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

Abstract

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.