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NHLBI project to study the causes of inhibitor formation in children with hemophilia A

RTI International is partnering with researchers from the University of Washington and Emory University in the seven-year cooperative agreement

RESEARCH TRIANGLE PARK, N.C. — The National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) has awarded $9.3 million to RTI International, the University of Washington and Emory University to create the Hemophilia A Analytic Research Program (HARP). HARP will study the causes of inhibitor formation in children suffering from hemophilia. The project will take place between October 2022 and 2029.

Hemophilia A is a rare congenital X-linked bleeding disorder caused by any of several mutations that result in the absence of activity in clotting factor VIII. Hemophilia A affects nearly 16,000 individuals in the U.S. The disease is typically diagnosed in children between 1 and 3 years old.

Recombinant FVIII protiens are now widely used to prevent, rather than treat, hemorrhage. However, in approximately 30% of severe cases, children develop an inhibitor that disables the effectiveness of recombinant FVIII proteins and can result in more serious illness and increased risk of death.

The research consortium for the project includes RTI’s Grier Page, Ph.D., as well as Jill Johnsen, M.D. from the University of Washington and Shannon Meeks, M.D. from Emory University, both of whom have extensive expertise in Hemophilia A. The research team will recruit a diverse group of mothers and children with Hemophilia A and follow them for several years, starting at or before birth. Throughout the study, researchers will collect health information and a variety of biological samples from the participants to generate environmental and multi-omics data.

The research team will seek to understand the causes of inhibitor formation, which could lead to its prevention or the development of effective treatment. The study will also create a biorepository of samples and data that can be used as a resource for further research into hemophilia.

“Inhibitor formation in Hemophilia A patients is devastating to the patient and their family when treatments that have previously been effective suddenly fail,” said Dr. Page. “We hope the study data can answer questions about the formation of inhibitors and ways to prevent and treat inhibitor formation.”

The research consortium will work closely with various hemophilia organizations across the United States. In addition, the research team will partner with 141 CDC-supported Hemophilia Treatment Centers for recruitment of participants.

Learn more about RTI’s omics work