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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

Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Carlos De Castro, Phillip Scheinberg, Bing Han, Susan Vallow, Georgina Bermann, Marion Dahlke, Zhixin Wang, Rakesh Kumar, Gavin Dickie, Caitlyn Lowe, Kaelyn Rupinski, Amber Nieves, Regis Peffault De Latour

Blood (2023) 142 (Supplement 1): 7339.

https://doi.org/10.1182/blood-2023-186840

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

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

Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Pooled Results from the Phase III COMMODORE 1, COMMODORE 2, and COMMODORE 3 Studies

Alexander Roeth, Rong FU, Guangsheng He, Hazza A Alzahrani, Sheng-Chieh Chou, Yosr Hicheri, Maciej Kazmierczak, Viviane Lacorte Recova, Michihiro Uchiyama, ANA Maria Vladareanu, Leigh Beveridge, Simon Buatois, Muriel Buri, Dayu Shi, Nadiesh Balachandran, Sasha Srekovic, Phillip Scheinberg

Blood (2023) 142 (Supplement 1): 575.

https://doi.org/10.1182/blood-2023-177618

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

Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes

Blood (2023) 141 (17): 2047–2061.

https://doi.org/10.1182/blood.2022018604

Published: 2023

Includes: Supplemental data

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

Five-Year Results and Overall Survival Update from the Phase 3 Randomized Study Augment: Lenalidomide Plus Rituximab (R 2 ) Vs Rituximab Plus Placebo in Patients with Relapsed/Refractory Indolent Non-Hodgkin Lymphoma

John P. Leonard, Marek Trneny, Fritz Offner, Jiri Mayer, Huilai Zhang, Grzegorz S. Nowakowski, Phillip Scheinberg, Argyrios Gkasiamis, Joanna Mikita-Geoffroy, Everton Rowe, John G. Gribben

Blood (2022) 140 (Supplement 1): 561–563.

https://doi.org/10.1182/blood-2022-158631

Published: 2022

Journal Articles

Efficacy and Safety of Eltrombopag Combined with Cyclosporine As First-Line Therapy in Adults with Severe Acquired Aplastic Anemia: Results of the Interventional Phase 2 Single-Arm Soar Study

Carlos Vallejo, Jun Ho Jang, Carlo Finelli, Efreen Montaño Figueroa, Lalita Norasetthada, Rodrigo T. Calado, Mehmet Turgut, Regis Peffault De Latour, Ulrike Kriemler-Krahn, Jens Haenig, Joan Maier, Phillip Scheinberg

Blood (2021) 138 (Supplement 1): 2174.

https://doi.org/10.1182/blood-2021-147733

Published: 2021

Journal Articles

Nanatinostat (Nstat) and Valganciclovir (VGCV) in Relapsed/Refractory (R/R) Epstein-Barr Virus-Positive (EBV + ) Lymphomas: Final Results from the Phase 1b/2 VT3996-201 Study

Bradley M. Haverkos, Onder Alpdogan, Robert Baiocchi, Jonathan E Brammer, Tatyana A. Feldman, Marcelo Capra, Elizabeth A Brem, Santosh M Nair, Phillip Scheinberg, Juliana Pereira, Leyla Shune, Erel Joffe, Afton Katkov, Robert McRae, Ivor Royston, Lisa Rojkjaer, Pierluigi Porcu

Blood (2021) 138 (Supplement 1): 623.

https://doi.org/10.1182/blood-2021-152603

Published: 2021

Journal Articles

Eltrombopag Specifically Expands Hematopoietic Multipotent Progenitors in Human Aplastic Anemia

Bruno Quintino Oliveira, brunoqolvr@gmail.com, Bárbara Santana, PhD, Luiz Fernando Bazzo Catto, MDPhD, Maria Florencia Tellechea, PhD, Priscila Santos Scheucher, PhD, Phillip Scheinberg, MD, Rodrigo T. Calado, MD PhD

Blood (2020) 136 (Supplement 1): 23.

https://doi.org/10.1182/blood-2020-142746

Published: 2020

Journal Articles

Oral Nanatinostat (Nstat) and Valganciclovir (VGCV) in Patients with Recurrent Epstein-Barr Virus (EBV)-Positive Lymphomas: Initial Phase 2 Results

Pierluigi Porcu, MD, Bradley Haverkos, MD MPH, MS, Onder Alpdogan, MD, Robert Baiocchi, MD PhD, Jonathan E Brammer, MD, Tatyana A. Feldman, MD, Marcelo Capra, MD PhD, Elizabeth A Brem, MD, Phillip Scheinberg, MD, Juliana Pereira, MD PhD, Leyla Shune, MD, Afton Katkov, MSc, Robert McRae, Lisa Rojkjaer, MD, Ivor Royston, MD, Douglas V. Faller, MD PhD

Blood (2020) 136 (Supplement 1): 7–8.

https://doi.org/10.1182/blood-2020-140843

Published: 2020

Journal Articles

Why do Tregs suddenly disappear in aplastic anemia?

Phillip Scheinberg

Blood (2020) 136 (7): 779–780.

https://doi.org/10.1182/blood.2020006446

Published: 2020

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

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

Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag

Thomas Winkler, Xing Fan, James Cooper, Ronan Desmond, David J. Young, Danielle M. Townsley, Phillip Scheinberg, Sophia Grasmeder, Andre Larochelle, Marie Desierto, Janet Valdez, Jennifer Lotter, Colin Wu, Ruba N. Shalhoub, Katherine R. Calvo, Neal S. Young, Cynthia E. Dunbar

Blood (2019) 133 (24): 2575–2585.

https://doi.org/10.1182/blood.2019000478

Published: 2019

Journal Articles

AUGMENT: A Phase III Randomized Study of Lenalidomide Plus Rituximab (R 2 ) Vs Rituximab/Placebo in Patients with Relapsed/Refractory Indolent Non-Hodgkin Lymphoma

Blood (2018) 132 (Supplement 1): 445.

https://doi.org/10.1182/blood-2018-99-110861

Published: 2018

NARROW

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

Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

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)

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

Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Pooled Results from the Phase III COMMODORE 1, COMMODORE 2, and COMMODORE 3 Studies

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

Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes

Five-Year Results and Overall Survival Update from the Phase 3 Randomized Study Augment: Lenalidomide Plus Rituximab (R 2 ) Vs Rituximab Plus Placebo in Patients with Relapsed/Refractory Indolent Non-Hodgkin Lymphoma

Efficacy and Safety of Eltrombopag Combined with Cyclosporine As First-Line Therapy in Adults with Severe Acquired Aplastic Anemia: Results of the Interventional Phase 2 Single-Arm Soar Study

Nanatinostat (Nstat) and Valganciclovir (VGCV) in Relapsed/Refractory (R/R) Epstein-Barr Virus-Positive (EBV + ) Lymphomas: Final Results from the Phase 1b/2 VT3996-201 Study

Eltrombopag Specifically Expands Hematopoietic Multipotent Progenitors in Human Aplastic Anemia

Oral Nanatinostat (Nstat) and Valganciclovir (VGCV) in Patients with Recurrent Epstein-Barr Virus (EBV)-Positive Lymphomas: Initial Phase 2 Results

Why do Tregs suddenly disappear in aplastic anemia?

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

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

Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag

AUGMENT: A Phase III Randomized Study of Lenalidomide Plus Rituximab (R 2 ) Vs Rituximab/Placebo in Patients with Relapsed/Refractory Indolent Non-Hodgkin Lymphoma

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American Society of Hematology

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American Society of Hematology

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