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Journal Articles

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

Régis Peffault de Latour, Jeffrey Szer, Koo Wilson, Piotr Wojciechowski, Zalmai Hakimi, Jameel Nazir, Barbara Czech, Austin G. Kulasekararaj

Blood (2024) 144 (Supplement 1): 7706.

https://doi.org/10.1182/blood-2024-209712

Published: 2024

Journal Articles

Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve Severe Aplastic Anemia: Results of the Diaamond-Ava-First Trial

Blood (2024) 144 (Supplement 1): 303.

https://doi.org/10.1182/blood-2024-209378

Published: 2024

Journal Articles

Cellular and Cell-Free Genomic Characterization of Patients Receiving Avatrombopag and Immunosuppressive Therapy for Treatment-Naïve Severe Aplastic Anemia

Blood (2024) 144 (Supplement 1): 1318.

https://doi.org/10.1182/blood-2024-208841

Published: 2024

Journal Articles

Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve and Relapsed/Refractory Severe Aplastic Anaemia - the Diaamond-Ava-First and Diaamond-Ava-Next Bayesian Optimal Phase II Trials

Blood (2022) 140 (Supplement 1): 2931–2932.

https://doi.org/10.1182/blood-2022-168963

Published: 2022

Journal Articles

Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria

Christopher J. Patriquin, Andrija Bogdanovic, Morag Griffin, Richard Kelly, Jaroslaw P. Maciejewski, Brian Mulherin, Régis Peffault de Latour, Alexander Roeth, Veena Selvaratnam, Jeffrey Szer, Jessica Savage, Regina Horneff, Lisa Tan, Michael Yeh, Jens Panse

Blood (2022) 140 (Supplement 1): 2921–2923.

https://doi.org/10.1182/blood-2022-163625

Published: 2022

Journal Articles

Longitudinal Genomic Characterization Using Cell-Free DNA in Patients with Idiopathic Aplastic Anemia

Georgina L Ryland, PhD, Lucy C Fox, MBBS, Xiangting Chen, PhD, Satwica Yerneni, MS, Jeffrey Szer, MBBS, Ashish Bajel, FRACP, Robin J Filshie, MBChB, PhD FRACP, FRCPA, Sue Morgan, FRACP, FRCPA, MBBS, Sarah-Jane Dawson, MD PhD, Piers Blombery, MBBS

Blood (2020) 136 (Supplement 1): 5–6.

https://doi.org/10.1182/blood-2020-140912

Published: 2020

Journal Articles

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

Alexander Röth, MD, Britta Hoechsmann, Morag Griffin, MBChB, FRCPath, Carlos M. de Castro, MD, Jeffrey Szer, MBBS, Kensuke Usuki, MD PhD, Juliette Soret, MD, Mohamed Hamdani, MS, Temitayo Ajayi, Sujata P. Sarda, PhD, Jens Panse, MD

Blood (2020) 136 (Supplement 1): 10–12.

https://doi.org/10.1182/blood-2020-134581

Published: 2020

Journal Articles

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 Years with Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension Studies

Regis Peffault De Latour, MD PhD, Jeffrey Szer, MBBS, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Jin Seok Kim, MD PhD, Caroline I. Piatek, MD, Alexander D. Kulagin, MD PhD, Anita Hill, Jimmy Wang, Ji Yu, Masayo Ogawa, MD FAAP, Hubert Schrezenmeier, Jong Wook Lee, MD, PhD

Blood (2020) 136 (Supplement 1): 42–43.

https://doi.org/10.1182/blood-2020-140777

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

Biomarker Analysis in Patients (pts) with Steroid-Refractory Acute Graft-Vs-Host Disease (aGVHD) Treated with Ruxolitinib (RUX) or Best Available Therapy (BAT) in the Randomized, Phase 3 REACH2 Study

Blood (2020) 136 (Supplement 1): 26–27.

https://doi.org/10.1182/blood-2020-140403

Published: 2020

Journal Articles

Pegcetacoplan Is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement

Regis Peffault De Latour, MD PhD, Carlos M. de Castro, MD, Jeffrey Szer, MBBS, Kensuke Usuki, MD PhD, Peter Hillmen, MBChB, PhD, Morag Griffin, MBChB, FRCPath, Mohamed Hamdani, MS, Temitayo Ajayi, Hisakazu Nishimori, MD PhD, Ilene C. Weitz

Blood (2020) 136 (Supplement 1): 32–33.

https://doi.org/10.1182/blood-2020-141061

Published: 2020

Journal Articles

The Global State of Hematopoietic Stem Cell Transplantation for Multiple Myeloma: An Analysis of the Worldwide Network of Blood and Marrow Transplantation (WBMT) Database and the Global Burden of Disease Study

Blood (2019) 134 (Supplement_1): 412.

https://doi.org/10.1182/blood-2019-126837

Published: 2019

Journal Articles

One and Half Million Hematopoietic Stem Cell Transplants (HSCT). Dissemination, Trends and Potential to Improve Activity By Telemedicine from the Worldwide Network for Blood and Marrow Transplantation (WBMT)

Blood (2019) 134 (Supplement_1): 2035.

https://doi.org/10.1182/blood-2019-125232

Published: 2019

Journal Articles

An Update of Australasian Trends in Allogeneic Stem Cell Transplantation for Myelofibrosis in the Molecular Era

Blood (2019) 134 (Supplement_1): 5719.

https://doi.org/10.1182/blood-2019-126547

Published: 2019

Journal Articles

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Shoshana Revel-Vilk, MD, Jeffrey Szer, MBBS, Michal Becker Cohen, Ari Zimran, MD

Blood (2019) 134 (Supplement_1): 4862.

https://doi.org/10.1182/blood-2019-131013

Published: 2019

Journal Articles

Results from a Phase 3, Multicenter, Non-Inferiority Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Eculizumab

Austin G. Kulasekararaj, MBBS, MD MRCP, FRCPath, Anita Hill, MD PhS, Scott T. Rottinghaus, MD, Saskia Langemeijer, MD PhD, Richard A. Wells, MD DPhil, F. Ataulfo Gonzalez-Fernandez, Anna Gaya, Jong-Wook Lee, MD PhD, Emilio Ojeda Gutierrez, Caroline I. Piatek, Jeffrey Szer, MBBS, Antonio M. Risitano, MD PhD, Shinji Nakao, MD PhD, Eric Bachman, Lori Shafner, PhD, Andrew I. Damokosh, PhD, Stephan Ortiz, RPh, PhD, Alexander Röth, MD, Regis Peffault De Latour

Blood (2018) 132 (Supplement 1): 625.

https://doi.org/10.1182/blood-2018-99-119147

Published: 2018

Journal Articles

Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter Studies

Regis Peffault De Latour, Robert A. Brodsky, MD, Stephan Ortiz, RPh, PhD, Antonio M. Risitano, MD PhD, Jun-Ho Jang, MD PhD, Peter Hillmen, MB ChB, PhD FRCP, FRCPath, Aleksandr Kulagin, Rajendra Pradhan, PhD, Scott T. Rottinghaus, MD, Rasha Aguzzi, MS, Richard A. Wells, MD DPhil, Jeffrey Szer, MBBS

Blood (2018) 132 (Supplement 1): 626.

https://doi.org/10.1182/blood-2018-99-110858

Published: 2018

Journal Articles

A Phase 3 Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria Naive to Complement Inhibitors: Results of a Subgroup Analysis with Patients Stratified By Baseline Hemolysis Level, Transfusion History, and Demographics

Ilene C. Weitz, MD, Aleksandr Kulagin, Shinji Nakao, MD PhD, Caroline I. Piatek, MD, Jeffrey Szer, MBBS, Scott T. Rottinghaus, MD, Lori Volles, MD, Andrew I. Damokosh, PhD, Rasha Aguzzi, MS, Loree Larratt, MD, Antonio M. Risitano, MD PhD

Blood (2018) 132 (Supplement 1): 627.

https://doi.org/10.1182/blood-2018-99-110623

Published: 2018

Journal Articles

Nivolumab for Relapsed or Residual Haematological Malignancies after Allogeneic Haematopoietic Stem Cell Transplantation (NIVALLO)

Eric Wong, MBBS, Emily Dawson, Joanne Davis, PhD, Rachel Koldej, PhD, Mandy Ludford-Menting, Matthew Lansdown, Heather Lambie, Jeffrey Szer, MBBS, Andrew P. Grigg, MBBS, FRACP, FRCPA, MD, David Ritchie, MBChB, PhD FRACP, FRCPA

Blood (2018) 132 (Supplement 1): 4633.

https://doi.org/10.1182/blood-2018-99-112310

Published: 2018

Journal Articles

A Local Experience of Pregnancy Outcomes in Sickle Cell Disease and Red Cell Exchange

Rose Turner, MBBS, Claire Dowsing, Nurse Practitioner, Gemma Crighton, BHB, MBChB, FRACP, FRCPA, Kylie D. Mason, MBBS, MD FRACP, FRCPA, PhD, Jeffrey Szer, MBBS

Blood (2018) 132 (Supplement 1): 4920.

https://doi.org/10.1182/blood-2018-99-116191

Published: 2018

NARROW

Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve Severe Aplastic Anemia: Results of the Diaamond-Ava-First Trial

Cellular and Cell-Free Genomic Characterization of Patients Receiving Avatrombopag and Immunosuppressive Therapy for Treatment-Naïve Severe Aplastic Anemia

Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve and Relapsed/Refractory Severe Aplastic Anaemia - the Diaamond-Ava-First and Diaamond-Ava-Next Bayesian Optimal Phase II Trials

Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria

Longitudinal Genomic Characterization Using Cell-Free DNA in Patients with Idiopathic Aplastic Anemia

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 Years with Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension Studies

Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Biomarker Analysis in Patients (pts) with Steroid-Refractory Acute Graft-Vs-Host Disease (aGVHD) Treated with Ruxolitinib (RUX) or Best Available Therapy (BAT) in the Randomized, Phase 3 REACH2 Study

Pegcetacoplan Is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement

The Global State of Hematopoietic Stem Cell Transplantation for Multiple Myeloma: An Analysis of the Worldwide Network of Blood and Marrow Transplantation (WBMT) Database and the Global Burden of Disease Study

One and Half Million Hematopoietic Stem Cell Transplants (HSCT). Dissemination, Trends and Potential to Improve Activity By Telemedicine from the Worldwide Network for Blood and Marrow Transplantation (WBMT)

An Update of Australasian Trends in Allogeneic Stem Cell Transplantation for Myelofibrosis in the Molecular Era

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Results from a Phase 3, Multicenter, Non-Inferiority Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Eculizumab

Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter Studies

A Phase 3 Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria Naive to Complement Inhibitors: Results of a Subgroup Analysis with Patients Stratified By Baseline Hemolysis Level, Transfusion History, and Demographics

Nivolumab for Relapsed or Residual Haematological Malignancies after Allogeneic Haematopoietic Stem Cell Transplantation (NIVALLO)

A Local Experience of Pregnancy Outcomes in Sickle Cell Disease and Red Cell Exchange

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American Society of Hematology

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