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Journal Articles

A natural DNMT1 mutation elevates the fetal hemoglobin level via epigenetic derepression of the γ-globin gene in β-thalassemia

Yi Gong, Xinhua Zhang, Qianqian Zhang, Yanxia Zhang, Yuhua Ye, Wenxia Yu, Congwen Shao, Tizhen Yan, Jin Huang, Jianmei Zhong, Li Wang, Yaoyun Li, Liren Wang, Xiangmin Xu

Blood (2021) 137 (12): 1652–1657.

https://doi.org/10.1182/blood.2020006425

Published: 2021

Includes: Supplemental data

Journal Articles

Less (Fe) is more (Hb) in SCA

Carlo Brugnara

Blood (2021) 137 (11): 1446–1447.

https://doi.org/10.1182/blood.2020010131

Published: 2021

Journal Articles

Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche

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

Blood (2020) 136 (5): 610–622.

https://doi.org/10.1182/blood.2019002721

Published: 2020

Includes: Supplemental data

Journal Articles

Antibodies against the erythroferrone N-terminal domain prevent hepcidin suppression and ameliorate murine thalassemia

João Arezes, Niall Foy, Kirsty McHugh, Doris Quinkert, Susan Benard, Anagha Sawant, Joe N. Frost, Andrew E. Armitage, Sant-Rayn Pasricha, Pei Jin Lim, May S. Tam, Edward Lavallie, Debra D. Pittman, Orla Cunningham, Matthew Lambert, John E. Murphy, Simon J. Draper, Reema Jasuja, Hal Drakesmith

Blood (2020) 135 (8): 547–557.

https://doi.org/10.1182/blood.2019003140

Published: 2020

Includes: Supplemental data

Journal Articles

Single-cell analysis of bone marrow–derived CD34 + cells from children with sickle cell disease and thalassemia

Peng Hua, Noemi Roy, Josu de la Fuente, Guanlin Wang, Supat Thongjuea, Kevin Clark, Anindita Roy, Bethan Psaila, Neil Ashley, Yvonne Harrington, Claus Nerlov, Suzanne M. Watt, Irene Roberts, James O. J. Davies

Blood (2019) 134 (23): 2111–2115.

https://doi.org/10.1182/blood.2019002301

Published: 2019

Includes: Supplemental data

Journal Articles

Heme-regulated eIF2α kinase in erythropoiesis and hemoglobinopathies

Jane-Jane Chen, Shuping Zhang

Blood (2019) 134 (20): 1697–1707.

https://doi.org/10.1182/blood.2019001915

Published: 2019

Journal Articles

Efficacy and Safety of Sevuparin, a Novel Non-Anti-Coagulant Heparinoid, in Patients with Acute Painful Vaso-Occlusive Crisis; A Global, Multicenter Double-Blind, Randomized, Placebo-Controlled Phase 2 Trial (TVOC01)

Bart J. Biemond, MD PhD, Anil Tombak, MD, Yurdanur Kilinc, MD, Murtadha Al-Khabori, MD, Miguel R. Abboud, MD, Mohammed Nafea, MD, Adlette Inati, MD, Yasser Ahmed Mohamed Soliman Wali, MD, Jens Kristensen, MD PhD, Ellen Donnelly, PhD, John Ohd, MD PhD

Blood (2019) 134 (Supplement_1): 614.

https://doi.org/10.1182/blood-2019-124653

Published: 2019

Journal Articles

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone in Iron-Overloaded Patients with Sickle Cell Disease or Other Anemias

Blood (2019) 134 (Supplement_1): 618.

https://doi.org/10.1182/blood-2019-122062

Published: 2019

Journal Articles

Neurocognitive Impairment Predicts Poor Transition Outcomes Among Patients with Sickle Cell Disease

Anjelica C. Saulsberry, BA, Marita Partanen, PhD, Jerlym S. Porter, PhD MPH, Pradeep S. B. Podila, PhD MS, Jason R. Hodges, PhD MA, Allison A. King, MD MPH, PhD, Winfred Wang, MD, Xiwen Zhao, MSPH, Guolian Kang, PhD, Lisa M. Jacola, PhD ABPP-CN, Jane S. Hankins, MD MS

Blood (2019) 134 (Supplement_1): 519.

https://doi.org/10.1182/blood-2019-121617

Published: 2019

Journal Articles

Double-Blind, Randomized Study of Canakinumab Treatment in Pediatric and Young Adult Patients with Sickle Cell Anemia

Blood (2019) 134 (Supplement_1): 615.

https://doi.org/10.1182/blood-2019-123355

Published: 2019

Journal Articles

Integrated Anemia Detection and Hemoglobin Variant Identification Using Point-of-Care Microchip Electrophoresis

Ran An, PhD, Muhammad Noman Hasan, MS, Yuncheng Man, Umut A. Gurkan, PhD

Blood (2019) 134 (Supplement_1): 378.

https://doi.org/10.1182/blood-2019-126096

Published: 2019

Journal Articles

Incidence, Risk Factors and Outcomes of Veno-Occlusive Disease / Sinusoidal Obstruction Syndrome (VOD/SOS) in Children with Severe Thalassemia (ST) Conditioned with Busulfan- Cyclophosphamide (Bu-Cy) Based Regimen

Stalin Ramprakash, MD MBBS, Rajat Kumar Agarwal, BS, C P Raghuram, MD, Pallavi Mehta, Deepa Trivedi, MD, Priya Marwah, Rajpreet Soni, Rakesh Dhanya, BE, Lawrence Faulkner, MD

Blood (2019) 134 (Supplement_1): 4499.

https://doi.org/10.1182/blood-2019-130841

Published: 2019

Journal Articles

Affect of Age on Survival and Complications after Hematopoietic Stem Cell Transplantation in Children with β-Thalassemia Major

Yiqi Xu, MD, Yongsheng Ruan, MD, Yuelin He, MD, Jianyun Wen, Xiaoqin Feng, Xuan Liu, Haixia Guo, MD PhD, Chunfu Li, MD, Qareen Haque, Xuedong Wu, MD PhD

Blood (2019) 134 (Supplement_1): 4591.

https://doi.org/10.1182/blood-2019-126596

Published: 2019

Journal Articles

Peripheral Blood to Marrow Collection White Cell Count Ratio As an Indicator of Harvest Quality and Transplant Outcomes in G-CSF-Primed Marrow Grafts

Rajat Kumar Agarwal, BE, Rakesh Dhanya, BE, Stalin Ramprakash, MD MBBS, C P Raghuram, MD, Pallavi Mehta, MD DM, Deepa Trivedi, MD, Shruthi Jayapal, Sandeep S, Lawrence Faulkner, MD

Blood (2019) 134 (Supplement_1): 4473.

https://doi.org/10.1182/blood-2019-125111

Published: 2019

Journal Articles

Complementary Transplantation with Unrelated Cord Blood Following Haploidentical Stem Cells for Juvenile Myelomonocytic Leukemia:Double-Centers Study from China

Zhiyong Peng, MD, Xiaoqin Feng, Huaying Liu, MD, Jianyun Liao, MD, Chaoke Bu, Yuelin He, MD, Jianmin Zhang, MD, Jingyu Gao, Chunfu Li, MD

Blood (2019) 134 (Supplement_1): 4181.

https://doi.org/10.1182/blood-2019-128331

Published: 2019

Journal Articles

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD

Blood (2019) 134 (Supplement_1): 5754.

https://doi.org/10.1182/blood-2019-129931

Published: 2019

Journal Articles

Hematopoietic Stem Cell Transplantation in Βthalassemia Major: The Egyptian Outcome

Amal El-Beshlawy, MD, Amina AbdelSalam, MD, Gamal Fathy, MD, Khaled M. Salamaa, MD, Mona El ghamrawy, MD, Samar S. Ramadan, M.Sc., Alaa M. Elhaddad, MD

Blood (2019) 134 (Supplement_1): 5732.

https://doi.org/10.1182/blood-2019-124398

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

Patient Education Interventions for Improving Self-Management in Adults with Hemoglobinopathies: A Systematic Review and Meta-Analysis

Maha Munawar, BSc, Richard Ward, MSc, MRCP, FRCPath, Kate Uchendu, BScN, MN, NP-Adult, Rouhi Fazelzad, BSc, MISt, Janet Papadakos, Kevin H. M. Kuo, MDMSc,FRCPC

Blood (2019) 134 (Supplement_1): 5783.

https://doi.org/10.1182/blood-2019-131718

Published: 2019

Journal Articles

A Global Systematic Literature Review on the Burden of Illness in Transfusion-Dependent β-Thalassemia

Monica Turner, Sohan Deshpande, Madhura Chitnis, Michael Byrnes, Christopher Pelligra, Amy Rines-MacEachern, Jessica Morison, Derek Tang, PhD BSPharm

Blood (2019) 134 (Supplement_1): 5786.

https://doi.org/10.1182/blood-2019-124307

Published: 2019

NARROW

A natural DNMT1 mutation elevates the fetal hemoglobin level via epigenetic derepression of the γ-globin gene in β-thalassemia

Less (Fe) is more (Hb) in SCA

Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche

Antibodies against the erythroferrone N-terminal domain prevent hepcidin suppression and ameliorate murine thalassemia

Single-cell analysis of bone marrow–derived CD34 + cells from children with sickle cell disease and thalassemia

Heme-regulated eIF2α kinase in erythropoiesis and hemoglobinopathies

Efficacy and Safety of Sevuparin, a Novel Non-Anti-Coagulant Heparinoid, in Patients with Acute Painful Vaso-Occlusive Crisis; A Global, Multicenter Double-Blind, Randomized, Placebo-Controlled Phase 2 Trial (TVOC01)

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone in Iron-Overloaded Patients with Sickle Cell Disease or Other Anemias

Neurocognitive Impairment Predicts Poor Transition Outcomes Among Patients with Sickle Cell Disease

Double-Blind, Randomized Study of Canakinumab Treatment in Pediatric and Young Adult Patients with Sickle Cell Anemia

Integrated Anemia Detection and Hemoglobin Variant Identification Using Point-of-Care Microchip Electrophoresis

Incidence, Risk Factors and Outcomes of Veno-Occlusive Disease / Sinusoidal Obstruction Syndrome (VOD/SOS) in Children with Severe Thalassemia (ST) Conditioned with Busulfan- Cyclophosphamide (Bu-Cy) Based Regimen

Affect of Age on Survival and Complications after Hematopoietic Stem Cell Transplantation in Children with β-Thalassemia Major

Peripheral Blood to Marrow Collection White Cell Count Ratio As an Indicator of Harvest Quality and Transplant Outcomes in G-CSF-Primed Marrow Grafts

Complementary Transplantation with Unrelated Cord Blood Following Haploidentical Stem Cells for Juvenile Myelomonocytic Leukemia:Double-Centers Study from China

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Hematopoietic Stem Cell Transplantation in Βthalassemia Major: The Egyptian Outcome

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

Patient Education Interventions for Improving Self-Management in Adults with Hemoglobinopathies: A Systematic Review and Meta-Analysis

A Global Systematic Literature Review on the Burden of Illness in Transfusion-Dependent β-Thalassemia

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American Society of Hematology

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American Society of Hematology

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