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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

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

The Effect of Oral Iptacopan Monotherapy on Hematological Parameters in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Is Consistent Regardless of the Type of Prior Anti-C5 Treatment Received: A Post Hoc Analysis of 24-Week Data from the Randomized Phase III APPLY-PNH Trial

Regis Peffault De Latour, Austin G. Kulasekararaj, Phillip Scheinberg, Yasutaka Ueda, Carlos de Castro, Samopriyo Maitra, Philippe Ferber, Marion Dahlke, Antonio M Risitano

Blood (2024) 144 (Supplement 1): 4087.

https://doi.org/10.1182/blood-2024-200763

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

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

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

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

Cyclosporine Versus Cyclosporine Plus Mycophenolate Mofetil in Reduced Intensity Allogeneic Stem Cell Transplantation for Intermediate-Risk AML: A Multi-Center Randomized Study from the Acute Leukemia French Intergroup, on Behalf of the Filo, ALFA, and SFGM-TC Study Groups

Blood (2024) 144 (Supplement 1): 4909.

https://doi.org/10.1182/blood-2024-203964

Published: 2024

Journal Articles

Incremental Effectiveness of Iptacopan Compared with C5 Inhibitors in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from a Modelling Analysis

Shreyans Gandhi, Regis Peffault De Latour, Katharina Pannagl, Maria-Magdalena Balp, Anggie Wiyani, Cheryl Druchok, Paul Lebeau, Richard J. Kelly

Blood (2024) 144 (Supplement 1): 5012.

https://doi.org/10.1182/blood-2024-201424

Published: 2024

Journal Articles

The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry: Highlights of the Data from the First Three Years

David Araten, Austin G. Kulasekararaj, Jun-ichi Nishimura, Regis Peffault De Latour, Ilene C. Weitz, Azalea Candelaria, Aliza Fink, Alice Houk, Elizabeth W Kottke, Brad A Wong

Blood (2024) 144 (Supplement 1): 3706.

https://doi.org/10.1182/blood-2024-208575

Published: 2024

Journal Articles

Allogeneic Hematopoietic Cell Transplantation for Myelodysplastic Neoplasms with Accompanying Paroxysmal Nocturnal Hemoglobinuria - a Study on Behalf of Cmwp and Saawp of the EBMT

Blood (2024) 144 (Supplement 1): 4949.

https://doi.org/10.1182/blood-2024-203009

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

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

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

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

NARROW

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

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

The Effect of Oral Iptacopan Monotherapy on Hematological Parameters in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Is Consistent Regardless of the Type of Prior Anti-C5 Treatment Received: A Post Hoc Analysis of 24-Week Data from the Randomized Phase III APPLY-PNH Trial

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)

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

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

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

Cyclosporine Versus Cyclosporine Plus Mycophenolate Mofetil in Reduced Intensity Allogeneic Stem Cell Transplantation for Intermediate-Risk AML: A Multi-Center Randomized Study from the Acute Leukemia French Intergroup, on Behalf of the Filo, ALFA, and SFGM-TC Study Groups

Incremental Effectiveness of Iptacopan Compared with C5 Inhibitors in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from a Modelling Analysis

The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry: Highlights of the Data from the First Three Years

Allogeneic Hematopoietic Cell Transplantation for Myelodysplastic Neoplasms with Accompanying Paroxysmal Nocturnal Hemoglobinuria - a Study on Behalf of Cmwp and Saawp of the EBMT

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

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

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

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

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American Society of Hematology

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American Society of Hematology

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