denotes an abstract that is clinically relevant.
denotes that this is a recommended PHD Trainee Session.
denotes that this is a ticketed session.Hematology Disease Topics & Pathways:
Research, Sickle Cell Disease, Clinical Practice (Health Services and Quality), Translational Research, CHIP, Genetic Disorders, Clinical Research, health outcomes research, Diversity, Equity, and Inclusion (DEI) , Hemoglobinopathies, Diseases, Biological Processes
Description:
Sickle cell disease is associated with extensive morbidity and early mortality. Adults with heart, lung, and kidney damage are at exceptionally high risk of dying early. Individuals with SCD are also at an increased risk of developing leukemia. Hematopoietic cell transplant offers a curative option for patients with SCD. While new data are emerging, insufficient data exist to systematically explore whether HCT improves, stabilizes, or worsens organ function in patients with SCD. Further, some individuals with SCD are at an increased risk of secondary malignancies in specific curative therapy settings.
Dr. Courtney Fitzhugh will discuss an unexpectedly high incidence of myeloid malignancies after graft failure and lentivirus-based gene therapy employing busulfan for SCD. She will also discuss the importance of identifying genetic risk factors and performing a benefit/risk assessment to help patients identify the best individualized curative approach.
Dr. Shalini Shenoy will review the indications for curative therapy in patients with SCD and the impact of curative therapies on those indications. She will focus on measures tracking improvements in disease symptoms and outcomes in the first two years after curative interventions.
Dr. Debra Friedman will describe what is known about the long-term health effects of curative therapy for SCD with a focus on the heart, lung, kidney, and reproductive systems. She will also stress the need for continued research on the long-term health outcomes following curative treatments for SCD.
Dr. Courtney Fitzhugh will discuss an unexpectedly high incidence of myeloid malignancies after graft failure and lentivirus-based gene therapy employing busulfan for SCD. She will also discuss the importance of identifying genetic risk factors and performing a benefit/risk assessment to help patients identify the best individualized curative approach.
Dr. Shalini Shenoy will review the indications for curative therapy in patients with SCD and the impact of curative therapies on those indications. She will focus on measures tracking improvements in disease symptoms and outcomes in the first two years after curative interventions.
Dr. Debra Friedman will describe what is known about the long-term health effects of curative therapy for SCD with a focus on the heart, lung, kidney, and reproductive systems. She will also stress the need for continued research on the long-term health outcomes following curative treatments for SCD.