The innovations in hemostasis and thrombosis discussed in this article, which are demonstrations of remarkable ingenuity and advances in biotechnology, should inspire admiration as well as enthusiasm and hope for our patients.
Among these advances, the development of a chimeric Factor X capable of bypassing and restoring hemostasis in patients treated with direct oral anticoagulants (DOAC) targeting Factor X is noteworthy. A molecule (Inno8) composed of two nanobodies that recognize Factor IXa and Factor X, respectively, bind to albumin in the bloodstream, and are able to cross the digestive barrier is expected to enable oral treatment for hemophilia A patients – an unimaginable ambition until recently. Furthermore, modified B lymphocytes engineered to produce Factor IX and hematopoietic stem cells integrating the Factor VIII gene open exceptional perspectives for gene therapy in these two diseases. Finally, a large French study has highlighted the benefits of prioritizing a reduced dose of DOACs in patients with a history of venous thromboembolism who require prolonged anticoagulation.
These data and innovations, whether already applicable in clinical practice or requiring further validation, will have a significant impact on the future of a rapidly evolving field.
Keywords
Direct oral anticoagulant, reversal agent, hemophilia, gene therapy