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Journal Articles

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Blood (2024) 144 (12): 1329–1342.

https://doi.org/10.1182/blood.2023022751

Published: 2024

Includes: Supplemental data

Journal Articles

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Blood (2024) 144 (3): 323–333.

https://doi.org/10.1182/blood.2024024173

Published: 2024

Journal Articles

Transplant Outcomes for Acute Myeloid Leukemia or Myelodysplastic Syndromes Secondary to Acquired Aplastic Anemia or Paroxysmal Nocturnal Hemoglobinuria: A Report from the EBMT Severe Aplastic Anemia Working Party

Blood (2023) 142 (Supplement 1): 704.

https://doi.org/10.1182/blood-2023-184783

Published: 2023

Journal Articles

Atgam Efficacy and Safety in Moderate-to-Very Severe Acquired Aplastic Anemia: Outcome of a Large Multicenter Cohort of 634 Children and Adults from the French Authorization for Temporary Use Surveillance Program

Flore Sicre De Fontbrune, Mony Fahd, Édouard Forcade, Suzanne Tavitian, Cécile Moluçon Chabrot, Fiorenza Barraco, Yosr Hicheri, Delphine Lebon, Sébastien Maury, Anne-Lise Menard, Barbara Możejko-Pastewka, Kevin D Wolter, Bruno Valtier, Thierry Leblanc, Regis Peffault De Latour

Blood (2023) 142 (Supplement 1): 1347.

https://doi.org/10.1182/blood-2023-186493

Published: 2023

Journal Articles

Resolution of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) with Defibrotide Following HCT in Adult and Pediatric Patients: Pooled Analysis of Defifrance and EBMT PASS Registries

Mohamad Mohty, Franco Locatelli, Didier Blaise, Regis Peffault De Latour, Hélène Labussière-Wallet, Vian Amber, Virginie Gandemer, Deborah Gutierrez, Nalina Dronamraju, Jean-Hugues Dalle, Micha Srour

Blood (2023) 142 (Supplement 1): 3527.

https://doi.org/10.1182/blood-2023-185963

Published: 2023

Journal Articles

Relapse Incidence Post Unrelated Allogeneic Stem Cell Transplantation with Post-Transplant Cyclophosphamide (PTCy) Versus Conventional Anti-Graft Versus Host Disease Prophylaxis in Patients with Acute Myeloid Leukemia: A Study on Behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Blood (2023) 142 (Supplement 1): 237.

https://doi.org/10.1182/blood-2023-174020

Published: 2023

Journal Articles

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry

Austin Kulasekararaj, Hubert Schrezenmeier, Kensuke Usuki, Aleksander Kulagin, Sandra Fatima Gualandro, Rosario Notaro, Masayo Ogawa, Ji Yu, Ami Patel, Yogesh Patel, Wolfgang Füreder, Regis Peffault De Latour, Jong-Wook Lee

Blood (2023) 142 (Supplement 1): 2714.

https://doi.org/10.1182/blood-2023-189150

Published: 2023

Journal Articles

Outcomes and Late Complications after Umbilical Cord Blood Transplantation for Fanconi Anemia: A Study on Behalf of Eurocord and Severe Aplastic Anemia Working Party - EBMT

Hanadi Rafii, Regis Peffault De Latour, Marc Bierings, Jean-Hugues Dalle, Mouhab F. Ayas, Rawad Rihani, Edoardo Lanino, Chantal Kenzey, Fernanda Volt, Monica M. M Rivera Franco, Barbara Cappelli, Graziana Scigliuolo, Vanderson Rocha, Annalisa Ruggeri, Antonio M Risitano, Éliane Gluckman

Blood (2023) 142 (Supplement 1): 4948.

https://doi.org/10.1182/blood-2023-185841

Published: 2023

Journal Articles

Trends in Allogeneic Stem Cell Transplantation for Good Risk Acute Myelogenous Leukemia in First Complete Remission: A Longitudinal Study of > 15 Years from the ALWP/EBMT

Blood (2023) 142 (Supplement 1): 3525.

https://doi.org/10.1182/blood-2023-174599

Published: 2023

Journal Articles

Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Carlos De Castro, Phillip Scheinberg, Bing Han, Susan Vallow, Georgina Bermann, Marion Dahlke, Zhixin Wang, Rakesh Kumar, Gavin Dickie, Caitlyn Lowe, Kaelyn Rupinski, Amber Nieves, Regis Peffault De Latour

Blood (2023) 142 (Supplement 1): 7339.

https://doi.org/10.1182/blood-2023-186840

Published: 2023

Journal Articles

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Regis Peffault De Latour, Austin Kulasekararaj, Phillip Scheinberg, Philippe Ferber, Tomasz Lawniczek, Christine Thorburn, Marion Dahlke, Antonio M Risitano

Blood (2023) 142 (Supplement 1): 1338.

https://doi.org/10.1182/blood-2023-179377

Published: 2023

Journal Articles

How Much Have Post-Transplant Outcomes Improved Since 2000 for Patients with Philadelphia-Positive Acute Lymphoblastic Leukemia in First Remission? a Study from the EBMT Acute Leukemia Working Party

Ali Bazarbachi, Myriam Labopin, Iman Abou Dalle, Ibrahim Yakoub-Agha, Regis Peffault De Latour, Thomas Schroeder, Didier Blaise, Xavier Poire, Marie Balsat, Urpu Salmenniemi, Nicolaus Kröger, Aleksandr Kulagin, Sr., Eva Wagner Drouet, Depei Wu, Eolia Brissot, Arnon Nagler, Sebastian Giebel, Fabio Ciceri, Mohamad Mohty

Blood (2023) 142 (Supplement 1): 2234.

https://doi.org/10.1182/blood-2023-179578

Published: 2023

Journal Articles

UM171 Expanded Cord Blood Transplantation Outcomes Compare Favorably to All Different Stem Cell Sources: An EBMT Registry Case-Control Analysis

Blood (2023) 142 (Supplement 1): 4977.

https://doi.org/10.1182/blood-2023-173727

Published: 2023

Journal Articles

Treatment of Adverse-Risk and Refractory/Relapsed Acute Myeloid Leukemia (AML) Patients with FLAG-IDA ± Venetoclax and CLAG-M: A Monocentric Experience

Ramy Rahmé, Valérie Vidal, Thomas Hueso, Lucie Le Meur, Marthe Rigal, Sarah Ivanoff, Sabine Brechignac, Regis Peffault De Latour, Claude Gardin, Thorsten Braun

Blood (2023) 142 (Supplement 1): 1520.

https://doi.org/10.1182/blood-2023-174356

Published: 2023

Journal Articles

Current Results of Intensive Therapy in Younger Adults with Acute Myeloid Leukemia (AML): The Large Randomized French Backbone Intergroup (BIG)-1 Study on Behalf of the Filo, ALFA, and SFGM-TC Study Groups

Blood (2023) 142 (Supplement 1): 967.

https://doi.org/10.1182/blood-2023-179376

Published: 2023

Journal Articles

Efficacy and Safety Is Maintained in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan for up to 3 Years

Carlos M de Castro, Brian Mulherin, Christopher J. Patriquin, Veena Selvaratnam, Raymond SM Wong, Richard J. Kelly, Lisa Tan, Peter Hillmen, Dale Zhang, Jessica Savage, Regis Peffault De Latour

Blood (2023) 142 (Supplement 1): 574.

https://doi.org/10.1182/blood-2023-181054

Published: 2023

Journal Articles

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Blood (2023) 142 (Supplement 1): 571.

https://doi.org/10.1182/blood-2023-180780

Published: 2023

Journal Articles

Changes in the Immunogenetic Landscape with the Age of Disease Onset in Acquired Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria: An Analysis of a Cohort of Adult and Pediatric Patients from the French National Reference Center for Aplastic Anemia

Vincent Allain, Juliette Villemonteix, Thierry Leblanc, Florian Chevillon, Catherine Fortier, Mony Fahd, Jean-Hugues Dalle, Gerard Socié, Regis Peffault De Latour, Sophie Caillat-Zucman, Flore Sicre De Fontbrune

Blood (2023) 142 (Supplement 1): 1339.

https://doi.org/10.1182/blood-2023-185528

Published: 2023

Journal Articles

Exposure-Response Relationships between the Complement Factor B Inhibitor Iptacopan and Lactate Dehydrogenase (LDH) and Hemoglobin (Hb) in Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Antonio M Risitano, Regis Peffault De Latour, Jun Ho Jang, Eng-Soo Yap, Christian Bartels, Guido Junge, Marion Dahlke, Irina Baltcheva

Blood (2023) 142 (Supplement 1): 5643.

https://doi.org/10.1182/blood-2023-179960

Published: 2023

Journal Articles

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

Antonio M Risitano, Bing Han, Austin Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw P. Maciejewski, Josefin Snellman, Ranjan Tiwari, Marion Dahlke, Régis Peffault de Latour

Blood (2023) 142 (Supplement 1): 4084.

https://doi.org/10.1182/blood-2023-180866

Published: 2023

NARROW

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Transplant Outcomes for Acute Myeloid Leukemia or Myelodysplastic Syndromes Secondary to Acquired Aplastic Anemia or Paroxysmal Nocturnal Hemoglobinuria: A Report from the EBMT Severe Aplastic Anemia Working Party

Atgam Efficacy and Safety in Moderate-to-Very Severe Acquired Aplastic Anemia: Outcome of a Large Multicenter Cohort of 634 Children and Adults from the French Authorization for Temporary Use Surveillance Program

Resolution of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) with Defibrotide Following HCT in Adult and Pediatric Patients: Pooled Analysis of Defifrance and EBMT PASS Registries

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry

Outcomes and Late Complications after Umbilical Cord Blood Transplantation for Fanconi Anemia: A Study on Behalf of Eurocord and Severe Aplastic Anemia Working Party - EBMT

Trends in Allogeneic Stem Cell Transplantation for Good Risk Acute Myelogenous Leukemia in First Complete Remission: A Longitudinal Study of > 15 Years from the ALWP/EBMT

Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

How Much Have Post-Transplant Outcomes Improved Since 2000 for Patients with Philadelphia-Positive Acute Lymphoblastic Leukemia in First Remission? a Study from the EBMT Acute Leukemia Working Party

UM171 Expanded Cord Blood Transplantation Outcomes Compare Favorably to All Different Stem Cell Sources: An EBMT Registry Case-Control Analysis

Treatment of Adverse-Risk and Refractory/Relapsed Acute Myeloid Leukemia (AML) Patients with FLAG-IDA ± Venetoclax and CLAG-M: A Monocentric Experience

Current Results of Intensive Therapy in Younger Adults with Acute Myeloid Leukemia (AML): The Large Randomized French Backbone Intergroup (BIG)-1 Study on Behalf of the Filo, ALFA, and SFGM-TC Study Groups

Efficacy and Safety Is Maintained in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan for up to 3 Years

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Changes in the Immunogenetic Landscape with the Age of Disease Onset in Acquired Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria: An Analysis of a Cohort of Adult and Pediatric Patients from the French National Reference Center for Aplastic Anemia

Exposure-Response Relationships between the Complement Factor B Inhibitor Iptacopan and Lactate Dehydrogenase (LDH) and Hemoglobin (Hb) in Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

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American Society of Hematology

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