RFI from NHLBI: To Improve Treatment of Patients with the Severe Hemophilia A Phenotype

October 23, 2018

Soliciting Input for future research at the National Heart, Lung, and Blood Institute on Factor VIII (FVIII) immunogenicity and FVIII inhibitor prevention/eradication in patients with Hemophilia A

The NHLBI has released an RFI soliciting perspectives and comments from the community on scientific opportunities, critical needs, strategies, and infrastructure that would be conducive to the establishment of a US-based national ante-, peri-, and post-natal cohort, to foster research into the pathophysiology underlying alloantibody formation and to improve the treatment of patients with the severe Hemophilia A phenotype [Factor VIII (FVIII) activity level of <1% of normal]. The major complication of FVIII replacement is the development of neutralizing alloantibodies (inhibitors) which can eventually preclude FVIII administration. 

The scientific priorities for this potential resource include elucidation of the mechanisms of immunogenicity and tolerance to FVIII exposure in severe Hemophilia A patients, as well as the development of prediction tools to 1) identify severe Hemophilia A children who are at risk for developing inhibitors; 2) mitigate modifiable risk factors; and 3) inform novel inhibitor prevention and treatment strategies. This RFI intends to understand the landscape of current and desired resources required to enable research that fully elucidates the determinants of alloantibody formation in this population. We would greatly appreciate feedback on the scientific priorities for the establishment of this resource from experts and interested individuals within and beyond the hemophilia community. Responses can be submitted via e-mail ( until November 12, 2018.

Contacts for this RFI are:

Donna Di Michele, M.D.
Simone Glynn M.D., M.Sc., M.P.H
Iman Martin Ph.D, M.P.H; M.Sc.
Division of Blood Diseases and Resources 
National Heart, Lung, and Blood Institute (NHLBI)
Telephone: 301-435-0065