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Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program

Silvia Sanz, Abdulfaheem Khan, Sviatlana Rizk, Erik Houtsma, Tim Costigan, Luca Monaco

Blood (2025) 146 (Supplement 1): 3202.

https://doi.org/10.1182/blood-2025-3202

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

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

The IRON2 Study. A Retrospective Observational Study to Describe the Evolution of Iron Overload in Patients with Low-Risk Myelodysplastic Syndrome

Angel Remacha, MD, PhD, Beatriz Arrizabalaga, MD, Ana Villegas, Maria Soledad Duran, Lourdes Hermosin, MD, Raquel de Paz, Marta García, Regina García, Consuelo del Cañizo, MD, Silvia Sanz, Guillermo Sanz

Blood (2012) 120 (21): 1723.

https://doi.org/10.1182/blood.V120.21.1723.1723

Published: 2012

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Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program

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

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

The IRON2 Study. A Retrospective Observational Study to Describe the Evolution of Iron Overload in Patients with Low-Risk Myelodysplastic Syndrome

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ASH Publications

American Society of Hematology

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Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program

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

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

The IRON2 Study. A Retrospective Observational Study to Describe the Evolution of Iron Overload in Patients with Low-Risk Myelodysplastic Syndrome

Email alerts

ASH Publications

American Society of Hematology

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