Future-proofing thalassaemia care: harnessing scalable therapies, artificial intelligence, and global collaboration.
TL;DR
The emerging role of repurposed low-cost therapies, particularly hydroxyurea and thalidomide, as disease-modifying options in resource-limited settings are discussed and opportunities to democratise access to curative gene therapies through technological developments, decentralised manufacturing, regional collaborations, and simplified treatment platforms are examined.
Abstract
Advances in the treatment of thalassaemia have led to major improvements in survival and quality of life over the past 30 years. In addition to developments in stem-cell transplantation, transfusion safety, iron overload monitoring, and iron-chelation therapy, novel disease-modifying and gene therapies are also emerging. However, substantial disparities in access to specialised care and novel therapies persist, particularly in low-income and middle-income countries, where most people with thalassaemia reside. This Series paper explores complementary strategies aimed at improving global thalassaemia outcomes beyond conventional therapeutic innovation. We discuss the emerging role of repurposed low-cost therapies, particularly hydroxyurea and thalidomide, as disease-modifying options in resource-limited settings. We further examine opportunities to democratise access to curative gene therapies through technological developments, decentralised manufacturing, regional collaborations, and simplified treatment platforms. In addition, the rapidly expanding role of artificial intelligence in screening, diagnosis, prediction of complications, and remote care delivery is reviewed. Finally, we highlight the importance of international collaborations, multidisciplinary training, and research networks in strengthening equitable global thalassaemia care.