Current and future approaches to overcoming the challenges of hemophilia treatment personalization

ABSTRACTIntroduction: Hemophilia is a rare congenital bleeding disorder that requires coagulation factor replacement therapy to treat or, better, to prevent life- and limb-threatening bleeding and its complications. Personalized treatment of patients with hemophilia is crucial for improving outcomes in an environment with increasing health budget constraints.Areas covered: Unmet needs for a more effective and efficient use of factor concentrates are examined. The current approach is described with its limits and recent improvements, i.e. pharmacokinetic assessments and dosing tools/models. Future approaches to personalization were researched in the literature and discussed, including biomarkers, genotyping, decision-making tools, goal attainment tools and digital health systems.Expert opinion: A paradigm shift in hemophilia management personalization is necessary in order to improve outcome and ensure long-term sustainability in a landscape of growing and aging patient populations, increasing expectations...