Sickle cell disease (SCD) is an inherited condition that affects about 100,000 individuals in the United States. This disorder is familiar to the transfusion medicine professionals (Physicians; Technologists) since it relies heavily on transfusion of blood products - either via simple transfusion or RBC exchange. In recent years (2017-2019), newer potentially disease-modifying options were identified and approved by the FDA for use including l-glutamin, anyipolomerization agents (voxelotor) and P-selectin inhibitors (crizanlizumab). Furthermore, gene therapy might be a real option for cure of the disease. This session will concentrate on the education of Transfusion Medicine professionals on these advances and their possible implications for clinical practice and subsequent potential effect on the transfusion service.
Describe the recent advances in gene therapy as a potential cure for Sickle Cell Disease (SCD)
Summarize the newly approved drugs for SCD and their respective mechanism of action.
Recognize the advances in the practice of RBC exchange for SCD - indications as well as desired parameters ( Hematocrit ; FCR)