Αlpha-thalassemia: A practical overview
Issued Date
2024-01-01
Resource Type
ISSN
0268960X
eISSN
15321681
Scopus ID
2-s2.0-85184718027
Pubmed ID
38182489
Journal Title
Blood Reviews
Rights Holder(s)
SCOPUS
Bibliographic Citation
Blood Reviews (2024)
Suggested Citation
Musallam K.M., Cappellini M.D., Coates T.D., Kuo K.H.M., Al-Samkari H., Sheth S., Viprakasit V., Taher A.T. Αlpha-thalassemia: A practical overview. Blood Reviews (2024). doi:10.1016/j.blre.2023.101165 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/97246
Title
Αlpha-thalassemia: A practical overview
Corresponding Author(s)
Other Contributor(s)
Abstract
α-Thalassemia is an inherited blood disorder characterized by decreased synthesis of α-globin chains that results in an imbalance of α and β globin and thus varying degrees of ineffective erythropoiesis, decreased red blood cell (RBC) survival, chronic hemolytic anemia, and subsequent comorbidities. Clinical presentation varies depending on the genotype, ranging from a silent or mild carrier state to severe, transfusion-dependent or lethal disease. Management of patients with α-thalassemia is primarily supportive, addressing either symptoms (eg, RBC transfusions for anemia), complications of the disease, or its transfusion-dependence (eg, chelation therapy for iron overload). Several novel therapies are also in development, including curative gene manipulation techniques and disease modifying agents that target ineffective erythropoiesis and chronic hemolytic anemia. This review of α-thalassemia and its various manifestations provides practical information for clinicians who practice beyond those regions where it is found with high frequency.