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Title: Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
Authors: Hiba Tariq Wally, Muhammad Hassan Zulfi, Elahi Sana Jilani
Journal: Journal of Pakistan Medical Association
Publisher: Pakistan Medical Association.
Country: Pakistan
Year: 2023
Volume: 73
Issue: 10
Language: en
DOI: 10.47391/JPMA.9532
Respected Editor, Beta thalassaemia is an autosomal recessive disorder that leads to abnormal production of functional adult haemoglobin because of either inadequate or absent production of the B globin chain (beta+/beta0).1 Transfusion-dependent thalassemia (TDT) is a rare and most severe form requiring life-long blood transfusions. To mitigate the effects of TDT, life-long blood transfusions every 2-5 weeks are required, which can elicit transfusion-based reactions and lead to an iron overload state, which can further cause widespread organ damage despite the use of iron chelation therapy.2
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