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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.

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

Published: 2020

Journal Articles

Final Primary Results from the Defifrance Registry Study: Effectiveness and Safety of Defibrotide in the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation

Mohamad Mohty, MD PhD, Ibrahim Yakoub-Agha, Myriam Labopin, MD, Didier Blaise, MD, Delphine Lebon, MD, Virginie Gandemer, MD PhD, Sébastien Maury, Cecile Renard, Charlotte Jubert, Robert J. Ryan, Virginie Bouvatier, Jean-Hugues Dalle, MD PhD, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 35–36.

https://doi.org/10.1182/blood-2020-136685

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.

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

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.

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

Published: 2020

Journal Articles

An Optimized Crovalimab Dose and Regimen Reduced the Formation of Drug-Target-Drug Complexes in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Phase I/II COMPOSER Trial

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-136265

Published: 2020

Journal Articles

Risk Factors for Thrombotic Events in Patients with PNH: A Nested Case-Control Study in the International PNH Registry

Britta Hoechsmann, Regis Peffault De Latour, MD PhD, Anita Hill, Alexander Röth, MD, Timothy Devos, MD, Christopher Patriquin, Wen-Chien Chou, MD PhD, Deepak Jain, Ke Zu, Jong Wook Lee, MD, PhD

Blood (2020) 136 (Supplement 1): 6–8.

https://doi.org/10.1182/blood-2020-136767

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.

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

Published: 2020

Journal Articles

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 Years with Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension Studies

Regis Peffault De Latour, MD PhD, Jeffrey Szer, MBBS, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Jin Seok Kim, MD PhD, Caroline I. Piatek, MD, Alexander D. Kulagin, MD PhD, Anita Hill, Jimmy Wang, Ji Yu, Masayo Ogawa, MD FAAP, Hubert Schrezenmeier, Jong Wook Lee, MD, PhD

Blood (2020) 136 (Supplement 1): 42–43.

https://doi.org/10.1182/blood-2020-140777

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.

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

Published: 2020

Journal Articles

Pegcetacoplan Is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement

Regis Peffault De Latour, MD PhD, Carlos M. de Castro, MD, Jeffrey Szer, MBBS, Kensuke Usuki, MD PhD, Peter Hillmen, MBChB, PhD, Morag Griffin, MBChB, FRCPath, Mohamed Hamdani, MS, Temitayo Ajayi, Hisakazu Nishimori, MD PhD, Ilene C. Weitz

Blood (2020) 136 (Supplement 1): 32–33.

https://doi.org/10.1182/blood-2020-141061

Published: 2020

Journal Articles

Efficacy and Safety of Concomitant Use of Ravulizumab and IST in Patients with Paroxysmal Nocturnal Hemoglobinuria up to 52 Weeks

Hubert Schrezenmeier, Jong Wook Lee, MD, PhD, Anita Hill, Vadim V Ptushkin, Viviani Pessoa, Rosario Notaro, Jimmy Wang, Masayo Ogawa, MD FAAP, Shinichiro Okamoto, MD PhD, Lily LL Wong, MD FRCPath, Regis Peffault De Latour, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath

Blood (2020) 136 (Supplement 1): 37–38.

https://doi.org/10.1182/blood-2020-142453

Published: 2020

Journal Articles

Crovalimab for Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Complement C5 Polymorphism - Experience from the Phase I/II COMPOSER Study

Jun-ichi Nishimura, MD PhD, Kensuke Usuki, MD PhD, Julia Ramos, MD PhD, Satoshi Ichikawa, MD PhD, Jules Hernández-Sánchez, PhD, Anna Kiialainen, PhD, Alexandre Sostelly, PhD, Regis Peffault De Latour, MD PhD, Ido Paz-Priel, MD, Alexander Röth, MD

Blood (2020) 136 (Supplement 1): 5–6.

https://doi.org/10.1182/blood-2020-136048

Published: 2020

Journal Articles

COMPOSER Part 4: An Optimized Dosing Strategy for Crovalimab in the Treatment of Complement Inhibitor-Naïve or -Experienced Patients with Paroxysmal Nocturnal Hemoglobinuria

Regis Peffault De Latour, MD PhD, Simona Sica, MD PhD, Julia Ramos, MD, Jules Hernández-Sánchez, PhD, Jin Seok Kim, MD PhD, Anna Kiialainen, PhD, Sung-Soo Yoon, MD PhD, Sasha Sreckovic, MD, Antoine Soubret, PhD, Haruhiko Ninomiya, MD PhD, Alexandre Sostelly, PhD, Jens Panse, MD, Simon Buatois, PharmD, PhD, Hubert Schrezenmeier, Ido Paz-Priel, MD, Jun-ichi Nishimura, MD PhD, Alexander Röth, MD

Blood (2020) 136 (Supplement 1): 6–7.

https://doi.org/10.1182/blood-2020-135841

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.

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

Published: 2020

Journal Articles

Immunogenomics of Aplastic Anemia: The Role of HLA Somatic Mutations and the HLA Evolutionary Divergence

Simona Pagliuca, MD, Carmelo Gurnari, MD, Hassan Awada, MD, Cassandra M Kerr, MS, Bhumika J. Patel, MD, Laila Terkawi, Misam Zawit, MBBS, Sunisa Kongkiatkamon, MD, Pedro H Prata, MD, Ashwin Kishtagari, MBBS, Adam Wahida, Thomas Laframboise, PhD, Valeria Visconte, PhD, Regis Peffault De Latour, MD PhD, Jaroslaw P. Maciejewski, MD PhD

Blood (2020) 136 (Supplement 1): 20–21.

https://doi.org/10.1182/blood-2020-142501

Published: 2020

Journal Articles

Breakthrough Hemolysis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab: Results of a 52-Week Extension from Two Phase 3 Studies

Anita Hill, MD PhD, Caroline I. Piatek, Régis Peffault de Latour, MD PhD, Lily LL Wong, MD FRCPath, Richard A. Wells, MD DPhil, Robert A. Brodsky, MD, Jin Seok Kim, MD PhD, Junichi Nishimura, MD PhD, Philip Kuriakose, MD, Rodrigo Pavani, Peng Liu, Stephan Ortiz, RPh, PhD, Hubert Schrezenmeier, MD, Jong-Wook Lee, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath

Blood (2019) 134 (Supplement_1): 952.

https://doi.org/10.1182/blood-2019-128929

Published: 2019

Journal Articles

Persistence of PNH Clones over Time: Insights from the Mid-Term Analysis of the French Nation-Wide Multicenter Prospective Observational Study

Magali Le Garff-Tavernier, Agathe Pruvot Debliquis, PharmD, Thomas Boyer, MD, Jean-Philippe Vial, MD, Véronique Harrivel, MD, Anne-Catherine Lhoumeau, PhD PharmD, Marie-Christine Jacob, MD, Gerard Socie, MD PhD, Regis Peffault de Latour, MD PhD, Bernard Drenou, MD PhD, Orianne Wagner Ballon, MD PhD

Blood (2019) 134 (Supplement_1): 1218.

https://doi.org/10.1182/blood-2019-123415

Published: 2019

Journal Articles

Prophylactic Antibiotic Use and Risk of Meningococcal Infections in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Eculizumab Who Received Meningococcal Vaccination: Results from the International PNH Registry

Christopher J. Patriquin, MDFRCPC,MSc, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Régis Peffault de Latour, MD PhD, Jun-Ho Jang, MD PhD, Saskia Langemeijer, MD PhD, Alexey A. Maschan, MD, Amanda Wilson, PhD, Deepak Jain, Jaroslaw P. Maciejewski, MD PhD

Blood (2019) 134 (Supplement_1): 4802.

https://doi.org/10.1182/blood-2019-127005

Published: 2019

Journal Articles

Stem Cell Transplantation for Diamond-Blackfan Anemia. a Retrospective Study on Behalf of Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

Blood (2019) 134 (Supplement_1): 44.

https://doi.org/10.1182/blood-2019-124644

Published: 2019

Journal Articles

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Pierre-Edouard Debureaux, MD, Fabiana Cacace, Bruno G.P. Silva, MD, Federica Barone, MD, Rodrigo T Calado, MD, Flore Sicre de Fontbrune, Juliette Soret-Dulphy, MD, Patrizia Ricci, Michela Sica, PhD, Rosario Notaro, MD, Phillip Scheinberg, MD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Antonio M. Risitano, MD PhD, Régis Peffault de Latour, MD PhD, Camilla Frieri, MD

Blood (2019) 134 (Supplement_1): 3517.

https://doi.org/10.1182/blood-2019-125917

Published: 2019

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

Final Primary Results from the Defifrance Registry Study: Effectiveness and Safety of Defibrotide in the Treatment of Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation

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

An Optimized Crovalimab Dose and Regimen Reduced the Formation of Drug-Target-Drug Complexes in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Phase I/II COMPOSER Trial

Risk Factors for Thrombotic Events in Patients with PNH: A Nested Case-Control Study in the International PNH Registry

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)

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 Years with Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension Studies

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

Pegcetacoplan Is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement

Efficacy and Safety of Concomitant Use of Ravulizumab and IST in Patients with Paroxysmal Nocturnal Hemoglobinuria up to 52 Weeks

Crovalimab for Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Complement C5 Polymorphism - Experience from the Phase I/II COMPOSER Study

COMPOSER Part 4: An Optimized Dosing Strategy for Crovalimab in the Treatment of Complement Inhibitor-Naïve or -Experienced Patients with Paroxysmal Nocturnal Hemoglobinuria

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

Immunogenomics of Aplastic Anemia: The Role of HLA Somatic Mutations and the HLA Evolutionary Divergence

Breakthrough Hemolysis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab: Results of a 52-Week Extension from Two Phase 3 Studies

Persistence of PNH Clones over Time: Insights from the Mid-Term Analysis of the French Nation-Wide Multicenter Prospective Observational Study

Prophylactic Antibiotic Use and Risk of Meningococcal Infections in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Eculizumab Who Received Meningococcal Vaccination: Results from the International PNH Registry

Stem Cell Transplantation for Diamond-Blackfan Anemia. a Retrospective Study on Behalf of Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

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American Society of Hematology

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American Society of Hematology

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