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Journal Articles

Long-Term Efficacy and Safety of Danicopan as Add-On Therapy to Ravulizumab or Eculizumab in PNH With Significant EVH

Austin G Kulasekararaj, Morag Griffin, Caroline I Piatek, Jamile Shammo, Jun-ichi Nishimura, Christopher J Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jens Peter Panse, Anna Gaya, Yogesh Patel, Peng Liu, Gleb Filippov, Flore Sicre de Fontbrune, Antonio Maria Risitano, Jong Wook Lee

Blood blood.2024026299.

https://doi.org/10.1182/blood.2024026299

Published: 2024

Includes: Supplemental data

Journal Articles

Involvement of Expert Patients in Simulation Courses to Train Residents on Communication of Bad News

Juliette Lambert, Paul Roux, Guy Bouguet, Flore Sicre de Fontbrune, Philippe Rousselot, Caroline Besson

Blood (2024) 144 (Supplement 1): 7954.

https://doi.org/10.1182/blood-2024-206857

Published: 2024

Journal Articles

Upfront HSCT with an Alemtuzumab Based Conditioning Regimen in Children with Acquired Aplastic Anemia: A Retrospective Study from the SFGM-TC

Arthur Sterin, Cécile Renard, Virginie Gandemer, Mony Fahd, Benedicte Bruno, Gaelle Stofleth, Nimrod Buchbinder, Marie Angoso, Cecile Pochon, Fanny Rialland Battisti, Marie Robin, Stéphanie Nguyen-Quoc, Jacques-Emanuel Galimard, Jean-Hugues Dalle, Regis Peffault De Latour, Flore Sicre De Fontbrune

Blood (2024) 144 (Supplement 1): 4075.

https://doi.org/10.1182/blood-2024-194915

Published: 2024

Journal Articles

Dynamics of Oligoclonal Hematopoiesis in Severe Aplastic Anemia Patients Undergoing Immunosuppressive Treatment: Longitudinal Somatic Mutation Analysis from the EBMT Race Trial

Blood (2024) 144 (Supplement 1): 33.

https://doi.org/10.1182/blood-2024-206632

Published: 2024

Journal Articles

Challenging the Adverse Label: Diverse Outcomes of ELN 2022 Adverse Cytogenetic Subgroups in Acute Myeloid Leukemia Patients Undergoing Allogeneic Hematopoietic Cell Transplantation in First Remission: On Behalf of the EBMT Acute Leukemia Working Party

Blood (2024) 144 (Supplement 1): 851.

https://doi.org/10.1182/blood-2024-201380

Published: 2024

Journal Articles

Eltrombopag Added to Standard Immunosuppressive Treatment Improves Long-Term Outcomes As Front-Line Therapy for Severe Aplastic Anemia: Final 2-Year Analysis of EBMT-Saawp Race Study

Antonio M Risitano, Simona Iacobelli, Austin G. Kulasekararaj, Marleen Van Os, Sophie Terwel, Morag Griffin, Constantijn J.M. Halkes, Christian Recher, Fiorenza Barraco, Edouard Forcade, Carlos Vallejo, Beatrice Drexler, Jean-Baptiste Méar, Alexander Smith, Emanuele Angelucci, Reinier Raymakers, Marco R De Groot, Etienne Daguindau, Erfan Nur, Wilma Barcellini, Nigel H. Russell, Louis Terriou, Anna Paola Iori, Walter Barberi, Anna Sureda Balari, Isabel Sanchez-Ortega, Blanca Xicoy, Isidro Jarque Ramos, Jamie Cavenagh, Flore Sicre De Fontbrune, Camilla Frieri, Talha Munir, Jennifer Tjon, Suzanne Tavitian, Aline Praire, Laurence Clement, Florence Rabian, Luana Marano, Anita Hill, Elena Palmisani, Petra Muus, Serena Marotta, Fabiana Cacace, Marica Laurino, Jakob Passweg, Sr., Judith Marsh, Gerard Socie, Ghulam Mufti, Carlo Dufour, Regis Peffault De Latour

Blood (2024) 144 (Supplement 1): 302.

https://doi.org/10.1182/blood-2024-194699

Published: 2024

Journal Articles

High Proportion of PNH Type II Neutrophils, I.e. Relative Percentage ≥3%, Is Associated with Thrombosis in Patients Displaying a PNH Clone >1%: Evidence from Analysis of the 5-Year French Nation-Wide Multicenter Observational Study

Blood (2024) 144 (Supplement 1): 4080.

https://doi.org/10.1182/blood-2024-204647

Published: 2024

Journal Articles

Ptcy Versus High Dose of ATG in HLA-Mismatched Unrelated Stem Cell Transplantation for Hematological Malignancies

Jonathan Bordat, Eléonore Kaphan, Marie Robin, Flore Sicre de Fontbrune, Florian Chevillon, Gerard Socie, Alienor Xhaard, Regis Peffault de Latour, David Michonneau

Blood (2024) 144 (Supplement 1): 7343.

https://doi.org/10.1182/blood-2024-208643

Published: 2024

Journal Articles

Long-Term Outcome of Unrelated Cord Blood Transplantation in Patients with Idiopathic Refractory Severe Aplastic Anemia: A Nationwide Phase 2 Study

Blood (2024) 144 (Supplement 1): 596.

https://doi.org/10.1182/blood-2024-194780

Published: 2024

Journal Articles

Outcome of Patients with HTLV-1 Adult T-Cell Leukemia/Lymphoma (ATL) after Allogeneic SCT: Results of the EBMT LWP

Patricia Lopez Pereira, Ali Bazarbachi, Ambroise Marçais, Maud Ngoya, Irma Khvedelidze, Ben Carpenter, Victoria Potter, Flore Sicre de Fontbrune, Alina Daniela Tanase, Lucy Cook, Francesca Kinsella, Emma Nicholson, Annoek E. C. Broers, Jennifer Clay, Sr., Hélène Labussière-Wallet, Fabio Ciceri, Bertram Glass, Olivier Hermine, Ana Maria Sureda Balari

Blood (2024) 144 (Supplement 1): 4950.

https://doi.org/10.1182/blood-2024-207307

Published: 2024

Journal Articles

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Patient-Reported Outcomes from the Phase 3 ALPHA Trial

Caroline Piatek, Jong Wook Lee, Morag Griffin, Jun-Ichi Nishimura, Christopher Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jamile Shammo, Anna Gaya, Mohammed Mahdi, Peng Liu, Karl-Johan Myren, Andrew Messali, Flore Sicre de Fontbrune, Jens Panse, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 2692.

https://doi.org/10.1182/blood-2024-199506

Published: 2024

Journal Articles

Efficacy and Safety of Pegcetacoplan in PNH Patients with Extra-Vascular Hemolysis Under Anti-C5: The Real-Life Experience of the National French Reference Center

Flore Sicre de Fontbrune, Edouard Forcade, Louis Terriou, Alice Garnier, Vincent Alcazer, Nicolas Daguindau, Lynda Maafa, Isabelle Brindel, Regis Peffault de Latour, Sylvie Chevret

Blood (2024) 144 (Supplement 1): 1321.

https://doi.org/10.1182/blood-2024-199472

Published: 2024

Journal Articles

Role of Age and Donor Type in 3646 Severe Aplastic Anemia Patients Undergoing Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-Saawp Study

Blood (2024) 144 (Supplement 1): 595.

https://doi.org/10.1182/blood-2024-194713

Published: 2024

Journal Articles

Recursive Partitioning As a Tool for Differential Diagnosis between Acquired and Inherited Bone Marrow Failure Syndromes (BMF)

Eléonore Kaphan, Anouk Walter Petrich, Lise Larcher, Thierry Leblanc, Mony Fahd, Benedicte Bruno, Edouard Forcade, Cecile Renard, Arthur Sterin, Michael Loschi, Elodie Lainey, Jean Soulier, Caroline Kannengiesser, Gerard Socie, Regis Peffault De Latour, Jerome Lambert, Flore Sicre de Fontbrune

Blood (2024) 144 (Supplement 1): 1314.

https://doi.org/10.1182/blood-2024-201084

Published: 2024

Journal Articles

Hematological Landscape of Adult Patients with Diamond-Blackfan Anaemia Syndrome (DBAS): Results from the French DBA Cohort

Nicolas Lecornec, Flore Sicre de Fontbrune, Loïc Garçon, Edouard Forcade, Louis Terriou, Isabelle Marie, Régis Peffault de Latour, Jean-Hugues Dalle, Lydie Da Costa, Thierry Leblanc

Blood (2024) 144 (Supplement 1): 1329.

https://doi.org/10.1182/blood-2024-209769

Published: 2024

Journal Articles

Allogeneic Stem-Cell Transplantation for Relapsed/Refractory ALK-Positive Anaplastic Large-Cell Lymphoma: A Study from the Société Francophone De Greffe De Moelle Et De Thérapie Cellulaire (SFGM-TC)

Blood (2024) 144 (Supplement 1): 4946.

https://doi.org/10.1182/blood-2024-212055

Published: 2024

Journal Articles

Up-Front Matched Unrelated Donor Transplantation in Pediatric Patients with Idiopathic Aplastic Anemia: A Phase II Feasibility Study, on Behalf of Société Francophone De Greffe De Moelle Et De Thérapie Cellulaire (SFGM-TC) and French Reference National Center of Severe Aplastic Anemia

Jean-Hugues Dalle, Sylvie Chevret, Cécile Renard, Virginie Gandemer, Benedicte Bruno, Flore Sicre De Fontbrune, Marie Angoso, Fanny Rialland Battisti, Lynda Maafa, Isabelle Brindel, Nimrod Buchbinder, Mony Fahd, Regis Peffault De Latour

Blood (2024) 144 (Supplement 1): 4859.

https://doi.org/10.1182/blood-2024-198369

Published: 2024

Journal Articles

Germ line ERG haploinsufficiency defines a new syndrome with cytopenia and hematological malignancy predisposition

Jiarna R. Zerella, Claire C. Homan, Peer Arts, Xuzhu Lin, Sam J. Spinelli, Parvathy Venugopal, Milena Babic, Peter J. Brautigan, Lynda Truong, Luis Arriola-Martinez, Sarah Moore, Rachel Hollins, Wendy T. Parker, Hung Nguyen, Karin S. Kassahn, Susan Branford, Simone Feurstein, Lise Larcher, Flore Sicre de Fontbrune, Serwet Demirdas, Sonja de Munnik, Hélène Antoine-Poirel, Benedicte Brichard, Sahar Mansour, Kristiana Gordon, Marcin W. Wlodarski, Ashwin Koppayi, Sara Dobbins, Pim G. N. J. Mutsaers, Kim E. Nichols, Ninad Oak, Desiree DeMille, Rong Mao, Ali Crawford, Julie McCarrier, Donald Basel, Josue Flores-Daboub, Michael W. Drazer, Kerry Phillips, Nicola K. Poplawski, Graeme M. Birdsey, Daniela Pirri, Pia Ostergaard, Annet Simons, Lucy A. Godley, David M. Ross, Devendra K. Hiwase, Jean Soulier, Anna L. Brown, Catherine L. Carmichael, Hamish S. Scott, Christopher N. Hahn

Blood (2024) 144 (17): 1765–1780.

https://doi.org/10.1182/blood.2024024607

Published: 2024

Includes: Supplemental data

Journal Articles

ERG Is a New Predisposition Gene for Bone Marrow Failure and Hematological Malignancy

Blood (2023) 142 (Supplement 2): LBA-6.

https://doi.org/10.1182/blood-2023-191986

Published: 2023

Journal Articles

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis: Phase 3 Long-Term Data

Austin Kulasekararaj, Morag Griffin, Caroline I Piatek, Jamile Shammo, Jun-Ichi Nishimura, Christopher J. Patriquin, Hubert Schrezenmeier, Anna Gaya, Yogesh Patel, Peng Liu, Gleb Filippov, Flore Sicre De Fontbrune, Antonio M Risitano, Jong-Wook Lee

Blood (2023) 142 (Supplement 1): 576.

https://doi.org/10.1182/blood-2023-189863

Published: 2023

NARROW

Long-Term Efficacy and Safety of Danicopan as Add-On Therapy to Ravulizumab or Eculizumab in PNH With Significant EVH

Involvement of Expert Patients in Simulation Courses to Train Residents on Communication of Bad News

Upfront HSCT with an Alemtuzumab Based Conditioning Regimen in Children with Acquired Aplastic Anemia: A Retrospective Study from the SFGM-TC

Dynamics of Oligoclonal Hematopoiesis in Severe Aplastic Anemia Patients Undergoing Immunosuppressive Treatment: Longitudinal Somatic Mutation Analysis from the EBMT Race Trial

Challenging the Adverse Label: Diverse Outcomes of ELN 2022 Adverse Cytogenetic Subgroups in Acute Myeloid Leukemia Patients Undergoing Allogeneic Hematopoietic Cell Transplantation in First Remission: On Behalf of the EBMT Acute Leukemia Working Party

Eltrombopag Added to Standard Immunosuppressive Treatment Improves Long-Term Outcomes As Front-Line Therapy for Severe Aplastic Anemia: Final 2-Year Analysis of EBMT-Saawp Race Study

High Proportion of PNH Type II Neutrophils, I.e. Relative Percentage ≥3%, Is Associated with Thrombosis in Patients Displaying a PNH Clone >1%: Evidence from Analysis of the 5-Year French Nation-Wide Multicenter Observational Study

Ptcy Versus High Dose of ATG in HLA-Mismatched Unrelated Stem Cell Transplantation for Hematological Malignancies

Long-Term Outcome of Unrelated Cord Blood Transplantation in Patients with Idiopathic Refractory Severe Aplastic Anemia: A Nationwide Phase 2 Study

Outcome of Patients with HTLV-1 Adult T-Cell Leukemia/Lymphoma (ATL) after Allogeneic SCT: Results of the EBMT LWP

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Patient-Reported Outcomes from the Phase 3 ALPHA Trial

Efficacy and Safety of Pegcetacoplan in PNH Patients with Extra-Vascular Hemolysis Under Anti-C5: The Real-Life Experience of the National French Reference Center

Role of Age and Donor Type in 3646 Severe Aplastic Anemia Patients Undergoing Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-Saawp Study

Recursive Partitioning As a Tool for Differential Diagnosis between Acquired and Inherited Bone Marrow Failure Syndromes (BMF)

Hematological Landscape of Adult Patients with Diamond-Blackfan Anaemia Syndrome (DBAS): Results from the French DBA Cohort

Allogeneic Stem-Cell Transplantation for Relapsed/Refractory ALK-Positive Anaplastic Large-Cell Lymphoma: A Study from the Société Francophone De Greffe De Moelle Et De Thérapie Cellulaire (SFGM-TC)

Up-Front Matched Unrelated Donor Transplantation in Pediatric Patients with Idiopathic Aplastic Anemia: A Phase II Feasibility Study, on Behalf of Société Francophone De Greffe De Moelle Et De Thérapie Cellulaire (SFGM-TC) and French Reference National Center of Severe Aplastic Anemia

Germ line ERG haploinsufficiency defines a new syndrome with cytopenia and hematological malignancy predisposition

ERG Is a New Predisposition Gene for Bone Marrow Failure and Hematological Malignancy

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis: Phase 3 Long-Term Data

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American Society of Hematology

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American Society of Hematology

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