Evolution and Earthquake in the Therapeutic Landscape for Hemophilia A & B, An Issue of Hematology/Oncology Clinics of North America

In this issue of Hematology/Oncology Clinics, guest editors Drs. Jean M. Connors and Donna DiMichele bring their considerable expertise to the topic of Evolution and Earthquake in the Therapeutic Landscape for Hemophilia A & B. The therapeutic landscape for Hemophilia A and B has evolved dramatically from standard factor replacement to a diverse toolkit including extended half-life factor concentrates, non-factor therapies, and gene therapy. These innovations have shifted prophylaxis toward less frequent dosing and improved quality of life, while gene therapy offers the prospect of a functional cure with durable factor expression up to four years post-infusion. Despite transformative progress, challenges around long-term durability, AAV re-dosing limitations, variable patient outcomes, and global access disparities remain active areas of investigation. In this issue, top experts discuss key topics in this rapidly advancing field.