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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

Journal Articles

Eltrombopag: More Than Just a Thrombopoietin Receptor Agonist (TPO-RA) in Immune Thrombocytopenia (ITP)

Anwar A. Sayed, MBBS, MSc, PhD, Amna Malik, PhD, Grace Ayoola, BSc., Elisa Lucchini, MD, Sasfia Candrianita, MD, Michelle H Tan, BSc, Camelia Vladescu, MSc, Deena Paul, Alice C J Hart, MBBS, BSc, MRCP, FRCPath(Haem), Ahmad Khoder, MD PhD MRCP, ASCP, Nesrina Imami, PhD, Nichola Cooper, MD MRCP

Blood (2019) 134 (Supplement_1): 2364.

https://doi.org/10.1182/blood-2019-122848

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

Assessment By MRI T2*of Iron Accumulation and Removal in Transfusion Dependent MDS Patients

Drorit Grizim Merkel, MD, Abraham Avigdor, MD, Arnon Nagler, MD MSc, Ohad Benjamini, MD, Yaron Davidovitz, MD, Orly Goitein, MD

Blood (2019) 134 (Supplement_1): 5432.

https://doi.org/10.1182/blood-2019-125279

Published: 2019

Journal Articles

Increased Incidence of Infection in Patients with Myelofibrosis and Transfusion-Associated Iron Overload: A Monocentric Experience

Giovanni Caocci, MD, Maria Pina Simula, Silvia Ghiani, Olga Mulas, Giorgia Mainas, Sandra Atzeni, Martina Pettinau, Emilio Usala, Giorgio La Nasa, MD

Blood (2019) 134 (Supplement_1): 4186.

https://doi.org/10.1182/blood-2019-122006

Published: 2019

Journal Articles

Combined Retro- and Prospective Analysis of Adherence to Guidelines and Patient-Tailored Therapeutic Recommendations in Patients with Myelodysplastic Syndromes (MDS) at a Tertiary Care Centre

Annika Kasprzak, Kathrin Nachtkamp, MD, Mustafa Kondakci, MD, Thomas Schroeder, MD, Guido Kobbe, MD, Andrea Kündgen, MD, Jennifer Kaivers, MD, Christina Rautenberg, MD, Rainer Haas, MD, Norbert Gattermann, MD, Nicolas Bonadies, MD, Ulrich Germing, MD

Blood (2019) 134 (Supplement_1): 4250.

https://doi.org/10.1182/blood-2019-124512

Published: 2019

Journal Articles

A Multicenter, Italian Trial of Early Iron Chelation Therapy with Low Dose Deferasirox (Exjade®) in Patients with Low/Intermediate-1 Risk MDS at the Beginning of Transfusional Story

Emanuele Angelucci, MD, Mario Capasso, PhD, Matteo Giovanni Della Porta, MD, Gian Luca Forni, Domenico Girelli, MD, PhD, Esther Natalie Oliva, MD, Federica Pilo, MD, Marino Clavio, MD, Marta Riva, MD, Annamaria Pelizzari, MD, Pasquale Niscola, MD PhD, Daniela Cilloni, MD PhD, Gianni Binotto, MD, Elena Crisà, MD, Valeria Santini, MD

Blood (2019) 134 (Supplement_1): 4256.

https://doi.org/10.1182/blood-2019-125575

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

Eltrombopag Creates a Transient Chemical Phenocopy of TET2 Loss of Function That Contributes to Hematopoietic Precursor Expansion

Yihong Guan, PhD, Bhumika J. Patel, MD, Metis Hasipek, PhD, Dale Grabowski, BS, Hassan Awada, MD, Cassandra M Kerr, MS, Valeria Visconte, PhD, Aziz Nazha, MD, Yogenthiran Saunthararajah, MD, Mikkael A. Sekeres, MD MS, Jaroslaw P. Maciejewski, MD PhD, Babal K. Jha, PhD

Blood (2019) 134 (Supplement_1): 453.

https://doi.org/10.1182/blood-2019-131525

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

Sickle Cell Disease Burden in North Lebanon

Adlette Inati, MD, Chadi Al Alam, Cristel El Ojaimi, Taghrid Hamad, Hemanth Kanakamedala, Virginia Pilipovic, Ramzieh Sabah

Blood (2019) 134 (Supplement_1): 1022.

https://doi.org/10.1182/blood-2019-128311

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

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

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

Eltrombopag: More Than Just a Thrombopoietin Receptor Agonist (TPO-RA) in Immune Thrombocytopenia (ITP)

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

Assessment By MRI T2*of Iron Accumulation and Removal in Transfusion Dependent MDS Patients

Increased Incidence of Infection in Patients with Myelofibrosis and Transfusion-Associated Iron Overload: A Monocentric Experience

Combined Retro- and Prospective Analysis of Adherence to Guidelines and Patient-Tailored Therapeutic Recommendations in Patients with Myelodysplastic Syndromes (MDS) at a Tertiary Care Centre

A Multicenter, Italian Trial of Early Iron Chelation Therapy with Low Dose Deferasirox (Exjade®) in Patients with Low/Intermediate-1 Risk MDS at the Beginning of Transfusional Story

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+)

Eltrombopag Creates a Transient Chemical Phenocopy of TET2 Loss of Function That Contributes to Hematopoietic Precursor Expansion

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

Sickle Cell Disease Burden in North Lebanon

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

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

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