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A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?

Cappellini, MD; Porter, JB; Viprakasit, V; Taher, AT; (2018) A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies? Blood Reviews , 32 (4) pp. 300-311. 10.1016/j.blre.2018.02.001. Green open access

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Abstract

Beta-thalassaemia causes defective haemoglobin synthesis leading to ineffective erythropoiesis, chronic haemolytic anaemia, and subsequent clinical complications. Blood transfusion and iron chelation allow long-term disease control, and haematopoietic stem cell transplantation offers a potential cure for some patients. Nonetheless, there are still many challenges in the management of beta-thalassaemia. The main treatment option for most patients is supportive care; furthermore, the long-term efficacy and safety of current therapeutic strategies are limited and adherence is suboptimal. An increasing understanding of the underlying molecular and cellular disease mechanisms plus an awareness of limitations of current management strategies are driving research into novel therapeutic options. Here we provide an overview of the current pathophysiology, clinical manifestations, and global burden of beta-thalassaemia. We reflect on what has been achieved to date, describe the challenges associated with currently available therapy, and discuss how these issues might be addressed by novel therapeutic approaches in development.

Type: Article
Title: A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.blre.2018.02.001
Publisher version: http://doi.org/10.1016/j.blre.2018.02.001
Language: English
Additional information: Copyright © 2018 The Authors. Published by Elsevier Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/BY-NC-ND/4.0/).
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/10050298
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