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Journal Articles

Long-Term Efficacy and Safety of Danicopan as Add-On Therapy to Ravulizumab or Eculizumab in PNH With Significant EVH

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

Blood blood.2024026299.

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

Published: 2024

Includes: Supplemental data

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

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

Characterization of Complement and Immunoglobulin Deposition in Bone Marrow Trephine Biopsies of Patients with Cytopenias

Cecilia Anna Fidanza, Marta Bortolotti, Giorgio Alberto Croci, Wilma Barcellini, Bruno Fattizzo

Blood (2024) 144 (Supplement 1): 5666.

https://doi.org/10.1182/blood-2024-198268

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

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

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

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

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

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

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

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

Cold Agglutinin Disease (CAD) Real-World Evidence (CADENCE) Registry: Update to the Study Design of the First International, Prospective CAD Registry

Alexander Roeth, Wilma Barcellini, Ulrich Jaeger, Yasutaka Ueda, Marc Michel, Quentin A. Hill, Shruti Srivastava, Ronnie Yoo, Catherine M. Broome

Blood (2023) 142 (Supplement 1): 7341.

https://doi.org/10.1182/blood-2023-180678

Published: 2023

Journal Articles

Clonal Hematopoiesis in Patients with Immune Thrombocytopenia: Preliminary Results of a Cross-Sectional Study

Alessandro Bosi, Marta Canzi, Marco Capecchi, Juri Alessandro Giannotta, Andrea Artoni, Wilma Barcellini, Bruno Fattizzo

Blood (2023) 142 (Supplement 1): 2581.

https://doi.org/10.1182/blood-2023-185087

Published: 2023

Journal Articles

Efficacy and Safety of Subcutaneous Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous Eculizumab: 2-Year Follow-up

Caroline I Piatek, Flore Sicre De Fontbrune, Fahri Sahin, Wolfgang Füreder, Sandra Fatima Gualandro, Wilma Barcellini, Masayo Ogawa, Stephan Ortiz, Ayca Ozol-Godfrey, Jeff Szer, Mustafa Yenerel

Blood (2023) 142 (Supplement 1): 2713.

https://doi.org/10.1182/blood-2023-190060

Published: 2023

Journal Articles

A Phase III Clinical Trial Program Investigating the Efficacy and Safety of Ianalumab in Patients with Primary Immune Thrombocytopenia (VAYHIT1 and VAYHIT2) and Warm Autoimmune Hemolytic Anemia (VAYHIA)

Hanny Al-Samkari, Wilma Barcellini, Nichola Cooper, Waleed Ghanima, Marc Michel, Raymond SM Wong, Francesco Zaja, Fengkui Zhang, Patrick Urban, Alex Allepuz, Justyna Fronczek-Sokol, Jens Haenig, Pascal Edrich, Adam Cuker

Blood (2023) 142 (Supplement 1): 5456.

https://doi.org/10.1182/blood-2023-180647

Published: 2023

Journal Articles

Ianalumab, a Novel Anti-B-Cell Activating Factor (BAFF) Receptor (BAFF-R) Monoclonal Antibody (mAb) in Development for Immune Thrombocytopenia (ITP) and Warm Autoimmune Hemolytic Anemia (wAIHA), Has Demonstrated a Favorable Safety Profile in Sjögren's Syndrome (SjS), Systemic Lupus Erythematosus (SLE) and Chronic Lymphocytic Leukemia (CLL)

Adam Cuker, Hanny Al-Samkari, Wilma Barcellini, Nichola Cooper, Waleed Ghanima, Marc Michel, Raymond SM Wong, Francesco Zaja, Fengkui Zhang, Patrick Urban, Roberto Abi Rached, David J Kuter

Blood (2023) 142 (Supplement 1): 5427.

https://doi.org/10.1182/blood-2023-180055

Published: 2023

NARROW

Long-Term Efficacy and Safety of Danicopan as Add-On Therapy to Ravulizumab or Eculizumab in PNH With Significant EVH

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

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

Characterization of Complement and Immunoglobulin Deposition in Bone Marrow Trephine Biopsies of Patients with Cytopenias

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

Mixed Type Autoimmune Hemolytic Anemia: A Single Center Observational Study

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

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

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

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

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

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

Cold Agglutinin Disease (CAD) Real-World Evidence (CADENCE) Registry: Update to the Study Design of the First International, Prospective CAD Registry

Clonal Hematopoiesis in Patients with Immune Thrombocytopenia: Preliminary Results of a Cross-Sectional Study

Efficacy and Safety of Subcutaneous Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous Eculizumab: 2-Year Follow-up

A Phase III Clinical Trial Program Investigating the Efficacy and Safety of Ianalumab in Patients with Primary Immune Thrombocytopenia (VAYHIT1 and VAYHIT2) and Warm Autoimmune Hemolytic Anemia (VAYHIA)

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American Society of Hematology

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American Society of Hematology

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