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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

Monotherapy Treatment with Zaltenibart (OMS906), an Alternative Pathway Masp-3 Inhibitor, Improved Key Hematologic Parameters in Patients with PNH with a Suboptimal Response to Ravulizumab: Interim Results from a Phase 2 Proof-of-Concept Study

Morag Griffin, Richard J. Kelly, Mathilde Gavillet, Petra Muus, Britta Hoechsmann, W. Jason Cummings, John Efthimiou, William Pullman, Hubert Schrezenmeier, Jens Panse

Blood (2024) 144 (Supplement 1): 4072.

https://doi.org/10.1182/blood-2024-205866

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

Efficacy and Safety of Pozelimab Plus Cemdisiran Vs Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Are Naïve to Complement Inhibition

Christopher Patriquin, Jun-Ho Jang, Lisa Aurand, Deepak Taneja, Andrew Magyar, Bradley Dain, Karoline Meagher, Lorah Perlee, Amal Souttou, Morag Griffin

Blood (2024) 144 (Supplement 1): 306.

https://doi.org/10.1182/blood-2024-194610

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

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

Population Pharmacokinetics/Pharmacodynamics and Clinical Pharmacology of Zaltenibart (OMS906) in Healthy Subjects and Patients with PNH

W. Jason Cummings, Yi Li, Siobhan Hayes, Richard Mills, Colm Farrell, Morag Griffin, J. Steve Whitaker, William Pullman

Blood (2024) 144 (Supplement 1): 4081.

https://doi.org/10.1182/blood-2024-206009

Published: 2024

Journal Articles

A Single-Centre Experience of Methotrexate in the Management of Pure Red Cell Aplasia and Pure White Cell Aplasia

Laura Batey, Richard J. Kelly, Talha Munir, Petra Muus, Abraham Mullasseril Varghese, Louise M Arnold, Catherine Barnfield, Isla Storey, Nora Youngs, Jessica Molyneux, Morag Griffin

Blood (2024) 144 (Supplement 1): 5684.

https://doi.org/10.1182/blood-2024-203945

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

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

Treatment outcomes of complement protein C5 inhibition in 509 UK patients with paroxysmal nocturnal hemoglobinuria

Richard J. Kelly, Matthew Holt, Jennifer Vidler, Louise M. Arnold, Joanna Large, Briony Forrest, Catherine Barnfield, Alexandra Pike, Morag Griffin, Talha Munir, Petra Muus, Sateesh K. Nagumantry, Abraham Varghese, John R. Davies, Roochi Trikha, Austin G. Kulasekararaj, Lindsay Mitchell, Shreyans Gandhi

Blood (2024) 143 (12): 1157–1166.

https://doi.org/10.1182/blood.2023021762

Published: 2024

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

Paroxysmal Nocturnal Haemoglobinuria (PNH) Arising from Non-Canonical Mutations

Alexandra Pike, Stephen John Richards, Claire E McKinley, Louise M Arnold, Abraham Mullasseril Varghese, Talha Munir, Petra Muus, Peter Hillmen, Morag Griffin, Richard J Kelly, Darren Newton

Blood (2023) 142 (Supplement 1): 4097.

https://doi.org/10.1182/blood-2023-188755

Published: 2023

Journal Articles

52-Week Open-Label Extension Data from a Phase 2 Study Evaluating the Safety and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switched from Eculizumab

Richard J. Kelly, Nicola Houghton, Talha Munir, Petra Muus, Nora Youngs, Lisa Aurand, Kosalai Mohan, Brian Senk, Rodrigo Pavani, Lorah Perlee, Amal Souttou, Morag Griffin

Blood (2023) 142 (Supplement 1): 2716.

https://doi.org/10.1182/blood-2023-188671

Published: 2023

Journal Articles

Sars-Cov-2 Antibody and T-Cell Responses in Patients with Paroxysmal Nocturnal Hemoglobinuria and Aplastic Anemia after Four COVID-19 Vaccinations

Blood (2023) 142 (Supplement 1): 2712.

https://doi.org/10.1182/blood-2023-190393

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

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

Experience of compassionate-use pegcetacoplan for paroxysmal nocturnal hemoglobinuria

Morag Griffin, Petra Muus, Talha Munir, Sateesh Nagumantry, Alexandra Pike, Louise Arnold, Briony Forrest, Catherine Barnfield, Nicola Houghton, Nora Youngs, Richard Kelly

Blood (2023) 141 (1): 116–120.

https://doi.org/10.1182/blood.2022017266

Published: 2023

Journal Articles

Pregnancy in Paroxysmal Nocturnal Hemoglobinuria - a Systematic Review

James E. Manning, Etienne Ciantar, Morag Griffin, Richard Kelly

Blood (2022) 140 (Supplement 1): 11438–11440.

https://doi.org/10.1182/blood-2022-166545

Published: 2022

Journal Articles

Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)

Blood (2022) 140 (Supplement 1): 5799–5800.

https://doi.org/10.1182/blood-2022-169086

Published: 2022

NARROW

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

Monotherapy Treatment with Zaltenibart (OMS906), an Alternative Pathway Masp-3 Inhibitor, Improved Key Hematologic Parameters in Patients with PNH with a Suboptimal Response to Ravulizumab: Interim Results from a Phase 2 Proof-of-Concept Study

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

Efficacy and Safety of Pozelimab Plus Cemdisiran Vs Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Are Naïve to Complement Inhibition

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

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

Population Pharmacokinetics/Pharmacodynamics and Clinical Pharmacology of Zaltenibart (OMS906) in Healthy Subjects and Patients with PNH

A Single-Centre Experience of Methotrexate in the Management of Pure Red Cell Aplasia and Pure White Cell Aplasia

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

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

Treatment outcomes of complement protein C5 inhibition in 509 UK patients with paroxysmal nocturnal hemoglobinuria

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

Paroxysmal Nocturnal Haemoglobinuria (PNH) Arising from Non-Canonical Mutations

52-Week Open-Label Extension Data from a Phase 2 Study Evaluating the Safety and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switched from Eculizumab

Sars-Cov-2 Antibody and T-Cell Responses in Patients with Paroxysmal Nocturnal Hemoglobinuria and Aplastic Anemia after Four COVID-19 Vaccinations

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

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

Experience of compassionate-use pegcetacoplan for paroxysmal nocturnal hemoglobinuria

Pregnancy in Paroxysmal Nocturnal Hemoglobinuria - a Systematic Review

Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)

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