AM24-SN-17-O: Sickle Cell Disease: Inching Towards Transfusion Independence. Exploring the Journey from Chronic Transfusions to the Novel Biotherapeutics (Enduring)

CABP CE Eligible

Sickle cell disease (SCD) is the most common inherited hemoglobinopathy that causes a variety of complications over the course of a patient’s life span. Hydroxyurea and chronic simple/exchange transfusions have been the cornerstone for management of SCD. For many decades, clinicians treating patients with SCD did not have many options for disease-modifying therapies that prevented complications of SCD. Newer FDA approved therapies for SCD, including L-glutamine, Voxelotor, and Crizanlizumab, focus on decreasing either hemolysis and the resulting anemia and thus aim at decreasing transfusion dependence. Therapies with a curative intent like stem cell transplantation are changing the landscape of management of Sickle Cell Disease. Newly approved Gene therapies in 2023 are the most revolutionary addition to the therapeutic armamentarium of Sickle Cell Disease. Blood Banking and Biotherapies professionals are at the centerstage of various treatments including chronic transfusions, red cell exchanges, stem cell transplantation and now gene therapies! This session engages expertise from faculty serving in varied roles as hematologists/oncologists and transplant physicians to blood banking professionals to explain the State-of-the-Art in the management of this chronic disabling disease as it slowly inches towards transfusion independence. The session will cover chronic transfusion protocols for sickle cell disease (SCD) and iron overload as a consequence of simple transfusion therapy without chelation. It will elaborate on the role of erythracytapheresis as an iron overload mitigation strategy and the overall best practices in chronic transfusion management of SCD. The session will next transition to therapeutic targets and new FDA-approved sickle cell disease modifying therapies which could lead to transfusion independence. The viral vector-based and gene editing approaches to treat sickle cell disease and the relative risks vs benefits of one approach over another will be discussed. The session will end with summarizing the current status and future direction for overall transfusion, transplantation as well as biotherapies-based management of Sickle Cell Disease.