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Journal Articles

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Blood (2024) 144 (3): 323–333.

https://doi.org/10.1182/blood.2024024173

Published: 2024

Journal Articles

Patient-Reported Improvements in Fatigue and Health-Related Quality of Life in the Phase 3 Studies Apply-PNH and Appoint-PNH Evaluating the Use of Iptacopan in C5 Inhibitor-Treated and Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

Antonio M Risitano, Carlos M de Castro, Bing Han, Austin Kulasekararaj, Jaroslaw P. Maciejewski, Phillip Scheinberg, Yasutaka Ueda, Susan Vallow, Georgina Bermann, Marion Dahlke, Rakesh Kumar, Regis Peffault de Latour

Blood (2023) 142 (Supplement 1): 487.

https://doi.org/10.1182/blood-2023-179035

Published: 2023

Journal Articles

Impact of Somatic Second Hit and Comutations in Myeloid Neoplasms with Germline DDX41 Mutations

Guilherme Mendes Sapinho, Pramila Krishnamurthy, Guy Hannah, Shreyans Gandhi, Victoria Potter, Mili Shah, Nicholas Lea, Clair Engelbrecht, Heidre Bezuidenhout, Katie Snape, Steve Best, Austin Kulasekararaj

Blood (2023) 142 (Supplement 1): 6446.

https://doi.org/10.1182/blood-2023-189451

Published: 2023

Journal Articles

Transplant Outcomes for Acute Myeloid Leukemia or Myelodysplastic Syndromes Secondary to Acquired Aplastic Anemia or Paroxysmal Nocturnal Hemoglobinuria: A Report from the EBMT Severe Aplastic Anemia Working Party

Blood (2023) 142 (Supplement 1): 704.

https://doi.org/10.1182/blood-2023-184783

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

Biomarker Analyses in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 Trial

Pontus Lundberg, Silvia de la Iglesia, Richard J Kelly, Austin Kulasekararaj, Jun-Ichi Nishimura, Antonio M Risitano, Alexander Roeth, Simon Buatois, Sammy Chebon, Himika Patel, Anna Kiialainen

Blood (2023) 142 (Supplement 1): 4088.

https://doi.org/10.1182/blood-2023-178694

Published: 2023

Journal Articles

Alternative Donor Transplantation for Severe Aplastic Anemia: A Comparative Study of the Saawp EBMT

Blood (2023) 142 (Supplement 1): 1047.

https://doi.org/10.1182/blood-2023-174724

Published: 2023

Journal Articles

Deciphering the Differential Diagnosis of Inherited Versus Acquired Bone Marrow Failure Syndromes: A Large Cohort Study

Marta Bortolotti, Roochi Trikha, Sarah Salter, Joanna Large, Shreyans Gandhi, Bruno Fattizzo, Wilma Barcellini, Austin Kulasekararaj

Blood (2023) 142 (Supplement 1): 1360.

https://doi.org/10.1182/blood-2023-186516

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

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Regis Peffault De Latour, Austin Kulasekararaj, Phillip Scheinberg, Philippe Ferber, Tomasz Lawniczek, Christine Thorburn, Marion Dahlke, Antonio M Risitano

Blood (2023) 142 (Supplement 1): 1338.

https://doi.org/10.1182/blood-2023-179377

Published: 2023

Journal Articles

Data-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS)

Luca Lanino, Somedeb Ball, Jan Philipp Bewersdorf, Monia Marchetti, Giulia Maggioni, Erica Travaglino, Najla H Al Ali, Pierre Fenaux, Uwe Platzbecker, Valeria Santini, Maria Diez-Campelo, Avani M Singh, Akriti Gupta Jain, Luis E. Aguirre, Sara M. Tinsley-Vance, Zaker I. Schwabkey, Onyee Chan, Zhuoer Xie, Andrew M. Brunner, Andrew T. Kuykendall, John M. Bennett, Rena Buckstein, Rafael Bejar, Hetty E. Carraway, Amy E. DeZern, Elizabeth A. Griffiths, Stephanie Halene, Robert Hasserjian, Jeffrey Lancet, Alan F. List, Sanam Loghavi, Olatoyosi Odenike, Eric Padron, Mrinal M. Patnaik, Gail J. Roboz, Maximilian Stahl, Mikkael A. Sekeres, David Peter Steensma, Michael R. Savona, Justin Taylor, Mina Xu, Kendra Sweet, David A Sallman, Stephen D. Nimer, Christopher S. Hourigan, Andrew H. Wei, Elisabetta Sauta, Saverio D'Amico, Gianluca Asti, Gastone Castellani, Uma M. Borate, Guillermo Sanz, Fabio Efficace, Steven D. Gore, Tae Kon Kim, Naval Daver, Guillermo Garcia-Manero, María Rozman, Alberto Orfao, Sa A. Wang, M. Kathy Foucar, Ulrich Germing, Torsten Haferlach, Phillip Scheinberg, Yasushi Miyazaki, Marcelo Iastrebner, Austin Kulasekararaj, Thomas Cluzeau, Shahram Kordasti, Arjan A. van de Loosdrecht, Lionel Ades, Amer M. Zeidan, Rami S. Komrokji, Matteo Giovanni Della Porta

Blood (2023) 142 (Supplement 1): 998.

https://doi.org/10.1182/blood-2023-186580

Published: 2023

Journal Articles

Pathogenetic Subtypes of Pure Red Cell Aplasia and Therapeutic Management Strategies : A Single Centre Experience in the United Kingdom of 75 Patients over 10 Years

Sukanya Gogoi, Jennifer Vidler, Manujasri Wimalachandra, Wasanthi Wickramasinghe, Austin Kulasekararaj

Blood (2023) 142 (Supplement 1): 2719.

https://doi.org/10.1182/blood-2023-190395

Published: 2023

Journal Articles

Efficacy of Parallel and Crossover Analysis As Well As Pharmacokinetic Similarity Were Confirmed between ABP 959 and Eculizumab Reference Product in Patients with PNH

Austin Kulasekararaj, Francesco Lanza, Alexandros Arvanitakis, Saskia MC Langemeijer, Satheesh Chonat, Jia Cao, Vincent Chow, Haby Henary

Blood (2023) 142 (Supplement 1): 4092.

https://doi.org/10.1182/blood-2023-186362

Published: 2023

Journal Articles

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

Antonio M Risitano, Bing Han, Austin Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw P. Maciejewski, Josefin Snellman, Ranjan Tiwari, Marion Dahlke, Régis Peffault de Latour

Blood (2023) 142 (Supplement 1): 4084.

https://doi.org/10.1182/blood-2023-180866

Published: 2023

Journal Articles

Outcome Prediction in DDX41 -Mutant Myelodysplastic Syndromes Is Not Possible with General Disease Schemes and Requires a Dedicated Risk Scoring System

Carmelo Gurnari, Hideki Makishima, Arda Durmaz, Ryunosuke Saiki, Guilherme Mendes Sapinho, Alex Bataller, Lukasz P. Gondek, Yasuhito Nannya, Steve Best, Pramila Krishnamurthy, Hussein Awada, Enrico Attardi, Valeria Visconte, Maria Teresa Voso, Amy E. DeZern, Guillermo Garcia-Manero, Austin Kulasekararaj, Jaroslaw P. Maciejewski, Seishi Ogawa

Blood (2023) 142 (Supplement 1): 316.

https://doi.org/10.1182/blood-2023-178116

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 Preferences and Treatment Satisfaction in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Approved C5 Inhibitors in the Phase III Randomized COMMODORE 1 and COMMODORE 2 Trials

Austin Kulasekararaj, Fernando Ataulfo Gonzalez Fernandez, Phillip Scheinberg, Nicole Straetmans, Yasutaka Ueda, Brittany Gentile, Jennifer Stefani, Marianne Uguen, Alexander Roeth

Blood (2023) 142 (Supplement 1): 5628.

https://doi.org/10.1182/blood-2023-178610

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

Germline Risks and Clinical Impacts of DDX41 Mutations in Myeloid Malignancies

Hideki Makishima, Ryunosuke Saiki, Yasuhito Nannya, Sophia C. Korotev, Carmelo Gurnari, June Takeda, Yukihide Momozawa, Steve Best, Pramila Krishnamurthy, Tetsuichi Yoshizato, Yoshiko Atsuta, Yusuke Shiozawa, Yuka Iijima-Yamashita, Kenichi Yoshida, Yuichi Shiraishi, Yasunobu Nagata, Nobuyuki Kakiuchi, Makoto Onizuka, Kenichi Chiba, Hiroko Tanaka, Ayana Kon, Yotaro Ochi, Masahiro Marshall Nakagawa, Rurika Okuda, Takuto Mori, Akinori Yoda, Hidehiro Itonaga, Yasushi Miyazaki, Masashi Sanada, Takayuki Ishikawa, Shigeru Chiba, Hisashi Tsurumi, Senji Kasahara, Carsten Müller-Tidow, Akifumi Takaori-Kondo, Kazuma Ohyashiki, Toru Kiguchi, Fumihiko Matsuda, Joop H. Jansen, Chantana Polprasert, Piers Blombery, Yoichiro Kamatani, Satoru Miyano, Luca Malcovati, Torsten Haferlach, Michiaki Kubo, Mario Cazzola, Austin Kulasekararaj, Lucy A. Godley, Jaroslaw P. Maciejewski, Seishi Ogawa

Blood (2022) 140 (Supplement 1): 208–210.

https://doi.org/10.1182/blood-2022-158851

Published: 2022

NARROW

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Patient-Reported Improvements in Fatigue and Health-Related Quality of Life in the Phase 3 Studies Apply-PNH and Appoint-PNH Evaluating the Use of Iptacopan in C5 Inhibitor-Treated and Treatment-Naïve Patients with Paroxysmal Nocturnal Hemoglobinuria

Impact of Somatic Second Hit and Comutations in Myeloid Neoplasms with Germline DDX41 Mutations

Transplant Outcomes for Acute Myeloid Leukemia or Myelodysplastic Syndromes Secondary to Acquired Aplastic Anemia or Paroxysmal Nocturnal Hemoglobinuria: A Report from the EBMT Severe Aplastic Anemia Working Party

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

Biomarker Analyses in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 Trial

Alternative Donor Transplantation for Severe Aplastic Anemia: A Comparative Study of the Saawp EBMT

Deciphering the Differential Diagnosis of Inherited Versus Acquired Bone Marrow Failure Syndromes: A Large Cohort Study

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

Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III Apply-PNH and Appoint-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Data-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS)

Pathogenetic Subtypes of Pure Red Cell Aplasia and Therapeutic Management Strategies : A Single Centre Experience in the United Kingdom of 75 Patients over 10 Years

Efficacy of Parallel and Crossover Analysis As Well As Pharmacokinetic Similarity Were Confirmed between ABP 959 and Eculizumab Reference Product in Patients with PNH

Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Naïve Patients in the Phase III APPOINT-PNH Trial

Outcome Prediction in DDX41 -Mutant Myelodysplastic Syndromes Is Not Possible with General Disease Schemes and Requires a Dedicated Risk Scoring System

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 Preferences and Treatment Satisfaction in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Approved C5 Inhibitors in the Phase III Randomized COMMODORE 1 and COMMODORE 2 Trials

Germline Risks and Clinical Impacts of DDX41 Mutations in Myeloid Malignancies

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American Society of Hematology

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American Society of Hematology

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