Arihant SENTHIL, Tashvin PAUL, Karanbir SINGH, Mahima KAMBLE, Jinpyo HONG, Mayank DHALANI, Vasu BANSAL, Rohit JAIN
Turkish Journal of Internal Medicine - 2026;8(1):122-136
Objective: Hemophilia, a genetic bleeding disorder caused by deficiencies in clotting factors VIII, IX, or XI, has transitioned from a fatal disease to a chronic but manageable condition. Traditional management with plasma-derived and recombinant factor replacement improved survival and reduced bleeding but remains limited by frequent infusions, inhibitor development, and incomplete protection against joint damage. Methods: This narrative review was based on a literature search of PubMed/MEDLINE and Google Scholar, focusing on recent clinical studies, guidelines, and landmark publications on hemophilia therapies. Results: Recent therapeutic advances have transformed hemophilia management by reducing treatment burden, improving bleed prevention, and enhancing patient quality of life. Extended half-life factor concentrates have decreased infusion frequency, while non-factor therapies have expanded effective prophylaxis for patients with and without inhibitors through convenient subcutaneous administration. Gene therapy has demonstrated sustained endogenous factor expression with substantial reductions in bleeding episodes, offering the potential for long-term disease modification. Despite these advances, challenges related to treatment durability, immune responses, patient eligibility, accessibility, and cost continue to limit widespread implementation. Conclusion: This narrative review outlines the evolution of hemophilia therapy from replacement strategies to innovative mechanism-based approaches, emphasizing clinical outcomes, emerging challenges, and the imperative to translate advances into equitable, sustainable care.
