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Journal Articles

Real-world clinical characteristics and treatment outcomes in PNH patients prescribed pegcetacoplan across europe, the United States and Canada

Jens Panse, Koo Wilson, Brianne Kerr, Sam Williamson, Susanna Libby, Yasmin Taylor, Peter Hillmen, David Dingli

Blood (2025) 146 (Supplement 1): 4970.

https://doi.org/10.1182/blood-2025-4970

Published: 2025

Journal Articles

Vexas syndrome in Germany: Insights from a prospective registry on clinical presentation, management and outcomes

Blood (2025) 146 (Supplement 1): 4968.

https://doi.org/10.1182/blood-2025-4968

Published: 2025

Journal Articles

Efficacy and safety results from the primary analysis of the pivotal summit trial: Bezuclastinib in adults with non-advanced systemic mastocytosis

Lindsay Rein, Nathan Boggs, Prithviraj Bose, Brian Modena, Vito Sabato, Karin Hartmann, Cem Akin, Tracy George, Cecilia Arana Yi, Hanneke Oude Elberink, Deepti Radia, Andreas Reiter, Miguel Piris-Villaespesa, Ingunn Dybedal, Jens Panse, Stephen Oh, Pankit Vachhani, Anthony Hunter, Mariana Castells, Cristina Bulai Livideanu, Paul van Daele, Arnold Kirshenbaum, Ivan Alvarez-Twose, Jennifer Vaughn, Minakshi Taparia, Sonia Cerquozzi, Andrzej Mital, Marek Hus, ALESSANDRA ROMANO, John Fahrenholz, Frederick Lansigan, Cristina Papayannidis, Helena Pomares, Michela Rondoni, Celalettin Ustun, Richard Herrscher, Michael Manning, Stéphane Barete, Mar Guilarte, Candido Rivera, Jonathan Bernstein, Peter Vadas, Chiara Elena, Derek McCulloch, Nina Orfali, Clodagh Keohane, Francesco Mannelli, Philippe Schafhausen, Amanda Pilla, Jenna Zhang, Lei Sun, Nisha Shah, Hina Jolin, Rachael Easton, Jessica Sachs, Frank Siebenhaar, Daniel Deangelo

Blood (2025) 146 (Supplement 1): 80.

https://doi.org/10.1182/blood-2025-80

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

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

Avapritinib achieves deep and durable symptom control with a well-tolerated safety profile in ism: Long-term outcomes from pioneer

Blood (2025) 146 (Supplement 1): 2024.

https://doi.org/10.1182/blood-2025-2024

Published: 2025

Journal Articles

Outcome of adult B-precursor acute lymphoblastic leukemia (ALL) treated with blinatumomab in addition to chemotherapy independent of MRD: First results from the national GMALL registry

Blood (2025) 146 (Supplement 1): 5124.

https://doi.org/10.1182/blood-2025-5124

Published: 2025

Journal Articles

An analysis of clonal dynamics in patients with indolent systemic mastocytosis treated with avapritinib in the pioneer study

Jens Panse, Tsewang Tashi, Jason Gotlib, Mariana Castells, Ivan Alvarez-Twose, Cem Akin, Cristina Bulai Livideanu, Vito Sabato, Juliana Schwaab, Andreas Reiter, Sigurd Broesby-Olsen, Frank Siebenhaar, Hui-Min Lin, Janet Hong, Benjamin Lampson, Guang Yang, Tracy George, Deepti Radia

Blood (2025) 146 (Supplement 1): 5573.

https://doi.org/10.1182/blood-2025-5573

Published: 2025

Journal Articles

Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH

Austin Kulasekararaj, Morag Griffin, Caroline Piatek, Jamile Shammo, Jun-ichi Nishimura, Christopher Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jens Panse, Anna Gaya, Yogesh Patel, Peng Liu, Gleb Filippov, Flore Sicre de Fontbrune, Antonio Risitano, Jong Wook Lee

Blood (2025) 145 (8): 811–822.

https://doi.org/10.1182/blood.2024026299

Published: 2025

Includes: Supplemental data

Journal Articles

Apex Part 2 Trial in Progress: A Phase 2 Open-Label Clinical Study of Bezuclastinib in Adult Patients with Advanced Systemic Mastocytosis

Blood (2024) 144 (Supplement 1): 4569.1.

https://doi.org/10.1182/blood-2024-194550

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

Real-World Adherence with Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria Compared with Previously Reported Oral Medication Adherence Rates

David Dingli, Brian Mulherin, Peter Hillmen, Barbara Czech, Jens Panse

Blood (2024) 144 (Supplement 1): 5680.

https://doi.org/10.1182/blood-2024-203541

Published: 2024

Journal Articles

Monotherapy Treatment with Zaltenibart (OMS906), an Alternative Pathway Masp-3 Inhibitor, Improved Key Hematologic Parameters in Patients with PNH with a Suboptimal Response to Ravulizumab: Interim Results from a Phase 2 Proof-of-Concept Study

Morag Griffin, Richard J. Kelly, Mathilde Gavillet, Petra Muus, Britta Hoechsmann, W. Jason Cummings, John Efthimiou, William Pullman, Hubert Schrezenmeier, Jens Panse

Blood (2024) 144 (Supplement 1): 4072.

https://doi.org/10.1182/blood-2024-205866

Published: 2024

Journal Articles

The Impact of Body Mass Index (BMI) in Patients with Aplastic Anemia Receiving Weight-Adjusted Horse Anti-Thymocyte Globulin (hATG) on Outcome, Response and Relapse Rate

Jeanette Walter, Benjamin Rolles, Yannic Schumacher, Frauke Theis, Leo Hansmann, Markus P. Radsak, Michael Heuser, Julia-Annabell Georgi, Jörg Chromik, Beatrice Drexler, Jörg Schubert, Kim Kricheldorf, Jens Panse, Thomas Stiehl, Tim H. H. Brummendorf, Bhavisha A. Patel, Neal S. Young, Emma M. Groarke, Fabian Beier

Blood (2024) 144 (Supplement 1): 4082.

https://doi.org/10.1182/blood-2024-207190

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

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Patient-Reported Outcomes from the Phase 3 ALPHA Trial

Caroline Piatek, Jong Wook Lee, Morag Griffin, Jun-Ichi Nishimura, Christopher Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jamile Shammo, Anna Gaya, Mohammed Mahdi, Peng Liu, Karl-Johan Myren, Andrew Messali, Flore Sicre de Fontbrune, Jens Panse, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 2692.

https://doi.org/10.1182/blood-2024-199506

Published: 2024

Journal Articles

Characterization of Erythrocytes in PNH: Can We Predict Response to Complement Inhibition Based on Different Erythrocyte Markers?

Elena E. Solomou, Alkmini Anastasiadi, Maria Papaioannou, Maria Angelopoulou, Panagiotis Zikos, Ioannis Assimakopoulos, Maria Dimou, Panagiotis Tsaftaridis, Jens Panse, Vasileios Tzounakas

Blood (2024) 144 (Supplement 1): 1317.

https://doi.org/10.1182/blood-2024-206170

Published: 2024

Journal Articles

The Association of Anemia and Patient Reported Outcomes in the New Era of PNH Treatment

Blood (2024) 144 (Supplement 1): 2321.

https://doi.org/10.1182/blood-2024-204693

Published: 2024

Journal Articles

Thrombosis and Encapsulated Bacterial Infection Rates in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Pegcetacoplan: Nearly 3 Years of Post-Marketing Experience

Gloria F. Gerber, Richard J. Kelly, Hisakazu Nishimori, Regina Horneff, Peter Hillmen, Uchendu O. Uchendu, Yiwei Zhang, Stacie Lallier, Jens Panse

Blood (2024) 144 (Supplement 1): 5698.

https://doi.org/10.1182/blood-2024-201443

Published: 2024

Journal Articles

Real-World Clinical Outcomes for Complement Inhibitor Experienced and Naïve Paroxysmal Nocturnal Hemoglobinuria Patients Prescribed Pegcetacoplan in Europe and Canada

Jens Panse, Juan Carlos Vallejo Llamas, Koo Wilson, Zalmai Hakimi, Barbara Czech, Brianne Kerr, Sam Williamson, Jade Garratt-Wheeldon, Yasmin Taylor, Niall Hatchell, Christopher Patriquin

Blood (2024) 144 (Supplement 1): 5085.

https://doi.org/10.1182/blood-2024-209732

Published: 2024

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Real-world clinical characteristics and treatment outcomes in PNH patients prescribed pegcetacoplan across europe, the United States and Canada

Vexas syndrome in Germany: Insights from a prospective registry on clinical presentation, management and outcomes

Efficacy and safety results from the primary analysis of the pivotal summit trial: Bezuclastinib in adults with non-advanced systemic mastocytosis

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

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)

Avapritinib achieves deep and durable symptom control with a well-tolerated safety profile in ism: Long-term outcomes from pioneer

Outcome of adult B-precursor acute lymphoblastic leukemia (ALL) treated with blinatumomab in addition to chemotherapy independent of MRD: First results from the national GMALL registry

An analysis of clonal dynamics in patients with indolent systemic mastocytosis treated with avapritinib in the pioneer study

Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH

Apex Part 2 Trial in Progress: A Phase 2 Open-Label Clinical Study of Bezuclastinib in Adult Patients with Advanced Systemic Mastocytosis

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

Real-World Adherence with Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria Compared with Previously Reported Oral Medication Adherence Rates

Monotherapy Treatment with Zaltenibart (OMS906), an Alternative Pathway Masp-3 Inhibitor, Improved Key Hematologic Parameters in Patients with PNH with a Suboptimal Response to Ravulizumab: Interim Results from a Phase 2 Proof-of-Concept Study

The Impact of Body Mass Index (BMI) in Patients with Aplastic Anemia Receiving Weight-Adjusted Horse Anti-Thymocyte Globulin (hATG) on Outcome, Response and Relapse Rate

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

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Patient-Reported Outcomes from the Phase 3 ALPHA Trial

Characterization of Erythrocytes in PNH: Can We Predict Response to Complement Inhibition Based on Different Erythrocyte Markers?

The Association of Anemia and Patient Reported Outcomes in the New Era of PNH Treatment

Thrombosis and Encapsulated Bacterial Infection Rates in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Pegcetacoplan: Nearly 3 Years of Post-Marketing Experience

Real-World Clinical Outcomes for Complement Inhibitor Experienced and Naïve Paroxysmal Nocturnal Hemoglobinuria Patients Prescribed Pegcetacoplan in Europe and Canada

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