{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T12:15:54Z","timestamp":1791375354458,"version":"4.3.1"},"reference-count":106,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T00:00:00Z","timestamp":1790812800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","thelancet.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["The Lancet Haematology"],"published-print":{"date-parts":[[2026,10]]},"DOI":"10.1016\/s2352-3026(26)00173-0","type":"journal-article","created":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T23:06:05Z","timestamp":1791327965000},"update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":1,"title":["The expanding therapeutic landscape of \u03b2-thalassaemia"],"prefix":"10.1016","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3935-903X","authenticated-orcid":false,"given":"Khaled M","family":"Musallam","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7640-1244","authenticated-orcid":false,"given":"Sujit","family":"Sheth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7976-3654","authenticated-orcid":false,"given":"Franco","family":"Locatelli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5975-1309","authenticated-orcid":false,"given":"Farrukh T","family":"Shah","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0938-6558","authenticated-orcid":false,"given":"Stefano","family":"Rivella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6744-9238","authenticated-orcid":false,"given":"Kevin H M","family":"Kuo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9833-1016","authenticated-orcid":false,"given":"Gian Luca","family":"Forni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5178-0655","authenticated-orcid":false,"given":"Antonis","family":"Kattamis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6194-6826","authenticated-orcid":false,"given":"Yesim","family":"Aydinok","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2082-0738","authenticated-orcid":false,"given":"Andreas","family":"Glenth\u00f8j","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7103-3406","authenticated-orcid":false,"given":"Janet L","family":"Kwiatkowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8676-6864","authenticated-orcid":false,"given":"Maria Domenica","family":"Cappellini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8515-2238","authenticated-orcid":false,"given":"Ali T","family":"Taher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/S2352-3026(26)00173-0_bib1","doi-asserted-by":"crossref","first-page":"2310","DOI":"10.1016\/S0140-6736(22)00536-0","article-title":"Thalassaemia","volume":"399","author":"Kattamis","year":"2022","journal-title":"Lancet"},{"key":"10.1016\/S2352-3026(26)00173-0_bib2","doi-asserted-by":"crossref","first-page":"727","DOI":"10.1056\/NEJMra2021838","article-title":"\u03b2-Thalassemias","volume":"384","author":"Taher","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib3","series-title":"Guidelines for the management of non-transfusion-dependent \u03b2-thalassaemia","author":"Taher","year":"2023"},{"key":"10.1016\/S2352-3026(26)00173-0_bib4","series-title":"Guidelines for the management of transfusion-dependent \u03b2-thalassaemia","author":"Taher","year":"2025"},{"key":"10.1016\/S2352-3026(26)00173-0_bib5","doi-asserted-by":"crossref","first-page":"1886","DOI":"10.1182\/blood-2009-09-243154","article-title":"Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study","volume":"115","author":"Taher","year":"2010","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib6","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1182\/blood-2018-07-815928","article-title":"Iron metabolism under conditions of ineffective erythropoiesis in \u03b2-thalassemia","volume":"133","author":"Rivella","year":"2019","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib7","doi-asserted-by":"crossref","first-page":"E78","DOI":"10.1002\/ajh.26428","article-title":"Risk of mortality from anemia and iron overload in nontransfusion-dependent \u03b2-thalassemia","volume":"97","author":"Musallam","year":"2022","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib8","doi-asserted-by":"crossref","first-page":"e218","DOI":"10.3324\/haematol.2013.097220","article-title":"Serum ferritin level and morbidity risk in transfusion-independent patients with \u03b2-thalassemia intermedia: the ORIENT study","volume":"99","author":"Musallam","year":"2014","journal-title":"Haematologica"},{"key":"10.1016\/S2352-3026(26)00173-0_bib9","doi-asserted-by":"crossref","first-page":"1605","DOI":"10.3324\/haematol.2011.047852","article-title":"Elevated liver iron concentration is a marker of increased morbidity in patients with \u03b2 thalassemia intermedia","volume":"96","author":"Musallam","year":"2011","journal-title":"Haematologica"},{"key":"10.1016\/S2352-3026(26)00173-0_bib10","doi-asserted-by":"crossref","first-page":"970","DOI":"10.1182\/blood-2012-02-412692","article-title":"Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study","volume":"120","author":"Taher","year":"2012","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib11","doi-asserted-by":"crossref","first-page":"1578","DOI":"10.1111\/bjh.70047","article-title":"Impact of lifetime anaemia and iron control on outcomes in \u03b2-thalassaemia: data from the longitudinal de-LIGHT study","volume":"207","author":"Musallam","year":"2025","journal-title":"Br J Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib12","doi-asserted-by":"crossref","first-page":"2489","DOI":"10.3324\/haematol.2021.278684","article-title":"Survival and causes of death in 2,033 patients with non-transfusion-dependent \u03b2-thalassemia","volume":"106","author":"Musallam","year":"2021","journal-title":"Haematologica"},{"key":"10.1016\/S2352-3026(26)00173-0_bib13","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1007\/s00277-020-04370-2","article-title":"Morbidity-free survival and hemoglobin level in non-transfusion-dependent \u03b2-thalassemia: a 10-year cohort study","volume":"101","author":"Musallam","year":"2022","journal-title":"Ann Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib14","doi-asserted-by":"crossref","first-page":"1903","DOI":"10.1007\/s00277-021-04456-5","article-title":"Variations in hemoglobin level and morbidity burden in non-transfusion-dependent \u03b2-thalassemia","volume":"100","author":"Musallam","year":"2021","journal-title":"Ann Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib15","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1038\/s41598-026-46504-y","article-title":"Efficacy and safety of thalidomide in \u03b2-thalassaemia: a systematic review and meta-analysis","volume":"16","author":"Yasara","year":"2026","journal-title":"Sci Rep"},{"key":"10.1016\/S2352-3026(26)00173-0_bib16","article-title":"Efficacy and safety of hydroxyurea therapy on patients with \u03b2-thalassemia: a systematic review and meta-analysis","volume":"11","author":"Huang","year":"2025","journal-title":"Front Med (Lausanne)"},{"key":"10.1016\/S2352-3026(26)00173-0_bib17","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1002\/ajh.27194","article-title":"\u2018Phenoconversion\u2019 in adult patients with \u03b2-thalassemia","volume":"99","author":"Musallam","year":"2024","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib18","doi-asserted-by":"crossref","DOI":"10.1016\/j.blre.2023.101138","article-title":"Management of transfusion-dependent \u03b2-thalassemia (TDT): expert insights and practical overview from the Middle East","volume":"63","author":"El-Beshlawy","year":"2024","journal-title":"Blood Rev"},{"key":"10.1016\/S2352-3026(26)00173-0_bib19","doi-asserted-by":"crossref","first-page":"930","DOI":"10.1182\/blood.2023022460","article-title":"Pretransfusion hemoglobin level and mortality in adults with transfusion-dependent \u03b2-thalassemia","volume":"143","author":"Musallam","year":"2024","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib20","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1002\/ajh.70100","article-title":"Quantifying morbidity risk attributed to red-cell transfusion volume in optimally transfused patients with \u03b2-thalassemia","volume":"101","author":"Musallam","year":"2026","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib21","doi-asserted-by":"crossref","first-page":"381","DOI":"10.1002\/ajh.26798","article-title":"Overall and complication-free survival in a large cohort of patients with \u03b2-thalassemia major followed over 50\u2008years","volume":"98","author":"Forni","year":"2023","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib22","doi-asserted-by":"crossref","first-page":"e134","DOI":"10.1016\/S2214-109X(21)00446-0","article-title":"Survival and complications in patients with haemoglobin E thalassaemia in Sri Lanka: a prospective, longitudinal cohort study","volume":"10","author":"Premawardhena","year":"2022","journal-title":"Lancet Glob Health"},{"key":"10.1016\/S2352-3026(26)00173-0_bib23","doi-asserted-by":"crossref","first-page":"E129","DOI":"10.1002\/ajh.25429","article-title":"Clinical and economic burden of regularly transfused adult patients with \u03b2-thalassemia in the United States: a retrospective cohort study using payer claims","volume":"94","author":"Weiss","year":"2019","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib24","doi-asserted-by":"crossref","DOI":"10.1177\/20406207241270872","article-title":"Systematic literature review of the indirect costs and humanistic burden of \u03b2-thalassemia","volume":"15","author":"Aydinok","year":"2024","journal-title":"Ther Adv Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib25","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1038\/bmt.2015.293","article-title":"Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000\u20132010","volume":"51","author":"Baronciani","year":"2016","journal-title":"Bone Marrow Transplant"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib26","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1182\/blood-2018-168","article-title":"Hematopoietic cell transplantation in thalassemia and sickle cell disease: report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000\u20132017","volume":"132","author":"Baronciani","year":"2018","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib27","doi-asserted-by":"crossref","first-page":"2562","DOI":"10.1182\/bloodadvances.2019000291","article-title":"Related and unrelated donor transplantation for \u03b2-thalassemia major: results of an international survey","volume":"3","author":"Li","year":"2019","journal-title":"Blood Adv"},{"key":"10.1016\/S2352-3026(26)00173-0_bib28","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/j.hoc.2022.12.009","article-title":"Hematopoietic stem cell transplantation in thalassemia","volume":"37","author":"Algeri","year":"2023","journal-title":"Hematol Oncol Clin North Am"},{"key":"10.1016\/S2352-3026(26)00173-0_bib29","doi-asserted-by":"crossref","first-page":"1356","DOI":"10.1002\/ajh.24914","article-title":"Safety and pharmacokinetics of the oral iron chelator SP-420 in \u03b2-thalassemia","volume":"92","author":"Taher","year":"2017","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib30","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1038\/nm.3512","article-title":"Transforming growth factor-\u03b2 superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis","volume":"20","author":"Suragani","year":"2014","journal-title":"Nat Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib31","doi-asserted-by":"crossref","first-page":"3864","DOI":"10.1182\/blood-2013-06-511238","article-title":"Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine \u03b2-thalassemia","volume":"123","author":"Suragani","year":"2014","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib32","doi-asserted-by":"crossref","first-page":"6162","DOI":"10.1111\/jcmm.15243","article-title":"Smad2\/3-pathway ligand trap luspatercept enhances erythroid differentiation in murine \u03b2-thalassaemia by increasing GATA-1 availability","volume":"24","author":"Martinez","year":"2020","journal-title":"J Cell Mol Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib33","doi-asserted-by":"crossref","first-page":"477","DOI":"10.3324\/haematol.2018.198887","article-title":"Sotatercept, a novel transforming growth factor \u03b2 ligand trap, improves anemia in \u03b2-thalassemia: a phase II, open-label, dose-finding study","volume":"104","author":"Cappellini","year":"2019","journal-title":"Haematologica"},{"key":"10.1016\/S2352-3026(26)00173-0_bib34","doi-asserted-by":"crossref","first-page":"766","DOI":"10.1002\/ajh.23732","article-title":"A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers","volume":"89","author":"Attie","year":"2014","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib35","doi-asserted-by":"crossref","DOI":"10.1177\/20406207221134404","article-title":"Long-term safety and erythroid response with luspatercept treatment in patients with \u03b2-thalassemia","volume":"13","author":"Piga","year":"2022","journal-title":"Ther Adv Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib36","doi-asserted-by":"crossref","first-page":"1219","DOI":"10.1056\/NEJMoa1910182","article-title":"A phase 3 trial of luspatercept in patients with transfusion-dependent \u03b2-thalassemia","volume":"382","author":"Cappellini","year":"2020","journal-title":"N Engl J Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib37","doi-asserted-by":"crossref","first-page":"1010","DOI":"10.1016\/S0140-6736(26)00874-3","article-title":"Efficacy and safety of mitapivat in adults with transfusion-dependent \u03b1-thalassaemia or \u03b2-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial","volume":"408","author":"Cappellini","year":"2026","journal-title":"Lancet"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib38","doi-asserted-by":"crossref","first-page":"4699","DOI":"10.1182\/blood-2025-4699","article-title":"Efficacy of mitapivat in patients with transfusion-dependent alpha-thalassemia: subgroup analysis from the ENERGIZE-T trial","volume":"146","author":"Lal","year":"2025","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib39","doi-asserted-by":"crossref","first-page":"e733","DOI":"10.1016\/S2352-3026(22)00208-3","article-title":"Luspatercept for the treatment of anaemia in non-transfusion-dependent \u03b2-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial","volume":"9","author":"Taher","year":"2022","journal-title":"Lancet Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib40","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/S0140-6736(25)00635-X","article-title":"Mitapivat in adults with non-transfusion-dependent \u03b1-thalassaemia or \u03b2-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial","volume":"406","author":"Taher","year":"2025","journal-title":"Lancet"},{"key":"10.1016\/S2352-3026(26)00173-0_bib41","doi-asserted-by":"crossref","first-page":"e180","DOI":"10.1016\/S2352-3026(24)00376-4","article-title":"Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent \u03b2-thalassaemia (BELIEVE): final results from a phase 3 randomised trial","volume":"12","author":"Cappellini","year":"2025","journal-title":"Lancet Haematol"},{"issue":"suppl 2","key":"10.1016\/S2352-3026(26)00173-0_bib42","first-page":"631","article-title":"EP1306: benefit of continuing therapy with luspatercept in patients with beta-thalassemia who do not achieve \u226533% reduction in red blood cell (RBC) transfusion burden (TB) in weeks 13\u201324 in the BELIEVE trial","volume":"5","author":"Piga","year":"2021","journal-title":"HemaSphere"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib43","first-page":"2816","article-title":"P1522: characterizing patterns of transfusion burden (TB) reduction in patients (pts) with transfusion-dependent (TD) beta-thalassemia treated with luspatercept in the BELIEVE Trial","volume":"8","author":"Kuo","year":"2023","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib44","doi-asserted-by":"crossref","first-page":"1651","DOI":"10.1002\/ajh.27758","article-title":"Luspatercept for transfusion-dependent beta-thalassemia: real-world experience in a large cohort of patients from Italy","volume":"100","author":"Origa","year":"2025","journal-title":"Am J Hematol"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib45","article-title":"Improvement of iron overload parameters in patients with transfusion-dependent \u03b2-thalassemia treated with luspatercept: data from the phase 3b long-term rollover study following the BELIEVE Trial","volume":"144","author":"Porter","year":"2024","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib46","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1002\/ajh.27102","article-title":"Luspatercept stimulates erythropoiesis, increases iron utilization, and redistributes body iron in transfusion-dependent thalassemia","volume":"99","author":"Garbowski","year":"2024","journal-title":"Am J Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib47","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1111\/bjh.70227","article-title":"Achieving clinically meaningful changes in haemoglobin levels in patients with non-transfusion-dependent \u03b2-thalassaemia treated with luspatercept: a post hoc analysis of the phase 2 BEYOND trial","volume":"208","author":"Musallam","year":"2026","journal-title":"Br J Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib48","doi-asserted-by":"crossref","first-page":"6108","DOI":"10.1182\/bloodadvances.2025016554","article-title":"Efficacy and safety of luspatercept in non-transfusion-dependent \u03b2-thalassemia: long-term results from the BEYOND study","volume":"9","author":"Taher","year":"2025","journal-title":"Blood Adv"},{"key":"10.1016\/S2352-3026(26)00173-0_bib49","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1111\/ejh.70161","article-title":"Patient-reported outcomes with luspatercept through 5\u2008years of treatment in patients with non-transfusion-dependent \u03b2-thalassemia treated in the BEYOND trial","volume":"117","author":"Musallam","year":"2026","journal-title":"Eur J Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib50","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1111\/bjh.18801","article-title":"Management of luspatercept therapy in patients with transfusion-dependent \u03b2-thalassaemia","volume":"201","author":"Sheth","year":"2023","journal-title":"Br J Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib51","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1182\/hematology.2024000567","article-title":"Luspatercept: a treatment for ineffective erythropoiesis in thalassemia","volume":"2024","author":"Musallam","year":"2024","journal-title":"Hematology (Am Soc Hematol Educ Program)"},{"key":"10.1016\/S2352-3026(26)00173-0_bib54","doi-asserted-by":"crossref","first-page":"1383","DOI":"10.1016\/S0140-6736(26)00150-9","article-title":"Pyruvate kinase activators in hereditary haemolytic anaemias: current evidence and clinical potential","volume":"407","author":"Doeven","year":"2026","journal-title":"Lancet"},{"key":"10.1016\/S2352-3026(26)00173-0_bib55","doi-asserted-by":"crossref","first-page":"1292","DOI":"10.1016\/j.exphem.2005.07.008","article-title":"Ineffective erythropoiesis in mutant mice with deficient pyruvate kinase activity","volume":"33","author":"Aizawa","year":"2005","journal-title":"Exp Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib56","doi-asserted-by":"crossref","first-page":"2818","DOI":"10.1182\/bloodadvances.2024013591","article-title":"Mitapivat metabolically reprograms human \u03b2-thalassemic erythroblasts, increasing their responsiveness to oxidation","volume":"9","author":"Siciliano","year":"2025","journal-title":"Blood Adv"},{"key":"10.1016\/S2352-3026(26)00173-0_bib57","doi-asserted-by":"crossref","first-page":"2535","DOI":"10.3324\/haematol.2022.282614","article-title":"Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in \u03b2-thalassemic mice","volume":"108","author":"Matt\u00e8","year":"2023","journal-title":"Haematologica"},{"key":"10.1016\/S2352-3026(26)00173-0_bib58","doi-asserted-by":"crossref","DOI":"10.1172\/JCI144206","article-title":"The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a \u03b2-thalassemia mouse model","volume":"131","author":"Matte","year":"2021","journal-title":"J Clin Invest"},{"key":"10.1016\/S2352-3026(26)00173-0_bib59","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1016\/S0140-6736(22)01337-X","article-title":"Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent \u03b1-thalassaemia or \u03b2-thalassaemia: an open-label, multicentre, phase 2 study","volume":"400","author":"Kuo","year":"2022","journal-title":"Lancet"},{"key":"10.1016\/S2352-3026(26)00173-0_bib60","doi-asserted-by":"crossref","first-page":"e35","DOI":"10.1016\/S2352-3026(24)00319-3","article-title":"Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial","volume":"12","author":"Idowu","year":"2025","journal-title":"Lancet Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib61","series-title":"Inc. AQVESME (mitapivat) prescribing information","author":"Pharmaceuticals","year":"2025"},{"key":"10.1016\/S2352-3026(26)00173-0_bib62","doi-asserted-by":"crossref","DOI":"10.1016\/j.exphem.2024.104673","article-title":"FT-4202, a selective pyruvate kinase R activator for sickle cell disease","volume":"141","author":"Ericsson","year":"2025","journal-title":"Exp Hematol"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib64","first-page":"457","article-title":"S297: etavopivat improves hemoglobin levels in adults and adolescents with non-transfusion-dependent alpha- or beta-thalassemia: results from the open-label phase 2 GLADIOLUS study","volume":"10","author":"Sheth","year":"2026","journal-title":"HemaSphere"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib65","first-page":"463","article-title":"S299: etavopivat reduces transfusion requirements in adults and adolescents with transfusion-dependent alpha- or beta-thalassemia: results from the open-label phase 2 GLADIOLUS study","volume":"10","author":"Lal","year":"2026","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib68","first-page":"110","article-title":"A hepcidin mimetic, PTG-300, demonstrates pharmacodynamic effects indicating reduced iron availability in transfusion-dependent beta-thalassemia subjects","volume":"4","author":"Lal","year":"2020","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib69","doi-asserted-by":"crossref","DOI":"10.1182\/bloodadvances.2026019799","article-title":"Phase 2a randomized study to evaluate sapablursen in patients with non-transfusion dependent \u03b2-thalassemia intermedia","author":"Taher","year":"2026","journal-title":"Blood Adv"},{"key":"10.1016\/S2352-3026(26)00173-0_bib71","doi-asserted-by":"crossref","first-page":"608","DOI":"10.1186\/s13023-025-04119-y","article-title":"Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent \u03b2-thalassemia: results from a randomized phase 2a study","volume":"20","author":"Kattamis","year":"2025","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/S2352-3026(26)00173-0_bib72","doi-asserted-by":"crossref","first-page":"474","DOI":"10.1111\/bjh.17479","article-title":"Haematological effects of oral administration of bitopertin, a glycine transport inhibitor, in patients with non-transfusion-dependent \u03b2-thalassaemia","volume":"194","author":"Taher","year":"2021","journal-title":"Br J Haematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib73","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1182\/blood-2017-06-790121","article-title":"Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study","volume":"131","author":"Taher","year":"2018","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib74","doi-asserted-by":"crossref","first-page":"1479","DOI":"10.1056\/NEJMoa1705342","article-title":"Gene therapy in patients with transfusion-dependent \u03b2-thalassemia","volume":"378","author":"Thompson","year":"2018","journal-title":"N Engl J Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib75","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1038\/s41591-021-01650-w","article-title":"Long-term outcomes of lentiviral gene therapy for the \u03b2-hemoglobinopathies: the HGB-205 trial","volume":"28","author":"Magrin","year":"2022","journal-title":"Nat Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib76","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101155","article-title":"Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for \u03b2-thalassemia","volume":"31","author":"Whitney","year":"2023","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/S2352-3026(26)00173-0_bib77","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1056\/NEJMoa2113206","article-title":"Betibeglogene autotemcel gene therapy for non-\u03b20\/\u03b20 genotype \u03b2-thalassemia","volume":"386","author":"Locatelli","year":"2022","journal-title":"N Engl J Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib78","doi-asserted-by":"crossref","first-page":"2175","DOI":"10.1016\/S0140-6736(24)01884-1","article-title":"Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype \u03b2-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial","volume":"404","author":"Kwiatkowski","year":"2024","journal-title":"Lancet"},{"key":"10.1016\/S2352-3026(26)00173-0_bib79","doi-asserted-by":"crossref","first-page":"2203","DOI":"10.1182\/blood.2025029196","article-title":"Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent \u03b2-thalassemia","volume":"147","author":"Kwiatkowski","year":"2026","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib80","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1182\/bloodadvances.2024014104","article-title":"Gene therapy in transfusion-dependent non-\u03b20\/\u03b20 genotype \u03b2-thalassemia: first real-world experience of beti-cel","volume":"9","author":"Mirza","year":"2025","journal-title":"Blood Adv"},{"key":"10.1016\/S2352-3026(26)00173-0_bib81","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1016\/j.stem.2024.04.021","article-title":"Modified lentiviral globin gene therapy for pediatric \u03b20\/\u03b20 transfusion-dependent \u03b2-thalassemia: a single-center, single-arm pilot trial","volume":"31","author":"Li","year":"2024","journal-title":"Cell Stem Cell"},{"key":"10.1016\/S2352-3026(26)00173-0_bib82","doi-asserted-by":"crossref","first-page":"2611","DOI":"10.1016\/j.ymthe.2026.02.017","article-title":"Safety and efficacy of hemobeglogene autotemcel (hemo-cel) gene therapy in five patients with transfusion-dependent \u03b2-thalassemia","volume":"34","author":"Liu","year":"2026","journal-title":"Mol Ther"},{"key":"10.1016\/S2352-3026(26)00173-0_bib83","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1038\/s41591-018-0301-6","article-title":"Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent \u03b2-thalassemia","volume":"25","author":"Marktel","year":"2019","journal-title":"Nat Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib84","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1038\/s41591-021-01554-9","article-title":"Lentiviral globin gene therapy with reduced-intensity conditioning in adults with \u03b2-thalassemia: a phase 1 trial","volume":"28","author":"Boulad","year":"2022","journal-title":"Nat Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib85","doi-asserted-by":"crossref","first-page":"1625","DOI":"10.1016\/j.ymthe.2020.12.036","article-title":"Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies","volume":"29","author":"Breda","year":"2021","journal-title":"Mol Ther"},{"key":"10.1016\/S2352-3026(26)00173-0_bib86","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 gene editing for sickle cell disease and \u03b2-thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib87","doi-asserted-by":"crossref","first-page":"1839","DOI":"10.1126\/science.1165409","article-title":"Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A","volume":"322","author":"Sankaran","year":"2008","journal-title":"Science"},{"key":"10.1016\/S2352-3026(26)00173-0_bib88","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1056\/NEJMoa2309673","article-title":"Exagamglogene autotemcel for transfusion-dependent \u03b2-thalassemia","volume":"390","author":"Locatelli","year":"2024","journal-title":"N Engl J Med"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib89","article-title":"Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent \u03b2-thalassemia","volume":"146","author":"Locatelli","year":"2025","journal-title":"Blood"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib90","first-page":"4278","article-title":"PS2333: long-term follow-up confirms durable clinical benefits of exagamglogene autotemcel in transfusion-dependent \u03b2-thalassemia: final results of the CLIMB THAL-111 study","volume":"10","author":"Meisel","year":"2026","journal-title":"HemaSphere"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib91","doi-asserted-by":"crossref","first-page":"512","DOI":"10.1182\/blood-2024-205779","article-title":"Durable clinical benefits with exagamglogene autotemcel for transfusion-dependent \u03b2-thalassemia","volume":"144","author":"Locatelli","year":"2024","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib92","doi-asserted-by":"crossref","first-page":"6502","DOI":"10.1182\/bloodadvances.2025016702","article-title":"Improvements in health-related quality of life in patients with transfusion-dependent \u03b2-thalassemia after exagamglogene autotemcel","volume":"9","author":"Fuente","year":"2025","journal-title":"Blood Adv"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib93","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1182\/blood-2025-379","article-title":"First results of exagamglogene autotemcel in pediatric patients aged 5\u201311 years with transfusion-dependent \u03b2-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises","volume":"146","author":"Frangoul","year":"2025","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib94","doi-asserted-by":"crossref","first-page":"947","DOI":"10.1056\/NEJMoa2603387","article-title":"Exa-cel in children with transfusion-dependent \u03b2-thalassemia or sickle cell disease","volume":"395","author":"Frangoul","year":"2026","journal-title":"N Engl J Med"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib95","first-page":"446","article-title":"S290: efficacy and safety of BRL-101 in TDT and SCD","volume":"9","author":"Zheng","year":"2025","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib96","doi-asserted-by":"crossref","first-page":"1573","DOI":"10.1038\/s41591-022-01906-z","article-title":"CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric \u03b20\/\u03b20 transfusion-dependent \u03b2-thalassemia","volume":"28","author":"Fu","year":"2022","journal-title":"Nat Med"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib97","first-page":"3953","article-title":"PS2181: updated safety and efficacy results of RM-001, autologous HBG1\/2 promoter-modified CD34+ hematopoietic stem and progenitor cells, in treating transfusion-dependent \u03b2-thalassemia","volume":"9","author":"Liu","year":"2025","journal-title":"HemaSphere"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib98","article-title":"Updated results of a phase 1\/2 clinical study of zinc finger nuclease-mediated editing of BCL11A in autologous hematopoietic stem cells for transfusion-dependent beta thalassemia","volume":"138","author":"Walters","year":"2021","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib99","doi-asserted-by":"crossref","first-page":"1292","DOI":"10.1056\/NEJMoa2501277","article-title":"CRISPR-Cas12a gene editing of HBG1 and HBG2 promoters to treat \u03b2-thalassemia","volume":"394","author":"Frangoul","year":"2026","journal-title":"N Engl J Med"},{"key":"10.1016\/S2352-3026(26)00173-0_bib100","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1038\/s41586-026-10342-9","article-title":"Clinical application of base editing for treating \u03b2-thalassaemia","volume":"653","author":"Lai","year":"2026","journal-title":"Nature"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib101","article-title":"Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent \u03b2-thalassemia participants: an autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)","volume":"146","author":"Chen","year":"2025","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib102","doi-asserted-by":"crossref","first-page":"436","DOI":"10.1126\/science.ade6967","article-title":"In vivo hematopoietic stem cell modification by mRNA delivery","volume":"381","author":"Breda","year":"2023","journal-title":"Science"},{"key":"10.1016\/S2352-3026(26)00173-0_bib103","doi-asserted-by":"crossref","DOI":"10.1177\/20406207231195594","article-title":"Luspatercept for transfusion-dependent \u03b2-thalassemia: time to get real","volume":"14","author":"Musallam","year":"2023","journal-title":"Ther Adv Hematol"},{"key":"10.1016\/S2352-3026(26)00173-0_bib104","doi-asserted-by":"crossref","first-page":"e49","DOI":"10.1016\/S2352-3026(25)00320-5","article-title":"Management of transfusion-dependent \u03b2-thalassaemia in the era of novel therapies: a prioritisation-based matrix for settings with limited resources","volume":"13","author":"Musallam","year":"2026","journal-title":"Lancet Haematol"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib105","first-page":"3965","article-title":"PS2186: real world data on improving anemia without transfusions with luspatercept in \u03b2-thalassemic patients with comorbidities and\/or alloimmunization, who convert to transfusion-dependent late in adult life","volume":"9","author":"Chatzidavid","year":"2025","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib106","doi-asserted-by":"crossref","DOI":"10.1002\/hem3.70165","article-title":"Luspatercept versus mitapivat for non-transfusion-dependent \u03b2-thalassemia: dare to compare?","volume":"9","author":"Musallam","year":"2025","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib107","doi-asserted-by":"crossref","first-page":"e555","DOI":"10.1097\/HS9.0000000000000555","article-title":"Selecting \u03b2-thalassemia patients for gene therapy: a decision-making algorithm","volume":"5","author":"Baronciani","year":"2021","journal-title":"HemaSphere"},{"key":"10.1016\/S2352-3026(26)00173-0_bib108","doi-asserted-by":"crossref","first-page":"2320","DOI":"10.1111\/bjh.70437","article-title":"Real-world evidence supports the safety and efficacy profile of luspatercept in clinically complex and heavily iron-overloaded patients","volume":"208","author":"Gianesin","year":"2026","journal-title":"Br J Haematol"},{"issue":"suppl 1","key":"10.1016\/S2352-3026(26)00173-0_bib109","article-title":"Revisiting erythroid response in the phase 3 BELIEVE trial of luspatercept in patients with transfusion-dependent \u03b2-thalassemia using real-world criteria","volume":"146","author":"Musallam","year":"2025","journal-title":"Blood"},{"key":"10.1016\/S2352-3026(26)00173-0_bib110","doi-asserted-by":"crossref","first-page":"1134","DOI":"10.1080\/13696998.2026.2649528","article-title":"Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with transfusion-dependent \u03b2-thalassemia in the United States","volume":"29","author":"Udeze","year":"2026","journal-title":"J Med Econ"},{"key":"10.1016\/S2352-3026(26)00173-0_bib111","doi-asserted-by":"crossref","DOI":"10.1080\/20016689.2021.1922028","article-title":"Economic evaluation of betibeglogene autotemcel (beti-cel) gene addition therapy in transfusion-dependent \u03b2-thalassemia","volume":"9","author":"Kansal","year":"2021","journal-title":"J Mark Access Health Policy"},{"key":"10.1016\/S2352-3026(26)00173-0_bib114","doi-asserted-by":"crossref","DOI":"10.1016\/S2352-3026(26)00197-3","article-title":"Future-proofing thalassaemia care: harnessing scalable therapies, artificial intelligence, and global collaboration","author":"Al-Samkari","year":"2026","journal-title":"Lancet Haematol"}],"container-title":["The Lancet Haematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2352302626001730?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2352302626001730?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T23:06:16Z","timestamp":1791327976000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2352302626001730"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10]]},"references-count":106,"alternative-id":["S2352302626001730"],"URL":"https:\/\/doi.org\/10.1016\/s2352-3026(26)00173-0","relation":{},"ISSN":["2352-3026"],"issn-type":[{"value":"2352-3026","type":"print"}],"subject":[],"published":{"date-parts":[[2026,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The expanding therapeutic landscape of \u03b2-thalassaemia","name":"articletitle","label":"Article Title"},{"value":"The Lancet Haematology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/S2352-3026(26)00173-0","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"https:\/\/doi.org\/10.1016\/S2352-3026(26)00222-X","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"https:\/\/doi.org\/10.1016\/S2352-3026(26)00227-9","name":"associatedlink","label":"CrossRef DOI link to the associated document"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 Elsevier Ltd. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}]}}