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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

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

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

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: 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

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

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

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

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

Complement inhibition at the level of C3 or C5: mechanistic reasons for ongoing terminal pathway activity

Marco Mannes, Arthur Dopler, Oliver Zolk, Sophia J. Lang, Rebecca Halbgebauer, Britta Höchsmann, Arne Skerra, Christian K. Braun, Markus Huber-Lang, Hubert Schrezenmeier, Christoph Q. Schmidt

Blood (2021) 137 (4): 443–455.

https://doi.org/10.1182/blood.2020005959

Published: 2021

Includes: Supplemental data

Journal Articles

Upfront Alternative Donor Transplant Versus Immunosuppressive Therapy in Patients with Severe Aplastic Anemia Who Lack Fully HLA Matched Related Donor: Systematic Review and Meta-Analysis of Retrospective Studies. on Behalf of the Severe Aplastic Anemia Working Party of European Group for Blood and Marrow Transplantation (SAAWP of EBMT)

Hind Alotaibi, MBBS, Mahmoud Aljurf, Regis Peffault De Latour, MD PhD, Shahid Iqbal, Andrea Bacigalupo, Judith Marsh, Hubert Schrezenmeier, Eliane Gluckman, Mansour Alfayez, Britta Hoechsmann, Constantijn Halkes, Josu de la Fuente, Nawal AlShehry, Simone Cesaro, Jakob Passweg, Carlo Dufour, Antonio Risitano, John F. DiPersio, MD PhD, Ibraheem Motabi

Blood (2020) 136 (Supplement 1): 6–7.

https://doi.org/10.1182/blood-2020-140846

Published: 2020

Journal Articles

Frequency and Stimulation of NPM1-Specific Immune Responses By Anti-PD1 Antibodies in NPM1-Mutated AML

Jochen Greiner, MD, Vanessa Schneider, Dr. rer. nat., Hubert Schrezenmeier, Markus Wiesneth, MD, Susanne Hofmann, MD, Marlies Götz

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

https://doi.org/10.1182/blood-2020-134876

Published: 2020

Journal Articles

Integration of Hi-C and Nanopore Sequencing for Structural Variant Analysis in AML with a Complex Karyotype: (Chromothripsis)²

Marius-Konstantin Klever, Eric Sträng, PhD, Julius Jungnitsch, Uirá Souto Melo, Sara Hetzel, Anna Dolnik, Robert Schöpflin, Jens F. Schrezenmeier, Olga Blau, Jörg Westermann, Konstanze Döhner, Hubert Schrezenmeier, Malte Spielmann, Alexander Meissner, PhD, Stefan Mundlos, Lars Bullinger, MD

Blood (2020) 136 (Supplement 1): 28.

https://doi.org/10.1182/blood-2020-133787

Published: 2020

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

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 Years with Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension Studies

Regis Peffault De Latour, MD PhD, Jeffrey Szer, MBBS, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Jin Seok Kim, MD PhD, Caroline I. Piatek, MD, Alexander D. Kulagin, MD PhD, Anita Hill, Jimmy Wang, Ji Yu, Masayo Ogawa, MD FAAP, Hubert Schrezenmeier, Jong Wook Lee, MD, PhD

Blood (2020) 136 (Supplement 1): 42–43.

https://doi.org/10.1182/blood-2020-140777

Published: 2020

NARROW

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

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

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

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

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

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)

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

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

Complement inhibition at the level of C3 or C5: mechanistic reasons for ongoing terminal pathway activity

Frequency and Stimulation of NPM1-Specific Immune Responses By Anti-PD1 Antibodies in NPM1-Mutated AML

Integration of Hi-C and Nanopore Sequencing for Structural Variant Analysis in AML with a Complex Karyotype: (Chromothripsis)²

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

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 Years with Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension Studies

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American Society of Hematology

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American Society of Hematology

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