The CDC's Division of Blood Disorders is proud to offer this webinar series, providing evidence-based information on new research, emerging issues of interest in blood disorders, as well as innovative approaches to collaboration.
Bone Marrow Transplantation and Other Curative Approaches in Thalassemia
June 24, 2021 • 2:00–3:00 PM ET
Alexis A. Thompson, MD, MPH
Hematology Section Head, Watson and Sarah Armour Endowed Chair for Blood Diseases and Cancer, Ann & Robert H. Lurie Children’s Hospital of Chicago
Professor of Pediatrics, Northwestern University Feinberg School of Medicine
Associate Director for Equity and Minority Health, Robert H. Lurie Comprehensive Cancer Center and Northwestern University Feinberg School of Medicine
The thalassemia syndromes are a group of genetic blood disorders in which the production of one of the two different globin proteins (alpha or beta) that make up hemoglobin is reduced or absent. The result is anemia due to both ineffective erythropoiesis and chronic hemolysis, which typically begins in early childhood and lasts throughout life.
The clinical severity of thalassemia and interventions needed depend upon the globin chain affected and the type of genetic mutation. Persons with the most severe forms of thalassemia often require regular transfusions to treat the anemia and chelation therapy to prevent iron overload and associated sequelae. However, many patients with transfusion-dependent thalassemia (TDT) have struggled with adherence to the ongoing, intensive treatment regimen. Recent innovations, such as oral iron chelation agents, have helped improve adherence, and thus, prognoses (i.e., survival and quality of life in persons with TDT). Nonetheless, some patients and families are interested in definitive, potentially curative treatments.
In this webinar, Dr. Thompson will describe the considerations for stem cell transplantation, including patient characteristics, donor stem cell sources, and preparative regimens that impact clinical outcomes. She will provide an overview of genomic therapies, such as gene addition and gene editing, and review the results of clinical studies on thalassemia. While most of her presentation will focus on beta thalassemia, she will also describe curative approaches to alpha thalassemia.
- Describe the options for stem cell transplantation for thalassemia.
- List the key considerations in choosing curative therapies.
- Provide updates on advances in genomic therapies for thalassemia.
This webinar is free and open to public health professionals, clinicians, and researchers. Advance registration is required.
Please preregister here: http://bit.ly/ThalBMT
For more information please contact Cynthia Sayers: firstname.lastname@example.org