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Journal Articles

Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Rachael F. Grace, MD, Audra Boscoe, PhD, Chris Bowden, MD, Bertil Glader, MD PhD, Hitoshi Kanno, MDPhD, D. Mark Layton, MB BS, Eduard van Beers, MD PhD, Joan-Lluis Vives Corrons, MD PhD, Yan Yan, MS, Paola Bianchi, BSc, PhD

Blood (2020) 136 (Supplement 1): 39–40.

https://doi.org/10.1182/blood-2020-136634

Published: 2020

Journal Articles

Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

Hanny Al-Samkari, MD, Rachael F. Grace, MD, Andreas Glenthoej, MD, Oliver Andres, MD, Wilma Barcellini, Frédéric Galactéros, MDPhD, Kevin H.M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Marta Morado, PhD, Vip Viprakasit, Yan Dong, PhD, Feng Tai, PhD, Peter Hawkins, PhD, Sarah Gheuens, MD PhD, Chris Bowden, MD, John B. Porter, MA, MD, FRCP, FRCPath, Eduard van Beers, MD PhD

Blood (2020) 136 (Supplement 1): 30–32.

https://doi.org/10.1182/blood-2020-136598

Published: 2020

Journal Articles

Characteristics of Children and Adults Treated with Rituximab for Immune Thrombocytopenia (ITP)

Emily M. Harris, MD, Kirsty Hillier, MD, Hanny Al-Samkari, MD, Peter W. Forbes, BS, Rachael F. Grace, MD

Blood (2020) 136 (Supplement 1): 38–39.

https://doi.org/10.1182/blood-2020-138856

Published: 2020

Journal Articles

Tapering Thrombopoietin Receptor Agonists in Primary Immune Thrombocytopenia: Recommendations Based on the RAND/UCLA Modified Delphi Panel Method

Adam Cuker, MD, Jenny M. Despotovic, DO, Rachael F. Grace, MD, Caroline Kruse, Michele P. Lambert, MD MSTR, Howard Liebman, MD, Roger M. Lyons, MD, Keith R McCrae, MD, Vinod A. Pullarkat, MD, Jeffrey Wasser, MD, David Beenhouwer, MD, Sarah N Gibbs, MPH, Irina Yermilov, MD MPH, MS, Michael S Broder, MD MSHS

Blood (2020) 136 (Supplement 1): 6–8.

https://doi.org/10.1182/blood-2020-133262

Published: 2020

Journal Articles

Extensive Variability in Platelet Count, Bleeding, and Quality of Life Outcome Measures in Adult and Pediatric Immune Thrombocytopenia: An Appraisal from a Critical Review of the Literature

Hanny Al-Samkari, MD, Alexandria Cronin, MLIS, Donald M. Arnold, MD MSc, Francesco Rodeghiero, MD, Rachael F. Grace, MD

Blood (2020) 136 (Supplement 1): 45–46.

https://doi.org/10.1182/blood-2020-138694

Published: 2020

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

The Effect of "Pathway" to Diagnosis for Childhood ITP on Caregiver Quality of Life at Time of Diagnosis

Michelle Neier, MD, Michele P. Lambert, MD, Rachael F. Grace, MD, Kerry Hege, MD, Stephanie Chiu, MPH, Julianne Thompson, Robert J Klaassen, MD FRCPC

Blood (2019) 134 (Supplement_1): 2174.

https://doi.org/10.1182/blood-2019-125686

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

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

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

A Phase 3 Study of Eltrombopag Vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia in Children

Kristin A. Shimano, MD, Rachael F. Grace, MD, Carolyn M. Bennett, MDMSc, Robert J Klaassen, MD FRCPC, Cindy Neunert, MD, Ellis J. Neufeld, MD, Clement Ma, PhD, Wendy B. London, PhD, Jenny M. Despotovic, DO, MS

Blood (2019) 134 (Supplement_1): 2369.

https://doi.org/10.1182/blood-2019-125487

Published: 2019

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

Targeting Immune Dysregulation in Childhood Evans Syndrome

Amanda Bell Grimes, MD, Taylor Kim, MD, Susan E. Kirk, PA-C, Michele P. Lambert, MD, Rachael F. Grace, MD, Jenny M. Despotovic, DO, MS

Blood (2018) 132 (Supplement 1): 3564.

https://doi.org/10.1182/blood-2018-99-110734

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

Journal Articles

Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

Blood (2018) 132 (Supplement 1): 4807.

https://doi.org/10.1182/blood-2018-99-113206

Published: 2018

Journal Articles

Expression Differences Distinguish Pediatric Acute and Chronic ITP Using RNA Sequencing

Taylor Kim, MD, Hyunjae Kim, PhD, Pavel Sumazin, PhD, Amanda Bell Grimes, MD, Katherine Jenson, BA, Rachael F. Grace, MD, Michele P. Lambert, MD, Jonathan Flanagan, PhD, Jenny M. Despotovic, DO, MS

Blood (2018) 132 (Supplement 1): 127.

https://doi.org/10.1182/blood-2018-99-118581

Published: 2018

Journal Articles

Genotype-Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency: Data from the PKD Natural History Study

Paola Bianchi, BSc, PhD, Elisa Fermo, PhD, Kimberly Lezon-Geyda, PhD, Patrick G. Gallagher, MD, Richard van Wijk, PhD, Eduard J. Van Beers, MD, Hassan M. Yaish, MD, Janet L. Kwiatkowski, MD, Peter E. Newburger, MD, Yaddanapudi Ravindranath, MBBS, Oliver Andres, MD, Wilma Barcellini, MD, D. Holmes Morton, MD, Bertil Glader, MD, Stefan W. Eber, MD, Satheesh Chonat, MD, Nina Kollmar, MD, Jenny M. Despotovic, DO, MS, Christine M. Knoll, MD, Kevin H.M. Kuo, MD MSc, FRCPC, Dagmar Pospisilova, Prof. MD PhD, Yves D. Pastore, MD, Alexis A Thompson, MD MPH, Winfred Wang, MD, Marcin W. Wlodarski, MD PhD, Jennifer A. Rothman, MD, Heng Wang, MD PhD, Suzanne Holzhauer, MD MSc, Vicky R. Breakey, BSc,MDFRCPC, Joachim B. Kunz, MD, Sujit Sheth, MD, Mukta Sharma, MD MPH, Melissa J. Rose, DO, Heather A. Bradeen, MD, Melissa N. McNaull, MD, Gabrielle P. Ooyama, Nolan Neu, BS, Hasan Al-Sayegh, Wendy B. London, PhD, Rachael F. Grace, MD

Blood (2017) 130 (Supplement 1): 3479.

https://doi.org/10.1182/blood.V130.Suppl_1.3479.3479

Published: 2017

Journal Articles

Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency

Blood (2017) 130 (Supplement 1): 2194.

https://doi.org/10.1182/blood.V130.Suppl_1.2194.2194

Published: 2017

NARROW

Baseline Characteristics of Patients in Peak: A Global, Longitudinal Registry of Patients with Pyruvate Kinase Deficiency

Early-Onset Osteopenia and Osteoporosis in Patients with Pyruvate Kinase Deficiency

Characteristics of Children and Adults Treated with Rituximab for Immune Thrombocytopenia (ITP)

Tapering Thrombopoietin Receptor Agonists in Primary Immune Thrombocytopenia: Recommendations Based on the RAND/UCLA Modified Delphi Panel Method

Extensive Variability in Platelet Count, Bleeding, and Quality of Life Outcome Measures in Adult and Pediatric Immune Thrombocytopenia: An Appraisal from a Critical Review of the Literature

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

The Effect of "Pathway" to Diagnosis for Childhood ITP on Caregiver Quality of Life at Time of Diagnosis

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

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

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

A Phase 3 Study of Eltrombopag Vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia in Children

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

Targeting Immune Dysregulation in Childhood Evans Syndrome

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

Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

Expression Differences Distinguish Pediatric Acute and Chronic ITP Using RNA Sequencing

Genotype-Phenotype Correlation and Molecular Heterogeneity in Pyruvate Kinase Deficiency: Data from the PKD Natural History Study

Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency

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American Society of Hematology

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ASH Publications

American Society of Hematology

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