No clinical trials are currently ongoing for F5F8D, as no FV concentrates are available to treat patients affected with FV deficiency. For this reason, and to improve the quality of life for F5F8D patients who still do not have a specific product that addresses their medical needs, drug companies are urged to produce and test a FV concentrate. A specific plasma-derived FV product has been developed by Kedrion (Italy) which has been shown to correct laboratory parameters (PT, PTT and thrombin generation) in severe FV-deficient plasma.36 However, it is not yet commercially available and outcomes in FV deficient patients need to be clarified.
In contrast, the products for the treatment of FVIII deficiency have developed substantially over the past 20 years, resulting in the production of modified molecules with improved potency, stability, and half-life. Both standard and global coagulation assays are now being used to test the efficacy and safety of these products. An assessment of whether inhibitor assays should be established for these novel products is necessary.
Rare Coagulation Disorders Resource Room, an extension of the RBDD Registry (eu.rbdd.org), was developed in collaboration with the Indiana Hemophilia & Thrombosis Center (www.innovativehematology.org).
The Resource Room serves as a platform to promote international collaboration to increase knowledge of these very rare disorders and to improve the care of affected individuals. The international RBDD mission, under the leadership of Dr. Peyvandi, will continue to be expanded over time.
The Resource Room thanks all contributing authors of the website articles that have made this valuable information available to the global community of both individuals affected with rare coagulation disorders and their healthcare providers.
