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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

Exposure-Response Relationships between the Complement Factor B Inhibitor Iptacopan and Lactate Dehydrogenase (LDH) and Hemoglobin (Hb) in Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Antonio M Risitano, Regis Peffault De Latour, Jun Ho Jang, Eng-Soo Yap, Christian Bartels, Guido Junge, Marion Dahlke, Irina Baltcheva

Blood (2023) 142 (Supplement 1): 5643.

https://doi.org/10.1182/blood-2023-179960

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

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

Outcomes and Late Complications after Umbilical Cord Blood Transplantation for Fanconi Anemia: A Study on Behalf of Eurocord and Severe Aplastic Anemia Working Party - EBMT

Hanadi Rafii, Regis Peffault De Latour, Marc Bierings, Jean-Hugues Dalle, Mouhab F. Ayas, Rawad Rihani, Edoardo Lanino, Chantal Kenzey, Fernanda Volt, Monica M. M Rivera Franco, Barbara Cappelli, Graziana Scigliuolo, Vanderson Rocha, Annalisa Ruggeri, Antonio M Risitano, Éliane Gluckman

Blood (2023) 142 (Supplement 1): 4948.

https://doi.org/10.1182/blood-2023-185841

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

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

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

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

PNH and complement gene variants

Antonio M. Risitano

Blood (2023) 141 (15): 1780–1782.

https://doi.org/10.1182/blood.2022019576

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

HLA in AA: innocent bystander or culprit?

Antonio M. Risitano

Blood (2021) 138 (26): 2744–2745.

https://doi.org/10.1182/blood.2021014535

Published: 2021

Journal Articles

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab

Austin G. Kulasekararaj, Antonio M. Risitano, Jaroslaw P. Maciejewski, Rosario Notaro, Peter Browett, Jong Wook Lee, Mingjun Huang, Michael Geffner, Robert A. Brodsky

Blood (2021) 138 (20): 1928–1938.

https://doi.org/10.1182/blood.2021011388

Published: 2021

Includes: Supplemental data

Journal Articles

Stem Cell Transplantation for Diamond-Blackfan Anemia. a Retrospective Study on Behalf of Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

Blood (2019) 134 (Supplement_1): 44.

https://doi.org/10.1182/blood-2019-124644

Published: 2019

Journal Articles

A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Austin Kulasekararaj, MBBS MDMRCP FRCPath, Antonio M. Risitano, MD PhD, Jaroslaw P. Maciejewski, MD PhD, Rosario Notaro, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA, Jong-Wook Lee, MD, Mingjun Huang, PhD, Michael Geffner, MD, Robert A. Brodsky, MD

Blood (2019) 134 (Supplement_1): 3514.

https://doi.org/10.1182/blood-2019-124748

Published: 2019

Journal Articles

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Pierre-Edouard Debureaux, MD, Fabiana Cacace, Bruno G.P. Silva, MD, Federica Barone, MD, Rodrigo T Calado, MD, Flore Sicre de Fontbrune, Juliette Soret-Dulphy, MD, Patrizia Ricci, Michela Sica, PhD, Rosario Notaro, MD, Phillip Scheinberg, MD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Antonio M. Risitano, MD PhD, Régis Peffault de Latour, MD PhD, Camilla Frieri, MD

Blood (2019) 134 (Supplement_1): 3517.

https://doi.org/10.1182/blood-2019-125917

Published: 2019

Journal Articles

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Antonio M. Risitano, MD PhD, Jong Wook Lee, MD PhD, Rosario Notaro, MD, Robert A. Brodsky, MD, Austin Kulasekararaj, MBBS MDMRCP FRCPath, Jaroslaw P. Maciejewski, MD PhD, Mingjun Huang, PhD, Michael Geffner, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA

Blood (2019) 134 (Supplement_1): 2226.

https://doi.org/10.1182/blood-2019-124837

Published: 2019

Journal Articles

Subcutaneous Alemtuzumab Has Activity in Treatment-Naïve Patients with Acquired Aplastic Anemia

Ana Rita Da Fonseca, MD, Anita Hill, MD PhD, Patricia Eiko Yamakawa, MD, Iara Baldim Rabelo, MD PhD, Andre Domingues Pereira, MD, Vinicius Campos de Molla, MD, Matheus Vescovi Gonçalves, MD PhD, Morag Griffin, MD, Talha Munir, MD, Peter Hillmen, MD PhD, Louise Arnold, MD, Kathryn Riley, MD, Antonio M. Risitano, MD PhD, Phillip Scheinberg, MD, Celso Arrais-Rodrigues, MD PhD

Blood (2019) 134 (Supplement_1): 2503.

https://doi.org/10.1182/blood-2019-130719

Published: 2019

Journal Articles

T-PLL: harmonizing criteria for research

Antonio M. Risitano

Blood (2019) 134 (14): 1113–1114.

https://doi.org/10.1182/blood.2019002303

Published: 2019

NARROW

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)

Exposure-Response Relationships between the Complement Factor B Inhibitor Iptacopan and Lactate Dehydrogenase (LDH) and Hemoglobin (Hb) in Patients (Pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

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

Outcomes and Late Complications after Umbilical Cord Blood Transplantation for Fanconi Anemia: A Study on Behalf of Eurocord and Severe Aplastic Anemia Working Party - EBMT

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

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

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

PNH and complement gene variants

HLA in AA: innocent bystander or culprit?

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab

Stem Cell Transplantation for Diamond-Blackfan Anemia. a Retrospective Study on Behalf of Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)

A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Subcutaneous Alemtuzumab Has Activity in Treatment-Naïve Patients with Acquired Aplastic Anemia

T-PLL: harmonizing criteria for research

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American Society of Hematology

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American Society of Hematology

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