Research graph
References from The expanding therapeutic landscape of β-thalassaemia. Local targets link to admitted publications; unresolved targets remain external evidence.
Thalassaemia
10.1016/s0140-6736(22)00536-0 · 2022 · External reference
β-Thalassemias
10.1056/nejmra2021838 · 2021 · External reference
Unresolved reference
2023 · External reference
Unresolved reference
2025 · External reference
Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study
10.1182/blood-2009-09-243154 · 2010 · External reference
Iron metabolism under conditions of ineffective erythropoiesis in β-thalassemia
10.1182/blood-2018-07-815928 · 2019 · External reference
Risk of mortality from anemia and iron overload in nontransfusion-dependent β-thalassemia
10.1002/ajh.26428 · 2022 · External reference
Serum ferritin level and morbidity risk in transfusion-independent patients with β-thalassemia intermedia: the ORIENT study
10.3324/haematol.2013.097220 · 2014 · External reference
Elevated liver iron concentration is a marker of increased morbidity in patients with β thalassemia intermedia
10.3324/haematol.2011.047852 · 2011 · External reference
Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study
10.1182/blood-2012-02-412692 · 2012 · External reference
Impact of lifetime anaemia and iron control on outcomes in β-thalassaemia: data from the longitudinal de-LIGHT study
10.1111/bjh.70047 · 2025 · External reference
Survival and causes of death in 2,033 patients with non-transfusion-dependent β-thalassemia
10.3324/haematol.2021.278684 · 2021 · External reference
Morbidity-free survival and hemoglobin level in non-transfusion-dependent β-thalassemia: a 10-year cohort study
10.1007/s00277-020-04370-2 · 2022 · External reference
Variations in hemoglobin level and morbidity burden in non-transfusion-dependent β-thalassemia
10.1007/s00277-021-04456-5 · 2021 · External reference
Efficacy and safety of thalidomide in β-thalassaemia: a systematic review and meta-analysis
10.1038/s41598-026-46504-y · 2026 · External reference
Efficacy and safety of hydroxyurea therapy on patients with β-thalassemia: a systematic review and meta-analysis
2025 · External reference
‘Phenoconversion’ in adult patients with β-thalassemia
10.1002/ajh.27194 · 2024 · External reference
Management of transfusion-dependent β-thalassemia (TDT): expert insights and practical overview from the Middle East
10.1016/j.blre.2023.101138 · 2024 · External reference
Pretransfusion hemoglobin level and mortality in adults with transfusion-dependent β-thalassemia
10.1182/blood.2023022460 · 2024 · External reference
Quantifying morbidity risk attributed to red-cell transfusion volume in optimally transfused patients with β-thalassemia
10.1002/ajh.70100 · 2026 · External reference
Overall and complication-free survival in a large cohort of patients with β-thalassemia major followed over 50 years
10.1002/ajh.26798 · 2023 · External reference
Survival and complications in patients with haemoglobin E thalassaemia in Sri Lanka: a prospective, longitudinal cohort study
10.1016/s2214-109x(21)00446-0 · 2022 · External reference
Clinical and economic burden of regularly transfused adult patients with β-thalassemia in the United States: a retrospective cohort study using payer claims
10.1002/ajh.25429 · 2019 · External reference
Systematic literature review of the indirect costs and humanistic burden of β-thalassemia
10.1177/20406207241270872 · 2024 · External reference
Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010
10.1038/bmt.2015.293 · 2016 · External reference
Hematopoietic cell transplantation in thalassemia and sickle cell disease: report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000–2017
10.1182/blood-2018-168 · 2018 · External reference
Related and unrelated donor transplantation for β-thalassemia major: results of an international survey
10.1182/bloodadvances.2019000291 · 2019 · External reference
Hematopoietic stem cell transplantation in thalassemia
10.1016/j.hoc.2022.12.009 · 2023 · External reference
Safety and pharmacokinetics of the oral iron chelator SP-420 in β-thalassemia
10.1002/ajh.24914 · 2017 · External reference
Transforming growth factor-β superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis
10.1038/nm.3512 · 2014 · External reference
Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine β-thalassemia
10.1182/blood-2013-06-511238 · 2014 · External reference
Smad2/3-pathway ligand trap luspatercept enhances erythroid differentiation in murine β-thalassaemia by increasing GATA-1 availability
10.1111/jcmm.15243 · 2020 · External reference
Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study
10.3324/haematol.2018.198887 · 2019 · External reference
A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers
10.1002/ajh.23732 · 2014 · External reference
Long-term safety and erythroid response with luspatercept treatment in patients with β-thalassemia
10.1177/20406207221134404 · 2022 · External reference
A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia
10.1056/nejmoa1910182 · 2020 · External reference
Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial
10.1016/s0140-6736(26)00874-3 · 2026 · External reference
Efficacy of mitapivat in patients with transfusion-dependent alpha-thalassemia: subgroup analysis from the ENERGIZE-T trial
10.1182/blood-2025-4699 · 2025 · External reference
Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial
10.1016/s2352-3026(22)00208-3 · 2022 · External reference
Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial
10.1016/s0140-6736(25)00635-x · 2025 · External reference
Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trial
10.1016/s2352-3026(24)00376-4 · 2025 · External reference
EP1306: benefit of continuing therapy with luspatercept in patients with beta-thalassemia who do not achieve ≥33% reduction in red blood cell (RBC) transfusion burden (TB) in weeks 13–24 in the BELIEVE trial
2021 · External reference
P1522: characterizing patterns of transfusion burden (TB) reduction in patients (pts) with transfusion-dependent (TD) beta-thalassemia treated with luspatercept in the BELIEVE Trial
2023 · External reference
Luspatercept for transfusion-dependent beta-thalassemia: real-world experience in a large cohort of patients from Italy
10.1002/ajh.27758 · 2025 · External reference
Improvement of iron overload parameters in patients with transfusion-dependent β-thalassemia treated with luspatercept: data from the phase 3b long-term rollover study following the BELIEVE Trial
2024 · External reference
Luspatercept stimulates erythropoiesis, increases iron utilization, and redistributes body iron in transfusion-dependent thalassemia
10.1002/ajh.27102 · 2024 · External reference
Achieving clinically meaningful changes in haemoglobin levels in patients with non-transfusion-dependent β-thalassaemia treated with luspatercept: a post hoc analysis of the phase 2 BEYOND trial
10.1111/bjh.70227 · 2026 · External reference
Efficacy and safety of luspatercept in non-transfusion-dependent β-thalassemia: long-term results from the BEYOND study
10.1182/bloodadvances.2025016554 · 2025 · External reference
Patient-reported outcomes with luspatercept through 5 years of treatment in patients with non-transfusion-dependent β-thalassemia treated in the BEYOND trial
10.1111/ejh.70161 · 2026 · External reference
Management of luspatercept therapy in patients with transfusion-dependent β-thalassaemia
10.1111/bjh.18801 · 2023 · External reference
Luspatercept: a treatment for ineffective erythropoiesis in thalassemia
10.1182/hematology.2024000567 · 2024 · External reference
Pyruvate kinase activators in hereditary haemolytic anaemias: current evidence and clinical potential
10.1016/s0140-6736(26)00150-9 · 2026 · External reference
Ineffective erythropoiesis in mutant mice with deficient pyruvate kinase activity
10.1016/j.exphem.2005.07.008 · 2005 · External reference
Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation
10.1182/bloodadvances.2024013591 · 2025 · External reference
Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice
10.3324/haematol.2022.282614 · 2023 · External reference
The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model
10.1172/jci144206 · 2021 · External reference
Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study
10.1016/s0140-6736(22)01337-x · 2022 · External reference
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
10.1016/s2352-3026(24)00319-3 · 2025 · External reference
Unresolved reference
2025 · External reference
FT-4202, a selective pyruvate kinase R activator for sickle cell disease
10.1016/j.exphem.2024.104673 · 2025 · External reference
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
2026 · External reference
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
2026 · External reference
A hepcidin mimetic, PTG-300, demonstrates pharmacodynamic effects indicating reduced iron availability in transfusion-dependent beta-thalassemia subjects
2020 · External reference
Phase 2a randomized study to evaluate sapablursen in patients with non-transfusion dependent β-thalassemia intermedia
10.1182/bloodadvances.2026019799 · 2026 · External reference
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
10.1186/s13023-025-04119-y · 2025 · External reference
Haematological effects of oral administration of bitopertin, a glycine transport inhibitor, in patients with non-transfusion-dependent β-thalassaemia
10.1111/bjh.17479 · 2021 · External reference
Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study
10.1182/blood-2017-06-790121 · 2018 · External reference
Gene therapy in patients with transfusion-dependent β-thalassemia
10.1056/nejmoa1705342 · 2018 · External reference
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
10.1038/s41591-021-01650-w · 2022 · External reference
Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia
10.1016/j.omtm.2023.101155 · 2023 · External reference
Betibeglogene autotemcel gene therapy for non-β0/β0 genotype β-thalassemia
10.1056/nejmoa2113206 · 2022 · External reference
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial
10.1016/s0140-6736(24)01884-1 · 2024 · External reference
Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia
10.1182/blood.2025029196 · 2026 · External reference
Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel
10.1182/bloodadvances.2024014104 · 2025 · External reference
Modified lentiviral globin gene therapy for pediatric β0/β0 transfusion-dependent β-thalassemia: a single-center, single-arm pilot trial
10.1016/j.stem.2024.04.021 · 2024 · External reference
Safety and efficacy of hemobeglogene autotemcel (hemo-cel) gene therapy in five patients with transfusion-dependent β-thalassemia
10.1016/j.ymthe.2026.02.017 · 2026 · External reference
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia
10.1038/s41591-018-0301-6 · 2019 · External reference
Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial
10.1038/s41591-021-01554-9 · 2022 · External reference
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies
10.1016/j.ymthe.2020.12.036 · 2021 · External reference
CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia
10.1056/nejmoa2031054 · 2021 · External reference
Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A
10.1126/science.1165409 · 2008 · External reference
Exagamglogene autotemcel for transfusion-dependent β-thalassemia
10.1056/nejmoa2309673 · 2024 · External reference
Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia
2025 · External reference
PS2333: long-term follow-up confirms durable clinical benefits of exagamglogene autotemcel in transfusion-dependent β-thalassemia: final results of the CLIMB THAL-111 study
2026 · External reference
Durable clinical benefits with exagamglogene autotemcel for transfusion-dependent β-thalassemia
10.1182/blood-2024-205779 · 2024 · External reference
Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel
10.1182/bloodadvances.2025016702 · 2025 · External reference
First results of exagamglogene autotemcel in pediatric patients aged 5–11 years with transfusion-dependent β-thalassemia or sickle cell disease with recurrent severe vaso-occlusive crises
10.1182/blood-2025-379 · 2025 · External reference
Exa-cel in children with transfusion-dependent β-thalassemia or sickle cell disease
10.1056/nejmoa2603387 · 2026 · External reference
S290: efficacy and safety of BRL-101 in TDT and SCD
2025 · External reference
CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia
10.1038/s41591-022-01906-z · 2022 · External reference
PS2181: updated safety and efficacy results of RM-001, autologous HBG1/2 promoter-modified CD34+ hematopoietic stem and progenitor cells, in treating transfusion-dependent β-thalassemia
2025 · External reference
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
2021 · External reference
CRISPR-Cas12a gene editing of HBG1 and HBG2 promoters to treat β-thalassemia
10.1056/nejmoa2501277 · 2026 · External reference
Clinical application of base editing for treating β-thalassaemia
10.1038/s41586-026-10342-9 · 2026 · External reference
Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: an autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)
2025 · External reference
In vivo hematopoietic stem cell modification by mRNA delivery
10.1126/science.ade6967 · 2023 · External reference
Luspatercept for transfusion-dependent β-thalassemia: time to get real
10.1177/20406207231195594 · 2023 · External reference
Management of transfusion-dependent β-thalassaemia in the era of novel therapies: a prioritisation-based matrix for settings with limited resources
10.1016/s2352-3026(25)00320-5 · 2026 · External reference
PS2186: real world data on improving anemia without transfusions with luspatercept in β-thalassemic patients with comorbidities and/or alloimmunization, who convert to transfusion-dependent late in adult life
2025 · External reference
Luspatercept versus mitapivat for non-transfusion-dependent β-thalassemia: dare to compare?
10.1002/hem3.70165 · 2025 · External reference
Selecting β-thalassemia patients for gene therapy: a decision-making algorithm
10.1097/hs9.0000000000000555 · 2021 · External reference
Real-world evidence supports the safety and efficacy profile of luspatercept in clinically complex and heavily iron-overloaded patients
10.1111/bjh.70437 · 2026 · External reference
Revisiting erythroid response in the phase 3 BELIEVE trial of luspatercept in patients with transfusion-dependent β-thalassemia using real-world criteria
2025 · External reference
Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with transfusion-dependent β-thalassemia in the United States
10.1080/13696998.2026.2649528 · 2026 · External reference
Economic evaluation of betibeglogene autotemcel (beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
10.1080/20016689.2021.1922028 · 2021 · External reference
10.1016/s2352-3026(26)00197-3 · 2026 · Admitted local publication
