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Journal Articles

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Blood (2024) 144 (12): 1329–1342.

https://doi.org/10.1182/blood.2023022751

Published: 2024

Includes: Supplemental data

Journal Articles

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Blood (2024) 144 (3): 323–333.

https://doi.org/10.1182/blood.2024024173

Published: 2024

Journal Articles

Dose-Exposure-Response Relationships of Biomarkers and Efficacy Measures with Iptacopan, a Complement Factor B Inhibitor, in Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) with or without Concomitant Anti-C5 Therapy

Antonio Risitano, Régis Peffault de Latour, Eng Soo Yap, Lily LL Wong, Bor-Sheng Ko, Sung-Soo Yoon, Alexander Roeth, Irina Baltcheva, Julie Milojevic, Niamh Fagan, Kenneth Kulmatycki, Prasanna K Nidamarthy, Guido Junge, Jun Ho Jang

Blood (2022) 140 (Supplement 1): 5803–5804.

https://doi.org/10.1182/blood-2022-160114

Published: 2022

Journal Articles

Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)

Blood (2022) 140 (Supplement 1): 5799–5800.

https://doi.org/10.1182/blood-2022-169086

Published: 2022

Journal Articles

Complement Biomarkers for Tracking Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated with Last-Generation Complement Inhibitors

Luana Marano, Patrizia Ricci, Camilla Frieri, Eleonora Urciuoli, Ada Sanseverino, Fabiana Cacace, Ilenia Manfra, Giovanna De Santis, Selenia Vitiello, Serena Marotta, Antonio Risitano

Blood (2022) 140 (Supplement 1): 5797–5798.

https://doi.org/10.1182/blood-2022-158329

Published: 2022

Journal Articles

Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party

Blood (2022) 140 (Supplement 1): 657–659.

https://doi.org/10.1182/blood-2022-163140

Published: 2022

Journal Articles

HLA-Haploidentical Stem Cell Transplantation in Children with Inherited Bone Marrow Failure Syndromes: A Retrospective Analysis on Behalf of EBMT Severe Aplastic Anemia Working Party

Blood (2022) 140 (Supplement 1): 5813–5815.

https://doi.org/10.1182/blood-2022-163219

Published: 2022

Journal Articles

Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study

Peter J. Browett, Austin Kulasekararaj, Rosario Notaro, Masayo Ogawa, Antonio Risitano, Ji Yu, Jong Wook Lee

Blood (2022) 140 (Supplement 1): 717–719.

https://doi.org/10.1182/blood-2022-169301

Published: 2022

Journal Articles

Haploidentical Versus Matched Sibling Donor Hematopoietic Stem Cell Transplantation for Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia: A Study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Arnon Nagler, Myriam Labopin, Pietro Pioltelli, Mutlu Arat, Ibrahim Yakoub-Agha, Aleksandr D. Kulagin, Emanuele Angelucci, Hakan Ozdogu, Antonio Risitano, Fabio Ciceri, Zubeyde Nur Ozkurt, Jaime Sanz, Eolia Brissot, Zinaida Peric, Sebastian Giebel, Mohamad Mohty

Blood (2021) 138 (Supplement 1): 1841.

https://doi.org/10.1182/blood-2021-144558

Published: 2021

Journal Articles

Two Currently Recruiting Randomized Phase III Trials: COMMODORE 1 and 2 Evaluating Crovalimab Vs Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Current Anti-Complement Therapy

Austin G. Kulasekararaj, Antonio Risitano, Alexander Roeth, Guangsheng He, Jeffrey Pu, Lilyan Wright, Anita Appius, Alexandre Sostelly, Sasha Sreckovic, Sven F Stanzel, Talha Munir, Jun-Ichi Nishimura

Blood (2021) 138 (Supplement 1): 4313.

https://doi.org/10.1182/blood-2021-152306

Published: 2021

Journal Articles

Categorized Hematologic Response to Pegcetacoplan and Correlations with Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from Phase 1b, Phase 2a, and Phase 3 Trials

Antonio Risitano, Raymond S Wong, Mohammed Al-Adhami, Crystal Chen, Regis Peffault De Latour

Blood (2021) 138 (Supplement 1): 1104.

https://doi.org/10.1182/blood-2021-147988

Published: 2021

Journal Articles

Transplantation for Congenital Sideroblastic Anaemia Is Feasible and Offers Outcomes Comparable to Other Transfusion Dependent Anaemias. a Joint Retrospective Study of the Paediatric Diseases and Severe Aplastic Anaemia Working Parties (PDWP/SAAWP) of EBMT

Blood (2020) 136 (Supplement 1): 45–47.

https://doi.org/10.1182/blood-2020-142397

Published: 2020

Journal Articles

Upfront Alternative Donor Transplant Versus Immunosuppressive Therapy in Patients with Severe Aplastic Anemia Who Lack Fully HLA Matched Related Donor: Systematic Review and Meta-Analysis of Retrospective Studies. on Behalf of the Severe Aplastic Anemia Working Party of European Group for Blood and Marrow Transplantation (SAAWP of EBMT)

Hind Alotaibi, MBBS, Mahmoud Aljurf, Regis Peffault De Latour, MD PhD, Shahid Iqbal, Andrea Bacigalupo, Judith Marsh, Hubert Schrezenmeier, Eliane Gluckman, Mansour Alfayez, Britta Hoechsmann, Constantijn Halkes, Josu de la Fuente, Nawal AlShehry, Simone Cesaro, Jakob Passweg, Carlo Dufour, Antonio Risitano, John F. DiPersio, MD PhD, Ibraheem Motabi

Blood (2020) 136 (Supplement 1): 6–7.

https://doi.org/10.1182/blood-2020-140846

Published: 2020

Journal Articles

Gvhd and Relapse Free Survival (GRFS) after Allogeneic Transplantation for Idiopathic Severe Aplastic Anemia: An Analysis from the Saawp Data Quality Initiative Program of EBMT

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-141757

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

Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS)

Antonio Risitano, Ilene C. Weitz, Carlos M. de Castro, MD, Jean-Jacques Kiladjian, Morag Griffin, MBChB, FRCPath, Hisakazu Nishimori, MD PhD, Mohamed Hamdani, Temitayo Ajayi, Scott B. Baver, PhD, Regis Peffault De Latour, MD PhD

Blood (2020) 136 (Supplement 1): 44–45.

https://doi.org/10.1182/blood-2020-142166

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

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 2 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Not Previously Treated with Complement Inhibitors

Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Guangsheng He, MD PhD, Talha Munir, MBBS, Jeffrey Pu, MDPhD, Antonio Risitano, Alexander Röth, MD, Camelia S. Sima, MD, Anita Appius, PhD, Alexandre Sostelly, PhD, Sasha Sreckovic, MD, Charlotte Vignal, PhD, Jun-Ichi Nishimura, MD PhD

Blood (2020) 136 (Supplement 1): 34.

https://doi.org/10.1182/blood-2020-136647

Published: 2020

Journal Articles

BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Austin Kulasekararaj, MD MRCP, FRCPath, Jacques Le Roux Malherbe, MD, Andrew McDonald, Melanie Cornpropst, PharmD, PhD, Phil Collis, PhD, Matthew Davidson, PharmD, Xilin Chen, MDPhD, Kristin Tower, Diane Gesty-Palmer, MD PhD, William P. Sheridan, MBBS, Antonio Risitano

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

https://doi.org/10.1182/blood-2020-138838

Published: 2020

NARROW

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Dose-Exposure-Response Relationships of Biomarkers and Efficacy Measures with Iptacopan, a Complement Factor B Inhibitor, in Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) with or without Concomitant Anti-C5 Therapy

Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)

Complement Biomarkers for Tracking Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated with Last-Generation Complement Inhibitors

Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party

HLA-Haploidentical Stem Cell Transplantation in Children with Inherited Bone Marrow Failure Syndromes: A Retrospective Analysis on Behalf of EBMT Severe Aplastic Anemia Working Party

Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study

Haploidentical Versus Matched Sibling Donor Hematopoietic Stem Cell Transplantation for Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia: A Study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Two Currently Recruiting Randomized Phase III Trials: COMMODORE 1 and 2 Evaluating Crovalimab Vs Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Current Anti-Complement Therapy

Categorized Hematologic Response to Pegcetacoplan and Correlations with Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from Phase 1b, Phase 2a, and Phase 3 Trials

Transplantation for Congenital Sideroblastic Anaemia Is Feasible and Offers Outcomes Comparable to Other Transfusion Dependent Anaemias. a Joint Retrospective Study of the Paediatric Diseases and Severe Aplastic Anaemia Working Parties (PDWP/SAAWP) of EBMT

Gvhd and Relapse Free Survival (GRFS) after Allogeneic Transplantation for Idiopathic Severe Aplastic Anemia: An Analysis from the Saawp Data Quality Initiative Program of EBMT

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

Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS)

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

Trial in Progress: The Phase III, Randomized, Open-Label, Multicenter COMMODORE 2 Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Adult and Adolescent Patients with Paroxysmal Nocturnal Hemoglobinuria Not Previously Treated with Complement Inhibitors

BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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American Society of Hematology

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