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Journal Articles

Efficacy of mitapivat in patients with transfusion-dependent alpha-thalassemia: Subgroup analysis from the ENERGIZE-T trial

Blood (2025) 146 (Supplement 1): 4699.

https://doi.org/10.1182/blood-2025-4699

Published: 2025

Journal Articles

Impact of long-term luspatercept treatment on iron parameters in patients with transfusion-dependent and non-transfusion-dependent β-thalassemia: Results from the phase 3b long-term follow-up study

Ali Taher, Khaled Musallam, Maria Domenica Cappellini, Maria Dimopoulou, Antonis Kattamis, Silverio Perrotta, Paolo Ricchi, Manuela Balocco, Patricia Martin-Regueira, Loyse Felber Medlin, George Zhang, Veronika Pozharskaya, John Porter

Blood (2025) 146 (Supplement 1): 4695.

https://doi.org/10.1182/blood-2025-4695

Published: 2025

Journal Articles

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Blood (2025) 146 (Supplement 1): 6089.

https://doi.org/10.1182/blood-2025-6089

Published: 2025

Journal Articles

Understanding health literacy among patients with thalassemia: Results from a global patient survey by the Thalassemia Advocacy Advisory Council

Sujit Sheth, Lily Cannon, Laurice Levine, Ralph Colasanti, Jill Deitrick, Tobias Larkin, Craig Butler, Dina Steagall, Eleni Michalaki, Holly John, Keely Gilroy, Maria Domenica Cappellini

Blood (2025) 146 (Supplement 1): 6421.

https://doi.org/10.1182/blood-2025-6421

Published: 2025

Journal Articles

Revisiting erythroid response in the phase 3 BELIEVE trial of luspatercept in patients with transfusion-dependent β-thalassemia using real-world criteria

Khaled Musallam, Maria Domenica Cappellini, Antonis Kattamis, Kevin Kuo, Sujit Sheth, Patricia Martin-Regueira, Loyse Felber Medlin, George Zhang, Christopher Westcott, Ali Taher

Blood (2025) 146 (Supplement 1): 1139.

https://doi.org/10.1182/blood-2025-1139

Published: 2025

Journal Articles

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

Blood (2025) 146 (Supplement 1): 379.

https://doi.org/10.1182/blood-2025-379

Published: 2025

Journal Articles

Luspatercept Improves Fatigue-Related Quality of Life through 5 Years of Treatment in Non-Transfusion Dependent Beta-Thalassemia

Khaled Musallam, Ali T. Taher, Ahmed Hnoosh, Laurie Eliason, Christopher G. Pelligra, Shien Guo, Yutian Mu, Maria Domenica Cappellini

Blood (2024) 144 (Supplement 1): 3857.

https://doi.org/10.1182/blood-2024-200066

Published: 2024

Journal Articles

Understanding Health Literacy Among Patients with Thalassemia: A Global Patient Survey by the Thalassemia Advocacy Advisory Council

Sujit Sheth, Lily Cannon, Laurice M Levine, Ralph Colasanti, Jill Deitrick, Tobias Larkin, Dina Steagall, Eleni Michalaki, Holly John, Keely S Gilroy, Louise Lombard, Maria Domenica Cappellini

Blood (2024) 144 (Supplement 1): 7949.

https://doi.org/10.1182/blood-2024-203302

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2024) 144 (Supplement 1): 512.

https://doi.org/10.1182/blood-2024-205779

Published: 2024

Journal Articles

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

Blood (2024) 144 (Supplement 1): 409.

https://doi.org/10.1182/blood-2024-200867

Published: 2024

Journal Articles

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

John B. Porter, Maria Domenica Cappellini, Kevin H.M. Kuo, Maria Dimopoulou, Marta Reverte, Loyse Felber Medlin, Shweta Sharma, Kefeng Wang, Jennie Zhang, Ali T. Taher

Blood (2024) 144 (Supplement 1): 2475.

https://doi.org/10.1182/blood-2024-200157

Published: 2024

Journal Articles

Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent β-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial

Ali T Taher, Vip Viprakasit, Antonis Kattamis, Silverio Perrotta, Paolo Ricchi, John B. Porter, Thomas D Coates, Gian Luca Forni, Khaled M. Musallam, Oriana Esposito, Ana Carolina Giuseppi, Wen-Ling Kuo, Marta Reverte, Richard Wei, Luciana Moro Bueno, Maria Domenica Cappellini

Blood (2023) 142 (Supplement 1): 3847.

https://doi.org/10.1182/blood-2023-174097

Published: 2023

Journal Articles

Investigating Health Literacy in Thalassemia: Founding a Patient-Led Research Approach

Sujit Sheth, Ralph Colasanti, Lily Cannon, Jill Deitrick, Holly John, Keely S Gilroy, Louise Lombard, Eleni Michalaki, Tobias Larkin, Laurice M Levine, Dina Steagall, Maria Domenica Cappellini

Blood (2023) 142 (Supplement 1): 5251.

https://doi.org/10.1182/blood-2023-181629

Published: 2023

Journal Articles

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2023) 142 (Supplement 1): 1053.

https://doi.org/10.1182/blood-2023-190534

Published: 2023

Journal Articles

Efficacy and Safety of Luspatercept in Patients Enrolled in the BELIEVE Trial: Data from the Phase 3b Long-Term Rollover Study

Maria Domenica Cappellini, Ali T Taher, Vip Viprakasit, Pencho Georgiev, Kevin H.M. Kuo, Natalia Holot, Valérie Vilmont, Loyse Felber Medlin, Wen-Ling Kuo, Yinzhi Lai, Luciana Moro Bueno, Sujit Sheth

Blood (2023) 142 (Supplement 1): 3849.

https://doi.org/10.1182/blood-2023-174089

Published: 2023

Journal Articles

Effect of Luspatercept on Erythropoiesis and Fetal Hemoglobin in β-Transfusion-Dependent Thalassemia Patients: A Real-Life Experience

Daniele Lello Panzieri, Guido Ausenda, Francesca Granata, Nancy Caponio, Dario Consonni, Lorena Duca, Elena Cassinerio, Silvia Elli, Marta Ferraresi, Giovanna Graziadei, Simona Leoni, Natalia Scaramellini, Valentina Brancaleoni, Maria Domenica Cappellini, Irene Motta

Blood (2023) 142 (Supplement 1): 2478.

https://doi.org/10.1182/blood-2023-187416

Published: 2023

Journal Articles

Durable Symptom Improvement for Patients with Non-Transfusion Dependent Thalassemia Treated with Luspatercept: Patient-Reported Outcomes from the BEYOND Study

Khaled M Musallam, Ali T Taher, Antonis Kattamis, Maria Domenica Cappellini, Jennifer Lord-Bessen, Mrudula Glassberg, Christopher Pelligra, Shien Guo, Loyse Felber Medlin, Luciana Moro Bueno, Kevin H.M. Kuo

Blood (2023) 142 (Supplement 1): 2474.

https://doi.org/10.1182/blood-2023-173366

Published: 2023

Journal Articles

Clinical Burden of β-Thalassemia: Findings from a Multinational Medical Record Abstraction Study

Maria Domenica Domenica Cappellini, Aylin Yucel, Juliana Meyers, Maria Jimenez, Tram Nham, Mrudula B. Glassberg, Ferras Alashkar

Blood (2022) 140 (Supplement 1): 10877–10878.

https://doi.org/10.1182/blood-2022-156549

Published: 2022

Journal Articles

Ferric Carboxymaltose for Long-Term Correction of Iron Deficiency Anemia in the Emergency Department

Paul Bernard Lemieux, Diletta Maira, Natalia Scaramellini, Margherita Migone De Amicis, Valeria Di Stefano, Marta Ferraresi, Elena Cassinerio, Maria Domenica Domenica Cappellini, Giovanna Graziadei, Irene Motta

Blood (2022) 140 (Supplement 1): 10771–10772.

https://doi.org/10.1182/blood-2022-170197

Published: 2022

Journal Articles

Effect of Luspatercept in β-Thalassemia Patients with β0/β0 Genotype: A Subgroup Analysis of the BELIEVE Study

Sujit Sheth, Olivier Hermine, Ali T. Taher, Kevin H.M. Kuo, John B. Porter, Antonio Piga, Thomas D. Coates, Antonis Kattamis, Loyse Felber Medlin, Wen-Ling Kuo, Natalia Holot, Maria Domenica Domenica Cappellini

Blood (2022) 140 (Supplement 1): 1946–1948.

https://doi.org/10.1182/blood-2022-157511

Published: 2022

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Efficacy of mitapivat in patients with transfusion-dependent alpha-thalassemia: Subgroup analysis from the ENERGIZE-T trial

Impact of long-term luspatercept treatment on iron parameters in patients with transfusion-dependent and non-transfusion-dependent β-thalassemia: Results from the phase 3b long-term follow-up study

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Understanding health literacy among patients with thalassemia: Results from a global patient survey by the Thalassemia Advocacy Advisory Council

Revisiting erythroid response in the phase 3 BELIEVE trial of luspatercept in patients with transfusion-dependent β-thalassemia using real-world criteria

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

Luspatercept Improves Fatigue-Related Quality of Life through 5 Years of Treatment in Non-Transfusion Dependent Beta-Thalassemia

Understanding Health Literacy Among Patients with Thalassemia: A Global Patient Survey by the Thalassemia Advocacy Advisory Council

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

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

Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent β-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial

Investigating Health Literacy in Thalassemia: Founding a Patient-Led Research Approach

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Efficacy and Safety of Luspatercept in Patients Enrolled in the BELIEVE Trial: Data from the Phase 3b Long-Term Rollover Study

Effect of Luspatercept on Erythropoiesis and Fetal Hemoglobin in β-Transfusion-Dependent Thalassemia Patients: A Real-Life Experience

Durable Symptom Improvement for Patients with Non-Transfusion Dependent Thalassemia Treated with Luspatercept: Patient-Reported Outcomes from the BEYOND Study

Clinical Burden of β-Thalassemia: Findings from a Multinational Medical Record Abstraction Study

Ferric Carboxymaltose for Long-Term Correction of Iron Deficiency Anemia in the Emergency Department

Effect of Luspatercept in β-Thalassemia Patients with β0/β0 Genotype: A Subgroup Analysis of the BELIEVE Study

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