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Journal Articles

Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

Blood (2025) 146 (Supplement 1): 2490.

https://doi.org/10.1182/blood-2025-2490

Published: 2025

Journal Articles

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

Antonio Risitano, Camilla Frieri, Bor-Sheng Ko, Lily Wong Lee Lee, Alexander Röth, Flore Sicre-de-Fontbrune, Eng Soo Yap, Sung-Soo Yoon, Philippe Ferber, Irina Baltcheva, Aurelie Verles, Silvia Sanz, Luca Monaco, Tim Costigan, Regis Peffault De Latour

Blood (2025) 146 (Supplement 1): 3198.

https://doi.org/10.1182/blood-2025-3198

Published: 2025

Journal Articles

Serum ferritin changes in iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria

Antonio Risitano, Carlos de Castro, Camilla Frieri, Bing Han, Richard Kelly, Austin Kulasekararaj, Jaroslaw Maciejewski, Vinod Pullarkat, Alexander Röth, Phillip Scheinberg, Flore Sicre-de-Fontbrune, Yasutaka Ueda, Tim Costigan, Marion Dahlke, Philippe Ferber, Tomasz Lawniczek, Stefanie Senger, Regis Peffault De Latour

Blood (2025) 146 (Supplement 1): 4974.

https://doi.org/10.1182/blood-2025-4974

Published: 2025

Journal Articles

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Austin Kulasekararaj, Morag Griffin, Robert Brodsky, Jun-ichi Nishimura, Christopher Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Anna Gaya, Yogesh Patel, Ying Tang, Gleb Filippov, Mohammed Mahdi, Flore Sicre-de-Fontbrune, Jong Wook Lee

Blood (2025) 146 (Supplement 1): 949.

https://doi.org/10.1182/blood-2025-949

Published: 2025

Journal Articles

Real-world use of oral iptacopan monotherapy in paroxysmal nocturnal hemoglobinuria

Blood (2025) 146 (Supplement 1): 1427.

https://doi.org/10.1182/blood-2025-1427

Published: 2025

Journal Articles

Thymoglobuline and grafalon show comparable transplant outcomes in patients with aplastic anemia undergoing allogeneic stem cell transplantation: A multicenter EBMT saawp study of 1603 patients

Blood (2025) 146 (Supplement 1): 3192.

https://doi.org/10.1182/blood-2025-3192

Published: 2025

Journal Articles

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial)

Britta Hoechsmann, Philippe Schafhausen, Fabian Beier, Michael Heuser, Joerg Westermann, Beatrice Drexler, Alexander Röth, Mathias Hänel, Flore Sicre-de-Fontbrune, Astrid Marx-Hofmann, Antonia Beitzen-Heineke, Jens Panse, Felicitas R Thol, Sixten Koerper, Tim H. Brummendorf, Hubert Schrezenmeier

Blood (2025) 146 (Supplement 1): 980.

https://doi.org/10.1182/blood-2025-980

Published: 2025

Journal Articles

Deep immune signature of immune-mediated aplastic anemia patients shows distinct subsets of regulatory T cells associated with response to treatment: Results from the phase 3, randomized EBMT race clinical trial

Blood (2025) 146 (Supplement 1): 26.

https://doi.org/10.1182/blood-2025-26

Published: 2025

Journal Articles

The CSMD1 gene is involved in hematopoietic stem cell HSC pruning in patients with acquired immune-mediated aplastic anemia

Antonio Risitano, Deniz Ece Kaya, Simona Iacobelli, Riley Cook, Giorgio Napolitani, Sila Gerlevik, Mohammad M Karimi, Nogayhan Seymen, Flore Sicre-de-Fontbrune, Morag Griffin, Camilla Frieri, Carlo Dufour, Regis Peffault De Latour, Ghulam Mufti, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 4982.

https://doi.org/10.1182/blood-2025-4982

Published: 2025

Journal Articles

Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dl on anti-C5 therapy: Subgroup analysis of the appulse-pnh Phase 3b trial

Blood (2025) 146 (Supplement 1): 4981.

https://doi.org/10.1182/blood-2025-4981

Published: 2025

Journal Articles

Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH

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

Blood (2025) 145 (8): 811–822.

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

Published: 2025

Includes: Supplemental data

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

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

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

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

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

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

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

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

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Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

Serum ferritin changes in iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Real-world use of oral iptacopan monotherapy in paroxysmal nocturnal hemoglobinuria

Thymoglobuline and grafalon show comparable transplant outcomes in patients with aplastic anemia undergoing allogeneic stem cell transplantation: A multicenter EBMT saawp study of 1603 patients

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial)

Deep immune signature of immune-mediated aplastic anemia patients shows distinct subsets of regulatory T cells associated with response to treatment: Results from the phase 3, randomized EBMT race clinical trial

The CSMD1 gene is involved in hematopoietic stem cell HSC pruning in patients with acquired immune-mediated aplastic anemia

Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dl on anti-C5 therapy: Subgroup analysis of the appulse-pnh Phase 3b trial

Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH

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

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

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

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)

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

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

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

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

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