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Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

DOI: https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Validation of Hemolytic Anemia Codes in the French Hospital Database

Julien Maquet, Hélène Derumeaux, MD PhD, Maryse Lapeyre-Mestre, MD PhD, Laurent Sailler, MD PhD, Guillaume Moulis, MD PhD

Blood (2019) 134 (Supplement_1): 3460.

DOI: https://doi.org/10.1182/blood-2019-124697

Published: 2019

Journal Articles

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Sule Unal, MD, Tekin Aksu, MD, Cagrı Coskun, MD, Fatma Gumruk, MD

Blood (2019) 134 (Supplement_1): 4794.

DOI: https://doi.org/10.1182/blood-2019-124272

Published: 2019

Journal Articles

Symptomatic Bilirubin Gallstones in Patients with PNH Treated with Eculizumab

David J. Araten, MD, H. Leon Pachter, Robert J Dring, Elliot Newman, Steven M Cohen

Blood (2019) 134 (Supplement_1): 4795.

DOI: https://doi.org/10.1182/blood-2019-124571

Published: 2019

Journal Articles

Optimizing Hydroxyurea Dosing in Sickle Cell Anemia: The Uganda MTD Study

Chandy C. John, MDMS, Robert Opoka, MMED, Teresa Latham, MA, Heather Hume, MDFRCPC, Maria Nakafeero, Phillip Kasirye, Christopher Ndugwa, Adam Lane, PhD, Russell E. Ware, MD PhD

Blood (2019) 134 (Supplement_1): 520.

DOI: https://doi.org/10.1182/blood-2019-125128

Published: 2019

Journal Articles

Glucocorticoids Induce the Maintenance and Expansion of an Immature CFU-E Erythroid Progenitor Population in Humans

Ryan Ashley, BS, MS, Hongxia Yan, MS, Nan Wang, PhD, John Hale, PhD, Brian M Dulmovits, MS, Julien Papoin, MS, Meagan Olive, Namrata Udeshi, PhD, Steven Carr, PhD, Adrianna Vlachos, MD, Jeffrey Michael M Lipton, MD PhD, Lydie M. Da Costa, MD PhD, Christopher D. Hillyer, MD, Sandrina Kinet, PhD, Naomi Taylor, MD PhD, Mohandas Narla, DSc, Anupama Narla, MD, Lionel Blanc, PhD

Blood (2019) 134 (Supplement_1): 943.

DOI: https://doi.org/10.1182/blood-2019-130710

Published: 2019

Journal Articles

Retrospective and Prospective Study of Childhood Autoimmune Hemolytic Anemia. a Preliminary Report from the Red Cell Working Group of the Paediatric Hemato-Oncology Italian Associations (AIEOP)

Blood (2019) 134 (Supplement_1): 947.

DOI: https://doi.org/10.1182/blood-2019-124735

Published: 2019

Journal Articles

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 949.

DOI: https://doi.org/10.1182/blood-2019-125352

Published: 2019

Journal Articles

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Charles T. Quinn, MD, Omar Niss, MD, Amanda Pfeiffer, Jennifer Korpik, Holly Bonar, Mary Reynaud, Russell E. Ware, MD PhD, Patrick T. McGann, MD

Blood (2019) 134 (Supplement_1): 892.

DOI: https://doi.org/10.1182/blood-2019-130414

Published: 2019

Journal Articles

Splenomegaly in Children with Sickle Cell Anemia Receiving Hydroxyurea in Sub-Saharan Africa

Leon Tshilolo, MD PhD, George A. Tomlinson, PhD BSc, MSc, Patrick T. McGann, MD, Teresa S. Latham, MA, Peter Olupot-Olupot, Brigida Santos, Banu Aygun, MD, Susan Stuber, MA, Adam Lane, PhD, Thomas N. Williams, Russell E. Ware, MD PhD

Blood (2019) 134 (Supplement_1): 993.

DOI: https://doi.org/10.1182/blood-2019-129937

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.

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

Published: 2019

Journal Articles

The Role of SOD2 in Maintaining Endothelial Cell Function in SCD

Atinuke Dosunmu-Ogunbi, Yingze Zhang, PhD, Seyed Mehdi Nouraie, PhD MD, Adam Straub, PhD

Blood (2019) 134 (Supplement_1): 3563.

DOI: https://doi.org/10.1182/blood-2019-131238

Published: 2019

Journal Articles

Decreased Activity and Stability of Pyruvate Kinase in Hereditary Hemolytic Anemia: A Potential Target for Therapy By AG-348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase

Minke A.E. Rab, MD, Brigitte A. van Oirschot, Stephanie van Straaten, MD PhD, Bart J. Biemond, MD PhD, Jennifer Bos, Penelope A. Kosinski, MS, Charles Kung, PhD, Eduard J. van Beers, MD PhD, Richard van Wijk, PhD

Blood (2019) 134 (Supplement_1): 3506.

DOI: https://doi.org/10.1182/blood-2019-129996

Published: 2019

Journal Articles

Normal Hemoglobin Concentrations in Obstructive Sleep Apnea and Associated Neocytolysis-Mediated Hemolysis and Inflammation Mediated Suppression of Expected Elevated Hemoglobin

Jihyun Song, PhD, Krishna M Sundar, MD, Monika Horvathova, MSc, PhD, Tsewang Tashi, MD, Carsten Lundby, PhD, Tomas Ganz, MD PhD, Josef T. Prchal, MD

Blood (2019) 134 (Supplement_1): 3507.

DOI: https://doi.org/10.1182/blood-2019-131269

Published: 2019

Journal Articles

Kidney Iron Deposition By R2* Is Associated with Hemolysis and Urinary Iron

Christopher C Denton, MD, Jon A Detterich, MD, Thomas Coates, MD, John C Wood, MD PhD

Blood (2019) 134 (Supplement_1): 3537.

DOI: https://doi.org/10.1182/blood-2019-131659

Published: 2019

Journal Articles

Rate of Sickle Hemoglobin Recovery in Sickle Cell Disease Patients Undergoing Red Blood Cell (RBC) Exchange Transfusion Is Associated with Age of Patients and Number of RBC Units Transfused

John Chinawaeze Aneke, MBBS, Aliraza Rajabali, Nafanta Fadiga, Stéphanie Forté, MD MSc, FRCPC, George A. Tomlinson, PhD BSc, MSc, Christine M. Cserti-Gazdewich, BSc, MD FRCPC, Christopher J. Patriquin, MDFRCPC,MSc, Richard Ward, MSc, MRCP, FRCPath, Jacob Pendergrast, MDFRCPC, David Barth, MD FRCPC, Kevin H. M. Kuo, MDMSc,FRCPC

Blood (2019) 134 (Supplement_1): 2170.

DOI: https://doi.org/10.1182/blood-2019-122072

Published: 2019

Journal Articles

Deficit of Circulating CD19 + CD24 hi CD38 hi Regulatory B Cells in Severe Aplastic Anemia

Yoshitaka Zaimoku, MD PhD, Bhavisha A Patel, MD, Sachiko Kajigaya, PhD, Xingmin Feng, PhD, Lemlem Alemu, BS, Diego Quinones Raffo, MSc, Emma M. Groarke, MD, Neal S. Young, MD

Blood (2019) 134 (Supplement_1): 1219.

DOI: https://doi.org/10.1182/blood-2019-127053

Published: 2019

Journal Articles

Concomitant Hydroxyurea and Voxelotor: Results from the HOPE Study

Russell E. Ware, MD PhD, Clark Brown, MD PhD, Mariane de Montalembert, MD PhD, Margaret Tonda, PharmD, Barbara Tong, PhD, Carolyn Hoppe, MD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Miguel R. Abboud, MD

Blood (2019) 134 (Supplement_1): 1003.

DOI: https://doi.org/10.1182/blood-2019-130767

Published: 2019

Journal Articles

Longitudinal Effect of Hydroxyurea Therapy on Left Ventricular Diastolic Function in Sickle Cell Anemia

Parul Rai, MD, Guolian Kang, PhD, Ronak Naik, MD, Jason Goldberg, MD, Xiwen Zhao, MSPH, Kenneth I. Ataga, MD, Vijaya Joshi, MD, Jane S. Hankins, MD MS

Blood (2019) 134 (Supplement_1): 1006.

DOI: https://doi.org/10.1182/blood-2019-130905

Published: 2019

Journal Articles

A Case of Mistaken Identity: TTP-like State Due to Heroin Overdose

Phani Borra, MD, Cory Edward Mellon, MD

Blood (2019) 134 (Supplement_1): 4905.

DOI: https://doi.org/10.1182/blood-2019-128537

Published: 2019

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NARROW

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Validation of Hemolytic Anemia Codes in the French Hospital Database

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Symptomatic Bilirubin Gallstones in Patients with PNH Treated with Eculizumab

Optimizing Hydroxyurea Dosing in Sickle Cell Anemia: The Uganda MTD Study

Glucocorticoids Induce the Maintenance and Expansion of an Immature CFU-E Erythroid Progenitor Population in Humans

Retrospective and Prospective Study of Childhood Autoimmune Hemolytic Anemia. a Preliminary Report from the Red Cell Working Group of the Paediatric Hemato-Oncology Italian Associations (AIEOP)

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Splenomegaly in Children with Sickle Cell Anemia Receiving Hydroxyurea in Sub-Saharan Africa

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

The Role of SOD2 in Maintaining Endothelial Cell Function in SCD

Decreased Activity and Stability of Pyruvate Kinase in Hereditary Hemolytic Anemia: A Potential Target for Therapy By AG-348 (Mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase

Normal Hemoglobin Concentrations in Obstructive Sleep Apnea and Associated Neocytolysis-Mediated Hemolysis and Inflammation Mediated Suppression of Expected Elevated Hemoglobin

Kidney Iron Deposition By R2* Is Associated with Hemolysis and Urinary Iron

Rate of Sickle Hemoglobin Recovery in Sickle Cell Disease Patients Undergoing Red Blood Cell (RBC) Exchange Transfusion Is Associated with Age of Patients and Number of RBC Units Transfused

Deficit of Circulating CD19 + CD24 hi CD38 hi Regulatory B Cells in Severe Aplastic Anemia

Concomitant Hydroxyurea and Voxelotor: Results from the HOPE Study

Longitudinal Effect of Hydroxyurea Therapy on Left Ventricular Diastolic Function in Sickle Cell Anemia

A Case of Mistaken Identity: TTP-like State Due to Heroin Overdose

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American Society of Hematology

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American Society of Hematology

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