Page 1 | Search Results | Blood | American Society of Hematology

1-20 of 570

Sort by

Sort Order Select

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

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

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent β-Thalassemia Treated at the Bambino Gesù Children's Hospital, Rome, Italy

Pietro Merli, MD, Annalisa Ruggeri, MD, Mattia Algeri, MD, Giuseppina Li Pira, PhD, Giulia Ceglie, MD, Katiana Gruppioni, MPH, Sanjeev Kommera, MS, Jenfue Maa, PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 969.

https://doi.org/10.1182/blood-2019-123440

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

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

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

Blood (2019) 134 (Supplement_1): 3550.

https://doi.org/10.1182/blood-2019-129930

Published: 2019

Journal Articles

Neutropenia in Children Treated with Deferiprone or Deferasirox: A Report of the Largest Randomized Trial of Oral Chelators in Transfusion-Dependent Pediatric Patients

Fernando Tricta, MD, Mariagrazia Felisi, PhD, Oscar Della Pasqua, Amal El-Beshlawy, MD, Hoda Hassab, MD, Antonis Kattamis, Manika Kreka, MD, Giorgio Reggiardo, Laila Metwally Sherief, MD, Michael Spino, PharmD, Bianca Tempesta, PhD, Yu-Chung Tsang, PhD, Adriana Ceci, MD

Blood (2019) 134 (Supplement_1): 3552.

https://doi.org/10.1182/blood-2019-127933

Published: 2019

Journal Articles

Fyn Specifically Regulates the Activity of Red Cell Glucose-6-Phosphate-Dehydrogenase

Blood (2019) 134 (Supplement_1): 3527.

https://doi.org/10.1182/blood-2019-125709

Published: 2019

Journal Articles

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia

Matthew Wake, PhD, Jonathan Papworth, BSc, Luke Bayliss, BSc, Benjamin Grimshaw, PhD, Natalie Rynkiewicz, PhD, Jemima Paterson, BSc, Alicia Poindron, MSc, Erin Spearing, Emily Carter, Robyne Hudson, BSc, Verena Petzer, MD, Amelie Largounez, BSc, Audrey Belot, BSc, Anais Palin, BSc, Igor Theurl, MD PhD, Volker Germaschewski, Delphine Meynard, PhD

Blood (2019) 134 (Supplement_1): 3532.

https://doi.org/10.1182/blood-2019-124075

Published: 2019

Journal Articles

Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

Sam Salek, PhDRPh FFPM MCMS MRPSGB FESCP, Audra N. Boscoe, PhD, Chris Evans, PhD, Shayna Egan, MPH, Ted Wells, Sarah Piantedosi, Jennifer Cohen, Robert J Klaassen, MD FRCPC, Rachael F. Grace, MD, Mike Storm, MSc

Blood (2019) 134 (Supplement_1): 3447.

https://doi.org/10.1182/blood-2019-128524

Published: 2019

Journal Articles

Uncertainty in the R2 Calibration Curve for High Iron Concentration

John C Wood, MD PhD, Eamon Doyle, PhD, Nilesh Ghugre, PhD, Thomas Coates, MD

Blood (2019) 134 (Supplement_1): 3541.

https://doi.org/10.1182/blood-2019-128886

Published: 2019

Journal Articles

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

Natalia Scaramellini, MD, Carola Arighi, MD, Alessia Marcon, MD, Dario Consonni, MD, Elena Cassinerio, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3542.

https://doi.org/10.1182/blood-2019-130237

Published: 2019

Journal Articles

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Rachael F. Grace, MD, D. Mark Layton, MB BS, FRCP, FRCPCH, Frédéric Galactéros, MD PhD, Wilma Barcellini, MD, Eduard J. van Beers, MD PhD, Hassan M. Yaish, MD, Yaddanapudi Ravindranath, MBBS, Kevin H. M. Kuo, MDMSc,FRCPC, Sujit Sheth, MD, Janet L. Kwiatkowski, MD MSCE, Lei Hua, PhD, Peter F. Hawkins, PhD, Chris Mix, MD, Bertil Glader, MD PhD

Blood (2019) 134 (Supplement_1): 3512.

https://doi.org/10.1182/blood-2019-123406

Published: 2019

Journal Articles

Quantification of Humoral Immune Response to Influenza Vaccination in MDS

Pankit Vachhani, MD, Kyle Wiatrowski, Pragya Srivastava, PhD, Lisa King, Jody Manischewitz, Hana Golding, Heather L Bashaw, BA, Ava R. Dimitroff, Amanda Przespolewski, DO, James E. Thompson, MD, Swapna Thota, MD, Eunice S. Wang, MD, Michael J. Nemeth, PhD, Elizabeth A. Griffiths, MD

Blood (2019) 134 (Supplement_1): 4756.

https://doi.org/10.1182/blood-2019-126176

Published: 2019

Journal Articles

I-Care for MDS: Development of Guidelines-Based Indicators for Appropriate Care in Adult Patients with Myelodysplastic Syndromes

Blood (2019) 134 (Supplement_1): 4752.

https://doi.org/10.1182/blood-2019-122285

Published: 2019

Journal Articles

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2019) 134 (Supplement_1): 3545.

https://doi.org/10.1182/blood-2019-122685

Published: 2019

Journal Articles

Altered Iron Metabolism Is a New Targetable Hallmark for Multiple Myeloma

Can Li, MD, Xuelian Tan, Qierra Brockman, BA, Yogesh Jethava, MBBS,MDFRCPath,MRCP, Marta Chesi, PhD, P. Leif Bergsagel, MD, Fangping Chen, MD PhD, Ivana Frech, DSc, PhD MBA, Fenghuang Zhan, MD PhD

Blood (2019) 134 (Supplement_1): 3059.

https://doi.org/10.1182/blood-2019-123473

Published: 2019

Journal Articles

Clinical Relevance of Individual Response to Iron Chelation Therapy (ICT) in Patients with Myelodysplastic Syndromes (MDS) and Transfusion Requirement

Blood (2019) 134 (Supplement_1): 3010.

https://doi.org/10.1182/blood-2019-129902

Published: 2019

Journal Articles

Persistent Red Blood Cell (RBC) Transfusion Is Associated with Increased Mortality Risk in Transfusion-Dependent (TD) Patients with Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS+)

Blood (2019) 134 (Supplement_1): 3012.

https://doi.org/10.1182/blood-2019-123684

Published: 2019

Journal Articles

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Rachael F. Grace, MD, Paola Bianchi, BSc, PhD, Bertil Glader, MD PhD, Andreas Glenthøj, MD PhD, Bryan Jones, PhD, Hitoshi Kanno, MD PhD, Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB ChB, Eduard J. van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Tu Xu, MS, PhD

Blood (2019) 134 (Supplement_1): 2223.

https://doi.org/10.1182/blood-2019-123059

Published: 2019

NARROW

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

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

Clinical Outcomes after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Transfusion-Dependent β-Thalassemia Treated at the Bambino Gesù Children's Hospital, Rome, Italy

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

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

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

Neutropenia in Children Treated with Deferiprone or Deferasirox: A Report of the Largest Randomized Trial of Oral Chelators in Transfusion-Dependent Pediatric Patients

Fyn Specifically Regulates the Activity of Red Cell Glucose-6-Phosphate-Dehydrogenase

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia

Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

Uncertainty in the R2 Calibration Curve for High Iron Concentration

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Quantification of Humoral Immune Response to Influenza Vaccination in MDS

I-Care for MDS: Development of Guidelines-Based Indicators for Appropriate Care in Adult Patients with Myelodysplastic Syndromes

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Altered Iron Metabolism Is a New Targetable Hallmark for Multiple Myeloma

Clinical Relevance of Individual Response to Iron Chelation Therapy (ICT) in Patients with Myelodysplastic Syndromes (MDS) and Transfusion Requirement

Persistent Red Blood Cell (RBC) Transfusion Is Associated with Increased Mortality Risk in Transfusion-Dependent (TD) Patients with Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS+)

An Ongoing Global, Longitudinal, Observational Study of Patients with Pyruvate Kinase Deficiency: The PEAK Registry

Email alerts

ASH Publications

American Society of Hematology

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only