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

1-20 of 82

Sort by

Sort Order Select

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

Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin

Richard A. Voit, Rachael F. Grace

Blood (2020) 136 (6): 770.

https://doi.org/10.1182/blood.2020006395

Published: 2020

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

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Thidarat Suksangpleng, PhD, Kalaya Tachavanich, M.Sc., Suchada Riolueang, MSc, Supachai Ekwattanakit, PhDMD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2220.

https://doi.org/10.1182/blood-2019-131720

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

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 2175.

https://doi.org/10.1182/blood-2019-123069

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Megan Lynch, MSN, Lei Hua, PhD, Chris Mix, MD, John B. Porter, MA MDFRCP FRCPath

Blood (2019) 134 (Supplement_1): 3526.

https://doi.org/10.1182/blood-2019-123107

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

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

Kathryn Addonizio, BA, Hanny Al-Samkari, MD, Bertil Glader, MD PhD, D Holmes Morton, MD, Satheesh Chonat, MD, Alexis A. Thompson, MD MPH, Kevin H. M. Kuo, MDMSc,FRCPC, Yaddanapudi Ravindranath, MBBS, Heng Wang, MD PhD, Jennifer A. Rothman, MD, Janet L. Kwiatkowski, MD MSCE, Charles Kung, PhD, Penelope A. Kosinski, MS, Hasan Al-Sayegh, MS, Wendy B. London, PhD, Rachael F. Grace, MD

Blood (2019) 134 (Supplement_1): 3515.

https://doi.org/10.1182/blood-2019-125248

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

Prevalence of Red Cell Pyruvate Kinase Deficiency: A Systematic Literature Review

Mike Storm, MSc, Matthew H Secrest, MSc, Courtney Carrington, MPH, Keely Gilroy, PhD, Leanne Pladson, RN BS, Audra N. Boscoe, PhD, Deborah Casso, MPH

Blood (2019) 134 (Supplement_1): 3513.

https://doi.org/10.1182/blood-2019-124381

Published: 2019

Journal Articles

Hereditary Hemolytic Anemias Other Than Thalassemia: Single Center Experience

Gul Nihal Ozdemir, MD, Mehmet Akif Kilic, MD

Blood (2019) 134 (Supplement_1): 4855.

https://doi.org/10.1182/blood-2019-121702

Published: 2019

Journal Articles

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Sule Unal, MD, Tekin Aksu, MD, Cagrı Coskun, MD, Fatma Gumruk, MD

Blood (2019) 134 (Supplement_1): 4794.

https://doi.org/10.1182/blood-2019-124272

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Eduard J. van Beers, MD PhD, Malia P. Judge, BS, RN, Lei Hua, PhD, Chris Mix, MD

Blood (2019) 134 (Supplement_1): 4791.

https://doi.org/10.1182/blood-2019-123100

Published: 2019

Journal Articles

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 949.

https://doi.org/10.1182/blood-2019-125352

Published: 2019

Journal Articles

Spectrum of Genetic Defects and Phenotype-Genotype Correlation in Dyserythropoietic Anemias: Bench to Bedside Approach in the Indian Scenario

Reena Das, MD, Manu Jamwal, PhD, Anu Aggarwal, PhD, Prashant Sharma, DM, Man Updesh Singh Sachdeva, MD, Deepak Bansal, MD, Sreejesh Sreedharanunni, DM, Narender Kumar, MD, Shano Naseem, MD, Jasmina Ahluwalia, MD, Neelam Varma, MD, Pankaj Malhotra, MD, Arindam Maitra, PhD

Blood (2019) 134 (Supplement_1): 950.

https://doi.org/10.1182/blood-2019-126453

Published: 2019

Journal Articles

Proton Pump Inhibition for Secondary Hemochromatosis in Hereditary Anemia, a Phase III Placebo Controlled Randomized Cross-over Trial in Progress

Annelies J. Van Vuren, MD, Bart J. Biemond, MD PhD, Jean-Louis Kerkhoffs, MD PhD, Saskia E.M. Schols, MD PhD, A. W. Rijneveld, MD PhD, Erfan Nur, MD PhD, Richard van Wijk, PhD, Tim Leiner, MD PhD, Joannes J.M. Marx, Emeritus Professor, Eduard J. van Beers, MD PhD

Blood (2019) 134 (Supplement_1): 960.

https://doi.org/10.1182/blood-2019-124059

Published: 2019

Journal Articles

Detection and Differential Diagnosis of Prekallikrein Deficiency: Genetic Study of New Families and Systematic Review of the Literature

Stefano Barco, Stefanie Sollfrank, Alice Trinchero, Luigi Tomao, Barbara Zieger, Johanna A. Kremer Hovinga, MD Prof, Laura Conti, Anke Adenäuer, Erwin Miloni, Karl J. Lackner, Heidi Rossmann, Bernhard Lammle, MD

Blood (2018) 132 (Supplement 1): 2496.

https://doi.org/10.1182/blood-2018-99-116030

Published: 2018

Journal Articles

Pklr Intron Splicing-Associated Mutations and Alternate Diagnoses Are Common in Pyruvate Kinase Deficient Patients with Single or No Pklr Coding Mutations

Kimberly Lezon-Geyda, PhD, Melissa J. Rose, DO, Melissa A. McNaull, MD, Christine M. Knoll, MD, Hassan M. Yaish, MD, Yves D. Pastore, MD, Elisa Fermi, Bertil Glader, MD, Paola Bianchi, BSc, PhD, Rachael F. Grace, MD, Patrick G. Gallagher, MD

Blood (2018) 132 (Supplement 1): 3607.

https://doi.org/10.1182/blood-2018-99-117805

Published: 2018

Journal Articles

Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency

Charles Kung, PhD, Penelope A. Kosinski, MS, Heidi Mangus, BS, Lei Hua, PhD, Gary Connor, Michelle Mobilia, BS, Karen Sullivan, BA, Sasha Frye, BS, Marie-Hélène Jouvin, MD, Rachael F. Grace, MD, Bertil Glader, MD, Chris Bowden, MD

Blood (2018) 132 (Supplement 1): 3621.

https://doi.org/10.1182/blood-2018-99-111166

Published: 2018

NARROW

Management of pyruvate kinase deficiency in children and adults

Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin

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

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

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

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

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

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

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

Prevalence of Red Cell Pyruvate Kinase Deficiency: A Systematic Literature Review

Hereditary Hemolytic Anemias Other Than Thalassemia: Single Center Experience

Pyruvate Kinase Deficiency Cases Mimicking Congenital Dyserythropoietic Anemia

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Spectrum of Genetic Defects and Phenotype-Genotype Correlation in Dyserythropoietic Anemias: Bench to Bedside Approach in the Indian Scenario

Proton Pump Inhibition for Secondary Hemochromatosis in Hereditary Anemia, a Phase III Placebo Controlled Randomized Cross-over Trial in Progress

Detection and Differential Diagnosis of Prekallikrein Deficiency: Genetic Study of New Families and Systematic Review of the Literature

Pklr Intron Splicing-Associated Mutations and Alternate Diagnoses Are Common in Pyruvate Kinase Deficient Patients with Single or No Pklr Coding Mutations

Genotype-Response Correlation in DRIVE PK, a Phase 2 Study of AG-348 in Patients with Pyruvate Kinase Deficiency

Email alerts

ASH Publications

American Society of Hematology

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only