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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

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

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

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

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

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

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

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

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

Mortality in β-thalassemia patients with confirmed pulmonary arterial hypertension on right heart catheterization

Valeria Maria Pinto, Khaled M. Musallam, Giorgio Derchi, Giovanna Graziadei, Marianna Giuditta, Raffaella Origa, Susanna Barella, Gavino Casu, Annamaria Pasanisi, Filomena Longo, Maddalena Casale, Roberta Miceli, Pierluigi Merella, Immacolata Tartaglione, Antonio Piga, Maria Domenica Cappellini, Barbara Gianesin, Gian Luca Forni, on behalf of the Webthal project

Blood (2022) 139 (13): 2080–2083.

https://doi.org/10.1182/blood.2021014862

Published: 2022

Journal Articles

Luspatercept Improves Quality of Life and Reduces Red Blood Cell Transfusion Burden in Patients with Non-Transfusion-Dependent β-Thalassemia in the BEYOND Trial

Antonis Kattamis, Vip Viprakasit, Maria Domenica Cappellini, Ersi Voskaridou, Antonio Piga, John B. Porter, Thomas D. Coates, Gian Luca Forni, Jeevan K. Shetty, Dimana Miteva, Oriana Esposito, Wen-Ling Kuo, Shien Guo, Christopher Pelligra, Jennifer Lord-Bessen, Aylin Yucel, Ali T. Taher

Blood (2021) 138 (Supplement 1): 3081.

https://doi.org/10.1182/blood-2021-148535

Published: 2021

Journal Articles

Inhibition of Fibroblast Growth Factor-23 (FGF-23) Rescues Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia, Uncovering the Missing Link between Hematopoiesis and Bone

Annamaria Aprile, Laura Raggi, Simona Bolamperti, Mariangela Storto, Isabella Villa, Gaia Morello, Sarah Marktel, Claudio Tripodo, Irene Motta, Maria Domenica Cappellini, Alessandro Rubinacci, Giuliana Ferrari

Blood (2021) 138 (Supplement 1): 572.

https://doi.org/10.1182/blood-2021-148366

Published: 2021

Journal Articles

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-139575

Published: 2020

Journal Articles

Inhibition of Fibroblast Growth Factor-23 (FGF-23) As a Novel Strategy to Target Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia

Annamaria Aprile, PhD, Laura Raggi, Mariangela Storto, Isabella Villa, PhD, Sarah Marktel, MD, Irene Motta, MD, Maria Domenica Cappellini, Alessandro Rubinacci, MD, Giuliana Ferrari, PhD

Blood (2020) 136 (Supplement 1): 2.

https://doi.org/10.1182/blood-2020-137461

Published: 2020

Journal Articles

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Maria Domenica Cappellini, Ali T. Taher, Antonio Piga, MD, Farrukh Shah, Ersi Voskaridou, Vip Viprakasit, John B. Porter, MA, MD, FRCP, FRCPath, Olivier Hermine, Ellis J. Neufeld, Alexis A. Thompson, MD, Derek Tang, Peiwen Yu, Shien Guo, Jeevan K. Shetty, Dimana Miteva, Tatiana Zinger, Jay T. Backstrom, Esther Natalie Oliva, MD

Blood (2020) 136 (Supplement 1): 8–9.

https://doi.org/10.1182/blood-2020-136312

Published: 2020

Journal Articles

High-Risk Population Screening for Gaucher Disease: Final Results and Key Learnings from an Italian Multicenter Observational Study

Irene Motta, MD, Christian Benedetto, Marina Stroppiano, Elena Cassinerio, MD, Dario Consonni, MD, Lorenza Borin, MD, Paola Ranalli, MD, Wilma Barcellini, Maria Domenica Cappellini, MD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-141527

Published: 2020

Journal Articles

Diagnostic Work-up of Pulmonary Hypertension in Non Transfusion Dependent Thalassemia Patients: Pathophysiologichal Mechanisms and Clinical Implications

Marta Mancarella, MD, Irene Motta, MD, Irene Rota, MD, Margherita Migone De Amicis, MD, Alessia Marcon, MD, Marta Ferraresi, MD, Marianna Giuditta, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Marco Vicenzi, MD

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-140819

Published: 2020

Journal Articles

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

Irene Motta, MD, Valeria Maria Pinto, MD, Filomena Longo, MD, Federico Bonetti, MD, Elisa De Michele, MD, Alberto Piperno, MD, Lorella Pitrolo, MD, Micol Quaresima, MD, Michela Ribersani, MD, Margherita Migone De Amicis, MD, Manuela Balocco, MD, Barbara Gianesin, PhD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Lucia De Franceschi, MD, Antonio Piga, MD, Gian Luca Forni, MD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-141610

Published: 2020

NARROW

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

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

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

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

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

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

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

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

Mortality in β-thalassemia patients with confirmed pulmonary arterial hypertension on right heart catheterization

Luspatercept Improves Quality of Life and Reduces Red Blood Cell Transfusion Burden in Patients with Non-Transfusion-Dependent β-Thalassemia in the BEYOND Trial

Inhibition of Fibroblast Growth Factor-23 (FGF-23) Rescues Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia, Uncovering the Missing Link between Hematopoiesis and Bone

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Inhibition of Fibroblast Growth Factor-23 (FGF-23) As a Novel Strategy to Target Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

High-Risk Population Screening for Gaucher Disease: Final Results and Key Learnings from an Italian Multicenter Observational Study

Diagnostic Work-up of Pulmonary Hypertension in Non Transfusion Dependent Thalassemia Patients: Pathophysiologichal Mechanisms and Clinical Implications

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

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American Society of Hematology

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