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Journal Articles

Patient Characteristics, Treatment Outcomes and Healthcare Resource Utilization across Europe in Multiple Myeloma Patients Ineligible for Stem Cell Transplantation Who Received Lenalidomide- or Bortezomib-Based Regimens

Elena Zamagni, MD, Sujith Dhanasiri, Adam Moore, Arun Ghale, Murielle Roussel, MD

Blood (2019) 134 (Supplement_1): 4772.

https://doi.org/10.1182/blood-2019-123629

Published: 2019

Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Blood (2019) 134 (Supplement_1): 4627.

https://doi.org/10.1182/blood-2019-129686

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Comparison of Lost Productivity Due to Eculizumab and Ravulizumab Treatments for Paroxysmal Nocturnal Hemoglobinuria in France, Germany, Italy, Russia, Spain, the United Kingdom, and the United States

Adrian R Levy, Laura Dysart, Yogesh Patel, Andrew Briggs, DPhil, John Schneider, Karl-Johan Myren, MSc, Ioannis Tomazos, PhD MBA

Blood (2019) 134 (Supplement_1): 4803.

https://doi.org/10.1182/blood-2019-127443

Published: 2019

Journal Articles

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Ingrid Grazielle Sousa, BSc, MSc, Priscila Keiko Matsumoto Martin, PhD, Dulcinéia Martins de Albuquerque, BSc, PhD, Carolina Lanaro, BSc, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Fernando Ferreira Costa, MD PhD

Blood (2019) 134 (Supplement_1): 968.

https://doi.org/10.1182/blood-2019-130920

Published: 2019

Journal Articles

Outcomes of Pregnancies in Patients with Sickle-Cell Disease : Update from European Non-Interventional, Multicentric, Prospective Escort-HU Study

Frédéric Galactéros, MD PhD, Giovanna Cannas, MD, Pablo Bartolucci, MD PhD, Ersi Voskaridou, Laure Joseph, MD, Emmanuelle Bernit, MD, Justine Gellen-Dautremer, MD, Corinne Charneau, MD, Stephanie Ngo, MD, Anoosha Habibi

Blood (2019) 134 (Supplement_1): 891.

https://doi.org/10.1182/blood-2019-125812

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

Analysis of Mutational Landscape in Systemic Anaplastic Large Cell Lymphoma Identifies Novel Prognostic Markers

Blood (2019) 134 (Supplement_1): 1490.

https://doi.org/10.1182/blood-2019-126501

Published: 2019

Journal Articles

Azacitidine for Pre-Emptive Treatment of Measurable-Residual Disease in MDS/AML Patients at High Risk of Hematological Relapse: Results of the Second Cohort of the RELAZA2 Trial

Blood (2019) 134 (Supplement_1): 644.

https://doi.org/10.1182/blood-2019-129926

Published: 2019

Journal Articles

Factor VIII Levels in Blood Group O and Non-O in Patients with Non-Severe Haemophilia a

Judit Rejtoe, MD, Oliver Königsbrügge, MD, Ella Grilz, MD, Stefanie Hofer, Johanna Gebhart, MD, Gerhard Schuster, MD, Clemens Feistritzer, MD, Peter Quehenberger, MD, Cihan Ay, MD, Ingrid Pabinger, MD

Blood (2019) 134 (Supplement_1): 1135.

https://doi.org/10.1182/blood-2019-123187

Published: 2019

Journal Articles

Sickle-Cell Disease Patients' Attitudes Towards Their Treatment with Hydroxycarbamide

Frédéric Galactéros, MD PhD, Ersi Voskaridou, Anoosha Habibi, Giovanna Cannas, MD, Laure Joseph, MD, Stephanie Ngo, MD, Nathalie Lemonne, Emmanuelle Bernit, MD, Justine Gellen-Dautremer, MD, Gylna Loko, MD

Blood (2019) 134 (Supplement_1): 1013.

https://doi.org/10.1182/blood-2019-125673

Published: 2019

Journal Articles

Physicians' and Patients' Perspectives on Treatments in ITP - a Multi-Country Perspective: Results from the ITP World Impact Survey (I-WISh)

James B. Bussel, MD, Waleed Ghanima, MD PhD, Yoshiaki Tomiyama, MD, Donald M. Arnold, MD, Drew Provan, MD, Ming Hou, MD PhD, Cristina Santoro, MD PhD, Serge Laborde, Alexandra Kruse, Caroline Kruse, Mervyn Morgan, Barbara Lovrencic, John Waller, PhD, Jens Haenig, Nichola Cooper, MD MRCP

Blood (2019) 134 (Supplement_1): 1097.

https://doi.org/10.1182/blood-2019-130591

Published: 2019

Journal Articles

Impact of Sickle Cell Disease Symptoms on Patients' Daily Lives: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

Ifeyinwa (ify) Osunkwo, MD MPH, Biree Andemariam, MD, Baba PD Inusa, MD, Fuad El-Rassi, Beverly Francis-Gibson, Alecia C. Nero, MD, Caterina P. Minniti, MD, Cassandra Trimnell, BA, Miguel R. Abboud, MD, Jean-Benoît Arlet, Raffaella Colombatti, Mariane de Montalembert, MD PhD, Suman Jain, Wasil Jastaniah, MBBS, FRCPC, Erfan Nur, MD PhD, Laurie DeBonnett, MD, John James, MSc

Blood (2019) 134 (Supplement_1): 2297.

https://doi.org/10.1182/blood-2019-124762

Published: 2019

Journal Articles

Safety, Efficacy, and PK of the BCL2 Inhibitor Venetoclax in Combination with Chemotherapy in Pediatric and Young Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia: Phase 1 Study

Seth E Karol, MD, Todd M Cooper, DO, Henrique Bittencourt, Lia Gore, MD, Maureen M O'Brien, MD, Christopher Fraser, MD, Marion Gambart, MD, Gunnar Cario, MD PhD, Christian Michel Zwaan, MD PhD, Jean-Pierre Bourquin, MD PhD, Mignon L. Loh, MD, Hubert Caron, MD PhD, Betty Prine, Ahmed Hamed Salem, PhD FCP, Kristina Unnebrink, Bo Tong, PhD, Tammy Palenski, PhD, Andrew E. Place, MD PhD

Blood (2019) 134 (Supplement_1): 2649.

https://doi.org/10.1182/blood-2019-129805

Published: 2019

Journal Articles

CXCR4-Targeted Positron Emission Tomography Imaging of Central Nervous System B-Cell Lymphoma

Peter Herhaus, MD, Jana Lipkova, Felicitas Lammer, MD, Julia Slotta-Huspenina, MD, Benedikt Wiestler, MD, Tibor Vag, MD, Stefan Habringer, MD, Igor Yakushev, MD, Constantin Lapa, MD, Tobias Pukrop, MD, Dirk Hellwig, MD, Andreas Buck, MD, Martina Deckert, MD, Hans-Jürgen Wester, PhD, Florian Bassermann, MD PhD, Wolfgang Weber, MD, Markus Schwaiger, MD, Björn Menze, PhD, Ulrich Keller, MD

Blood (2019) 134 (Supplement_1): 2900.

https://doi.org/10.1182/blood-2019-126024

Published: 2019

Journal Articles

A Prospective, Open-Label, Multicenter, Phase 2 Trial to Evaluate the Safety and Efficacy of the Combination of Tirabrutinib (ONO/GS-4059) and Idelalisib with and without Obinutuzumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL)

Nadine Kutsch, MD, Christian Pallasch, Thomas Decker, Holger Hebart, Kai Uwe Chow, Ullrich Graeven, Jens Kisro, Alexander Kroeber, Eugen Tausch, Clemens-Martin Wendtner, Michael J. Eckart, Stephan Stilgenbauer, Xi Huang, Juliane M. Jürgensmeier, Pankaj Bhargava, Michael Hallek, Barbara F. Eichhorst, MD

Blood (2019) 134 (Supplement_1): 3047.

https://doi.org/10.1182/blood-2019-131025

Published: 2019

Journal Articles

Impact of the Association between Tgl and EZH2 Mutations (Y641N/Y641F/Y641H/Y641S) on Disease Free Survival of Patients with Diffuse Large B-Cell Lymphoma

Myrna Candelaria, MD PharmD, Adriana Palacios Campos, MD, Olga Gutierrez Hernandez, QFB, Alejandro Aviles, MD, Uvi Cancino-Ramos, MD, Lucia Taja Chayeb, PhD, Osvaldo García-Perez, MD, David Cantú de León

Blood (2019) 134 (Supplement_1): 5222.

https://doi.org/10.1182/blood-2019-125906

Published: 2019

Journal Articles

Whole Body Diffusion Weighted MRI (WB DWI) for the Management of Multiple Myeloma: High Concordance between MRI Diffuse Pattern and BONE Marrow Plasma CELL Infiltration RATE

Silvia Mangiacavalli, Zacchino Michela, MD, Virginia Valeria Ferretti, Claudio Salvatore Cartia, MD, Giovanni Savietto, MD, Pietro Benvenuti, MD, Marzia Varettoni, Sara Rattotti, Brociner Marco, Gabriele Merati, MD, Luca Arcaini

Blood (2019) 134 (Supplement_1): 5495.

https://doi.org/10.1182/blood-2019-126422

Published: 2019

Journal Articles

An Increased Bone Mineral Density As an Adverse Prognostic Factor in Patients with Systemic Mastocytosis

Mohamad Jawhar, Juliana Schwaab, Nicole Naumann, Georgia Metzgeroth, Alice Fabarius, PhD, Stefan O Schoenberg, Wolf-Karsten Hofmann, MD PhD, Philipp Riffel, Andreas Reiter

Blood (2019) 134 (Supplement_1): 4185.

https://doi.org/10.1182/blood-2019-129798

Published: 2019

Journal Articles

A Prospective, Open-Label, Multicenter, Phase 2 Trial to Evaluate the Safety and Efficacy of the Combination of Tirabrutinib (ONO/GS-4059) and Entospletinib with and without Obinutuzumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL)

Nadine Kutsch, MD, Christian Pallasch, Eugen Tausch, Volkmar Boehme, Matthias Ritgen, Ruediger Liersch, Alexander Wacker, Georg Jacobs, Ralf Ulrich Trappe, Peter Dreger, Stephan Stilgenbauer, Danjie Zhang, Juliane M. Jürgensmeier, Pankaj Bhargava, Michael Hallek, Barbara F. Eichhorst, MD

Blood (2019) 134 (Supplement_1): 4297.

https://doi.org/10.1182/blood-2019-130912

Published: 2019

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Patient Characteristics, Treatment Outcomes and Healthcare Resource Utilization across Europe in Multiple Myeloma Patients Ineligible for Stem Cell Transplantation Who Received Lenalidomide- or Bortezomib-Based Regimens

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Comparison of Lost Productivity Due to Eculizumab and Ravulizumab Treatments for Paroxysmal Nocturnal Hemoglobinuria in France, Germany, Italy, Russia, Spain, the United Kingdom, and the United States

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Outcomes of Pregnancies in Patients with Sickle-Cell Disease : Update from European Non-Interventional, Multicentric, Prospective Escort-HU Study

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Analysis of Mutational Landscape in Systemic Anaplastic Large Cell Lymphoma Identifies Novel Prognostic Markers

Azacitidine for Pre-Emptive Treatment of Measurable-Residual Disease in MDS/AML Patients at High Risk of Hematological Relapse: Results of the Second Cohort of the RELAZA2 Trial

Factor VIII Levels in Blood Group O and Non-O in Patients with Non-Severe Haemophilia a

Sickle-Cell Disease Patients' Attitudes Towards Their Treatment with Hydroxycarbamide

Physicians' and Patients' Perspectives on Treatments in ITP - a Multi-Country Perspective: Results from the ITP World Impact Survey (I-WISh)

Impact of Sickle Cell Disease Symptoms on Patients' Daily Lives: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

Safety, Efficacy, and PK of the BCL2 Inhibitor Venetoclax in Combination with Chemotherapy in Pediatric and Young Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia: Phase 1 Study

CXCR4-Targeted Positron Emission Tomography Imaging of Central Nervous System B-Cell Lymphoma

A Prospective, Open-Label, Multicenter, Phase 2 Trial to Evaluate the Safety and Efficacy of the Combination of Tirabrutinib (ONO/GS-4059) and Idelalisib with and without Obinutuzumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL)

Impact of the Association between Tgl and EZH2 Mutations (Y641N/Y641F/Y641H/Y641S) on Disease Free Survival of Patients with Diffuse Large B-Cell Lymphoma

Whole Body Diffusion Weighted MRI (WB DWI) for the Management of Multiple Myeloma: High Concordance between MRI Diffuse Pattern and BONE Marrow Plasma CELL Infiltration RATE

An Increased Bone Mineral Density As an Adverse Prognostic Factor in Patients with Systemic Mastocytosis

A Prospective, Open-Label, Multicenter, Phase 2 Trial to Evaluate the Safety and Efficacy of the Combination of Tirabrutinib (ONO/GS-4059) and Entospletinib with and without Obinutuzumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL)

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