The Dilemma of Providing Advanced Hemophilia Treatments in Developing Countries - For Whom, by Whom and Where?

Abstract

Hemophilia, a congenital deficiency of factor VIII (hemophilia A) or factor IX (hemophilia B), leads to recurrent bleeding episodes that may cause progressive joint damage and long-term disability. Traditional management relies on intravenous factor replacement therapy; however, limited half-life, immunogenicity, venous access challenges, and the burden of frequent infusions have prompted the development of extended half-life (EHL) factor products. Although EHL therapies represent an important advancement, they only partially reduce treatment burden and do not fully meet expectations for improved convenience and sustained bleed protection. Recent innovations are reshaping the therapeutic landscape. Nonfactor subcutaneous therapies such as anti-TFPI molecules, antithrombin reducing agents, and anti-protein C agents offer simplified administration with the potential for improved adherence. Gene therapy provides the prospect of a long-term therapeutic effect in selected patients. In T & uuml;rkiye, hemophilia care remains largely factor-based; however, clinical trial participation and recent regulatory approvals for selected novel therapies have begun to expand real-world experience with these emerging treatment options. As global practice shifts toward individualized, less invasive treatment approaches, expanding availability of novel therapies and optimizing patient-specific treatment strategies will be essential. This review examines current and evolving treatment options, key challenges, and future opportunities shaping the trajectory of hemophilia management.