Leave no one behind: Optimal Care of all Patients with Haemophilia A

Background: Haemophilia is an inherited bleeding disorder due to failure of throm- bin generation resulting from clotting factor deficiency. In haemophilia A, the de- ficient factor is Factor VIII due to mutations in the F8 gene. In the past several decades, we have seen an unprecedented evolution o...

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Published inJornal de Assistência Farmacêutica e Farmacoeconomia Vol. 1; no. s3
Main Author Johnny Mahlangu
Format Journal Article
LanguageEnglish
Published Instituto Nacional de Assistência Farmacêutica e Farmacoeconomia 14.12.2022
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Summary:Background: Haemophilia is an inherited bleeding disorder due to failure of throm- bin generation resulting from clotting factor deficiency. In haemophilia A, the de- ficient factor is Factor VIII due to mutations in the F8 gene. In the past several decades, we have seen an unprecedented evolution of therapies for managing hae- mophilia A (Mahlangu, 2022) . These have included blood and blood products to plasma-derived concentrate, recombinant concentrate, concentrate with improved pharmacokinetics and, more recently, non-replacement therapies. Notwithstanding these advances in treatments, patients with haemophilia A (PwHA) remain with several unmet needs.
ISSN:2525-5010
2525-7323
DOI:10.22563/2525-7323.2022.v1.s3.4-7