Comprehensive Management of Hemophilia, Including Recent Therapeutic Advances

Hemophilia remains one of the most challenging inherited bleeding disorders, especially in low- and middle-income countries (LMICs) where late diagnosis, suboptimal factor availability, and limited access to novel therapies hinder optimal outcomes. This chapter presents an integrated approach to the management of Hemophilia A and B, encompassing pathophysiology, classification, and clinical manifestations across different age groups. Using a real-life illustrative case, the narrative explores the evolution of treatment strategies—from plasma-derived concentrates to recombinant factor replacement and novel non-factor therapies. The role of low-dose prophylaxis, the importance of early joint protection, and LMIC-specific implementation barriers are emphasized. Particular attention is paid to the advent of emicizumab and other non-factor therapies, which has transformed prophylaxis in both inhibitor and non-inhibitor patients, with insights from the HAVEN trials and Indian low-dose data. Gene therapy’s promise is critically evaluated, including limitations related to cost, vector immunity, and durability of response. A cost-effectiveness comparison between conventional therapy, emicizumab, and gene therapy is presented. The chapter also outlines current international treatment guidelines (WFH, ISTH) and how they can be contextualized for resource-constrained settings. This chapter aims to equip clinicians, researchers, and policymakers with a comprehensive yet practical understanding of hemophilia management tailored to regional needs and emerging global standards.