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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

Breakthrough Hemolysis Events in Patients with Paroxysmal Nocturnal Hemoglobinuria: Data from Danicopan and Pegcetacoplan Trials

Hubert Schrezenmeier, Mohammed Mahdi, Christoph Gasteyger, Cecile De Coster

Blood (2024) 144 (Supplement 1): 2694.

https://doi.org/10.1182/blood-2024-203195

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

Impact of Various Combinations of Potentially Immunogenic Therapeutics on the T Cell Response in Acute Myeloid Leukemia

Jochen Greiner, Patrick J Schuler, Hubert Schrezenmeier, Susanne Hofmann, Marlies Götz

Blood (2024) 144 (Supplement 1): 5792.

https://doi.org/10.1182/blood-2024-205110

Published: 2024

Journal Articles

Ravulizumab in Pregnant Women with Paroxysmal Nocturnal Hemoglobinuria (PNH) - Favourable Experience from a Retrospective Case Series

Britta Hoechsmann, Wilhelm Leopold, Ariane Schneider, Sixten Koerper, Ursula Kalhammer, Hubert Schrezenmeier

Blood (2024) 144 (Supplement 1): 5251.

https://doi.org/10.1182/blood-2024-209857

Published: 2024

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

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

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry

Austin Kulasekararaj, Hubert Schrezenmeier, Kensuke Usuki, Aleksander Kulagin, Sandra Fatima Gualandro, Rosario Notaro, Masayo Ogawa, Ji Yu, Ami Patel, Yogesh Patel, Wolfgang Füreder, Regis Peffault De Latour, Jong-Wook Lee

Blood (2023) 142 (Supplement 1): 2714.

https://doi.org/10.1182/blood-2023-189150

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

Patient-Reported Outcomes (PROs) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 and COMMODORE 1 Trials

Jens Panse, Jaroslav Cermak, Olena Kyselova, Akihiko Gotoh, Fahri Sahin, Hubert Schrezenmeier, Alice C. Chang, Brittany Gentile, Marianne Uguen, Bing Han

Blood (2023) 142 (Supplement 1): 4090.

https://doi.org/10.1182/blood-2023-177584

Published: 2023

Journal Articles

Real-World Data Suggest Effectiveness of Allogeneic Mesenchymal Stromal (MSC-FFM) Cells in Ruxolitinib-Refractory Acute Graft-Versus-Host Disease

Blood (2023) 142 (Supplement 1): 4927.

https://doi.org/10.1182/blood-2023-174492

Published: 2023

Journal Articles

Oral Monotherapy with Iptacopan, a Proximal Complement Inhibitor of Factor B, Has Superior Efficacy to Intravenous Terminal Complement Inhibition with Standard of Care Eculizumab or Ravulizumab and Favorable Safety in Patients with Paroxysmal Nocturnal Hemoglobinuria and Residual Anemia: Results from the Randomized, Active-Comparator-Controlled, Open-Label, Multicenter, Phase III Apply-PNH Study

Blood (2022) 140 (Supplement 2): LBA-2.

https://doi.org/10.1182/blood-2022-171469

Published: 2022

Journal Articles

Monitoring the in-Vivo Expansion and Persistence of CAR-T Cells As a Tool to Help Decision Making in Patients with Aggressive B-Cell Lymphoma

Daniel Fürst, Christine Neuchel, Adela Neagoie, Elisa Amann, Immanuel Rode, Angela Krauss, Hubert Schrezenmeier, Verena Wais, Hartmut Döhner, Andreas Viardot, Elisa Sala

Blood (2022) 140 (Supplement 1): 7527–7528.

https://doi.org/10.1182/blood-2022-169212

Published: 2022

Journal Articles

Complement and the prothrombotic state

Christoph Q. Schmidt, Hubert Schrezenmeier, David Kavanagh

Blood (2022) 139 (13): 1954–1972.

https://doi.org/10.1182/blood.2020007206

Published: 2022

Journal Articles

Constitutional PIGA mutations cause a novel subtype of hemochromatosis in patients with neurologic dysfunction

Lena Muckenthaler, Oriana Marques, Silvia Colucci, Joachim Kunz, Piotr Fabrowski, Thomas Bast, Sandro Altamura, Britta Höchsmann, Hubert Schrezenmeier, Monika Langlotz, Paulina Richter-Pechanska, Tobias Rausch, Nicole Hofmeister-Mielke, Nikolas Gunkel, Matthias W. Hentze, Andreas E. Kulozik, Martina U. Muckenthaler

Blood (2022) 139 (9): 1418–1422.

https://doi.org/10.1182/blood.2021013519

Published: 2022

Includes: Supplemental data

Journal Articles

Donor Genetic Determinant of Thymopoiesis rs2204985 Impacts Clinical Outcome after Single HLA Mismatched HSCT

Blood (2021) 138 (Supplement 1): 3903.

https://doi.org/10.1182/blood-2021-144748

Published: 2021

Journal Articles

Enhanced Stimulation of Antigen-Specific Immune Responses Against NPM1-Mutated AML

Jochen Marlies Greiner, Vanessa Schneider, Hubert Schrezenmeier, Susanne Hofmann, Marlies Götz

Blood (2021) 138 (Supplement 1): 1292.

https://doi.org/10.1182/blood-2021-146875

Published: 2021

Journal Articles

Clinically Important Difference for the FACIT-Fatigue Scale in Paroxysmal Nocturnal Hemoglobinuria: A Derivation from International PNH Registry Patient Data

David Cella, Peter Johansson, Yasutaka Ueda, Ioannis Tomazos, Philippe Gustovic, Alice Wang, Ami S. Patel, Hubert Schrezenmeier

Blood (2021) 138 (Supplement 1): 1952.

https://doi.org/10.1182/blood-2021-153127

Published: 2021

Journal Articles

Predictors for Improvement in Patient-Reported Outcomes: Post-Hoc Analysis of a Phase 3 Randomized, Open-Label Study of Eculizumab and Ravulizumab in Complement Inhibitor-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Hubert Schrezenmeier, Austin G. Kulasekararaj, Lindsay Mitchell, Regis Peffault De Latour, Timothy Devos, Shinichiro Okamoto, Richard Wells, Karissa Johnston, Evan Popoff, Antoinette Cheung, Jimmy Wang, Philippe Gustovic, Alice Wang, Ioannis Tomazos, Yogesh Patel, Jong Wook Lee

Blood (2021) 138 (Supplement 1): 2196.

https://doi.org/10.1182/blood-2021-144554

Published: 2021

NARROW

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

Breakthrough Hemolysis Events in Patients with Paroxysmal Nocturnal Hemoglobinuria: Data from Danicopan and Pegcetacoplan Trials

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

Impact of Various Combinations of Potentially Immunogenic Therapeutics on the T Cell Response in Acute Myeloid Leukemia

Ravulizumab in Pregnant Women with Paroxysmal Nocturnal Hemoglobinuria (PNH) - Favourable Experience from a Retrospective Case Series

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

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

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry

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

Patient-Reported Outcomes (PROs) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 and COMMODORE 1 Trials

Real-World Data Suggest Effectiveness of Allogeneic Mesenchymal Stromal (MSC-FFM) Cells in Ruxolitinib-Refractory Acute Graft-Versus-Host Disease

Monitoring the in-Vivo Expansion and Persistence of CAR-T Cells As a Tool to Help Decision Making in Patients with Aggressive B-Cell Lymphoma

Complement and the prothrombotic state

Constitutional PIGA mutations cause a novel subtype of hemochromatosis in patients with neurologic dysfunction

Donor Genetic Determinant of Thymopoiesis rs2204985 Impacts Clinical Outcome after Single HLA Mismatched HSCT

Enhanced Stimulation of Antigen-Specific Immune Responses Against NPM1-Mutated AML

Clinically Important Difference for the FACIT-Fatigue Scale in Paroxysmal Nocturnal Hemoglobinuria: A Derivation from International PNH Registry Patient Data

Predictors for Improvement in Patient-Reported Outcomes: Post-Hoc Analysis of a Phase 3 Randomized, Open-Label Study of Eculizumab and Ravulizumab in Complement Inhibitor-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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American Society of Hematology

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American Society of Hematology

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