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Journal Articles

Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities

Blood (2024) 144 (Supplement 2): LBA-5.

https://doi.org/10.1182/blood-2024-212967

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

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

Incidence, Time to Diagnosis, and Therapeutic Landscape of Aplastic Anemia: Results from a Large United States Claims Database

Phillip Scheinberg, Tim H. H. Brummendorf, Regis Peffault De Latour, Carlo Dufour, Raj Gokani, Morag Griffin, Juliana Hey-Hadavi, Yoshinobu Kanda, Eric Mitchell, Barbara Możejko-Pastewka, Andres Quintero, Patryk Skowron, Meltem Kurt Yüksel, Alicia Rovó

Blood (2024) 144 (Supplement 1): 2329.

https://doi.org/10.1182/blood-2024-204908

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

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

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

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

The Influence of HLA Matching of the Winning and Losing Units in Relapse of Patients Receiving Double Cord Blood Transplantation for Acute Leukemia or Myelodysplastic Syndrome

Blood (2024) 144 (Supplement 1): 925.

https://doi.org/10.1182/blood-2024-200785

Published: 2024

Journal Articles

Central Nervous System Disorders (CNSD) Following Hematopoietic Stem Cell Transplantation (HSCT): A Prospective Study from the Infectious Diseases Working Party (IDWP) and the Transplant Complications Working Party (TCWP), European Society of Blood and Marrow Transplantation (EBMT)

Blood (2024) 144 (Supplement 1): 3497.

https://doi.org/10.1182/blood-2024-194695

Published: 2024

Journal Articles

Impact of Donor Type on Outcomes after Allogeneic Hematopoietic Cell Transplantation in Myelofibrosis

Blood (2024) 144 (Supplement 1): 3559.

https://doi.org/10.1182/blood-2024-193992

Published: 2024

Journal Articles

Sequential Intensified Conditioning Regimen Allogeneic Transplantation in Younger Adult Patients with Adverse-Risk Acute Myeloid Leukemia: A Multi-Center Prospective Study from the Acute Leukemia French Intergroup on Behalf of the Filo, ALFA, and SFGM-TC Study Groups

Blood (2024) 144 (Supplement 1): 3483.

https://doi.org/10.1182/blood-2024-198603

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

Oral Iptacopan Monotherapy Leads to Long-Term Improvements in Patient (Pt)-Reported Health-Related Quality of Life (HRQoL) and Investigator-Assessed Signs and Symptoms of Paroxysmal Nocturnal Hemoglobinuria (PNH): 48-Week (Wk) Results from the Phase III APPLY-PNH and APPOINT-PNH Trials

Antonio M Risitano, Bing Han, Austin G. Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw Maciejewski, Josefin Snellman, Olivier Somenzi, Randall Winnette, Samopriyo Maitra, Shujie Li, Marion Dahlke, Regis Peffault De Latour

Blood (2024) 144 (Supplement 1): 4079.

https://doi.org/10.1182/blood-2024-203532

Published: 2024

Journal Articles

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

Régis Peffault de Latour, Jeffrey Szer, Koo Wilson, Piotr Wojciechowski, Zalmai Hakimi, Jameel Nazir, Barbara Czech, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 7706.

https://doi.org/10.1182/blood-2024-209712

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

A First Report from the COMPLETE Study on the Real-World Effectiveness of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Régis Peffault de Latour, Roochi Trikha, Carmen Gonzalez Gonzalez, Matthias Nölte, Håkan Malmström, Stefan Lethagen

Blood (2024) 144 (Supplement 1): 5669.

https://doi.org/10.1182/blood-2024-194458

Published: 2024

Journal Articles

SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post HLA Mismatched Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: Reset-02 First-in-Human Phase I/II

Raynier Devillier, Anne Huynh, Patrice Chevallier, Simona Piemontese, Regis Peffault de Latour, Patrizia Chiusolo, Aurelie Bauquet, Rahim Fandi, Sebastien Oster, Laura Simons, Isabelle Andre, Marina Cavazzana, Fabio Ciceri

Blood (2024) 144 (Supplement 1): 3457.

https://doi.org/10.1182/blood-2024-203203

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

Improved Outcomes in Paroxysmal Nocturnal Hemoglobinuria Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-Saawp Study

Blood (2024) 144 (Supplement 1): 305.

https://doi.org/10.1182/blood-2024-194716

Published: 2024

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Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities

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

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

Incidence, Time to Diagnosis, and Therapeutic Landscape of Aplastic Anemia: Results from a Large United States Claims Database

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

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

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

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

The Influence of HLA Matching of the Winning and Losing Units in Relapse of Patients Receiving Double Cord Blood Transplantation for Acute Leukemia or Myelodysplastic Syndrome

Central Nervous System Disorders (CNSD) Following Hematopoietic Stem Cell Transplantation (HSCT): A Prospective Study from the Infectious Diseases Working Party (IDWP) and the Transplant Complications Working Party (TCWP), European Society of Blood and Marrow Transplantation (EBMT)

Impact of Donor Type on Outcomes after Allogeneic Hematopoietic Cell Transplantation in Myelofibrosis

Sequential Intensified Conditioning Regimen Allogeneic Transplantation in Younger Adult Patients with Adverse-Risk Acute Myeloid Leukemia: A Multi-Center Prospective Study from the Acute Leukemia French Intergroup on Behalf of the Filo, ALFA, and SFGM-TC Study Groups

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

Oral Iptacopan Monotherapy Leads to Long-Term Improvements in Patient (Pt)-Reported Health-Related Quality of Life (HRQoL) and Investigator-Assessed Signs and Symptoms of Paroxysmal Nocturnal Hemoglobinuria (PNH): 48-Week (Wk) Results from the Phase III APPLY-PNH and APPOINT-PNH Trials

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

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

A First Report from the COMPLETE Study on the Real-World Effectiveness of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post HLA Mismatched Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: Reset-02 First-in-Human Phase I/II

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

Improved Outcomes in Paroxysmal Nocturnal Hemoglobinuria Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-Saawp Study

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