Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia

Abstract

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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