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Journal Articles

Transplantation for Congenital Sideroblastic Anaemia Is Feasible and Offers Outcomes Comparable to Other Transfusion Dependent Anaemias. a Joint Retrospective Study of the Paediatric Diseases and Severe Aplastic Anaemia Working Parties (PDWP/SAAWP) of EBMT

Blood (2020) 136 (Supplement 1): 45–47.

DOI: https://doi.org/10.1182/blood-2020-142397

Published: 2020

Journal Articles

Upfront Alternative Donor Transplant Versus Immunosuppressive Therapy in Patients with Severe Aplastic Anemia Who Lack Fully HLA Matched Related Donor: Systematic Review and Meta-Analysis of Retrospective Studies. on Behalf of the Severe Aplastic Anemia Working Party of European Group for Blood and Marrow Transplantation (SAAWP of EBMT)

Hind Alotaibi, MBBS, Mahmoud Aljurf, Regis Peffault De Latour, MD PhD, Shahid Iqbal, Andrea Bacigalupo, Judith Marsh, Hubert Schrezenmeier, Eliane Gluckman, Mansour Alfayez, Britta Hoechsmann, Constantijn Halkes, Josu de la Fuente, Nawal AlShehry, Simone Cesaro, Jakob Passweg, Carlo Dufour, Antonio Risitano, John F. DiPersio, MD PhD, Ibraheem Motabi

Blood (2020) 136 (Supplement 1): 6–7.

DOI: https://doi.org/10.1182/blood-2020-140846

Published: 2020

Journal Articles

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 2 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Not Previously Treated with Complement Inhibitors

Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Guangsheng He, MD PhD, Talha Munir, MBBS, Jeffrey Pu, MDPhD, Antonio Risitano, Alexander Röth, MD, Camelia S. Sima, MD, Anita Appius, PhD, Alexandre Sostelly, PhD, Sasha Sreckovic, MD, Charlotte Vignal, PhD, Jun-Ichi Nishimura, MD PhD

Blood (2020) 136 (Supplement 1): 34.

DOI: https://doi.org/10.1182/blood-2020-136647

Published: 2020

Journal Articles

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 1 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Complement Inhibitors

Antonio Risitano, Alexander Röth, MD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Jeffrey Pu, MDPhD, Jun-ichi Nishimura, MD PhD, Lilyan Wright, PhD, Anita Appius, PhD, Alexandre Sostelly, PhD, Sasha Sreckovic, MD, Charlotte Vignal, PhD, Talha Munir, MBBS

Blood (2020) 136 (Supplement 1): 43–44.

DOI: https://doi.org/10.1182/blood-2020-137471

Published: 2020

Journal Articles

Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS)

Antonio Risitano, Ilene C. Weitz, Carlos M. de Castro, MD, Jean-Jacques Kiladjian, Morag Griffin, MBChB, FRCPath, Hisakazu Nishimori, MD PhD, Mohamed Hamdani, Temitayo Ajayi, Scott B. Baver, PhD, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 44–45.

DOI: https://doi.org/10.1182/blood-2020-142166

Published: 2020

Journal Articles

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

Antonio Risitano, Jun-Ho Jang, MD, Lee Gyeong-Won, Wanchai Wanachiwanawin, Hubert Schrezenmeier, Yuji Yonemura, MDPhD, Talha Munir, MBBS, Rodrigo Pavani, Jimmy Wang, Alexander D. Kulagin, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Flore Sicre de Fontbrune, Alexander Röth, MD

Blood (2020) 136 (Supplement 1): 31–33.

DOI: https://doi.org/10.1182/blood-2020-140418

Published: 2020

Journal Articles

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Peter Hillmen, MBChB, PhD, Jeffrey Szer, MBBS, Ilene C. Weitz, Alexander Röth, MD, Britta Hoechsmann, Jens Panse, MD, Kensuke Usuki, MD PhD, Morag Griffin, MBChB, FRCPath, Jean-Jacques Kiladjian, MD PhD, Carlos M. de Castro, MD, Hisakazu Nishimori, MD PhD, Lisa Tan, Mohamed Hamdani, Pascal Deschatelets, PhD, Cedric Francois, MD PhD, Federico Grossi, MD, Antonio Risitano, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 35–37.

DOI: https://doi.org/10.1182/blood-2020-142174

Published: 2020

Journal Articles

BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Austin Kulasekararaj, MD MRCP, FRCPath, Jacques Le Roux Malherbe, MD, Andrew McDonald, Melanie Cornpropst, PharmD, PhD, Phil Collis, PhD, Matthew Davidson, PharmD, Xilin Chen, MDPhD, Kristin Tower, Diane Gesty-Palmer, MD PhD, William P. Sheridan, MBBS, Antonio Risitano

Blood (2020) 136 (Supplement 1): 14–15.

DOI: https://doi.org/10.1182/blood-2020-138838

Published: 2020

Journal Articles

Phase 3 Study of Danicopan, an Oral Complement Factor D Inhibitor, As Add-on Therapy to a C5 Inhibitor in Patients with Paroxysmal Nocturnal Hemoglobinuria with Clinically Evident Extravascular Hemolysis

Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Antonio Risitano, Jong Wook Lee, MD, PhD, Mingjun Huang, PhD, Jun-Ichi Nishimura, MD PhD, Alejandra Ramirez-Santiago, Robert Brodsky

Blood (2020) 136 (Supplement 1): 6–7.

DOI: https://doi.org/10.1182/blood-2020-134388

Published: 2020

Journal Articles

Upfront Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Idiopathic Aplastic Anemia: A Study on Behalf of the Saawp of EBMT

Audrey Françoise Petit, Dirk-Jan Eikema, Alexey Maschan, MD, Dalila Adjaoud, MD, Aleksander Kulagin, Alessandro Rambaldi, MD, Franca Fagioli, MD, Laimonas Griškevičius, MD PhD, John A Snowden, BSc (Hons), MBChB, MD FRCP, FRCPath, Jan-Erik Johansson, MD PhD, Jean-Hugues Dalle, MD PhD, Jenny Byrne, MD PhD, Antonio Risitano, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 11–13.

DOI: https://doi.org/10.1182/blood-2020-136657

Published: 2020

Journal Articles

Gvhd and Relapse Free Survival (GRFS) after Allogeneic Transplantation for Idiopathic Severe Aplastic Anemia: An Analysis from the Saawp Data Quality Initiative Program of EBMT

Blood (2020) 136 (Supplement 1): 3–4.

DOI: https://doi.org/10.1182/blood-2020-141757

Published: 2020

Journal Articles

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study

Austin G. Kulasekararaj, Anita Hill, Scott T. Rottinghaus, Saskia Langemeijer, Richard Wells, F. Ataulfo Gonzalez-Fernandez, Anna Gaya, Jong Wook Lee, Emilio Ojeda Gutierrez, Caroline I. Piatek, Jeff Szer, Antonio Risitano, Shinji Nakao, Eric Bachman, Lori Shafner, Andrew I. Damokosh, Stephan Ortiz, Alexander Röth, Regis Peffault de Latour

Blood (2019) 133 (6): 540–549.

DOI: https://doi.org/10.1182/blood-2018-09-876805

Published: 2019

Includes: Supplemental data

Journal Articles

HLA B1402, Cw0802 Haplotype and DRB1*1501 Allele Are Significantly Increased in Italian Paroxysmal Nocturnal Hemoglobinuria Affected Patients.

Fiorella Alfinito, Elena Cosentini, Rosa Camerlengo, Giuseppe Terrazzano, Michela Sica, Antonio Risitano, Stefania Costagliola, Roberta Della Pepa, Carmela Palladino, Lucio Luzzatto, Bruno Rotoli, Rosario Notaro, Giuseppina Ruggiero

Blood (2007) 110 (11): 4035.

DOI: https://doi.org/10.1182/blood.V110.11.4035.4035

Published: 2007

Journal Articles

Analysis of Immunogenetic Factors in Paroxysmal Nocturnal Hemoglobinuria Reveal Increased Frequency of IL-10 Low Secretor Genotype and Correlates with Induced IL-10 Production.

Giridharan Ramsingh, MD, Zachary Nearman, Chris Hung, Serio Bianca, M.D, Antonio Risitano, M.D, Jaroslaw Maciejewski, M.D., Ph.D

Blood (2006) 108 (11): 983.

DOI: https://doi.org/10.1182/blood.V108.11.983.983

Published: 2006

Journal Articles

Distinct clinical outcomes for cytogenetic abnormalities evolving from aplastic anemia

Jaroslaw P. Maciejewski, Antonio Risitano, Elaine M. Sloand, Olga Nunez, Neal S. Young

Blood (2002) 99 (9): 3129–3135.

DOI: https://doi.org/10.1182/blood.V99.9.3129

Published: 2002

NARROW

Transplantation for Congenital Sideroblastic Anaemia Is Feasible and Offers Outcomes Comparable to Other Transfusion Dependent Anaemias. a Joint Retrospective Study of the Paediatric Diseases and Severe Aplastic Anaemia Working Parties (PDWP/SAAWP) of EBMT

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 2 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Not Previously Treated with Complement Inhibitors

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 1 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Complement Inhibitors

Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS)

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3 Study of Danicopan, an Oral Complement Factor D Inhibitor, As Add-on Therapy to a C5 Inhibitor in Patients with Paroxysmal Nocturnal Hemoglobinuria with Clinically Evident Extravascular Hemolysis

Upfront Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Idiopathic Aplastic Anemia: A Study on Behalf of the Saawp of EBMT

Gvhd and Relapse Free Survival (GRFS) after Allogeneic Transplantation for Idiopathic Severe Aplastic Anemia: An Analysis from the Saawp Data Quality Initiative Program of EBMT

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study

HLA B1402, Cw0802 Haplotype and DRB1*1501 Allele Are Significantly Increased in Italian Paroxysmal Nocturnal Hemoglobinuria Affected Patients.

Analysis of Immunogenetic Factors in Paroxysmal Nocturnal Hemoglobinuria Reveal Increased Frequency of IL-10 Low Secretor Genotype and Correlates with Induced IL-10 Production.

Distinct clinical outcomes for cytogenetic abnormalities evolving from aplastic anemia

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American Society of Hematology

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American Society of Hematology

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