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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

Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey

Blood (2018) 132 (Supplement 1): 2389.

https://doi.org/10.1182/blood-2018-99-115938

Published: 2018

Journal Articles

Hemoglobin Utilization in Asplenic and Non-Splenectomized Transfusion Dependent Thalassemia Patients Supported with Pathogen Reduced Red Blood Cell Concentrates in a Phase 3 Study (SPARC)

Yesim Aydinok, Antonio Piga, Raffaella Origa, MD, Nina Mufti, PhD, Anna Erikson, PhD, Anne North, PhD, Katie Waldhaus, Christine Ernst, MD, Jin-Sying Lin, PhD, Norman Huang, PhD, Richard J. Benjamin, MD, Laurence Corash, MD

Blood (2018) 132 (Supplement 1): 3812.

https://doi.org/10.1182/blood-2018-99-112685

Published: 2018

Journal Articles

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Blood (2018) 132 (Supplement 1): 3633.

https://doi.org/10.1182/blood-2018-99-111770

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

Journal Articles

Low Impact of Genetic Modifiers on the Phenotype of Homozygous Beta Thalassemia in the Last Decennial Cohort of Thalassemia Newborns in Sardinia

Paolo Moi, MD, Matteo Marcello, MD, Stefania Satta, PhD, Laura Manunza, PhD, Franca Rosa De Martis, Raffaella Origa, MD, Giovanni Caocci, MD, Susanna Barella, MD

Blood (2017) 130 (Supplement 1): 951.

https://doi.org/10.1182/blood.V130.Suppl_1.951.951

Published: 2017

Journal Articles

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Blood (2017) 130 (Supplement 1): 355.

https://doi.org/10.1182/blood.V130.Suppl_1.355.355

Published: 2017

Journal Articles

Safety and Efficacy of Direct-Acting Antiviral Drugs in Patients with Haemoglobinophaties and Chronic Hepatitis C Infection

Antonio Piga, MD, Paolo Rigano, MD, Raffaella Origa, MD, M. Domenica Cappellini, MD, Valeria Pinto, MD, Immacolata Tartaglione, MD, Aldo Filosa, MD, Domenico D'Ascola, MD, Vito Di Marco, Gian Luca Forni, MD

Blood (2016) 128 (22): 3627.

https://doi.org/10.1182/blood.V128.22.3627.3627

Published: 2016

Journal Articles

International Comparison Study of Toxic Iron Assays in Patients with Iron Overload Disorders

Louise De Swart, MD, Jan C.M. Hendriks, Lisa Van der Vorm, Ioav Z. Cabantchik, PhD, Patricia J. Evans, PhD, Eldad A. Hod, MD, Gary M. Brittenham, MD, Yael Furman, Mirian C.H. Janssen, MD, Bart J Biemond, MD, Marius A. MacKenzie, MD PhD, Vera E.J.M. Mattijssen, MD, Raffaella Origa, MD, Renzo Galanello, MD, Robert C. Hider, Dorine W. Swinkels, MD PhD

Blood (2014) 124 (21): 4033.

https://doi.org/10.1182/blood.V124.21.4033.4033

Published: 2014

Journal Articles

Deferasirox–Deferoxamine Combination Therapy Reduces Cardiac Iron With Rapid Liver Iron Removal In Patients With Severe Transfusional Iron Overload (HYPERION)

Yesim Aydinok, MD, Antonis Kattamis, MD, M Domenica Cappellini, MD, Amal El-Beshlawy, MD, Raffaella Origa, MD, Mohsen Elalfy, MD, Yurdanur Kilinç, MD, Silverio Perrotta, MD, Zeynep Karakas, MD, Vip Viprakasit, MD, Dany Habr, MD, Antje Wegener, MD, Junwu Shen, PhD, John B Porter, MD

Blood (2013) 122 (21): 2257.

https://doi.org/10.1182/blood.V122.21.2257.2257

Published: 2013

Journal Articles

A Phase 2a, Open-Label, Dose-Finding Study To Determine The Safety and Tolerability Of Sotatercept (ACE-011) In Adults With Beta (β)-Thalassemia: Interim Results

Maria-Domenica Cappellini, MD, John Porter, MD, Raffaella Origa, MD, Gian Luca Forni, MD, Adberrahmane Laadem, MD, Frédéric Galacteros, MD PhD, Dimana Miteva, PhD, Victoria Sung, PhD, Rajesh Chopra, MD, Jean-Benoît Arlet, MD, PhD, Jean-Antoine Ribeil, MD, PhD, Kenneth Klesczewski, PhD, Kenneth Attie, MD, Maciej Garbowski, MD, Olivier Hermine, MD, PhD

Blood (2013) 122 (21): 3448.

https://doi.org/10.1182/blood.V122.21.3448.3448

Published: 2013

Journal Articles

Onset of Cardiac Iron Loading in Pediatric Patients.

John C. Wood, MD, PhD, Raffaella Origa, MD, Annalisa Agus, MD, Gildo Matta, MD, Thomas D. Coates, MD, Renzo Galanello, MD

Blood (2007) 110 (11): 2765.

https://doi.org/10.1182/blood.V110.11.2765.2765

Published: 2007

Journal Articles

Glutathione S-Transferase Gene Polymorphism and Heart Iron Overload in Thalassemia Major.

Raffaella Origa, MD, Stefania Satta, PhD, M. Dolores Cipollina, PhD, Lucia Perseu, PhD, Annalisa Agus, MD, Renzo Galanello, MD

Blood (2007) 110 (11): 2767.

https://doi.org/10.1182/blood.V110.11.2767.2767

Published: 2007

Journal Articles

Lack of Heart Iron Overload in Patients with Thalassemia Intermedia.

Renzo Galanello, MD, Mark Tanner, MD, Gildo Matta, MD, Susanna Barella, MD, Patrizio Bina, MD, Raffaella Origa, MD

Blood (2006) 108 (11): 3822.

https://doi.org/10.1182/blood.V108.11.3822.3822

Published: 2006

Journal Articles

Urinary Hepcidin in Thalassemic Syndromes.

Elizabeta Nemeth, PhD, Raffaella Origa, MD, Tomas Ganz, PhD, MD, Renzo Galanello, MD

Blood (2005) 106 (11): 3589.

https://doi.org/10.1182/blood.V106.11.3589.3589

Published: 2005

Journal Articles

Neutropenia in Patients with Thalassemia Major.

Renzo Galanello, MD, Raffaella Origa, MD

Blood (2004) 104 (11): 3763.

https://doi.org/10.1182/blood.V104.11.3763.3763

Published: 2004

Journal Articles

Lack of Correlation between Serum Ferritin and Liver Iron Concentration in Beta-Zero Thalassemia Intermedia.

Renzo Galanello, MD, Nicolina Giagu, MD, Susanna Barella, MD, Liliana Maccioni, PhD, Raffaella Origa, MD

Blood (2004) 104 (11): 3620.

https://doi.org/10.1182/blood.V104.11.3620.3620

Published: 2004

NARROW

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

Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey

Hemoglobin Utilization in Asplenic and Non-Splenectomized Transfusion Dependent Thalassemia Patients Supported with Pathogen Reduced Red Blood Cell Concentrates in a Phase 3 Study (SPARC)

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

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

Low Impact of Genetic Modifiers on the Phenotype of Homozygous Beta Thalassemia in the Last Decennial Cohort of Thalassemia Newborns in Sardinia

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Safety and Efficacy of Direct-Acting Antiviral Drugs in Patients with Haemoglobinophaties and Chronic Hepatitis C Infection

International Comparison Study of Toxic Iron Assays in Patients with Iron Overload Disorders

Deferasirox–Deferoxamine Combination Therapy Reduces Cardiac Iron With Rapid Liver Iron Removal In Patients With Severe Transfusional Iron Overload (HYPERION)

A Phase 2a, Open-Label, Dose-Finding Study To Determine The Safety and Tolerability Of Sotatercept (ACE-011) In Adults With Beta (β)-Thalassemia: Interim Results

Onset of Cardiac Iron Loading in Pediatric Patients.

Glutathione S-Transferase Gene Polymorphism and Heart Iron Overload in Thalassemia Major.

Lack of Heart Iron Overload in Patients with Thalassemia Intermedia.

Urinary Hepcidin in Thalassemic Syndromes.

Neutropenia in Patients with Thalassemia Major.

Lack of Correlation between Serum Ferritin and Liver Iron Concentration in Beta-Zero Thalassemia Intermedia.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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