An update of the current pharmacotherapeutic armamentarium for hemophilia A

Emicizumab state‐of‐the‐art update

“…Elevation in the thrombin generation was observed following emicizumab prophylaxis, with lower values recorded in younger infants, yet as all bleedings were trauma‐related, laboratory monitoring could not predict bleeding risk 13 . Further longitudinal follow–up of the mixed Israeli cohort (107 patients with severe HA, including 58 children whose median (IQR) age was 6 1–11 years), followed for a median of 67 weeks (up to 144 weeks) disclosed zero bleeds in half of the patients. Most bleeding episodes (94%) among children were trauma‐related.…”

“…For several decades, replacement therapy with plasma‐derived or recombinant clotting factors was the standard of care in managing patients with haemophilia A 1 . However, patients on replacement therapy have several unmet needs, including the requirement for intravenous access to infuse clotting factor, the immunogenicity of replacement FVIII and clotting factor pharmacokinetics (PK) characterised by peaks and troughs, with the latter associated with breakthrough bleeds 2 . Non‐replacement therapies are a new treatment paradigm developed to address these unmet needs, and the first non‐factor therapeutic licenced is emicizumab.…”

“…by peaks and troughs, with the latter associated with breakthrough bleeds. 2 Non-replacement therapies are a new treatment paradigm developed to address these unmet needs, and the first non-factor therapeutic licenced is emicizumab.…”

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

“…In haemophilia A, the deficient 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 haemophilia 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.…”

“…The prophylaxis goals are achievable through several strategies. Firstly, we now have a variety of therapeutic agents we use, including products, recombinant products and non-replacement therapies (Mahlangu, 2022) . The estimated global FVIII consumption in 2020 was 12 billion units.…”