IMR Press / FBE / Volume 14 / Issue 3 / DOI: 10.31083/j.fbe1403018
Open Access Review
Benefits and Risks in Polypathology and Polypharmacotherapy Challenges in the Era of the Transition of Thalassaemia from a Fatal to a Chronic or Curable Disease
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1 Postgraduate Research Institute of Science, Technology, Environment and Medicine Limassol, 3021 Limassol, Cyprus
*Correspondence: (George J. Kontoghiorghes)
Academic Editor: Alessandro Poggi
Front. Biosci. (Elite Ed) 2022, 14(3), 18;
Submitted: 18 February 2022 | Revised: 25 April 2022 | Accepted: 26 April 2022 | Published: 12 July 2022
Copyright: © 2022 The Author(s). Published by IMR Press.
This is an open access article under the CC BY 4.0 license.

Beta thalassaemia major (TM), a potentially fatal haemoglobinopathy, has transformed from a fatal to a chronic disease in the last 30 years following the introduction of effective, personalised iron chelation protocols, in particular the use of oral deferiprone, which is most effective in the removal of excess iron from the heart. This transition in TM has been achieved by the accessibility to combination therapy with the other chelating drugs deferoxamine and deferasirox but also therapeutic advances in the treatment of related co-morbidities. The transition and design of effective personalised chelation protocols was facilitated by the development of new non-invasive diagnostic techniques for monitoring iron removal such as MRI T2*. Despite this progress, the transition in TM is mainly observed in developed countries, but not globally. Similarly, potential cures of TM with haemopoietic stem cell transplantation and gene therapy are available to selected TM patients but potentially carry high risk of toxicity. A global strategy is required for the transition efforts to become available for all TM patients worldwide. The same strategy could also benefit many other categories of transfusional iron loaded patients including other thalassaemias, sickle cell anaemia, myelodysplasia and leukaemia patients.

thalassaemia major
iron overload
iron toxicity
organ damage
Fig. 1.
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