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Journal Articles

Breakthrough Hemolysis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab: Results of a 52-Week Extension from Two Phase 3 Studies

Anita Hill, MD PhD, Caroline I. Piatek, Régis Peffault de Latour, MD PhD, Lily LL Wong, MD FRCPath, Richard A. Wells, MD DPhil, Robert A. Brodsky, MD, Jin Seok Kim, MD PhD, Junichi Nishimura, MD PhD, Philip Kuriakose, MD, Rodrigo Pavani, Peng Liu, Stephan Ortiz, RPh, PhD, Hubert Schrezenmeier, MD, Jong-Wook Lee, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath

Blood (2019) 134 (Supplement_1): 952.

https://doi.org/10.1182/blood-2019-128929

Published: 2019

Journal Articles

Rare Germline Mutations in Complement Regulatory Genes Make the Antiphospholipid Syndrome Catastrophic

Shruti Chaturvedi, MBBS, MS, Evan M Braunstein, MD PhD, Xuan Yuan, MD MSc, Hang Chen, Ravi Kumar Alluri, PhD, Michael B. Streiff, MD, Michelle Petri, MD MPH, Mark Crowther, MD MSc, FRCPC, Keith R McCrae, MD, Robert A. Brodsky, MD

Blood (2019) 134 (Supplement_1): 4.

https://doi.org/10.1182/blood-2019-129115

Published: 2019

Journal Articles

A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Austin Kulasekararaj, MBBS MDMRCP FRCPath, Antonio M. Risitano, MD PhD, Jaroslaw P. Maciejewski, MD PhD, Rosario Notaro, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA, Jong-Wook Lee, MD, Mingjun Huang, PhD, Michael Geffner, MD, Robert A. Brodsky, MD

Blood (2019) 134 (Supplement_1): 3514.

https://doi.org/10.1182/blood-2019-124748

Published: 2019

Journal Articles

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Antonio M. Risitano, MD PhD, Jong Wook Lee, MD PhD, Rosario Notaro, MD, Robert A. Brodsky, MD, Austin Kulasekararaj, MBBS MDMRCP FRCPath, Jaroslaw P. Maciejewski, MD PhD, Mingjun Huang, PhD, Michael Geffner, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA

Blood (2019) 134 (Supplement_1): 2226.

https://doi.org/10.1182/blood-2019-124837

Published: 2019

Journal Articles

One-Year Efficacy and Safety from a Phase 3 Trial of Ravulizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Prior Eculizumab Treatment

Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Anita Hill, MD PhD, Saskia Langemeijer, MD PhD, Richard A. Wells, MD DPhil, F. Ataulfo Gonzalez-Fernandez, Anna Gaya, Emilio Ojeda Gutierrez, Caroline I. Piatek, Lindsay D Mitchell, MBChB, MRCP(UK), FRCPath, Kensuke Usuki, Alberto Bosi, Robert A. Brodsky, MD, Masayo Ogawa, MD FAAP, Ji Yu, Stephan Ortiz, RPh, PhD, Alexander Röth, MD PhD, Jong-Wook Lee, MD PhD, Régis Peffault de Latour, MD PhD

Blood (2019) 134 (Supplement_1): 2231.

https://doi.org/10.1182/blood-2019-128746

Published: 2019

Journal Articles

The Path to Cure: Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Treatment-Naïve and Refractory Severe Aplastic Anemia (SAA)

Amy E. DeZern, MD, Marianna Zahurak, MS, Kenneth R. Cooke, MD, David Margolis, MD, Richard J. Jones, MD, Robert A. Brodsky, MD

Blood (2019) 134 (Supplement_1): 147.

https://doi.org/10.1182/blood-2019-121900

Published: 2019

Journal Articles

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease: BMT CTN Protocol 1507

Adetola A. Kassim, MDMS, Mark C. Walters, MD, Mary Eapen, MBBS, MS, Brianne Allison, MS, Adam M. Mendizabal, MS, Robert A. Brodsky, MD, Michael R. DeBaun, MDMPH

Blood (2019) 134 (Supplement_1): 802.

https://doi.org/10.1182/blood-2019-126321

Published: 2019

Journal Articles

PIG-a Gene Expression Deficiency Association with Reduced DNA Damage Checkpoint Response and Activation

Jeffrey J Pu, MD PhD, Shasha Lu, MD PhD, Matthew Nemesure, Emmanuel Kwame Teye, PhD, Emily Schleicher, George-Lucian Moldovan, PhD, Robert A. Brodsky, MD, Fangyuan Chen, MD PhD

Blood (2018) 132 (Supplement 1): 3875.

https://doi.org/10.1182/blood-2018-99-119177

Published: 2018

Journal Articles

An Alternative Pathway Specific Flow Cytometric Assay to Detect Complement Activation in Atypical Hemolytic Uremic Syndrome (aHUS)

Jia Yu, Xuan Yuan, MD MSc, Kang Zhou, Daniel White, Samuel A. Merrill, MD PhD, Robert A. Brodsky, MD

Blood (2018) 132 (Supplement 1): 3748.

https://doi.org/10.1182/blood-2018-99-119445

Published: 2018

Journal Articles

Chronic Kidney Disease, Hypertension and Cardiovascular Sequelae during Long Term Follow up of Adults with Atypical Hemolytic Uremic Syndrome

Shruti Chaturvedi, MBBS, MS, Alison R. Moliterno, MD, Samuel A. Merrill, MD PhD, Evan M Braunstein, MD PhD, Xuan Yuan, MD MSc, Christopher John Sperati, MD, Gebran Khneizer, Robert A. Brodsky, MD

Blood (2018) 132 (Supplement 1): 3754.

https://doi.org/10.1182/blood-2018-99-119999

Published: 2018

Journal Articles

A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria

Robert A. Brodsky, MD, Regis Peffault De Latour, Scott T. Rottinghaus, MD, Alexander Röth, MD, Antonio M. Risitano, MD PhD, Ilene C. Weitz, MD, Peter Hillmen, MB ChB, PhD FRCP, FRCPath, Jaroslaw P. Maciejewski, Jeffrey Szer, MBBS, Jong-Wook Lee, MD PhD, Austin G. Kulasekararaj, MBBS, MD MRCP, FRCPath, Lori Volles, MD, Andrew I. Damokosh, PhD, Stephan Ortiz, RPh, PhD, Lori Shafner, PhD, Anita Hill, MD PhS, Hubert Schrezenmeier, MD

Blood (2018) 132 (Supplement 1): 2330.

https://doi.org/10.1182/blood-2018-99-110874

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

Interim Analysis of Safety Outcomes during Treatment with Eculizumab: Results from the International Paroxysmal Nocturnal Hemoglobinuria Registry

Anita Hill, MBChB (Hons), PhD, Alexander Roeth, MD, Gérard Socié, MDPhD, Marie-Pierre Caby-Tosi, PharmD, PhD, Amanda Wilson, MPH, PhD, Philippe Gustovic, MD, Camille L. Bedrosian, MD, Robert A. Brodsky, MD

Blood (2017) 130 (Supplement 1): 3486.

https://doi.org/10.1182/blood.V130.Suppl_1.3486.3486

Published: 2017

Journal Articles

Efficacy of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and High Disease Activity with or without History of Aplastic Anemia in the International PNH Registry

Jong-Wook Lee, MD, Regis Peffault De Latour, MD PhD, Robert A. Brodsky, MD, Jun Ho Jang, MD PhD, Anita Hill, MBChB (Hons), PhD, Alexander Roeth, MD, Hubert Schrezenmeier, MD, Amanda Wilson, MPH, PhD, Jing L. Marantz, MD PhD, Jaroslaw P. Maciejewski, PhD MD FACP

Blood (2017) 130 (Supplement 1): 3487.

https://doi.org/10.1182/blood.V130.Suppl_1.3487.3487

Published: 2017

Journal Articles

In Vitro Combination Studies of ACH-4471 with Eculizumab to Assess a Potential "Switch" Treatment Approach for Paroxysmal Nocturnal Hemoglobinuria

Dharaben Patel, MS, Jane A Thanassi, BS, Guangwei Yang, PhD, Xuan Yuan, MD MSc, Robert A. Brodsky, MD, Mingjun Huang, PhD, Steven D Podos, PhD

Blood (2017) 130 (Supplement 1): 2198.

https://doi.org/10.1182/blood.V130.Suppl_1.2198.2198

Published: 2017

Journal Articles

Adult Hemophagocytic Lymphohistiocytosis-a Quality Improvement Initiative

Samuel A. Merrill, MD PhD, Rakhi P. Naik, MD, Evan M Braunstein, MDPhD, Michael B. Streiff, Satish Shanbhag, MD, Sophie Lanzkron, MD, Alison R. Moliterno, MD, Robert A. Brodsky, MD

Blood (2017) 130 (Supplement 1): 677.

https://doi.org/10.1182/blood.V130.Suppl_1.677.221

Published: 2017

Journal Articles

Haploidentical Bone Marrow Transplant with Post-Transplant Cytoxan Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Multicenter Learning Collaborative

Nathalie Dhedin, MD, Josu de la Fuente, Francoise Bernaudin, MD, Mathieu Kuentz, MD, Leena Karnik, MD FRCPath, MBBS,, Tatsuki Koyama, PhD, Robert A. Brodsky, MD, Michael DeBaun, MD, Adetola A. Kassim, MD MS

Blood (2016) 128 (22): 1233.

https://doi.org/10.1182/blood.V128.22.1233.1233

Published: 2016

Journal Articles

β-2-Glycoprotein Antibodies Activate the Alternative Pathway of Complement in Antiphospholipid Antibody Syndrome

Andrea C. Baines, MD PhD, Xuan Yuan, MD MSc, Eleni Gavriilaki, MD PhD, Alexander J. Ambinder, MD MPH, Alka Jain, PhD, Neal S. Fedarko, PhD, C. John Sperati, MD MHS, Alison R. Moliterno, MD, Rakhi P. Naik, MD MHS, Michael Streiff, MD, Wei Fu, MS, Michelle Petri, MD MPH, Robert A. Brodsky, MD

Blood (2016) 128 (22): 3818.

https://doi.org/10.1182/blood.V128.22.3818.3818

Published: 2016

Journal Articles

Second Blood or Marrow Transplant (BMT) for Relapse: Mismatch Haplotype Switch May Improve Outcome

Philip H Imus, MD, August R Dietrich, MD, Amanda Blackford, ScM, Robert A. Brodsky, MD, Heather J. Symons, Kenneth Cooke, MD, Richard J Jones, MD, Javier Bolaños-Meade, MD

Blood (2016) 128 (22): 2252.

https://doi.org/10.1182/blood.V128.22.2252.2252

Published: 2016

Journal Articles

Evaluation of Bacteria-Mediated Potential "Bystander" Hemolysis of PNH RED CELLS I n Vitro : NO Evidence of Significant Complement Classical or Lectin Pathway-Mediated Hemolysis Induced by Microorganisms

Xuan Yuan, MD MSc, Guangwei Yang, PhD, Jane A Thanassi, Manuel D Galvan, PhD, Steven D Podos, PhD, Mingjun Huang, PhD, Robert A. Brodsky, MD

Blood (2016) 128 (22): 2431.

https://doi.org/10.1182/blood.V128.22.2431.2431

Published: 2016

NARROW

Breakthrough Hemolysis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab: Results of a 52-Week Extension from Two Phase 3 Studies

Rare Germline Mutations in Complement Regulatory Genes Make the Antiphospholipid Syndrome Catastrophic

A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

One-Year Efficacy and Safety from a Phase 3 Trial of Ravulizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Prior Eculizumab Treatment

The Path to Cure: Using Haploidentical (haplo) Donors and High-Dose Post-Transplant Cyclophosphamide (PTCy) for Treatment-Naïve and Refractory Severe Aplastic Anemia (SAA)

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease: BMT CTN Protocol 1507

PIG-a Gene Expression Deficiency Association with Reduced DNA Damage Checkpoint Response and Activation

An Alternative Pathway Specific Flow Cytometric Assay to Detect Complement Activation in Atypical Hemolytic Uremic Syndrome (aHUS)

Chronic Kidney Disease, Hypertension and Cardiovascular Sequelae during Long Term Follow up of Adults with Atypical Hemolytic Uremic Syndrome

A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria

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

Interim Analysis of Safety Outcomes during Treatment with Eculizumab: Results from the International Paroxysmal Nocturnal Hemoglobinuria Registry

Efficacy of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and High Disease Activity with or without History of Aplastic Anemia in the International PNH Registry

In Vitro Combination Studies of ACH-4471 with Eculizumab to Assess a Potential "Switch" Treatment Approach for Paroxysmal Nocturnal Hemoglobinuria

Adult Hemophagocytic Lymphohistiocytosis-a Quality Improvement Initiative

Haploidentical Bone Marrow Transplant with Post-Transplant Cytoxan Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Multicenter Learning Collaborative

β-2-Glycoprotein Antibodies Activate the Alternative Pathway of Complement in Antiphospholipid Antibody Syndrome

Second Blood or Marrow Transplant (BMT) for Relapse: Mismatch Haplotype Switch May Improve Outcome

Evaluation of Bacteria-Mediated Potential "Bystander" Hemolysis of PNH RED CELLS I n Vitro : NO Evidence of Significant Complement Classical or Lectin Pathway-Mediated Hemolysis Induced by Microorganisms

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American Society of Hematology

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American Society of Hematology

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