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Beta Thalassemia: Monitoring and New Treatment Approaches

Eugene Khandros1, Janet L Kwiatkowski1

  • 1Division of Hematology, Children's Hospital of Philadelphia, 3401 Civic Center Boulevard, Colket Translational Research Building, Room 11024, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA, USA.

Hematology/Oncology Clinics of North America
|April 30, 2019
PubMed

Beta thalassemias are a significant global health problem. Globin chain imbalance leads to a complex physiologic cascade of hemolytic anemia, ineffective erythropoiesis, and iron overload. Management of the broad spectrum of phenotypes requires the careful use of red blood transfusions, supportive care, monitoring, and management of iron overload. In this article, the authors discuss recommendations for monitoring of individuals with thalassemia, as well as ongoing preclinical and clinical trials of therapies targeting different aspects of thalassemia pathophysiology.

Keywords:
Beta thalassemiaChelationGene therapyIron overload

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