Management of transfusion-dependent β-thalassaemia in the era of novel therapies: a prioritisation-based matrix for settings with limited resources.

Khaled M Musallam, Sujit Sheth, Maria Domenica Cappellini, Farrukh Shah, Stefano Rivella, Vijay G Sankaran, Kevin H M Kuo, Vip Viprakasit, Androulla Eleftheriou, Michael Angastiniotis, Franco Locatelli, Ali T Taher

Journal: The Lancet. Haematology 2026;13(1):e49-e54

PMID: 41482447

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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).

Address: Center for Research on Rare Blood Disorders and Thalassemia & Sickle Cell Center, Burjeel Cancer Institute, Burjeel Medical City, Abu Dhabi, United Arab Emirates; Department of Public Health & Epidemiology, Khalifa University, Abu Dhabi, United Arab Emirates; Division of Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA. Electronic address: [email protected].; Division of Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA.; Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan, Italy.; Center for Research on Rare Blood Disorders and Thalassemia & Sickle Cell Center, Burjeel Cancer Institute, Burjeel Medical City, Abu Dhabi, United Arab Emirates; Department of Medical Sciences, Khalifa University, Abu Dhabi, United Arab Emirates.; Division of Hematology, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Raymond G Perelman Center for Cellular and Molecular Therapeutics and Penn Center for Musculoskeletal Disorders, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Penn Institute for RNA Innovation, University of Pennsylvania, Philadelphia, PA, USA.; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA; Broad Institute of MIT and Harvard, Cambridge, MA, USA; Howard Hughes Medical Institute, Boston, MA, USA.; Division of Hematology, Department of Medicine, University of Toronto, Toronto, ON, Canada; Scarborough Health Network, Scarborough, Toronto, ON, Canada.; Department of Pediatrics, Siriraj Thalassemia Center, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.; Thalassaemia International Federation, Nicosia, Cyprus.; Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital, Rome, Italy; Catholic University of the Sacred Heart, Rome, Italy.; Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.

DOI: 10.1016/S2352-3026(25)00320-5

MeSH Terms: Humans, beta-Thalassemia, Blood Transfusion, Disease Management