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The International Factor IX Treatment Network
Hemophilia B is the second most common type of hemophilia, occurring in about one in 25,000 male births. This disease is in some ways more complex than hemophilia A, and is less well understood. Inhibitor issues in particular are somewhat different from those seen in hemophilia A. A variety of replacement products and treatment regimens are available and utilized for treatment of hemophilia B; the impact of these various replacement products and treatment regimens is not well documented in terms of outcomes.
The “royal disease”, a blood disorder transmitted through European royal families, is the most striking example of X-linking recessive inheritance. Recent DNA analysis revealed that the royal disease mutation was a severe form of hemophilia B (Science. 2009; 326(5954): 817).
The International Factor IX Treatment Network is a study group formed with the goals of investigating the treatment, outcomes and complications of hemophilia B, providing the community an improved understanding of the disease, and assisting in the introduction of new approaches to improve management and achieve optimal outcomes.
As a first step, a survey was conducted among hemophilia treatment centers worldwide, with the objectives of characterizing the population under care, and developing a factor IX investigators network. Data were collected without individual or identifying data, and included the number of patients with factor IX deficiency at each center, type(s) of treatment, type(s) of product used, number with inhibitors, proportion whose factor IX (F9) gene mutation has been determined, methods of replacement therapy for surgical procedures, and an assessment of investigators’ interest in Network initiatives. The survey was concluded on 31 December 2010.
Completed surveys were received from 156 centers in 30 countries located in Asia, Europe, Latin America, North America, and the Middle East, treating a total of 5,525 patients for factor IX deficiency. Results will be used to determine the future initiatives of the Network.
The International Factor IX Treatment Network is an investigator-initiated study funded by an unrestricted grant from GRIFOLS.
For questions about this study, please contact Jennifer Waters at Jennifer_Waters@rhoworld.com.