Updates in Thalassemia: New Challenges and Novel Treatment Approaches

Speakers: Alexis Thompson, MD, and Sujit Sheth, MD

Description:

For general hematologists with a foundational understanding of thalassemia, this episode discusses the changing demographics due to immigration and refugee resettlement. It covers the diagnosis process from newborn screening to older individuals, emphasizing genetic testing due to numerous mutations and varied phenotypes. The importance of correctly diagnosing older individuals previously misdiagnosed with iron deficiency refractory to treatment is highlighted. The episode also addresses regular transfusions, iron chelation, and monitoring for complications, along with advances in blood supply safety, reduced alloimmunization risks through extended typing, and MRI monitoring. Finally, novel agents in practice and development, including luspatercept, PK activators, and gene therapy, will be explored.
 

Learning Objectives:

1. Understand the changing landscape of thalassemia in the US. 
2. Revisit the state-of-the-art diagnostic and treatment approaches. 
3. Learn about novel therapeutics in the thalassemia field