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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

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

Antonio Risitano, Camilla Frieri, Bor-Sheng Ko, Lily Wong Lee Lee, Alexander Röth, Flore Sicre-de-Fontbrune, Eng Soo Yap, Sung-Soo Yoon, Philippe Ferber, Irina Baltcheva, Aurelie Verles, Silvia Sanz, Luca Monaco, Tim Costigan, Regis Peffault De Latour

Blood (2025) 146 (Supplement 1): 3198.

https://doi.org/10.1182/blood-2025-3198

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

Real-world safety of sutimlimab in patients with CAD/CAS: A multinational, multi-center, observational, prospective cohort Study

Alexander Röth, Marc Michel, Wilma Barcellini, Ulrich Jaeger, Yasutaka Ueda, Quentin A. Hill, Joan Cid, Kelley Dacus, Mounia Beloueche, Catherine Broome

Blood (2025) 146 (Supplement 1): 6242.

https://doi.org/10.1182/blood-2025-6242

Published: 2025

Journal Articles

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial)

Britta Hoechsmann, Philippe Schafhausen, Fabian Beier, Michael Heuser, Joerg Westermann, Beatrice Drexler, Alexander Röth, Mathias Hänel, Flore Sicre-de-Fontbrune, Astrid Marx-Hofmann, Antonia Beitzen-Heineke, Jens Panse, Felicitas R Thol, Sixten Koerper, Tim H. Brummendorf, Hubert Schrezenmeier

Blood (2025) 146 (Supplement 1): 980.

https://doi.org/10.1182/blood-2025-980

Published: 2025

Journal Articles

Switching between complement inhibitors in patients with PNH: A real-world analysis of strategy, efficacy, and safety.

Morag Griffin, Bruno Fattizzo, Jong Wook Lee, Richard Kelly, Roochi Trikha, Yasutaka Ueda, Jun-ichi Nishimura, Christopher Patriquin, Alexander Röth, Petra Muus, Jens Panse, Miguel Gómez Álvarez, Alexandra Pike, Talha Munir, Shreyans Gandhi, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 1114.

https://doi.org/10.1182/blood-2025-1114

Published: 2025

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

Phase III Randomized COMMODORE 2 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Naive to Complement Inhibition

Alexander Röth, Guangsheng He, Andres Brodsky, Chatree Chai-Adisaksopha, Narcisa Sonia Comia, Anna Gaya, Martin Höglund, Richard J. Kelly, Ahlam Naila Kori, Je-Hwan Lee, Naoshi Obara, Antonio M Risitano, Anita Appius, Leigh Beveridge, Brittany Gentile, Zilu Zhang, Bing Han, Sasha Sreckovic

Blood (2024) 144 (Supplement 1): 2687.

https://doi.org/10.1182/blood-2024-208072

Published: 2024

Journal Articles

First Interim Analysis of Patients with Cold Agglutinin Disease (CAD) Patients and the Sutimlimab-Treated Subgroup from the Cadence Registry

Catherine M. Broome, Marc Michel, Wilma Barcellini, Ulrich Jaeger, Yasutaka Ueda, Quentin A. Hill, Joan Cid, Jiangming Wu, Shruti Srivastava, Marek Wardęcki, Reena Patel, Ronnie Yoo, Alexander Röth

Blood (2024) 144 (Supplement 1): 7667.

https://doi.org/10.1182/blood-2024-206942

Published: 2024

Journal Articles

Iptacopan Monotherapy in Patients with Cold Agglutinin Disease: Phase II Study Results

Alexander Röth, Wilma Barcellini, Christine Ademokun, Shripad Chitnis, Sofiya Matviykiv, Alessandra Vitaliti, Chi Chen, Vasiliki Katsanou, Raghav Chawla, Hanny Al-Samkari

Blood (2024) 144 (Supplement 1): 2457.

https://doi.org/10.1182/blood-2024-199637

Published: 2024

Journal Articles

Safety, tolerability, and activity of the active C1s antibody riliprubart in cold agglutinin disease: a phase 1b study

Shirley D’Sa, Josephine M. I. Vos, Wilma Barcellini, Marek Wardęcki, Laurent Perrin, Graham Barker, Moshe Zilberstein, Michael Storek, Timothy Chow, Alexander Röth

Blood (2024) 143 (8): 713–720.

https://doi.org/10.1182/blood.2023022153

Published: 2024

Includes: Supplemental data

Journal Articles

Sutimlimab in patients with cold agglutinin disease: results of the randomized placebo-controlled phase 3 CADENZA trial

Alexander Röth, Sigbjørn Berentsen, Wilma Barcellini, Shirley D’Sa, Bernd Jilma, Marc Michel, Ilene C. Weitz, Masaki Yamaguchi, Jun-ichi Nishimura, Josephine M. I. Vos, Michael Storek, Nancy Wong, Parija Patel, Xiaoyu Jiang, Deepthi S. Vagge, Marek Wardęcki, Frank Shafer, Michelle Lee, Catherine M. Broome

Blood (2022) 140 (9): 980–991.

https://doi.org/10.1182/blood.2021014955

Published: 2022

Includes: Supplemental data

Journal Articles

Risk Factors for Thrombotic Events in Patients with PNH: A Nested Case-Control Study in the International PNH Registry

Britta Hoechsmann, Regis Peffault De Latour, MD PhD, Anita Hill, Alexander Röth, MD, Timothy Devos, MD, Christopher Patriquin, Wen-Chien Chou, MD PhD, Deepak Jain, Ke Zu, Jong Wook Lee, MD, PhD

Blood (2020) 136 (Supplement 1): 6–8.

https://doi.org/10.1182/blood-2020-136767

Published: 2020

Journal Articles

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 1 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Complement Inhibitors

Antonio Risitano, Alexander Röth, MD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Jeffrey Pu, MDPhD, Jun-ichi Nishimura, MD PhD, Lilyan Wright, PhD, Anita Appius, PhD, Alexandre Sostelly, PhD, Sasha Sreckovic, MD, Charlotte Vignal, PhD, Talha Munir, MBBS

Blood (2020) 136 (Supplement 1): 43–44.

https://doi.org/10.1182/blood-2020-137471

Published: 2020

Journal Articles

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

Antonio Risitano, Jun-Ho Jang, MD, Lee Gyeong-Won, Wanchai Wanachiwanawin, Hubert Schrezenmeier, Yuji Yonemura, MDPhD, Talha Munir, MBBS, Rodrigo Pavani, Jimmy Wang, Alexander D. Kulagin, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Flore Sicre de Fontbrune, Alexander Röth, MD

Blood (2020) 136 (Supplement 1): 31–33.

https://doi.org/10.1182/blood-2020-140418

Published: 2020

Journal Articles

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Peter Hillmen, MBChB, PhD, Jeffrey Szer, MBBS, Ilene C. Weitz, Alexander Röth, MD, Britta Hoechsmann, Jens Panse, MD, Kensuke Usuki, MD PhD, Morag Griffin, MBChB, FRCPath, Jean-Jacques Kiladjian, MD PhD, Carlos M. de Castro, MD, Hisakazu Nishimori, MD PhD, Lisa Tan, Mohamed Hamdani, Pascal Deschatelets, PhD, Cedric Francois, MD PhD, Federico Grossi, MD, Antonio Risitano, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 35–37.

https://doi.org/10.1182/blood-2020-142174

Published: 2020

Journal Articles

Effect of Sutimlimab Treatment on Healthcare Resource Utilization in Patients with Cold Agglutinin Disease

Alexander Röth, MD, Sophie Guillonneau, Nadege Narcisse, Paulo Carita, Jun Su, Florence Joly

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-139738

Published: 2020

Journal Articles

Long-Term Safety and Efficacy of Sutimlimab in Patients with Chronic Immune Thrombocytopenia

Catherine M. Broome, Alexander Röth, MD, David J. Kuter, MD, Marie Scully, MD, Roy Smith, Jennifer Wang, Xiaoyu Jiang, Caroline Reuter, Ahmed Daak, William Hobbs

Blood (2020) 136 (Supplement 1): 14–15.

https://doi.org/10.1182/blood-2020-139663

Published: 2020

Journal Articles

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

Alexander Röth, MD, Britta Hoechsmann, Morag Griffin, MBChB, FRCPath, Carlos M. de Castro, MD, Jeffrey Szer, MBBS, Kensuke Usuki, MD PhD, Juliette Soret, MD, Mohamed Hamdani, MS, Temitayo Ajayi, Sujata P. Sarda, PhD, Jens Panse, MD

Blood (2020) 136 (Supplement 1): 10–12.

https://doi.org/10.1182/blood-2020-134581

Published: 2020

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

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

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

Real-world safety of sutimlimab in patients with CAD/CAS: A multinational, multi-center, observational, prospective cohort Study

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial)

Switching between complement inhibitors in patients with PNH: A real-world analysis of strategy, efficacy, and safety.

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

Phase III Randomized COMMODORE 2 Trial: 2-Year Efficacy and Safety of Crovalimab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Naive to Complement Inhibition

First Interim Analysis of Patients with Cold Agglutinin Disease (CAD) Patients and the Sutimlimab-Treated Subgroup from the Cadence Registry

Iptacopan Monotherapy in Patients with Cold Agglutinin Disease: Phase II Study Results

Safety, tolerability, and activity of the active C1s antibody riliprubart in cold agglutinin disease: a phase 1b study

Sutimlimab in patients with cold agglutinin disease: results of the randomized placebo-controlled phase 3 CADENZA trial

Risk Factors for Thrombotic Events in Patients with PNH: A Nested Case-Control Study in the International PNH Registry

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 1 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Complement Inhibitors

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Effect of Sutimlimab Treatment on Healthcare Resource Utilization in Patients with Cold Agglutinin Disease

Long-Term Safety and Efficacy of Sutimlimab in Patients with Chronic Immune Thrombocytopenia

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

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