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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

Patient-Reported Outcomes: Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis

Caroline I Piatek, Jong-Wook Lee, Morag Griffin, Jun-Ichi Nishimura, Christopher J. Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jamile Shammo, Anna Gaya, Yogesh Patel, Peng Liu, Karl-Johan Myrén, Flore Sicre De Fontbrune, Jens Panse, Austin Kulasekararaj

Blood (2023) 142 (Supplement 1): 1346.

https://doi.org/10.1182/blood-2023-190015

Published: 2023

Journal Articles

Fertility and Pregnancy in Patients with Congenital Neutropenia: Experience of the French Severe Chronic Neutropenia Registry in 565 Patients Above Age of 15 Years

Aurelia Alimi, Evrard Nika, Blandine Beaupain, Claire Fieschi, Flore Sicre De Fontbrune, Nathalie Aladjidi, Despina Moshous, Aline Moignet.Autrel, Coralie Mallebranche, Martin Biosse Duplan, Christine Bellanne-Chantelot, Jean Donadieu

Blood (2023) 142 (Supplement 1): 2545.

https://doi.org/10.1182/blood-2023-184559

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

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

Efficacy and Safety of Subcutaneous Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous Eculizumab: 2-Year Follow-up

Caroline I Piatek, Flore Sicre De Fontbrune, Fahri Sahin, Wolfgang Füreder, Sandra Fatima Gualandro, Wilma Barcellini, Masayo Ogawa, Stephan Ortiz, Ayca Ozol-Godfrey, Jeff Szer, Mustafa Yenerel

Blood (2023) 142 (Supplement 1): 2713.

https://doi.org/10.1182/blood-2023-190060

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

IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria

Blood (2023) 141 (25): 3122–3125.

https://doi.org/10.1182/blood.2023020108

Published: 2023

Includes: Supplemental data

Journal Articles

The clinical picture of ERCC6L2 disease: from bone marrow failure to acute leukemia

Blood (2023) 141 (23): 2853–2866.

https://doi.org/10.1182/blood.2022019425

Published: 2023

Includes: Supplemental data

Journal Articles

Rare germline complement factor H variants in patients with paroxysmal nocturnal hemoglobinuria

Pedro Henrique Prata, Jacques-Emmanuel Galimard, Flore Sicre de Fontbrune, Anna Duval, Paula Vieira Martins, Stephane Roncelin, Pierre-Édouard Debureaux, Anne-Claire Lepretre, Lise Larcher, Rudy Birsen, Ygal Benhamou, Jean Soulier, Gérard Socié, Véronique Fremeaux-Bacchi, Régis Peffault de Latour

Blood (2023) 141 (15): 1812–1816.

https://doi.org/10.1182/blood.2022017019

Published: 2023

Includes: Supplemental data

Journal Articles

Molecular International Prognostic Scoring System (IPSS-M) in Myelodysplastic Syndromes Arising from Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Blood (2022) 140 (Supplement 1): 5788–5789.

https://doi.org/10.1182/blood-2022-160458

Published: 2022

Journal Articles

Immunogenetic, Molecular and Clinical Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Blood (2021) 138 (Supplement 1): 602.

https://doi.org/10.1182/blood-2021-147556

Published: 2021

Journal Articles

Clonal Hematopoiesis Driven By MDM4 Amplification Defines a Canonical Route Towards Secondary MDS/AML in Fanconi Anemia Patients

Blood (2021) 138 (Supplement 1): 860.

https://doi.org/10.1182/blood-2021-147811

Published: 2021

Journal Articles

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

Antonio Risitano, Jun-Ho Jang, MD, Lee Gyeong-Won, Wanchai Wanachiwanawin, Hubert Schrezenmeier, Yuji Yonemura, MDPhD, Talha Munir, MBBS, Rodrigo Pavani, Jimmy Wang, Alexander D. Kulagin, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Flore Sicre de Fontbrune, Alexander Röth, MD

Blood (2020) 136 (Supplement 1): 31–33.

https://doi.org/10.1182/blood-2020-140418

Published: 2020

Journal Articles

How Many Patients Have Congenital Neutropenia? a Population-Based Estimation from the Nationwide French Severe Chronic Neutropenia Registry

Blood (2020) 136 (Supplement 1): 40–41.

https://doi.org/10.1182/blood-2020-135912

Published: 2020

Journal Articles

Impact and Dynamics of TP53 Mutated Clones in Shwachman Diamond Syndrome in a Series of 80 Patients

Jean-Alain Martignoles, Pierre Hirsch, MD PhD, Blandine Beaupain, Thomas Longval, MD, Nawa Hachem, MsC, Mira El-Khoury, PhD, Sophie Kaltenbach, Jean Soulier, MDPhD, Thierry Leblanc, MD, Pierre Simon Rohrlich, MDPhD, Eric Jeziorski, MDPhD, Isabelle Meyts, Dalila Adjaoud, MD, Vincent Barlogis, MD, PhD, Marlène Pasquet, MD, PhD, Laetitia Largeaud, Flore Sicre de Fontbrune, Claire Fieschi, MD PhD, Virginie Gandemer, MD PhD, Nathalie Aladjidi, MD, Fanny Fouyssac, MD, Guy Leverger, MD, Wadih Abou Chahla, MD, Stephane Blanche, MD, Despina Moushous, MD PhD, Aline Moignet Autrel, MD, Yves Bertrand, MD, Cecile Renard, Pascale Flandrin-Gresta, MD, Jean Louis Stephan, MDPhD, Olivier Tournilhac, MD PhD, Liana Carausu, Frederic Millot, MD, Marielaure Couec, MD, Claude Preudhomme, MD PhD, Hélène Lapillonne, MD PhD, Vahid Asnafi, MD PhD, Patrick Revy, Christine Bellanne-Chantelot, PhD PharmD, Francois Delhommeau, MD PhD, Jean Donadieu, MDPhD

Blood (2020) 136 (Supplement 1): 22–23.

https://doi.org/10.1182/blood-2020-138540

Published: 2020

Journal Articles

Lenograstim and Filgrastim Have a Similar Efficacy and Safety Profile in the Treatment of Chronic Neutropenia. a Study for the French SCN Registry:

Jean Donadieu, MDPhD, Blandine Beaupain, Fares bou Mitri, Flore Sicre de Fontbrune, Despina Moushous, MD PhD, Thierry Lamy, PhD MD, Aline Moignet Autrel, MD, Elodie Gouache, Marlène Pasquet, MD, PhD, Christine Bellanne-Chantelot, PhD PharmD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-138476

Published: 2020

Journal Articles

Congenital Neutropenia Is Also Associated with a High Cancer Risk: A Study from the French Severe Chronic Neutropenia Registry

Blood (2020) 136 (Supplement 1): 15–16.

https://doi.org/10.1182/blood-2020-136053

Published: 2020

NARROW

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

Patient-Reported Outcomes: Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis

Fertility and Pregnancy in Patients with Congenital Neutropenia: Experience of the French Severe Chronic Neutropenia Registry in 565 Patients Above Age of 15 Years

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

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

Efficacy and Safety of Subcutaneous Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous Eculizumab: 2-Year Follow-up

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

IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria

The clinical picture of ERCC6L2 disease: from bone marrow failure to acute leukemia

Rare germline complement factor H variants in patients with paroxysmal nocturnal hemoglobinuria

Molecular International Prognostic Scoring System (IPSS-M) in Myelodysplastic Syndromes Arising from Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Immunogenetic, Molecular and Clinical Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Clonal Hematopoiesis Driven By MDM4 Amplification Defines a Canonical Route Towards Secondary MDS/AML in Fanconi Anemia Patients

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

How Many Patients Have Congenital Neutropenia? a Population-Based Estimation from the Nationwide French Severe Chronic Neutropenia Registry

Impact and Dynamics of TP53 Mutated Clones in Shwachman Diamond Syndrome in a Series of 80 Patients

Lenograstim and Filgrastim Have a Similar Efficacy and Safety Profile in the Treatment of Chronic Neutropenia. a Study for the French SCN Registry:

Congenital Neutropenia Is Also Associated with a High Cancer Risk: A Study from the French Severe Chronic Neutropenia Registry

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American Society of Hematology

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American Society of Hematology

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