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Journal Articles

Outcomes of HLA-matched unrelated versus haploidentical hematopoietic cell transplantation in acute leukemia: A multicenter observational study in Brazil facilitated by the CIBMTR

Blood (2025) 146 (Supplement 1): 5981.

https://doi.org/10.1182/blood-2025-5981

Published: 2025

Journal Articles

Healthcare inequities in Latin America outweigh the benefits of pediatric regimens in adult acute lymphoblastic leukemia

Blood (2025) 146 (Supplement 1): 288.

https://doi.org/10.1182/blood-2025-288

Published: 2025

Journal Articles

Qualitative assessment of predictors of response to immunosuppressive therapy in aplastic anemia: A systematic literature review

Phillip Scheinberg, Stephanie MacLeod, Meryem Bektas, Raj Gokani, Kevin Kallmes, Shahnaz Khan, Barbara Możejko-Pastewka, Alicia Rovo

Blood (2025) 146 (Supplement 1): 6717.

https://doi.org/10.1182/blood-2025-6717

Published: 2025

Journal Articles

Serum ferritin changes in iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria

Antonio Risitano, Carlos de Castro, Camilla Frieri, Bing Han, Richard Kelly, Austin Kulasekararaj, Jaroslaw Maciejewski, Vinod Pullarkat, Alexander Röth, Phillip Scheinberg, Flore Sicre-de-Fontbrune, Yasutaka Ueda, Tim Costigan, Marion Dahlke, Philippe Ferber, Tomasz Lawniczek, Stefanie Senger, Regis Peffault De Latour

Blood (2025) 146 (Supplement 1): 4974.

https://doi.org/10.1182/blood-2025-4974

Published: 2025

Journal Articles

Apply-PNH: Analysis of complement pathway biomarkers provides evidence for pharmacodynamic response in PNH patients who receive oral iptacopan monotherapy versus continuing anti-C5 therapy

Regis Peffault De Latour, Carlos de Castro, Richard Kelly, Austin Kulasekararaj, Alexander Röth, Phillip Scheinberg, Yasutaka Ueda, Douglas Applegate, Marion Dahlke, Philippe Ferber, Imelda Schuhmann, Antonio Risitano

Blood (2025) 146 (Supplement 1): 4979.

https://doi.org/10.1182/blood-2025-4979

Published: 2025

Journal Articles

The 2-year efficacy and safety of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria with a history of aplastic anemia on concomitant immunosuppressive therapy who entered the roll-over extension program

Regis Peffault De Latour, Carlos de Castro, Anna Gaya, Morag Griffin, Bing Han, Austin Kulasekararaj, Jaroslaw Maciejewski, Bhumika Patel, Alexander Röth, Phillip Scheinberg, Hubert Schrezenmeier, Yasutaka Ueda, Tim Costigan, Marion Dahlke, Philippe Ferber, Rajeshri Karki, Luca Monaco, Silvia Sanz, Antonio Risitano

Blood (2025) 146 (Supplement 1): 4978.

https://doi.org/10.1182/blood-2025-4978

Published: 2025

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

Autologous Stem Cell Transplantation (ASCT) for Light Chain (AL) Amyloidosis: A Single-Center Report

Natalia Pin Chuen Zing, Breno Gusmao, Juliana Matos Pessoa, Jose Ulysses A. Filho, Fabio R. Kerbauy, Hegta R. Figueiroa, Isabella Pittol, Paula Lucafo Zenero, Phillip Scheinberg, Germano Lima, André Américo, Fauze Lutfe Ayoub

Blood (2024) 144 (Supplement 1): 6900.

https://doi.org/10.1182/blood-2024-211920

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

Impact of Daratumumab-Containing Induction Therapy on Stem Cell Mobilization in Multiple Myeloma: A Retrospective, Single-Center Analysis

Hegta R. Figueiroa, Germano Medeiros, Juliana Matos Pessoa, Paula Lucafo Zenero, Isabella Pittol, Fauze Lutfe Ayoub, Pedro Paulo Faust Machado, Gabriela Camargo, Dimitra Oliveira Delijaicov, André Larubia, Arlett Edna Lazar, Jose Ulysses A. Filho, Breno Gusmao, Fabio R. Kerbauy, André Dias Americo, Phillip Scheinberg

Blood (2024) 144 (Supplement 1): 7262.

https://doi.org/10.1182/blood-2024-210191

Published: 2024

Journal Articles

Early Versus Standard Initiation of Immunosuppressive Therapy for Graft Versus Host Disease Prophylaxis in Haploidentical Allogeneic Stem Cell Transplantation: Incidence and Severity of Cytokine Release Syndrome and Related Outcomes

Luisa V. Carvalho, André Dias Americo, Camila Carminati Isoppo, Isabella Pittol, João A. G. G. Prats, Eurides L. Rosa, Germano Lima, Natalia Fumagalli, Paula Lucafo Zenero, Hegta R. Figueiroa, Fauze Lutfe Ayoub, Juliana Matos Pessoa, Jose Ulysses A. Filho, Fabio R. Kerbauy, Phillip Scheinberg

Blood (2024) 144 (Supplement 1): 7272.

https://doi.org/10.1182/blood-2024-193956

Published: 2024

Journal Articles

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease, Experience of a Brazilian Private Service

Hegta R. Figueiroa, Victor Gottardello Zecchin, Adriana Maria Paixao de Sousa da Silva, MArina Foltran Cela, Maria Lucia De Martino Lee, Fabricio Tera Romagnol, Fernanda Marinho Manga, Gabriela Camargo, Dimitra Oliveira Delijaicov, Leticia Santos Soares, Tomas Marzagão Barbuto, Ricardo Helman, Fabio R. Kerbauy, Phillip Scheinberg

Blood (2024) 144 (Supplement 1): 7322.

https://doi.org/10.1182/blood-2024-209967

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

Incidence, Time to Diagnosis, and Therapeutic Landscape of Aplastic Anemia: Results from a Large United States Claims Database

Phillip Scheinberg, Tim H. H. Brummendorf, Regis Peffault De Latour, Carlo Dufour, Raj Gokani, Morag Griffin, Juliana Hey-Hadavi, Yoshinobu Kanda, Eric Mitchell, Barbara Możejko-Pastewka, Andres Quintero, Patryk Skowron, Meltem Kurt Yüksel, Alicia Rovó

Blood (2024) 144 (Supplement 1): 2329.

https://doi.org/10.1182/blood-2024-204908

Published: 2024

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

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

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

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Outcomes of HLA-matched unrelated versus haploidentical hematopoietic cell transplantation in acute leukemia: A multicenter observational study in Brazil facilitated by the CIBMTR

Healthcare inequities in Latin America outweigh the benefits of pediatric regimens in adult acute lymphoblastic leukemia

Qualitative assessment of predictors of response to immunosuppressive therapy in aplastic anemia: A systematic literature review

Serum ferritin changes in iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria

Apply-PNH: Analysis of complement pathway biomarkers provides evidence for pharmacodynamic response in PNH patients who receive oral iptacopan monotherapy versus continuing anti-C5 therapy

The 2-year efficacy and safety of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria with a history of aplastic anemia on concomitant immunosuppressive therapy who entered the roll-over extension program

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

Autologous Stem Cell Transplantation (ASCT) for Light Chain (AL) Amyloidosis: A Single-Center Report

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

Impact of Daratumumab-Containing Induction Therapy on Stem Cell Mobilization in Multiple Myeloma: A Retrospective, Single-Center Analysis

Early Versus Standard Initiation of Immunosuppressive Therapy for Graft Versus Host Disease Prophylaxis in Haploidentical Allogeneic Stem Cell Transplantation: Incidence and Severity of Cytokine Release Syndrome and Related Outcomes

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease, Experience of a Brazilian Private Service

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

Incidence, Time to Diagnosis, and Therapeutic Landscape of Aplastic Anemia: Results from a Large United States Claims Database

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

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

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

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