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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

Subcutaneous Alemtuzumab Has Activity in Treatment-Naïve Patients with Acquired Aplastic Anemia

Ana Rita Da Fonseca, MD, Anita Hill, MD PhD, Patricia Eiko Yamakawa, MD, Iara Baldim Rabelo, MD PhD, Andre Domingues Pereira, MD, Vinicius Campos de Molla, MD, Matheus Vescovi Gonçalves, MD PhD, Morag Griffin, MD, Talha Munir, MD, Peter Hillmen, MD PhD, Louise Arnold, MD, Kathryn Riley, MD, Antonio M. Risitano, MD PhD, Phillip Scheinberg, MD, Celso Arrais-Rodrigues, MD PhD

Blood (2019) 134 (Supplement_1): 2503.

https://doi.org/10.1182/blood-2019-130719

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

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Pierre-Edouard Debureaux, MD, Fabiana Cacace, Bruno G.P. Silva, MD, Federica Barone, MD, Rodrigo T Calado, MD, Flore Sicre de Fontbrune, Juliette Soret-Dulphy, MD, Patrizia Ricci, Michela Sica, PhD, Rosario Notaro, MD, Phillip Scheinberg, MD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Antonio M. Risitano, MD PhD, Régis Peffault de Latour, MD PhD, Camilla Frieri, MD

Blood (2019) 134 (Supplement_1): 3517.

https://doi.org/10.1182/blood-2019-125917

Published: 2019

Journal Articles

Outcome of Transformed Fanconi Anaemia Patients after Hematopoietic Stem Cell Transplantation: Analysis on Behalf of European Group for Blood and Marrow Transplantation

Blood (2018) 132 (Supplement 1): 646.

https://doi.org/10.1182/blood-2018-99-115399

Published: 2018

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

Eltrombopag for Prolonged Post-Transplant Hyporigenerative Cytopenias

Luana Marano, Serena Marotta, Fabiana Cacace, Camilla Frieri, MD, Luigia Simeone, Fabio Trastulli, MD, Selenia Vitiello, Flora Cardano, Fabrizio Pane, Antonio M. Risitano, MD PhD

Blood (2018) 132 (Supplement 1): 5711.

https://doi.org/10.1182/blood-2018-99-115381

Published: 2018

Journal Articles

Long-Term Follow-up of the Randomized Controlled Study in Patients with Newly Diagnosed Severe Aplastic Anemia Treated with ATG, Cyclosporine, with or without G-CSF: On Behalf of the SAA Working Party of the EBMT

André Tichelli, MD, Regis Peffault De Latour, Jakob R. Passweg, MD PhD, Cora Knol, Gérard Socié, MDPhD, Judith Marsh, Britta Hoechsmann, MD, Hubert Schrezenmeier, MD, Andrea Bacigalupo, MD, Sujith Samarasinghe, Alicia Rovo, MD, Austin G. Kulasekararaj, MBBS, MD MRCP, FRCPath, Dirk-Jan Eikema, Paul Bosman, Peter Bader, MD PhD, Antonio M. Risitano, MD PhD, Carlo Dufour, MD

Blood (2018) 132 (Supplement 1): 1307.

https://doi.org/10.1182/blood-2018-99-111811

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

Evaluation of Novel Endpoints As Treatment Outcome in Allogeneic Hematopoietic Stem Cell Transplantation: A Comparison of "Classical" and "Refined" Definition of Graft-Versus Host Disease Free and Relapse Free Survival for Registry-Based Studies

Simona Pagliuca, Antonio M Risitano, MD PhD, Flore Sicre De Fontbrune, MD, David Michonneau, MD PhD, Alban Villate, MD, Rudy Birsen, Camille Rouzaud, Aurelien Sutra del Galy, Nabergoj Mitja, MD, Regis Peffault De Latour, MD PhD, Marie Robin, MD PhD, Gérard Socié, MDPhD

Blood (2017) 130 (Supplement 1): 3311.

https://doi.org/10.1182/blood.V130.Suppl_1.3311.3311

Published: 2017

Journal Articles

Outcomes of Hematopoietic Stem Cell Transplantation after Chronic Graft Versus Host Disease and Evaluation of Revised Failure Free Survival in a Retrospective Analysis from Two Referral Centers.

Simona Pagliuca, Flore Sicre De Fontbrune, MD, Antonio M Risitano, MD PhD, David Michonneau, MD PhD, Alban Villate, MD, Rudy Birsen, Camille Rouzaud, Aurelien Sutra del Galy, Nabergoj Mitja, MD, Regis Peffault De Latour, MD PhD, Gérard Socié, MDPhD, Marie Robin, MD PhD

Blood (2017) 130 (Supplement 1): 4578.

https://doi.org/10.1182/blood.V130.Suppl_1.4578.4578

Published: 2017

Journal Articles

Prophylaxis with ATLG Significantly Improves Chronic Graft-Versus-Host Disease / Relapse-Free Survival and Quality of Life after a Myeloablative HLA Identical Sibling Transplant for Acute Leukemia Patients in Complete Remission. Long-Term Follow-up of the Randomized ATG Familystudy

Blood (2017) 130 (Supplement 1): 519.

https://doi.org/10.1182/blood.V130.Suppl_1.519.519

Published: 2017

Journal Articles

Subcutaneous Low Dose Alemtuzumab: Role As a Salvage Therapy in Immune -Mediated Marrow Failure Conditions

Swapna Thota, MD, Srinivasa Reddy Sanikommu, MD, Bhumika Patel, MD, Meena Sadaps, Cassandra M. Hirsch, BSc, Michael J. Clemente, M.S., Serena Marotta, MD, Antonio M Risitano, MD PhD, Mikkael A. Sekeres, MD MS, Jaroslaw P Maciejewski, MD PhD FACP

Blood (2016) 128 (22): 1505.

https://doi.org/10.1182/blood.V128.22.1505.1505

Published: 2016

Journal Articles

CD59 Deficiency Is Critical for C3 Binding on Red Blood Cells of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) during Anti-C5 Treatment (eculizumab)

MIchela Sica, PhD, Tommaso Rondelli, Patrizia Ricci, PhD, Maria De Angioletti, PhD, Antonio M Risitano, MD PhD, Rosario Notaro, MD

Blood (2016) 128 (22): 401.

https://doi.org/10.1182/blood.V128.22.401.401

Published: 2016

Journal Articles

Aplastic Anemia in the Context of Hemolytic Paroxysmal Nocturnal Hemoglobinuria: Feasibility of Antibody-Based Intensive Immunosuppression during Eculizumab Treatment

Simona Pagliuca, MD, Flore Sicre de Fontbrune, MD, Serena Marotta, MD, Anna Paola Iori, Marie Robin, MD PhD, Gérard Socié, MD PhD, Regis Peffault de Latour, MD PhD, Antonio M Risitano, MD PhD

Blood (2016) 128 (22): 5074.

https://doi.org/10.1182/blood.V128.22.5074.5074

Published: 2016

Journal Articles

Evaluation of Graft Versus Host Disease and Relapse Free Survival As Novel Endpoint in Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Joint Naples-Paris Study

Simona Pagliuca, MD, Antonio M Risitano, MD PhD, Sylvie Chevret, Flore Sicre de Fontbrune, MD, Alienor Xhaard, MD, Tereza Coman, MD PhD, David Michonneau, MD PhD, Serena Marotta, MD, Mathilde Ruggiu, Swann Bredin, Regis Peffault de Latour, MD PhD, Gérard Socié, MD PhD, Marie Robin, MD PhD

Blood (2016) 128 (22): 2285.

https://doi.org/10.1182/blood.V128.22.2285.2285

Published: 2016

Journal Articles

HCT Outcome in Patients with Fanconi Anemia Transplanted at Adult Age

Blood (2016) 128 (22): 4691.

https://doi.org/10.1182/blood.V128.22.4691.4691

Published: 2016

Journal Articles

The Natural History of Fanconi Anemia: A Report from the Italian Fanconi Anemia Registry (RIAF)

Serena Marotta, MD, Antonio M Risitano, MD PhD, Rita Calzone, PhD, Oriana Catapano, PhD, Adriana Zatterale, PhD

Blood (2015) 126 (23): 1208.

https://doi.org/10.1182/blood.V126.23.1208.1208

Published: 2015

NARROW

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)

Subcutaneous Alemtuzumab Has Activity in Treatment-Naïve Patients with Acquired Aplastic Anemia

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

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Outcome of Transformed Fanconi Anaemia Patients after Hematopoietic Stem Cell Transplantation: Analysis on Behalf of European Group for Blood and Marrow Transplantation

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

Eltrombopag for Prolonged Post-Transplant Hyporigenerative Cytopenias

Long-Term Follow-up of the Randomized Controlled Study in Patients with Newly Diagnosed Severe Aplastic Anemia Treated with ATG, Cyclosporine, with or without G-CSF: On Behalf of the SAA Working Party of the EBMT

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

Evaluation of Novel Endpoints As Treatment Outcome in Allogeneic Hematopoietic Stem Cell Transplantation: A Comparison of "Classical" and "Refined" Definition of Graft-Versus Host Disease Free and Relapse Free Survival for Registry-Based Studies

Outcomes of Hematopoietic Stem Cell Transplantation after Chronic Graft Versus Host Disease and Evaluation of Revised Failure Free Survival in a Retrospective Analysis from Two Referral Centers.

Prophylaxis with ATLG Significantly Improves Chronic Graft-Versus-Host Disease / Relapse-Free Survival and Quality of Life after a Myeloablative HLA Identical Sibling Transplant for Acute Leukemia Patients in Complete Remission. Long-Term Follow-up of the Randomized ATG Familystudy

Subcutaneous Low Dose Alemtuzumab: Role As a Salvage Therapy in Immune -Mediated Marrow Failure Conditions

CD59 Deficiency Is Critical for C3 Binding on Red Blood Cells of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) during Anti-C5 Treatment (eculizumab)

Aplastic Anemia in the Context of Hemolytic Paroxysmal Nocturnal Hemoglobinuria: Feasibility of Antibody-Based Intensive Immunosuppression during Eculizumab Treatment

Evaluation of Graft Versus Host Disease and Relapse Free Survival As Novel Endpoint in Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Joint Naples-Paris Study

HCT Outcome in Patients with Fanconi Anemia Transplanted at Adult Age

The Natural History of Fanconi Anemia: A Report from the Italian Fanconi Anemia Registry (RIAF)

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