Identifying LDL Particles as a Novel Functional Carrier for Coagulation Factor VIII in Hemophilia A

Project Description Narrative:

Due to innovations in hemophilia therapies, patients are no longer routinely dying prematurely due to catastrophic bleeding events and thus the hemophilia patient population is aging, yet providers and patients lack understanding of their aging-associated complications, including cardiovascular diseases. However, the lipoprotein levels and the impact of low apoB lipoproteins in hemophilia A are unknown and require detailed investigation to understand cardiovascular risk in hemophilia A patients and carriers.

Wisconsin has the best opportunity to study the cardiovascular risk in the hemophilia A population. Although hemophilia A is a rare bleeding disorder, Wisconsin has one of the largest patient populations. One reason is that the Blood Center of Wisconsin, now Versiti Wisconsin, provides the most advanced patient care specialized to hemophilia. This project aims to enhance understanding of the cardiovascular risk factors in hemophilia patients and may also provide new knowledge in understanding the thrombosis risk in dyslipidemia patients, and how to provide appropriate treatments by lowering thrombosis risk without causing higher bleeding risk.

With the successful completion of this project, researchers hope to elucidate an important role of LDL in improving hemostasis. They anticipate that this knowledge will pave the path to conceive new strategies to balance the bleeding risk and cardiovascular risk in hemophilia A patients who receive prophylaxis treatment or gene therapy.