Extending health equity to people with moderate and mild hemophilia A: revisiting the HAVEN 6 trial

Abstract

Congenital hemophilia A (HA) disease severity has traditionally been categorized according to intrinsic factor (F)VIII levels, with <1% of normal indicating severe HA, 1% to 5% moderate HA, and 6% to 40% mild HA. However, mounting evidence illustrates considerable variability in bleeding phenotype regardless of FVIII level. Despite treatment advances, people with moderate or mild HA may be neglected, as treatment guidelines and established norms focus on FVIII levels, and many clinical trials do not include people with FVIII > 1%. Data from the HAVEN 6 trial demonstrated that people with moderate or mild HA, for whom prophylaxis was warranted by the treating physician’s judgment, experienced a clear clinical benefit from receiving emicizumab prophylaxis. A shift in treatment paradigms to incorporate clinical phenotypes alongside FVIII levels should be encouraged. This change in practice would allow treaters to extend health equity to people with moderate or mild HA.