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

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 req...

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Main Authors: Hiba Tariq Wally, Muhammad Hassan Zulfi, Elahi Sana Jilani
Format: Article
Language:English
Published: Pakistan Medical Association 2023-09-01
Series:Journal of the Pakistan Medical Association
Online Access:https://ojs.jpma.org.pk/index.php/public_html/article/view/9532
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author Hiba Tariq Wally
Muhammad Hassan Zulfi
Elahi Sana Jilani
author_facet Hiba Tariq Wally
Muhammad Hassan Zulfi
Elahi Sana Jilani
author_sort Hiba Tariq Wally
collection DOAJ
description 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 Continue...
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spelling doaj.art-a79577966e4d433ea2cc75fe39d799802023-09-15T04:52:50ZengPakistan Medical AssociationJournal of the Pakistan Medical Association0030-99822023-09-01731010.47391/JPMA.9532Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemiaHiba Tariq Wally0Muhammad Hassan Zulfi1Elahi Sana Jilani2Department of Orthopaedic Surgery, Jinnah Postgraduate Medical Centre, Karachi, PakistanDepartment of Orthopaedic Surgery, Jinnah Postgraduate Medical Centre, Karachi, PakistanDepartment of Orthopaedic Surgery, Jinnah Postgraduate Medical Centre, Karachi, Pakistan 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 Continue... https://ojs.jpma.org.pk/index.php/public_html/article/view/9532
spellingShingle Hiba Tariq Wally
Muhammad Hassan Zulfi
Elahi Sana Jilani
Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
Journal of the Pakistan Medical Association
title Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
title_full Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
title_fullStr Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
title_full_unstemmed Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
title_short Betibeglogene Autotemcel; A new hope for transfusion dependent beta-thalassaemia
title_sort betibeglogene autotemcel a new hope for transfusion dependent beta thalassaemia
url https://ojs.jpma.org.pk/index.php/public_html/article/view/9532
work_keys_str_mv AT hibatariqwally betibeglogeneautotemcelanewhopefortransfusiondependentbetathalassaemia
AT muhammadhassanzulfi betibeglogeneautotemcelanewhopefortransfusiondependentbetathalassaemia
AT elahisanajilani betibeglogeneautotemcelanewhopefortransfusiondependentbetathalassaemia