September 27, 2024
In honor of Sickle Cell Disease Awareness Month, recognized each September, AABB spoke with members of the blood and biotherapies community who are striving to improve our understanding of sickle cell disease and advance patient care.
Julia Z. Xu, MD, MScGH is an adult hematologist and an assistant professor of medicine in the University of Pittsburgh School of Medicine, specializing in the care of patients with sickle cell disease (SCD). Her research focuses on new drug therapies and biomarkers for improving anemia and other chronic complications of SCD. She is dedicated to improving anemia-related outcomes for individuals living with SCD in the U.S. and globally by optimizing treatment strategies for anemia and expanding global access to treatments.
Xu earned her Doctor of Medicine at the Columbia University College of Physicians and Surgeons. She continued her training with internal medicine-global health residency at Duke University School of Medicine, and a hematology fellowship at National Institutes of Health.
In 2023, Xu received an AABB Foundation Early-Career Scientific Research Grant to study the effect of transfusion modality on hemorheology and vascular function in SCD.
Sickle cell disease is not only a tremendous global burden but also a devastating disease that carries a high burden of symptoms, greatly affecting patients’ quality of life from childhood through adulthood. Acute episodes of pain and chronic anemia are the hallmarks of the disease, and transfusion therapy is one of the oldest and most effective treatments for sickle cell disease, yet we have a limited understanding of how to best leverage transfusion therapy to improve outcomes for patients with SCD.
My research compares the effects of simple red blood cell (RBC) transfusion versus RBC exchange transfusion on hemorheology, or factors affecting blood flow, such as blood viscosity and tissue oxygenation, in SCD. This is an important clinical question because although anemia causes hypoxia and poor quality of life in SCD, raising the hemoglobin level too high may increase the blood viscosity and paradoxically cause increased vaso-occlusion, decreased tissue perfusion and other negative effects. My research will help demonstrate the effect of simple transfusions versus exchange transfusions on hemorheology, answering critical questions on the selection of transfusion modality in SCD and helping to develop potential biomarker tools for optimizing transfusion management in SCD.
Once we understand the effect of different transfusion modalities on anemia in SCD, I would like to compare these transfusion strategies with approved and emerging drug therapies. Additionally, SCD is a very heterogeneous disease, and one treatment does not work for everyone. It is very important to find a way to select optimal therapies for patients, and biomarkers can help personalize therapy. I hope to identify hemorheological biomarkers that can tell us which patients will respond best to transfusion therapy versus drug therapy so that we can improve treatment success in the future.
We are currently in an extremely exciting time for the blood and biotherapies field, with rapid therapeutic development and the use of multi-omic and machine learning approaches to gain a deeper understanding of cellular biology than ever before. With these scientific advances, there is so much more to be done, such as translating new discoveries from bench to bedside and expanding access to transfusion therapy and biotherapies globally. Be strategic – develop a biological, clinical or methodological niche, use early-career funding to lay a foundation for your future grants, and have a vision of the impact you want to have on your field, as this will help give you direction and focus.
With the rapid scientific advancement and therapeutic development that the blood and biotherapies field is experiencing, we are more in need than ever of growing a pipeline of investigators who can continue moving the field forward. Early-career funding from the AABB Foundation is critical for allowing early-career investigators to pursue exciting new research directions and establish their research careers. The support I have received from the AABB Foundation has allowed me to set up my laboratory and conduct a key prospective study on transfusion therapy in SCD that will shed light on important clinical questions and lead to future studies. This award has made a big difference in setting me up for a successful academic career, and I am extremely grateful for this support.