A global study involving McMaster University researchers has trialled a potential new treatment that could be a game-changer for people living with hemophilia A, a life-threatening genetic blood disorder.
Co-principal investigator Davide Matino said that once-weekly injections of efanesoctocog alfa can prevent bleeding and promote near-normal activity by the clotting agent factor VIII, crucial for stopping the prolonged bleeding caused by hemophilia A. People who took efanesoctocog alfa also enjoyed improved joint function, better overall health and less pain.
Matino said that efanesoctocog alpha works by separating factor VIII from its associated protein carrier, known as the von Willebrand Factor, thereby overriding its ‘half-life’. This means people with hemophilia A only need once-weekly instead of thrice-weekly injections.
Researchers published their findings in The New England Journal of Medicine.
The trial, which concluded in 2021, found that 133 hemophilia patients treated weekly for a year improved their outcomes and quality of life compared to their previous treatment.
