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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

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

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

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

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

Sustained Reductions in Red Blood Cell (RBC) Transfusion Burden and Events in β-Thalassemia with Luspatercept: Longitudinal Results of the Believe Trial

Ali T. Taher, Vip Viprakasit, Olivier Hermine, John B. Porter, MA, MD, FRCP, FRCPath, Antonio Piga, MD, Kevin H.M. Kuo, MDMSc,FRCPC, Thomas D. Coates, MD, Ersi Voskaridou, Abderrahim Khelif, Antonis Kattamis, Jeevan K. Shetty, George Zhang, Yu (Olivia) Tian, Dimana Miteva, Tatiana Zinger, Derek Tang, Jay T. Backstrom, Maria Domenica Cappellini

Blood (2020) 136 (Supplement 1): 45–46.

https://doi.org/10.1182/blood-2020-140310

Published: 2020

Journal Articles

Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy (ICT) in Adult Patients (Pts) with β-Thalassemia in the Believe Trial

Olivier Hermine, Maria Domenica Cappellini, Ali T. Taher, Thomas D. Coates, MD, Vip Viprakasit, Ersi Voskaridou, Ashutosh Lal, MD, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Jeevan K. Shetty, George Zhang, Yu (Olivia) Tian, Dimana Miteva, Tatiana Zinger, Derek Tang, Jay T. Backstrom, John B. Porter, MA, MD, FRCP, FRCPath

Blood (2020) 136 (Supplement 1): 47–48.

https://doi.org/10.1182/blood-2020-136517

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

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

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Donatella Baronciani, MD, Maddalena Casale, MD, Lucia De Franceschi, MD, Giovanna Graziadei, MD, Filomena Longo, MD, Raffaella Origa, MD, Valeria Maria Pinto, MD, Paolo Rigano, MD, Monia Marchetti, MD, Antonia Gigante, Emanuele Angelucci, MD, Maria Domenica Cappellini, MD, Achille Iolascon, MD PhD, Antonio Piga, Gian Luca Forni

Blood (2019) 134 (Supplement_1): 972.

https://doi.org/10.1182/blood-2019-128320

Published: 2019

Journal Articles

Fyn Specifically Regulates the Activity of Red Cell Glucose-6-Phosphate-Dehydrogenase

Blood (2019) 134 (Supplement_1): 3527.

https://doi.org/10.1182/blood-2019-125709

Published: 2019

Journal Articles

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Paola Delbini, PhD, Viola Ghiandai, Maria Domenica Cappellini, MD, Lorena Duca, Isabella Nava, Elena Cassinerio, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3592.

https://doi.org/10.1182/blood-2019-130032

Published: 2019

NARROW

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

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

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

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

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

Sustained Reductions in Red Blood Cell (RBC) Transfusion Burden and Events in β-Thalassemia with Luspatercept: Longitudinal Results of the Believe Trial

Longitudinal Effect of Luspatercept Treatment on Iron Overload and Iron Chelation Therapy (ICT) in Adult Patients (Pts) with β-Thalassemia 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

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

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Fyn Specifically Regulates the Activity of Red Cell Glucose-6-Phosphate-Dehydrogenase

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

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American Society of Hematology

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