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Advances in the management of α-thalassemia major: reasons to be optimistic

10 Dec 2021

Abstract excerpt

α-Thalassemia major (ATM) is a severe disease resulting from deletions in all 4 copies of the α-globin gene. Although it is usually fatal before birth, the advent of in utero transfusions has enabled survival of a growing number of children. Postnatal therapy consists of chronic transfusions or stem cell transplantation, similar to patients with β-thalassemia major. In this review, we discuss the experience with...

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PMID: 34889445
DOI: 10.1182/hematology.2021000295

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