Characteristics of Factor IX Deficiency

The Unique Characteristics of Factor IX Deficiency

ISTH 2015 (Toronto, June 20-25) A session at the International Society on Thrombosis and Hemostasis (ISTH) 2015 Meeting discussed Factor IX deficiency, the factor responsible for Hemophilia B and how it may differ from Hemophilia A. Historically, severe hemophilia B has been compared to and treated similarly to severe hemophilia A. Lack of extensive clinical information due to the lower number of cases of severe Hemophilia B compared with Hemophilia A is the reason for this. Some studies have reported that Hemophilia B patients use lower amounts of factor, use prophylaxis less often, have less hospital admissions, and less joint disease. Findings from the PedNet (European Pediatric Network for Hemophilia Management) registry showed severity and types of bleeding are similar during the early stage of the disease in young boys with severe and moderate hemophilia A and B suggested that children with Hemophilia B should be observed and treated as carefully as those with Hemophilia A. Extended half-life FIX products may have the potential for improved patient management with less frequent infusions, higher troughs, simplified prophylaxis and increased patient adherence. Gene therapy may provide a cure for Hemophilia B and overcome the limitations of factor replacement therapy.