The expanding therapeutic landscape of β-thalassaemia. (PubMed, Lancet Haematol) - Oct 7, 2026 - "Over the past decade, the therapeutic landscape has expanded rapidly, with disease-modifying agents targeting erythroid maturation and red cell metabolism, including luspatercept and mitapivat, now available as options for both transfusion-dependent and non-transfusion-dependent β-thalassaemia. In parallel, gene addition and gene editing therapies, particularly beti-cel and exa-cel, have established proof of curative potential independent of donor availability. However, optimal patient selection, response definitions, long-term outcome assessment, affordability, and equitable global access remain central challenges for wide implementation. In this Series paper, we discuss advances in β-thalassaemia treatment over the past decade, including newly approved and late-stage therapies and their future implications, and highlight remaining research gaps."
Journal • Review • Beta-Thalassemia • Hematological Disorders • Transplantation
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Ali T Taher; Andreas Glenthøj; Antonis Kattamis; Farrukh T Shah; Franco Locatelli; Gian Luca Forni; Janet L Kwiatkowski; Kevin H M Kuo; Khaled M Musallam; Maria Domenica Cappellini; Stefano Rivella; Sujit Sheth; Yesim Aydinok
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