Management of transfusion-dependent β-thalassaemia in the era of novel therapies: a prioritisation-based matrix for settings with limited resources
Issued Date
2026-01-01
Resource Type
ISSN
24519960
eISSN
23523026
Scopus ID
2-s2.0-105026232475
Pubmed ID
41482447
Journal Title
Lancet Haematology
Volume
13
Issue
1
Start Page
e49
End Page
e54
Rights Holder(s)
SCOPUS
Bibliographic Citation
Lancet Haematology Vol.13 No.1 (2026) , e49-e54
Suggested Citation
Musallam K.M., Sheth S., Cappellini M.D., Shah F., Rivella S., Sankaran V.G., Kuo K.H.M., Viprakasit V., Eleftheriou A., Angastiniotis M., Locatelli F., Taher A.T. Management of transfusion-dependent β-thalassaemia in the era of novel therapies: a prioritisation-based matrix for settings with limited resources. Lancet Haematology Vol.13 No.1 (2026) , e49-e54. e54. doi:10.1016/S2352-3026(25)00320-5 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/114521
Title
Management of transfusion-dependent β-thalassaemia in the era of novel therapies: a prioritisation-based matrix for settings with limited resources
Author's Affiliation
Harvard Medical School
University of Pennsylvania
University of Toronto Faculty of Medicine
University of Pennsylvania Perelman School of Medicine
Weill Cornell Medicine
Boston Children's Hospital
Howard Hughes Medical Institute
The Children's Hospital of Philadelphia
Università Cattolica del Sacro Cuore, Campus di Roma
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
American University of Beirut
Khalifa University
Broad Institute
IRCCS Ospedale Pediatrico Bambino Gesù
Siriraj Hospital
Burjeel Medical City
Scarborough Health Network
Thalassaemia International Federation
University of Pennsylvania
University of Toronto Faculty of Medicine
University of Pennsylvania Perelman School of Medicine
Weill Cornell Medicine
Boston Children's Hospital
Howard Hughes Medical Institute
The Children's Hospital of Philadelphia
Università Cattolica del Sacro Cuore, Campus di Roma
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
American University of Beirut
Khalifa University
Broad Institute
IRCCS Ospedale Pediatrico Bambino Gesù
Siriraj Hospital
Burjeel Medical City
Scarborough Health Network
Thalassaemia International Federation
Corresponding Author(s)
Other Contributor(s)
Abstract
β-thalassaemia is an inherited haemoglobinopathy characterised by ineffective erythropoiesis and chronic anaemia of varying severity, which is predominant in the region extending from the Mediterranean basin and Middle East towards southeast Asia. Patients with severe phenotypes require lifelong transfusions, iron overload monitoring, and chelation. Suboptimal management due to access challenges continues to be directly linked to increased morbidity and mortality in many regions. In the past few decades, an improved understanding of the underlying pathogenesis of β-thalassaemia has led to the development of several disease-modifying therapies and curative gene manipulation techniques. However, global disparities in access and the need for specialised expertise hinder their wide implementation, especially in resource-limited countries where more than 80% of patients live. Uncertainty about which biomarkers can predict patient response further complicates the selection of patients for treatment. Beyond the need for access programmes and pragmatic national health policies, patient prioritisation by treating physicians, informed by available evidence and expert opinion, is crucial for ensuring that a resource-cautious management approach is implemented. This Viewpoint provides a decision matrix to prioritise interventions by need, benefit, and risk in settings with inadequate access, and to identify alternatives when standard options are unavailable. It draws on the Thalassaemia International Federation guidelines, best available trial and real-world evidence, and expert consensus from virtual discussions among the authors (haematologists, bone marrow transplantation physicians, patient group representatives, translational scientists, and trialists).