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Minerva Pediatrica 2018 Feb 23

DOI: 10.23736/S0026-4946.18.04872-7

Copyright © 2018 EDIZIONI MINERVA MEDICA

lingua: Inglese

Recent perspectives of pediatric β-thalassaemias

Qi AN, Conghai FAN , Shumei XU

Department of Hematology, Xuzhou Children’s Hospital, Xuzhou, Jiangsu, P.R. China


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β-thalassaemia is a potentially lethal hereditary anaemia, caused by reduced or absent expression of HBB polypeptide chains of adult haemoglobin (HbA: α2β2). Current curative treatments options are limited to few patients, while alternative, chronic palliative therapy consisting of frequent transfusions coupled with iron chelation therapy, are costly. The above treatments also affect quality of life of patients. A search was conducted in the electronic databases like medline, pubmed, etc. for screening studies reporting various aspects including gene therapy, prevention strategies, blood, transfusion and chelation therapy for the management of β-thalassaemia. Increased levels of fetal haemoglobin (HbF: α2γ2) were shown to lessen the severity of β-thalassaemia, highlighting the therapeutic potential of a gene-therapy-mediated increase in HBG1 and HBG2 (HBG) expression. The primary outcome of most of the above studies was the efficient management of β-thalassaemia, without any major complication. So, the present review is focused on the recent perspectives in the management of β-thalassaemia including combinatorial gene therapy for β-thalassaemia.


KEY WORDS: Thalassaemia - Pediatrics - Anemia - Gene therapy

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