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Journal Articles

Congenital dyserythropoietic anemias

Achille Iolascon, Immacolata Andolfo, Roberta Russo

Blood (2020) 136 (11): 1274–1283.

https://doi.org/10.1182/blood.2019000948

Published: 2020

Journal Articles

Diamond-Blackfan anemia

Lydie Da Costa, Thierry Leblanc, Narla Mohandas

Blood (2020) 136 (11): 1262–1273.

https://doi.org/10.1182/blood.2019000947

Published: 2020

Journal Articles

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Jing Zeng, MD, Yuxuan Wu, PhD, Chunyan Ren, PhD, Jasmine Bonanno, Anne H. Shen, Devlin Shea, Jason M Gehrke, PhD, John P Manis, MD, J. Keith Gehrke, Daniel E. Bauer, MD PhD

Blood (2019) 134 (Supplement_1): 612.

https://doi.org/10.1182/blood-2019-129173

Published: 2019

Journal Articles

Elucidating an Erythroblast Survival Mechanism: Differential Contributions of Exosome Complex Catalytic Subunits

Charu Mehta, MS, Isabela J Fraga de Andrade, Bachelors in Biological Science, Nobuyuki Tanimura, PhD, Gary M Wilson, Erik A. Ranheim, MD PhD, Joshua J Coon, PhD, Emery H Bresnick, PhD

Blood (2019) 134 (Supplement_1): 155.

https://doi.org/10.1182/blood-2019-126917

Published: 2019

Journal Articles

Hematologic Improvement-Neutrophil and -Platelet in the MEDALIST Trial: Multilineage Data from a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept to Treat Anemia in Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions

Guillermo Garcia-Manero, MD, Ghulam J. Mufti, Pierre Fenaux, MD PhD, Rena Buckstein, MD FRCPC, Valeria Santini, MD, María Díez-Campelo, MD PhD, Carlo Finelli, Mario Cazzola, MD, Osman Ilhan, Mikkael A. Sekeres, MD MS, Alan F. List, MD, Abderrahmane Laadem, Rodrigo Ito, Jennie Zhang, Anita Rampersad, Daniel Sinsimer, Peter G. Linde, Uwe Platzbecker, MD, Rami Komrokji

Blood (2019) 134 (Supplement_1): 4243.

https://doi.org/10.1182/blood-2019-123048

Published: 2019

Journal Articles

The Clinical, Economic, and Humanistic Burden of Patients with β-Thalassemia: A US-Centric Review of the Literature

Elizabeth S. Mearns, Chuka Udeze, Ronda Copher, Sujit Sheth, MD

Blood (2019) 134 (Supplement_1): 5855.

https://doi.org/10.1182/blood-2019-125676

Published: 2019

Journal Articles

An Open-Label, Phase 2 Study of KRT-232, a First-in-Class, Oral Small Molecule Inhibitor of MDM2, for the Treatment of Patients with Myelofibrosis (MF) Who Have Previously Received Treatment with a JAK Inhibitor

Regina Garcia-Delgado, MD, Donal P. McLornan, MB, BCh (Hons), MRCP, PhD FRCPath, László Rejtő, Eric Jourdan, MD, Haifa Kathrin Al-Ali, MD, Andrzej Pluta, MD, Marek Hus, MD PhD, Joanne Ewing, PhDBMBS BSc FRPATH, Irum Khan, MD, Holger Hebart, Philipp J. Jost, MD, Sebastian Grosicki, MD PhD, Wayne M. Rothbaum, Jesse McGreivy, Srdan Verstovsek, MD PhD

Blood (2019) 134 (Supplement_1): 2945.

https://doi.org/10.1182/blood-2019-123836

Published: 2019

Journal Articles

Cell-Intrinsic Inflammation Drives Progression from Myelodysplastic Syndromes to Leukemia

Laura Barreyro, PhD, Avery Sampson, BSc, Kathleen Hueneman, Kwangmin Choi, PhD, Susanne Christie, BSc, Dehua Wang, MD, Gang Huang, PhD, Daniel T. Starczynowski, PhD

Blood (2019) 134 (Supplement_1): 2983.

https://doi.org/10.1182/blood-2019-127307

Published: 2019

Journal Articles

Mutation of Murine Rpl5 reveals a New Model for Diamond Blackfan Anemia Characterized By Defective Erythropoiesis

Philippe Lemay, PhD, Morgan A Jones, MD PhD BS, MS, Fatma Mohamed, Marie-Claude Guyot, Alexander Ludlow, Qing Li, MD, James Douglas Engel, PhD, Renny Franceschi, PhD, Thomas L. Rothstein, MD PhD, Vesa Kaartinen, PhD, Zoha Kibar, PhD, Sharon A Singh, MD

Blood (2019) 134 (Supplement_1): 2495.

https://doi.org/10.1182/blood-2019-131526

Published: 2019

Journal Articles

Development of a Severity Score System for Thalassemia Syndromes

Blood (2019) 134 (Supplement_1): 2225.

https://doi.org/10.1182/blood-2019-124675

Published: 2019

Journal Articles

Manifestations of Reduced Kidney Function Occur at a Higher Estimated Glomerular Filtration Rate in Sickle Cell Anemia

Santosh L. Saraf, MD, Vimal K. Derebail, MD, Xu Zhang, PhD, Mark T Gladwin, MD, Victor R Gordeuk, MD, Jane A. Little, MD

Blood (2019) 134 (Supplement_1): 2268.

https://doi.org/10.1182/blood-2019-124737

Published: 2019

Journal Articles

A Novel Sandwich ELISA to Quantify Erythroferrone in Mouse Serum

Patrick Gutschow, MS, Huiling Han, MD, Gordana Olbina, PhD, Keith Westerman, BS, Eileen Westerman, BS, Marc Ruiz Martinez, PhD, Yelena Ginzburg, MD, Elizabeta Nemeth, PhD, Tomas Ganz, MD PhD, Vaughn Ostland, PhD

Blood (2019) 134 (Supplement_1): 2237.

https://doi.org/10.1182/blood-2019-130947

Published: 2019

Journal Articles

A Longitudinal Study in Elderly Patients with Non-Transfusion Dependent Thalassemia (NTDT) Emphasizes the Size of Splenomegaly Predicts Transfusion Requirement Later in Life

Supachai Ekwattanakit, PhDMD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2250.

https://doi.org/10.1182/blood-2019-122296

Published: 2019

Journal Articles

Functional Overlap between the SEC23 Paralogs Suggests a Novel Treatment Paradigm for Congenital Dyserythropoietic Anemia Type II

Richard King, MD, Ann Friedman, BS, Zesen Lin, BS, Rami Khoriaty, MD

Blood (2019) 134 (Supplement_1): 2221.

https://doi.org/10.1182/blood-2019-129669

Published: 2019

Journal Articles

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Katrin Uhlig, MD MS, Megan Lynch, MSN, NP, Li Liu, PhD, Elliott P. Vichinsky, MD

Blood (2019) 134 (Supplement_1): 2249.

https://doi.org/10.1182/blood-2019-123113

Published: 2019

Journal Articles

Erythroferrone Regulates Bone Remodeling in β-Thalassemia

Melanie Castro-Mollo, MD, Marc Ruiz Martinez, PhD, Maria Feola, PhD, Anisa Azatovna Gumerova, MD, Carla Casu, PhD, Robert E Fleming, MD, Stefano Rivella, PhD, Tony Yuen, PhD, Mone Zaidi, MD PhD, Yelena Ginzburg, MD

Blood (2019) 134 (Supplement_1): 2.

https://doi.org/10.1182/blood-2019-125822

Published: 2019

Journal Articles

Elucidating the Role of IL6 in Stress Erythropoiesis and in the Development of Anemia Under Inflammatory Conditions

Sayantani Sinha, PhD, Ritama Gupta, PhD, Jianbing Zhang, PhD, Amaliris Guerra, PhD, Ping La, Sara Gardenghi, PhD, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 940.

https://doi.org/10.1182/blood-2019-128826

Published: 2019

Journal Articles

Does Gene Therapy in Beta Thalassemia Normalize Novel Markers of Ineffective Erythropoiesis and Iron Homeostasis?

Alexis A. Thompson, MD MPH, Tomas Ganz, MD PhD, Mary Therese Forsyth, Elizabeta Nemeth, PhD, Sherif M. Badawy, MD

Blood (2019) 134 (Supplement_1): 816.

https://doi.org/10.1182/blood-2019-129658

Published: 2019

Journal Articles

Hematopoietic Stem Cell Function in β-Thalassemia Is Impaired and Is Rescued By Targeting the Bone Marrow Niche

Annamaria Aprile, PhD, Alessandro Gulino, PhD, Isabella Villa, PhD, Stefano Beretta, PhD, Ivan Merelli, PhD, Alessandro Rubinacci, MD, Maurilio Ponzoni, MD, Sarah Marktel, MD, Claudio Tripodo, MD, Maria Rosa Lidonnici, PhD, Giuliana Ferrari, PhD

Blood (2019) 134 (Supplement_1): 967.

https://doi.org/10.1182/blood-2019-126829

Published: 2019

Journal Articles

Experience with Low Dose Thalidomide in Transfusion Dependent Beta Thalassaemia in a Resource Limited Setting

Pallavi Mehta, MD DM, Neha Yadav, BDS , MBA, Priyanka Soni, MD FNB, Shinto Francis Thekuddan, MBBS,MDDM, Reema Singh, MSc, Vishvdeep Khushoo, MBBS,MDDM, Sumeet Prakash Mirgh, MBBS,MDDM, Narendra Agrawal, MD DM, Rayaz Ahmed, MDDM, Jyotsna Kapoor, MSc, Dinesh Bhurani, MD DM, FRCPA

Blood (2019) 134 (Supplement_1): 963.

https://doi.org/10.1182/blood-2019-131682

Published: 2019

NARROW

Congenital dyserythropoietic anemias

Diamond-Blackfan anemia

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Elucidating an Erythroblast Survival Mechanism: Differential Contributions of Exosome Complex Catalytic Subunits

The Clinical, Economic, and Humanistic Burden of Patients with β-Thalassemia: A US-Centric Review of the Literature

An Open-Label, Phase 2 Study of KRT-232, a First-in-Class, Oral Small Molecule Inhibitor of MDM2, for the Treatment of Patients with Myelofibrosis (MF) Who Have Previously Received Treatment with a JAK Inhibitor

Cell-Intrinsic Inflammation Drives Progression from Myelodysplastic Syndromes to Leukemia

Mutation of Murine Rpl5 reveals a New Model for Diamond Blackfan Anemia Characterized By Defective Erythropoiesis

Development of a Severity Score System for Thalassemia Syndromes

Manifestations of Reduced Kidney Function Occur at a Higher Estimated Glomerular Filtration Rate in Sickle Cell Anemia

A Novel Sandwich ELISA to Quantify Erythroferrone in Mouse Serum

A Longitudinal Study in Elderly Patients with Non-Transfusion Dependent Thalassemia (NTDT) Emphasizes the Size of Splenomegaly Predicts Transfusion Requirement Later in Life

Functional Overlap between the SEC23 Paralogs Suggests a Novel Treatment Paradigm for Congenital Dyserythropoietic Anemia Type II

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Erythroferrone Regulates Bone Remodeling in β-Thalassemia

Elucidating the Role of IL6 in Stress Erythropoiesis and in the Development of Anemia Under Inflammatory Conditions

Does Gene Therapy in Beta Thalassemia Normalize Novel Markers of Ineffective Erythropoiesis and Iron Homeostasis?

Hematopoietic Stem Cell Function in β-Thalassemia Is Impaired and Is Rescued By Targeting the Bone Marrow Niche

Experience with Low Dose Thalidomide in Transfusion Dependent Beta Thalassaemia in a Resource Limited Setting

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American Society of Hematology

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American Society of Hematology

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