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Hemophilia of Georgia Funds Two New Researchers

Published April 9, 2014

 

By Jeff Cornett RN MSN, Director of Training, Research, & Advocacy

This month, Hemophilia of Georgia begins funding two new physician researchers as part of its Clinical Scientist Development Grant program. These grants provide up to five years of funding. They are designed to allow a physician who has completed research training to advance to the status of an independent investigator. Our goal is for the achievements of these scientists to improve the lives of people with bleeding disorders. With the addition of these two researchers, 11 physicians have been funded since the grant program began in 2003. You can see a list of past grant recipients on our website at https://www.hog.org/programs/page/hemophilia-clinical-scientist-development-grantees.

Robert F. Sidonio Jr., MD MSc
Assistant Professor of Pediatrics
Vanderbilt University Medical Center, Nashville, Tennessee

Research Project: Prospective Feasibility Study of the Bleeding Genotype and Phenotype in Hemophilia A Carriers

It is well known that women can be carriers of hemophilia A (factor VIII deficiency) with the disorder potentially being seen in their male children. What is much less well studied is how these carriers can be affected by bleeding themselves. Dr. Sidonio is studying joint damage in women who are carriers. He has previously published that 16% of hemophilia A carriers report a history of joint bleeding. He plans to recruit adult carriers and compare their lab tests, bleeding history, and joint exams to those of women who are not carriers. If a carrier has reduced range of motion in a joint, an MRI will be done to look for joint damage. The loss of range of motion may be a sign of previous bleeding in the joint.

Sean R. Stowell, MD PhD
Post-Doctoral Fellow
Emory University, Atlanta

Research Project: Characterization of Factors Influencing the Development of Factor VIII Inhibitors

In order to prevent bleeding, people with hemophilia A inject a protein, factor VIII, into their blood. Typically infusing a protein this way does not cause a response from the body’s immune system. However, 30% of people with severe hemophilia A develop neutralizing antibodies called inhibitors to factor VIII. Inhibitors keep the factor from working correctly to stop bleeding. The goal of Dr. Stowell’s research is to develop a better understanding of the very early response of the immune system to the factor VIII protein. He will begin to address the types of immune cells involved in developing inhibitors and what conditions may enhance or decrease this response. His research may lead to new strategies to prevent the formation of factor VIII inhibitors in people with hemophilia A.