SUMMARY
The 2024 ASH Congress once again highlighted ground-breaking advancements in the field of haemostasis and thrombosis, with four studies standing out for their clinical and scientific significance. First, a study demonstrated that low-dose direct oral anticoagulants (DOACs) are not inferior to standard doses for long-term anticoagulation in patients with venous thromboembolism (VTED), offering a promising alternative therapeutic approach. Furthermore, VMX-C001 shows potential as a significant advancement in managing emergencies in patients on factor Xa inhibitors (FXa-DOACs), enabling rapid and effective anticoagulation reversal; however, its efficacy and safety still require validation in phase III clinical trials. Additionally, gene therapy for haemophilia A using giroctocogene fitelparvovec achieved sustained factor VIII expression, leading to a substantial reduction in bleeding episodes among adults with moderately severe to severe haemophilia A. Lastly, autologous transplantation of CD34+ hematopoietic stem cells transduced with a lentiviral vector carrying a novel F8 transgene (ET3) resulted in stable expression of factor VIII for up to two years, representing a transformative breakthrough in the treatment of haemophilia A. Together, these findings underscore the remarkable progress achieved this year in addressing these critical medical challenges.
(BELG J HEMATOL 2025;16(1):34–40)
