New therapies in hemophilia: extend the half-life, mimic, or rebalance?

Abstract

The treatment landscape for hemophilia has evolved rapidly over the past decade, with the approval of several therapies with novel mechanisms of action. Extended half-life factor concentrates and nonfactor therapies, including factor VIII mimetics and rebalancing agents, have reduced treatment burden while also improving bleed protection. Although these new therapies provide an unprecedented ability to personalize treatment and to meet changing needs across the life span for each individual, the complexity of these treatment considerations has made clinical decision-making and selection of the optimal treatment challenging for those living with hemophilia and their clinicians.