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Maria Domenica Cappellini
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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
Journal: Blood
Blood (2024) 144 (Supplement 1): 3857.
https://doi.org/10.1182/blood-2024-200066
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

Maria Domenica Cappellini, Sujit Sheth, Ali T. Taher, Hanny Al-Samkari, Ali Bülent Antmen, David Beneitez, Giovanna Cannas, Thomas D. Coates, Lauren Czapla, Jayme L. Dahlin, Jeremie H. Estepp, Elizabeth Feenstra, Pencho Georgiev, Sarah Gheuens, Keely S Gilroy, Andreas Glenthøj, Khaled Musallam, Kareem Osman, John B. Porter, Hui Shao, Katrin Uhlig, Eduard J. Van Beers, Vip Viprakasit, Kevin H.M. Kuo, Antonis Kattamis
Journal: Blood
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 2475.
https://doi.org/10.1182/blood-2024-200157
Published: 2024
Journal Articles

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

Franco Locatelli, Peter Lang, Roland Meisel, Donna Wall, Selim Corbacioglu, Amanda M Li, Josu de la Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Y Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan A. Grupp, Hayley Lynne Merkeley, Kevin H.M. Kuo, Joachim Rupprecht, Puja Kohli, Gang Xu, Leorah Ross, Yael Bobruff, Bo Tong, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2024) 144 (Supplement 1): 512.
https://doi.org/10.1182/blood-2024-205779
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 7949.
https://doi.org/10.1182/blood-2024-203302
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
Journal: Blood
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 5251.
https://doi.org/10.1182/blood-2023-181629
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 3849.
https://doi.org/10.1182/blood-2023-174089
Published: 2023
Journal Articles

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Franco Locatelli, Peter Lang, Selim Corbacioglu, Donna Wall, Roland Meisel, Amanda M Li, Josu de La Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan Grupp, Puja Kohli, Daoyuan Shi, Leorah Ross, Yael Bobruff, Christopher Simard, Lanju Zhang, Phuong Khanh Morrow, William Hobbs, Haydar Frangoul
Journal: Blood
Blood (2023) 142 (Supplement 1): 1053.
https://doi.org/10.1182/blood-2023-190534
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
Journal: Blood
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
Journal: Blood
Blood (2023) 142 (Supplement 1): 2474.
https://doi.org/10.1182/blood-2023-173366
Published: 2023
Journal Articles

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

Clinical Trials & Observations
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2021) 138 (Supplement 1): 572.
https://doi.org/10.1182/blood-2021-148366
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
Journal: Blood
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
Journal: Blood
Blood (2020) 136 (Supplement 1): 8–9.
https://doi.org/10.1182/blood-2020-136312
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

Haydar Frangoul, MD, Yael Bobruff, PhD, Maria Domenica Cappellini, Selim Corbacioglu, MD, Christine Marie Fernandez, RN,MSN, Josu de la Fuente, Stephan A Grupp, MD PhD, Rupert Handgretinger, MD, Tony W. Ho, MD, Suzan Imren, MD, Antonis Kattamis, Julie Lekstrom-Himes, MD, Franco Locatelli, MD PhD, Yimeng Lu, PhD, Markus Y Mapara, MD PhD, Mariane de Montalembert, MD PhD, Sarah D. Mulcahey, MS, Damiano Rondelli, MD, Niraj Shanbhag, MD PhD, Sujit Sheth, MD, Sandeep Soni, MBBS, Martin H. Steinberg, MD, Michael J. Weinstein, John K. Wu, MBBS, FRCPC, MS, Donna Wall, MDCCPE
Journal: Blood
Blood (2020) 136 (Supplement 1): 3–4.
https://doi.org/10.1182/blood-2020-139575
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2020) 136 (Supplement 1): 21–22.
https://doi.org/10.1182/blood-2020-140819
Published: 2020