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Updated: Jul 20, 2025

Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies
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Beta-thalassemia: is cure still a dream?

Mohammad H Hodroj1, Nathalie Akiki1, Rayan Bou-Fakhredin2

  • 1Division of Hematology and Oncology, Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.

Minerva Medica
|August 3, 2023
PubMed

β-thalassemia is a monogenic disorder characterized by decreased hemoglobin production, resulting in chronic anemia. There are several factors affecting the clinical presentation of patients with β-thalassemia, and several complications such as iron overload or ineffective erythropoiesis have been linked to this disease. Until nowadays, several conservative therapies namely blood transfusions, iron chelation, and the FDA-approved drug Luspatercept have been adopted alongside other debatable permanent cures. Other clinical trials are being conducted to develop better and safer management techniques for these patients. This review will discuss the different treatment strategies of β-thalassemia including novel therapies, besides all possible curative therapies that are being developed for this disease.

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