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Optimizing Therapy in Sickle Cell Disease

PhD Trainee
Program: Education Program
Saturday, December 5, 2015: 9:30 AM-11:00 AM
Chapin Theater (W320), Level 3 (Orange County Convention Center)
Sunday, December 6, 2015: 9:30 AM-11:00 AM
Hall E1, Level 2 (Orange County Convention Center)

Description:

Sickle cell disease is a severe hemoglobinopathy caused by a single nucleotide substitution in the beta globin gene. This change results in the production of an abnormal hemoglobin that forms polymers when red cells are deoxygenated. Red cells that contain substantial amounts of HbS polymer are not deformable, and therefore lodge in the end-arterioles and capillaries causing vaso-occlusive crisis and tissue infarction. The session will focus on evidenced-based, contemporary and investigative approaches to prevent or delay these pathological processes.

Dr. Sophie Lanzkron will provide the audience with an understanding of the pain experience of adults living with sickle cell disease. In addition the role of neurocognitive dysfunction, renal disease and venous thromboembolism will be reviewed. Finally, the risk of red cell alloimmunization and the appropriate use of red cell transfusions will be discussed.

Dr. Russell E. Ware will focus on current evidence and clinical experience regarding the optimal use of hydroxyurea for sickle cell anemia. Hydroxyurea has proven efficacy as a disease-modifying treatment for sickle cell anemia, but is currently under-utilized in clinical practice. To improve the effectiveness of hydroxyurea therapy, he will discuss how efforts should be directed toward increasing the indications, dosage, and benefits of treatment.

Dr. Joshua J. Field will discuss the role of selectin and invariant NKT (iNKT) cell-based therapies for the treatment of sickle cell disease. In pre-clinical models, vascular occlusion is reduced when selectin or iNKT cell activity is blocked. Based on these data, two selectin and two iNKT cell-based therapies have been developed.

Chair:
Griffin P. Rodgers, MD, National Institutes of Health

Disclosures:
No relevant conflicts of interest to declare.

Sophie Lanzkron, MD, MHS

Johns Hopkins School of Medicine, Baltimore, MD

Russell E. Ware, MD, PhD

Cincinnati Children's Hospital Medical Center, Cincinnati, OH

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