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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

Phase III COMMODORE 2 and 1 Trials: Characterization of Breakthrough Hemolysis Events in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab or Eculizumab

Austin G. Kulasekararaj, Jaroslaw Czyz, Catherine Flynn, Gabriela Roncone Gastal, Marek Hus, Chrysavgi Lalayanni, Erfan Nur, Phillip Scheinberg, Yasutaka Ueda, Alessandro Maria Vannucchi, Maria Vidriales Vicente, Iige Viigimaa, Simon Buatois, Alice C. Chang, Sammy Chebon, Alexander Röth

Blood (2024) 144 (Supplement 1): 1323.

https://doi.org/10.1182/blood-2024-200400

Published: 2024

Journal Articles

The Effect of Oral Iptacopan Monotherapy on Hematological Parameters in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Is Consistent Regardless of the Type of Prior Anti-C5 Treatment Received: A Post Hoc Analysis of 24-Week Data from the Randomized Phase III APPLY-PNH Trial

Regis Peffault De Latour, Austin G. Kulasekararaj, Phillip Scheinberg, Yasutaka Ueda, Carlos de Castro, Samopriyo Maitra, Philippe Ferber, Marion Dahlke, Antonio M Risitano

Blood (2024) 144 (Supplement 1): 4087.

https://doi.org/10.1182/blood-2024-200763

Published: 2024

Journal Articles

Role of Age and Donor Type in 3646 Severe Aplastic Anemia Patients Undergoing Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-Saawp Study

Blood (2024) 144 (Supplement 1): 595.

https://doi.org/10.1182/blood-2024-194713

Published: 2024

Journal Articles

Phase III Randomized COMMODORE 1 Trial: 2-Year Safety and Efficacy of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Eculizumab

Phillip Scheinberg, Diego Villa Cle, Berangere Devalet, Valentina Giai, Cristina Barrenetxea Lekue, Hsuan-Yu Lin, Zsolt Nagy, Jun-ichi Nishimura, Jens Panse, Agnieszka Piekarska, Jörg Schubert, Kazutaka Sunami, Mustafa Yenerel, Anita Appius, Brittany Gentile, Shino Iwase, Himika Patel, Sasha Sreckovic, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 1313.

https://doi.org/10.1182/blood-2024-200745

Published: 2024

Journal Articles

A Dedicated System Accounting for Specific Germline/Somatic Genomic Constellations Is Essential to Predict Outcomes in DDX41 -Mutant Myeloid Neoplasia

Blood (2024) 144 (Supplement 1): 1332.

https://doi.org/10.1182/blood-2024-194024

Published: 2024

Journal Articles

The Association of Anemia and Patient Reported Outcomes in the New Era of PNH Treatment

Blood (2024) 144 (Supplement 1): 2321.

https://doi.org/10.1182/blood-2024-204693

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

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

The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry: Highlights of the Data from the First Three Years

David Araten, Austin G. Kulasekararaj, Jun-ichi Nishimura, Regis Peffault De Latour, Ilene C. Weitz, Azalea Candelaria, Aliza Fink, Alice Houk, Elizabeth W Kottke, Brad A Wong

Blood (2024) 144 (Supplement 1): 3706.

https://doi.org/10.1182/blood-2024-208575

Published: 2024

Journal Articles

Oral Iptacopan Monotherapy Leads to Long-Term Improvements in Patient (Pt)-Reported Health-Related Quality of Life (HRQoL) and Investigator-Assessed Signs and Symptoms of Paroxysmal Nocturnal Hemoglobinuria (PNH): 48-Week (Wk) Results from the Phase III APPLY-PNH and APPOINT-PNH Trials

Antonio M Risitano, Bing Han, Austin G. Kulasekararaj, Yasutaka Ueda, Phillip Scheinberg, Carlos de Castro, Jaroslaw Maciejewski, Josefin Snellman, Olivier Somenzi, Randall Winnette, Samopriyo Maitra, Shujie Li, Marion Dahlke, Regis Peffault De Latour

Blood (2024) 144 (Supplement 1): 4079.

https://doi.org/10.1182/blood-2024-203532

Published: 2024

Journal Articles

Phase III COMMODORE 1 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Ravulizumab

Phillip Scheinberg, Hazza Alzahrani, Alejandro Contento Gonzalo, Martin Höglund, Jin Seok Kim, Jens Panse, Melissa J Rose, Jörg Schubert, Yasutaka Ueda, Eng Soo Yap, Muriel Buri, Nicolo' Compagno, Brittany Gentile, Sasha Sreckovic, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 4078.

https://doi.org/10.1182/blood-2024-200889

Published: 2024

Journal Articles

Correlation between the Integrated MDS Flow Cytometry Score and Genomic Abnormalities - a Single Centre Analysis from King's College Hospital

Henry Wood, Lauren Jamieson, Charles Manogaran, Katy Jones, Kar Lok Kong, Josephine Goodwin, Steve Best, Azim Mohamedali, Aytug Kizilors, Nicholas C Lea, Zoe Thorn, Frances Aldridge, Liron Barnea Slonim, Prudence Hardefeldt, Guy Hannah, Shireen Kassam, Deborah Yallop, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 6751.

https://doi.org/10.1182/blood-2024-211268

Published: 2024

Journal Articles

Role of Cidofovir in the Treatment of BK Virus Haemorrhagic Cystitis in Patients Undergoing T- Cell Deplete Stem Cell Transplant and Cellular Therapies

Shreyas Hanmantgad, Samitha Fernando, Mark Zuckerman, Pamella Paul, Yahya Almohmeed, Mili Shah, Pramila Krishnamurthy, Daniele Avenoso, Antonio Pagliuca, Austin G. Kulasekararaj, Victoria Potter, Varun Mehra

Blood (2024) 144 (Supplement 1): 7293.

https://doi.org/10.1182/blood-2024-208440

Published: 2024

Journal Articles

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

Régis Peffault de Latour, Jeffrey Szer, Koo Wilson, Piotr Wojciechowski, Zalmai Hakimi, Jameel Nazir, Barbara Czech, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 7706.

https://doi.org/10.1182/blood-2024-209712

Published: 2024

Journal Articles

A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes

Amer M. Zeidan, Mikkael A. Sekeres, Hanny Al-Samkari, Hetty E. Carraway, Amy E. DeZern, Moshe Mittelman, Meredith Little, Vanessa Beynon, Xiaoshu Dai, Sal Sommakia, Jenny M. Despotovic, Parija Patel, Melissa Dibacco, Bruno Fattizzo, Eytan M. Stein, David Sallman, Austin G. Kulasekararaj, Uwe Platzbecker, Pierre Fenaux

Blood (2024) 144 (Supplement 1): 6708.

https://doi.org/10.1182/blood-2024-203073

Published: 2024

Journal Articles

Aplastic Anemia in the Elderly: Treatment Patterns and Outcomes from an International Observational Study

Bruno Fattizzo, Carmelo Gurnari, Sabrina Giammarco, Antony Ricchiuti, Hussein Awada, Marta Bortolotti, Giacinto Luca Pedone, Francesco Versino, Dario Consonni, Roochi Trikha, Shreyans Gandhi, Simona Sica, Jaroslaw Maciejewski, Austin G. Kulasekararaj, Wilma Barcellini

Blood (2024) 144 (Supplement 1): 4076.

https://doi.org/10.1182/blood-2024-198321

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

Treatment of Idiopathic Aplastic Anemia (AA) with Expanded Autologous Regulatory T Cells: The Tiara Phase One Trial

Nazia Matto, Shreyans Gandhi, Marta Rzepkowska, Sila Gerlevik, Mohammad M Karimi, Austin G. Kulasekararaj, Abdel Douiri, Angela Miller, Lynne Duran, Haili Cui, Jen Lewis, Shahram Kordasti, Giovanna Lombardi, Giorgio Napolitani, Ghulam Jeelani Mufti

Blood (2024) 144 (Supplement 1): 301.

https://doi.org/10.1182/blood-2024-207101

Published: 2024

NARROW

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

Phase III COMMODORE 2 and 1 Trials: Characterization of Breakthrough Hemolysis Events in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab or Eculizumab

The Effect of Oral Iptacopan Monotherapy on Hematological Parameters in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Is Consistent Regardless of the Type of Prior Anti-C5 Treatment Received: A Post Hoc Analysis of 24-Week Data from the Randomized Phase III APPLY-PNH Trial

Role of Age and Donor Type in 3646 Severe Aplastic Anemia Patients Undergoing Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-Saawp Study

Phase III Randomized COMMODORE 1 Trial: 2-Year Safety and Efficacy of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Eculizumab

A Dedicated System Accounting for Specific Germline/Somatic Genomic Constellations Is Essential to Predict Outcomes in DDX41 -Mutant Myeloid Neoplasia

The Association of Anemia and Patient Reported Outcomes in the New Era of PNH Treatment

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

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

The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry: Highlights of the Data from the First Three Years

Oral Iptacopan Monotherapy Leads to Long-Term Improvements in Patient (Pt)-Reported Health-Related Quality of Life (HRQoL) and Investigator-Assessed Signs and Symptoms of Paroxysmal Nocturnal Hemoglobinuria (PNH): 48-Week (Wk) Results from the Phase III APPLY-PNH and APPOINT-PNH Trials

Phase III COMMODORE 1 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Switched from Ravulizumab

Correlation between the Integrated MDS Flow Cytometry Score and Genomic Abnormalities - a Single Centre Analysis from King's College Hospital

Role of Cidofovir in the Treatment of BK Virus Haemorrhagic Cystitis in Patients Undergoing T- Cell Deplete Stem Cell Transplant and Cellular Therapies

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes

Aplastic Anemia in the Elderly: Treatment Patterns and Outcomes from an International Observational Study

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

Treatment of Idiopathic Aplastic Anemia (AA) with Expanded Autologous Regulatory T Cells: The Tiara Phase One Trial

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American Society of Hematology

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