies for patients with and without FVIII inhibitors.
- To highlight advancements in hemophilia A treatment.
Main Methods:
- Literature review of hemophilia A management.
- Analysis of extended half-life products, emicizumab, and gene therapy.
- Discussion of inhibitor eradication and acute bleed management.
Main Results:
- Extended half-life products decrease injection frequency compared to conventional therapy.
- Emicizumab offers a treatment option for hemophilia A patients with or without inhibitors.
- Gene therapy shows promise for a potential one-time cure or significant phenotype modification.
Conclusions:
- Therapeutic options for hemophilia A have expanded significantly.
- Newer treatments aim to reduce treatment burden and improve patient outcomes.
- Gene therapy represents a paradigm shift in hemophilia A management.
Keywords:
Clotting factor concentratesEmicizumabExtended half-lifeGene therapyHemophilia AInhibitorManagementProphylaxisMore Related Videos
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