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Journal Articles

Defective palmitoylation of transferrin receptor triggers iron overload in Friedreich ataxia fibroblasts

Floriane Petit, Anthony Drecourt, Michaël Dussiot, Coralie Zangarelli, Olivier Hermine, Arnold Munnich, Agnès Rötig

Blood (2021) 137 (15): 2090–2102.

https://doi.org/10.1182/blood.2020006987

Published: 2021

Includes: Multimedia, Supplemental data

Journal Articles

Management of pyruvate kinase deficiency in children and adults

Rachael F. Grace, Wilma Barcellini

Blood (2020) 136 (11): 1241–1249.

https://doi.org/10.1182/blood.2019000945

Published: 2020

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

Hepatic transferrin plays a role in systemic iron homeostasis and liver ferroptosis

Yingying Yu, Li Jiang, Hao Wang, Zhe Shen, Qi Cheng, Pan Zhang, Jiaming Wang, Qian Wu, Xuexian Fang, Lingyan Duan, Shufen Wang, Kai Wang, Peng An, Tuo Shao, Raymond T. Chung, Shusen Zheng, Junxia Min, Fudi Wang

Blood (2020) 136 (6): 726–739.

https://doi.org/10.1182/blood.2019002907

Published: 2020

Includes: Supplemental data

Journal Articles

Correlation between Changes in Cardiac Iron and Hepatic Iron in Pediatric Patients with Thalassemia Major

Alessia Pepe, MD, Antonella Meloni, PhD, Aldo Filosa, Laura Pistoia, Tommaso Casini, Roberto Lisi, Maria Caterina Putti, MD, Aurelio Maggio, MD, Calogera Gerardi, MD, Priscilla Fina, Vincenzo Positano, Maddalena Casale, MD

Blood (2019) 134 (Supplement_1): 2241.

https://doi.org/10.1182/blood-2019-121730

Published: 2019

Journal Articles

Factor-Associated Risk Factors of Mild Cognitive Impairment in Thalassemia Patients : Probable Role of FGF21

Wasan Theerajangkhaphichai, MD, Adisak Tantiworawit, MD, Jirapas Sripetchwandee, PhD, Nattayaporn Apaijai, PhD, Sirawit Sriwichaiin, MD, Napatsorn Saiyasit, Kewarin Jinawong, Busarin Arunsak, Pokpong Piriyakhuntorn, MD, Thanawat Rattanathammethee, MD, Sasinee Hantrakool, MD, Chatree Chai-Adisaksopha, MD PhD, Ekarat Rattarittamrong, MD, Lalita Norasetthada, MD, Pimlak Charoenkwan, MD, Nipon Chattipakorn, MD PhD, Siriporn Chattipakorn, DDS, PhD

Blood (2019) 134 (Supplement_1): 2251.

https://doi.org/10.1182/blood-2019-125182

Published: 2019

Journal Articles

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

John Porter, Maria Domenica Cappellini, MD, Thomas Coates, MD, Olivier Hermine, Vip Viprakasit, MD DPhil, Ersi Voskaridou, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Abderrahmane Laadem, Jeevan K. Shetty, Wen-Ling Kuo, Dimana Miteva, Tatiana Zinger, Peter G. Linde, Daniel Sinsimer, Ali Taher

Blood (2019) 134 (Supplement_1): 2245.

https://doi.org/10.1182/blood-2019-122561

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

Pancreatic Iron Deposition Following the Role of Iron Loading Among Transfusion Dependent Sickle Cell Disease Egyptian Children and Young Adults

Mohsen Saleh Elalfy, MD, Khalid Allam, MD, Ahmed Ibrahim, MD, Basant Mosaad, MD, Fatma Soliman Elsayed Ebeid, MD

Blood (2019) 134 (Supplement_1): 4828.

https://doi.org/10.1182/blood-2019-123683

Published: 2019

Journal Articles

Glucose Metabolism in Sickle Cell Disease

Taif Hassan, Binal N. Shah, Xu Zhang, PhD, Victor R Gordeuk, MD

Blood (2019) 134 (Supplement_1): 4830.

https://doi.org/10.1182/blood-2019-131485

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.

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

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

Iron-Chelating Potential of Novel Phytochemicals in Poplar and Cedar Trees

Sarah Lane, BSc, Juergen Ehlting, MSc, PhD, Patrick B Walter, MSc, PhD

Blood (2019) 134 (Supplement_1): 4806.

https://doi.org/10.1182/blood-2019-127259

Published: 2019

Journal Articles

Increased Allele Frequency of GNPAT p.D519G in Compound HFE p.C282Y/p.H63D Heterozygotes with Elevated Serum Ferritin Levels

Eriza S. Secondes, BSc (Honors), Daniel F. Wallace, PhD, Gautam Rishi, PhD, Gordon D. McLaren, MD, Christine E. McLaren, PhD, Wen-Pin Chen, MS, Louise Ramm, BSc, Lawrie W. Powell, MD PhD, Grant A. Ramm, PhD, James C. Barton, MD, V. Nathan Subramaniam, PhD

Blood (2019) 134 (Supplement_1): 4807.

https://doi.org/10.1182/blood-2019-128240

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

Journal Articles

Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34 + Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major

Riguo Fang, PhD, Pengfei Yuan, PhD, Lingling Yu, Bachelor, Huihui Yang, Master, Jie Liu, Master, Jia Shi, PhD, Yongjian Zhang, PhD, Wei Zhang, PhD, Yiding Zhao, PhD, Zhiyong Peng, MD, Chunfu Li, MD, Yun Li, MD, Dong Wei, PhD

Blood (2019) 134 (Supplement_1): 965.

https://doi.org/10.1182/blood-2019-126499

Published: 2019

Journal Articles

Therapeutic Applications of a Unique Calcium Channel Blocker to Target SF3B1 MDS

Sunisa Kongkiatkamon, Anand Tiwari, Vera Adema, PhD, Cassandra M Kerr, MS, Bartlomiej Przychodzen, Hassan Awada, Jibran Durrani, Yvonne Parker, Drew Adams, Yuriy Fedorov, Hetty E. Carraway, MD MBA, Chao-Yie Yang, Mikkael A. Sekeres, MD MS, Daniel J. Lindner, James G Phillips, PhD, Jaroslaw P. Maciejewski, MD PhD, Valeria Visconte, PhD

Blood (2019) 134 (Supplement_1): 881.

https://doi.org/10.1182/blood-2019-128671

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

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

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Donatella Baronciani, MD, Maddalena Casale, MD, Lucia De Franceschi, MD, Giovanna Graziadei, MD, Filomena Longo, MD, Raffaella Origa, MD, Valeria Maria Pinto, MD, Paolo Rigano, MD, Monia Marchetti, MD, Antonia Gigante, Emanuele Angelucci, MD, Maria Domenica Cappellini, MD, Achille Iolascon, MD PhD, Antonio Piga, Gian Luca Forni

Blood (2019) 134 (Supplement_1): 972.

https://doi.org/10.1182/blood-2019-128320

Published: 2019

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Defective palmitoylation of transferrin receptor triggers iron overload in Friedreich ataxia fibroblasts

Management of pyruvate kinase deficiency in children and adults

Congenital dyserythropoietic anemias

Hepatic transferrin plays a role in systemic iron homeostasis and liver ferroptosis

Correlation between Changes in Cardiac Iron and Hepatic Iron in Pediatric Patients with Thalassemia Major

Factor-Associated Risk Factors of Mild Cognitive Impairment in Thalassemia Patients : Probable Role of FGF21

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

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

Pancreatic Iron Deposition Following the Role of Iron Loading Among Transfusion Dependent Sickle Cell Disease Egyptian Children and Young Adults

Glucose Metabolism in Sickle Cell Disease

Symptomatic Bilirubin Gallstones in Patients with PNH Treated with Eculizumab

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

Iron-Chelating Potential of Novel Phytochemicals in Poplar and Cedar Trees

Increased Allele Frequency of GNPAT p.D519G in Compound HFE p.C282Y/p.H63D Heterozygotes with Elevated Serum Ferritin Levels

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

Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34 + Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major

Therapeutic Applications of a Unique Calcium Channel Blocker to Target SF3B1 MDS

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

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

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

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