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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

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

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) 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

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

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

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

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

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

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

Journal Articles

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

Natalia Scaramellini, MD, Carola Arighi, MD, Alessia Marcon, MD, Dario Consonni, MD, Elena Cassinerio, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3542.

https://doi.org/10.1182/blood-2019-130237

Published: 2019

Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

Published: 2019

Journal Articles

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2019) 134 (Supplement_1): 3545.

https://doi.org/10.1182/blood-2019-122685

Published: 2019

Journal Articles

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

John Porter, Maria Domenica Cappellini, MD, Thomas Coates, MD, Olivier Hermine, Vip Viprakasit, MD DPhil, Ersi Voskaridou, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Abderrahmane Laadem, Jeevan K. Shetty, Wen-Ling Kuo, Dimana Miteva, Tatiana Zinger, Peter G. Linde, Daniel Sinsimer, Ali Taher

Blood (2019) 134 (Supplement_1): 2245.

https://doi.org/10.1182/blood-2019-122561

Published: 2019

Journal Articles

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Christophe Lechauve, PhD, Julia Keith, Eugene Khandros, MD PhD, Stephanie Fowler, Kalin Mayberry, Abdullah Freiwan, Christopher Thom, MD PhD, Jingjing Zhang, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Irene Motta, MD, Maria Domenica Cappellini, MD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 411.

https://doi.org/10.1182/blood-2018-99-113901

Published: 2018

Journal Articles

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2018) 132 (Supplement 1): 163.

https://doi.org/10.1182/blood-2018-163

Published: 2018

NARROW

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

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

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) As a Novel Strategy to Target Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

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

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

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

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

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

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

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American Society of Hematology

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ASH Publications

American Society of Hematology

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