Optimizing Clinical Outcomes: Inhibitors, Clotting Factors, and Gene Therapy in Hemophilia

Section 3 of a 3-part series. Hemophilia, primarily diagnosed in infancy and early childhood, is a genetic disorder characterized by spontaneous or excessive bleeding predominantly into the weight-bearing joints or muscles. Most individuals are impacted through adolescence, adulthood, and into old age. This CME-accredited activity will review recent breakthroughs addressing the limitations of regular, prophylactic infusion of missing coagulation factors VIII/IX, discuss current and emerging therapies and treatment regimens, highlight risks and prevention of progressive degenerative joint changes, and explore practical implications of novel developments, while addressing “transition strategies” for adolescent care in the changing landscape of hemophilia.