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Journal Articles

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Austin Kulasekararaj, Morag Griffin, Robert Brodsky, Jun-ichi Nishimura, Christopher Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Anna Gaya, Yogesh Patel, Ying Tang, Gleb Filippov, Mohammed Mahdi, Flore Sicre-de-Fontbrune, Jong Wook Lee

Blood (2025) 146 (Supplement 1): 949.

https://doi.org/10.1182/blood-2025-949

Published: 2025

Journal Articles

Early access program for danicopan (ALXN2040) as add-on treatment to eculizumab or ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH): Preliminary data from the Italian real-world survey.

Elisabetta Metafuni, Antonio De Vivo, Andrea Patriarca, Marta Venturi, Simona Raso, Amalia Figuera, Antonella Sau, Maria Bruna Greve, Milena Mirabile, Olga Mulas, Eloise Beggiato, Antonella Carbone, Martina Salvatori, Anna Paola Iori, Simona Sica, Wilma Barcellini, Bruno Fattizzo

Blood (2025) 146 (Supplement 1): 4665.

https://doi.org/10.1182/blood-2025-4665

Published: 2025

Journal Articles

Real world use of sutimlimab in patients with cold agglutinin disease: An international study

Blood (2025) 146 (Supplement 1): 1100.

https://doi.org/10.1182/blood-2025-1100

Published: 2025

Journal Articles

Characterization of bone marrow B-cells in patients with warm autoimmune hemolytic anemia by single-cell analysis

Bruno Fattizzo, Francesca Lazzaroni, Filippo Viviani, Emanuele Calvi, Giacinto Luca Pedone, Marta Bortolotti, Akihiro Maeda, Marta Lionetti, Alessio Marella, Antonio Matera, Niccolo Bolli, Francesco Passamonti, Wilma Barcellini, Matteo Claudio Da Vià

Blood (2025) 146 (Supplement 1): 1122.

https://doi.org/10.1182/blood-2025-1122

Published: 2025

Journal Articles

Decoding the ektacytometric landscape of hereditary spherocytosis: Insights from 204 cases

Anna Zaninoni, Elisa Fermo, cristina vercellati, Anna Marcello, Dario Consonni, Bruno Fattizzo, Wilma Barcellini, Paola Bianchi

Blood (2025) 146 (Supplement 1): 2889.

https://doi.org/10.1182/blood-2025-2889

Published: 2025

Journal Articles

Distinct patterns of bone marrow complement and immunoglobulin deposition define unique clinicopathologic phenotypes in myelodysplastic neoplasms

Giacinto Luca Pedone, Claudia Leoni, Marta Bortolotti, Wilma Barcellini, Matteo Claudio Da Vià, Bruno Fattizzo

Blood (2025) 146 (Supplement 1): 7368.

https://doi.org/10.1182/blood-2025-7368

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

Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice

Bruno Fattizzo, Francesco Versino, Wilma Barcellini

Blood (2025) 146 (4): 411–421.

https://doi.org/10.1182/blood.2024027574

Published: 2025

Includes: Supplemental data

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

Part a Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Patients with Warm Autoimmune Hemolytic Anemia (wAIHA): Multicenter, Open-Label, Phase 2b Study

Nichola Cooper, David J Kuter, Henrik Frederiksen, Bing Han, Frederick Chen, Wilma Barcellini, Joan Cid, Catherine M. Broome, Míriam Vara, Amitabha Mazumder, Alessandro Lucchesi, Xin Zhao, Antonio M Risitano, Sumit Sawhney, Quentin A. Hill, Deepthi Jayawardene, Remco Diab, Jing Zhao, Ahmed Daak

Blood (2024) 144 (Supplement 1): 3836.

https://doi.org/10.1182/blood-2024-200325

Published: 2024

Journal Articles

Toward the Creation of Diagnostic Algorithms for Congenital Hemolytic Anemia Using Advanced Parameters from Modern Hematology Analyzers

Paola Bianchi, Azita Sharif, Cristina Vercellati, Elisa Fermo, Maria Laura Chiaretto, Anna Marcello, Anna Zaninoni, Nadia Mirra, Simona Torricelli, Virna De Zordi, Stefania Bottalico, Francesco Passamonti, Wilma Barcellini, Matteo Vidali, Anna Chiara Migliorini

Blood (2024) 144 (Supplement 1): 1086.

https://doi.org/10.1182/blood-2024-203099

Published: 2024

Journal Articles

Not so Rare, Not so Mild Disease: Defects of Flippase Activity Due to ATP11C Mutations. Description of Three New Cases

Elisa Fermo, Elena Trombetta, Anna Marcello, Cristina Vercellati, Giulia Maria Ferrari, Anna Zaninoni, Valentina Brancaleoni, Marta Tornese, Wilma Barcellini, Tommaso Casini, Paola Consuelo Corti, Paola Bianchi

Blood (2024) 144 (Supplement 1): 2465.

https://doi.org/10.1182/blood-2024-204922

Published: 2024

Journal Articles

Reaction - Real Life Use of Ravulizumab in Italian Patients with Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study, Final Results

Blood (2024) 144 (Supplement 1): 2464.

https://doi.org/10.1182/blood-2024-204659

Published: 2024

Journal Articles

Effect of Obexelimab on the Production of Anti-Erythrocyte Autoantibodies in Vitro By Mitogen Stimulated Direct Anti-Globulin Test (MS-DAT)

Bruno Fattizzo, Mark Matijevic, Michele Youd, Xiao Feng, Wilma Barcellini, Anna Zaninoni

Blood (2024) 144 (Supplement 1): 5215.

https://doi.org/10.1182/blood-2024-198318

Published: 2024

Journal Articles

Aplastic Anemia in the Elderly: Treatment Patterns and Outcomes from an International Observational Study

Bruno Fattizzo, Carmelo Gurnari, Sabrina Giammarco, Antony Ricchiuti, Hussein Awada, Marta Bortolotti, Giacinto Luca Pedone, Francesco Versino, Dario Consonni, Roochi Trikha, Shreyans Gandhi, Simona Sica, Jaroslaw Maciejewski, Austin G. Kulasekararaj, Wilma Barcellini

Blood (2024) 144 (Supplement 1): 4076.

https://doi.org/10.1182/blood-2024-198321

Published: 2024

Journal Articles

Eltrombopag Added to Standard Immunosuppressive Treatment Improves Long-Term Outcomes As Front-Line Therapy for Severe Aplastic Anemia: Final 2-Year Analysis of EBMT-Saawp Race Study

Antonio M Risitano, Simona Iacobelli, Austin G. Kulasekararaj, Marleen Van Os, Sophie Terwel, Morag Griffin, Constantijn J.M. Halkes, Christian Recher, Fiorenza Barraco, Edouard Forcade, Carlos Vallejo, Beatrice Drexler, Jean-Baptiste Méar, Alexander Smith, Emanuele Angelucci, Reinier Raymakers, Marco R De Groot, Etienne Daguindau, Erfan Nur, Wilma Barcellini, Nigel H. Russell, Louis Terriou, Anna Paola Iori, Walter Barberi, Anna Sureda Balari, Isabel Sanchez-Ortega, Blanca Xicoy, Isidro Jarque Ramos, Jamie Cavenagh, Flore Sicre De Fontbrune, Camilla Frieri, Talha Munir, Jennifer Tjon, Suzanne Tavitian, Aline Praire, Laurence Clement, Florence Rabian, Luana Marano, Anita Hill, Elena Palmisani, Petra Muus, Serena Marotta, Fabiana Cacace, Marica Laurino, Jakob Passweg, Sr., Judith Marsh, Gerard Socie, Ghulam Mufti, Carlo Dufour, Regis Peffault De Latour

Blood (2024) 144 (Supplement 1): 302.

https://doi.org/10.1182/blood-2024-194699

Published: 2024

Journal Articles

Mixed Type Autoimmune Hemolytic Anemia: A Single Center Observational Study

Francesco Versino, Giacinto Luca Pedone, Marta Bortolotti, Wilma Barcellini, Bruno Fattizzo

Blood (2024) 144 (Supplement 1): 3844.

https://doi.org/10.1182/blood-2024-194219

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

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

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

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Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial

Early access program for danicopan (ALXN2040) as add-on treatment to eculizumab or ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH): Preliminary data from the Italian real-world survey.

Real world use of sutimlimab in patients with cold agglutinin disease: An international study

Characterization of bone marrow B-cells in patients with warm autoimmune hemolytic anemia by single-cell analysis

Decoding the ektacytometric landscape of hereditary spherocytosis: Insights from 204 cases

Distinct patterns of bone marrow complement and immunoglobulin deposition define unique clinicopathologic phenotypes in myelodysplastic neoplasms

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

Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice

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

Part a Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Patients with Warm Autoimmune Hemolytic Anemia (wAIHA): Multicenter, Open-Label, Phase 2b Study

Toward the Creation of Diagnostic Algorithms for Congenital Hemolytic Anemia Using Advanced Parameters from Modern Hematology Analyzers

Not so Rare, Not so Mild Disease: Defects of Flippase Activity Due to ATP11C Mutations. Description of Three New Cases

Reaction - Real Life Use of Ravulizumab in Italian Patients with Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study, Final Results

Effect of Obexelimab on the Production of Anti-Erythrocyte Autoantibodies in Vitro By Mitogen Stimulated Direct Anti-Globulin Test (MS-DAT)

Aplastic Anemia in the Elderly: Treatment Patterns and Outcomes from an International Observational Study

Eltrombopag Added to Standard Immunosuppressive Treatment Improves Long-Term Outcomes As Front-Line Therapy for Severe Aplastic Anemia: Final 2-Year Analysis of EBMT-Saawp Race Study

Mixed Type Autoimmune Hemolytic Anemia: A Single Center Observational Study

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

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

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

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