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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

Brief Report
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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

Janet L. Kwiatkowski, MD MSCE, Mohsen Saleh Elalfy, MD, Caroline Fradette, PhD, Mona Hamdy, MD, Amal El-Beshlawy, MD, Fatma Soliman Elsayed Ebeid, MD, Monica PINHEIRO DE ALMEIDA Verissimo, MD, Mohamed Ahmed Badr, MD, Alshehri Abdulrahman, MD, Julie Kanter, MD, Baba PD Inusa, MD, Mohamed Bejaoui, MD, Suzan Williams, MD, Yurdanur Kilinc, MD, Yu-Chung Tsang, PhD, Anne Stilman, BA, Anna Rozova, MD, Jodie Sinclair, MSc, Dian Shaw, RN, Theresa Chan, BSc, Noemi Toiber Temin, MA, David Lee, PhD, Michael Spino, PharmD, Fernando Tricta, MD
Journal: Blood
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
Journal: Blood
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

David C Rees, Yurdanur Kilinc, MD, Selma Unal, Carlton Dampier, MD, Betty S. Pace, MD, Banu Kaya, MD, Sara Trompeter, MBChB BSc MRCPCH FRCPath MSc, Isaac Odame, Johnny N Mahlangu, MBBCH, MMed, FCPath, Sule Unal, MD, Julie Brent, MD, Regine Grosse, MD, Beng R. Fuh, MD, Baba PD Inusa, MD, Ariel Koren, MD, Carina Levin, MD PhD, Steven Mortier, Elizabeth McNamara, Yue Li, Stephen John Oliver, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 615.
https://doi.org/10.1182/blood-2019-123355
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2.
https://doi.org/10.1182/blood-2019-125822
Published: 2019
Journal Articles

Patient-Reported Outcome Measures (PROMs) in Pediatric Non-Malignant Hematology: A Systematic Review

Robert J Klaassen, MD FRCPC, Julia Y. Kinahan, MDcal Student, Johann M. I. Graham, MDcal Student, Yamilée V. Hébert, MDcal Student, Katie O'Hearn, MSc Human Kinetics
Journal: Blood
Blood (2019) 134 (Supplement_1): 2180.
https://doi.org/10.1182/blood-2019-127248
Published: 2019
Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2066.
https://doi.org/10.1182/blood-2019-131318
Published: 2019
Journal Articles

Pediatric Hematologist/Oncologists' Beliefs about Hematopoietic Stem Cell Transplant for Children with Sickle Cell Disease: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) Study

Emily Riehm Meier, MD MSHS, Allistair Abraham, MD, Alexander I. Ngwube, MD, Isaac Janson, PhD, Gregory M.T. Guilcher, MD, John T. Horan, MDMPH, Kimberly A. Kasow, DO
Journal: Blood
Blood (2019) 134 (Supplement_1): 2164.
https://doi.org/10.1182/blood-2019-130543
Published: 2019
Journal Articles

Methodology for the Start-up of a Pediatric Hematopoietic Stem Cell Transplant Unit (pHSCTU) in Middle-Income Countries (MICs): An Exportable Model?

Marta Verna, MD, Attilio Rovelli, MD, Valentino Conter, MD, Marta Canesi, Nurse, Ignazio Majolino, MD, Dosti Othman, MD, Vian Faeq, MD, Jabibi Noguera, MD, Maria Liz Benitez, MD, Othman Dastan, MD, Andrea Biondi, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2044.
https://doi.org/10.1182/blood-2019-121922
Published: 2019
Journal Articles

Excellent Survival and Immune Reconstitution Following Matched/Mismatched Unrelated and Mismatched Related Transplantation in Adult Patients with Sickle Cell Disease: Updated Results from a Single Center Experience

Amer Assal, MD, Diane George, MD, Monica Bhatia, MD, Divaya Bhutani, MD, Christian Gordillo, BS, Suzanne Lentzsch, MD PhD, Ran Reshef, MDMSc, Markus Y Mapara, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2043.
https://doi.org/10.1182/blood-2019-132113
Published: 2019
Journal Articles

An Educational Study Promoting the Delivery of TCD Screening in Paediatric Sickle Cell Disease: A European Multi-Centre Perspective

Baba PD Inusa, MD, Corrina Macmahon, MD, Laura Sainati, MD, Raffaella Colombatti, MD PhD, Maddalena Casale, MD PhD, Patrizia Rampazzo, MD, Silverio Perrotta, Soundrie Padayachee
Journal: Blood
Blood (2019) 134 (Supplement_1): 2172.
https://doi.org/10.1182/blood-2019-130390
Published: 2019
Journal Articles

Academic Challenges and Concerns for Children with Sickle Cell Disease: Analysis of a Hospital-Based School Intervention Program

Kristine Anne Karkoska, MD, Kenneth Haber, MS, Patrick T. McGann, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2165.
https://doi.org/10.1182/blood-2019-128279
Published: 2019
Journal Articles

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent β-Thalassaemia in the United Kingdom: A Mixed Methods Observational Study

Farrukh Shah, MDMBBS,FRCPath, Paul Telfer, DM, FRCP, Mark Velangi, Shivan Pancham, Robert Wynn, Sally Pollard, Elizabeth Chalmers, Jonathan Kell, MD, Joe Hickey, PhD, Clark Paramore, MPH, Minesh Jobanputra, MD, Kate Ryan, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3550.
https://doi.org/10.1182/blood-2019-129930
Published: 2019
Journal Articles

Pancreatic Iron and Glucidic Metabolism in Thalassemia Major

Alessia Pepe, MD, Laura Pistoia, Saveria Campisi, MD, Roberto Sarli, Piera Giovangrossi, Giulia Guerrini, Roberto Mattei, Aurelio Maggio, MD, Domenico Visceglie, Valentina Carrai, MD, Giuseppe Peritore, Vincenzo Positano, Antonella Meloni, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3551.
https://doi.org/10.1182/blood-2019-121876
Published: 2019