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Journal Articles

The Phenotypic Spectrum of Pyruvate Kinase Deficiency (PKD) from the PKD Natural History Study (NHS): Description of Four Severity Groups By Anemia Status

Blood (2015) 126 (23): 2136.

https://doi.org/10.1182/blood.V126.23.2136.2136

Published: 2015

Journal Articles

Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New Variants

Blood (2015) 126 (23): 3337.

https://doi.org/10.1182/blood.V126.23.3337.3337

Published: 2015

Journal Articles

Epidemiologic and Clinical Characteristics of Thalassemia (Thal) Intermedia (TI) in the United States

Elliott P. Vichinsky, MD, Janet Kwiatkowski, MD, Patricia J. Giardina, MD, Carole Paley, MD, Francis Vekeman, MA, Wendy Y. Cheng, MPH, MPhil, Joseph Damron, BA, Nora McCormick, MSc, Medha Sasane, PhDBPharm,MS, Ying Qiu, PhD, Mei Sheng Duh, MPH, ScD, Alexis A. Thompson, MD

Blood (2015) 126 (23): 3279.

https://doi.org/10.1182/blood.V126.23.3279.3279

Published: 2015

Journal Articles

A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD)

Blood (2013) 122 (1): 112–123.

https://doi.org/10.1182/blood-2012-08-439083

Published: 2013

Includes: Supplemental data

Journal Articles

Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort

Janet L. Kwiatkowski, Hae-Young Kim, Alexis A. Thompson, Charles T. Quinn, Brigitta U. Mueller, Isaac Odame, Patricia J. Giardina, Elliott P. Vichinsky, Jeanne M. Boudreaux, Alan R. Cohen, John B. Porter, Thomas Coates, Nancy F. Olivieri, Ellis J. Neufeld, for the Thalassemia Clinical Research Network

Blood (2012) 119 (12): 2746–2753.

https://doi.org/10.1182/blood-2011-04-344507

Published: 2012

Includes: Supplemental data

Journal Articles

Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report

Claudia R. Morris, Hae-Young Kim, Felicia Trachtenberg, John Wood, Charles T. Quinn, Nancy Sweeters, Janet L. Kwiatkowski, Alexis A. Thompson, Patricia J. Giardina, Jeanne Boudreaux, Nancy F. Olivieri, John B. Porter, Ellis J. Neufeld, Elliott P. Vichinsky

Blood (2011) 118 (14): 3794–3802.

https://doi.org/10.1182/blood-2010-11-319152

Published: 2011

Journal Articles

How I treat thalassemia

Eliezer A. Rachmilewitz, Patricia J. Giardina

Blood (2011) 118 (13): 3479–3488.

https://doi.org/10.1182/blood-2010-08-300335

Published: 2011

Journal Articles

Serial Measurements of Cardiac and Hepatic Iron with MRI T2* In Transfusion Dependent Patients with Beta-Thalassemia Major Receiving Deferasirox

Srikanth R. Ambati, MBBS, MD, Dorothy A. Kleinert, NP, MPH, Patricia J. Giardina, MD

Blood (2010) 116 (21): 2051.

https://doi.org/10.1182/blood.V116.21.2051.2051

Published: 2010

Journal Articles

Optimizing the Use of Deferasirox: Evidence of Synergy When Combined with Deferoxamine

Robert W Grady, PhD, Renzo Galanello, MD, Dorothy A Kleinert, NP, MPH, Carole S. Paley, MD, Patricia J. Giardina, MD

Blood (2010) 116 (21): 5163.

https://doi.org/10.1182/blood.V116.21.5163.5163

Published: 2010

Journal Articles

Bone loss caused by iron overload in a murine model: importance of oxidative stress

Jaime Tsay, Zheiwei Yang, F. Patrick Ross, Susanna Cunningham-Rundles, Hong Lin, Rhima Coleman, Philipp Mayer-Kuckuk, Stephen B. Doty, Robert W. Grady, Patricia J. Giardina, Adele L. Boskey, Maria G. Vogiatzi

Blood (2010) 116 (14): 2582–2589.

https://doi.org/10.1182/blood-2009-12-260083

Published: 2010

Includes: Supplemental data

Journal Articles

Renal Dysfunction in Thalassemia.

Charles T Quinn, MD, MS, Valerie L Johnson, MD, PhD, Hae-Young Kim, MS, DrPh, Felicia Trachtenberg, PhD, Maria G Vogiatzi, MD, Janet L Kwiatkowski, MD, MSCE, Ellis J Neufeld, MD, PhD, Ellen Fung, PhD, RD, Nancy F Olivieri, MD, Melanie Kirby-Allen, MD, Patricia J Giardina, MD

Blood (2009) 114 (22): 2008.

https://doi.org/10.1182/blood.V114.22.2008.2008

Published: 2009

Journal Articles

Pulmonary Hypertension in Thalassemia Assessed by Echocardiography: A Report From Baseline Data of the Thalassemia Clinical Research Network Longitudinal Cohort Study.

Claudia R. Morris, MD, Hae-Young Kim, MS, DrPh, Felicia Trachtenberg, PhD, Charles T. Quinn, MD, MS, Nancy F Olivieri, MD, Nancy Sweeters, RN, PNP, Patricia J Giardina, MD, Janet L Kwiatkowski, MD, MSCE, Alexis A. Thompson, MD, Jeanne Boudreaux, MD, Ellis J Neufeld, MD, PhD, Elliott P Vichinsky, MD

Blood (2009) 114 (22): 2016.

https://doi.org/10.1182/blood.V114.22.2016.2016

Published: 2009

Journal Articles

Following Beta-Globin Gene Transfer, the Production of Hemoglobin Depends Upon the Beta-Thalassemia Genotype.

Laura Breda, PhD, Carla Casu, PhD, Laura Casula, PhD, Dorothy A. Kleinert, NP, MPH, Nicoletta Bianchi, PhD, Evgenia Prus, PhD, Luca Cartegni, PhD, Eitan Fibach, PhD, Lawrence B. Gardner, MD, Patricia J Giardina, MD, Roberto Gambari, PhD, Stefano Rivella, PhD

Blood (2009) 114 (22): 978.

https://doi.org/10.1182/blood.V114.22.978.978

Published: 2009

Journal Articles

Chelation Choices and Iron Burden Among Patients with Thalassemia in the 21st Century: a Report From the Thalassemia Clinical Research Network (TCRN) Longitudinal Cohort.

Janet L Kwiatkowski, MD, MSCE, Hae-Young Kim, MS, DrPh, Alexis A. Thompson, Charles T. Quinn, MD, MS, Brigitta U Mueller, Isaac Odame, MBChB, Patricia J Giardina, MD, Elliott P Vichinsky, MD, Jeanne Boudreaux, MD, John B Porter, Thomas Coates, Nancy F Olivieri, MD, Ellis J Neufeld, MD, PhD

Blood (2009) 114 (22): 4056.

https://doi.org/10.1182/blood.V114.22.4056.4056

Published: 2009

Journal Articles

Symptoms of Anxiety and Depression Among Teens and Adults in the Thalassemia Longitudinal Cohort Study.

Lauren Mednick, PhD, Shuli Yu, PhD, Felicia Trachtenberg, PhD, Dorothy A. Kleinert, NP, MPH, Patricia J Giardina, MD, Janet L Kwiatkowski, MD, MSCE, Dru Foote, RN, PNP, Vivekanandan Thayalasuthan, MD, John B Porter, MD, Alexis A. Thompson, MD, Leann Schilling, MPH, Charles T. Quinn, MD, MS, Ellis J Neufeld, MD, PhD, Robert Yamashita, PhD

Blood (2009) 114 (22): 555.

https://doi.org/10.1182/blood.V114.22.555.555

Published: 2009

Journal Articles

Quality of Life in Adolescents and Adults with Thalassemia: A Report of the Thalassemia Longitudinal Cohort.

Amy Sobota, MD, Robert Yamashita, PhD, Yan Xu, PhD, Felicia Trachtenberg, PhD, Dorothy A. Kleinert, NP, MPH, Patricia J Giardina, MD, Janet L Kwiatkowski, MD, MSCE, Dru Foote, RN, PNP, Vivekanandan Thayalasuthan, MD, John B Porter, Alexis A. Thompson, MD, Leann Schilling, MPH, Charles T. Quinn, MD, MS, Ellis J Neufeld, MD, PhD

Blood (2009) 114 (22): 556.

https://doi.org/10.1182/blood.V114.22.556.556

Published: 2009

Journal Articles

Initial Liver Iron Predicts Cardiac Chelation Efficacy of Deferasirox (Exjade®) Monotherapy in Chronically Transfused β-Thalassemia (β-Thal) Patients: 18- and 24-Month Data.

John C. Wood, Alexis A. Thompson, Carole Paley, Tara Glynos, Barinder Kang, Patricia J. Giardina, Paul R. Harmatz, Thomas Coates

Blood (2009) 114 (22): 4069.

https://doi.org/10.1182/blood.V114.22.4069.4069

Published: 2009

Journal Articles

Iron Chelation with Deferasirox in Thalassemia Major Patients with Low Iron Burdens and Moderate to High Transfusion Regimens.

Elizabeth Evans, Dorothy A. Kleinert, NP, Kevin Mennitt, MD, Robert W. Grady, PhD, Patricia J. Giardina, MD

Blood (2009) 114 (22): 4070.

https://doi.org/10.1182/blood.V114.22.4070.4070

Published: 2009

Journal Articles

The Impact of the Child with Thalassemia On the Family: Parental Assessment by Child Health Questionnaire.

Robert Yamashita, PhD, Amy Sobota, MD, Felicia Trachtenberg, PhD, Yan Xu, MS, Zahra Pakbaz, MD, Isaac Odame, MBChB, Alexis A. Thompson, MD, Dru Foote, RN, PNP, Dorothy A. Kleinert, NP, MPH, Patricia J Giardina, MD, Charles T. Quinn, MD, MS, Janet L Kwiatkowski, MD, MSCE, Ellis J Neufeld, MD, PhD, Leann Schilling, MPH, Jeanne Boudreaux, MD

Blood (2009) 114 (22): 1371.

https://doi.org/10.1182/blood.V114.22.1371.1371

Published: 2009

Journal Articles

Use of Jak2 Inhibitors to Limit Ineffective Erythropoiesis and Iron Absorption in Mice Affected by β-Thalassemia and Other Disorders of Red Cell Production.

Luca Melchiori, Sara Gardenghi, PhD, Ella C. Guy, Eliezer Rachmilewitz, M.D, Patricia J Giardina, MD, Robert W. Grady, PhD, Mohandas Narla, DSc, Xiuli An, MD, PhD, Stefano Rivella, PhD

Blood (2009) 114 (22): 2020.

https://doi.org/10.1182/blood.V114.22.2020.2020

Published: 2009

NARROW

The Phenotypic Spectrum of Pyruvate Kinase Deficiency (PKD) from the PKD Natural History Study (NHS): Description of Four Severity Groups By Anemia Status

Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New Variants

Epidemiologic and Clinical Characteristics of Thalassemia (Thal) Intermedia (TI) in the United States

A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD)

Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort

Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report

How I treat thalassemia

Serial Measurements of Cardiac and Hepatic Iron with MRI T2* In Transfusion Dependent Patients with Beta-Thalassemia Major Receiving Deferasirox

Optimizing the Use of Deferasirox: Evidence of Synergy When Combined with Deferoxamine

Bone loss caused by iron overload in a murine model: importance of oxidative stress

Renal Dysfunction in Thalassemia.

Pulmonary Hypertension in Thalassemia Assessed by Echocardiography: A Report From Baseline Data of the Thalassemia Clinical Research Network Longitudinal Cohort Study.

Following Beta-Globin Gene Transfer, the Production of Hemoglobin Depends Upon the Beta-Thalassemia Genotype.

Chelation Choices and Iron Burden Among Patients with Thalassemia in the 21st Century: a Report From the Thalassemia Clinical Research Network (TCRN) Longitudinal Cohort.

Symptoms of Anxiety and Depression Among Teens and Adults in the Thalassemia Longitudinal Cohort Study.

Quality of Life in Adolescents and Adults with Thalassemia: A Report of the Thalassemia Longitudinal Cohort.

Initial Liver Iron Predicts Cardiac Chelation Efficacy of Deferasirox (Exjade®) Monotherapy in Chronically Transfused β-Thalassemia (β-Thal) Patients: 18- and 24-Month Data.

Iron Chelation with Deferasirox in Thalassemia Major Patients with Low Iron Burdens and Moderate to High Transfusion Regimens.

The Impact of the Child with Thalassemia On the Family: Parental Assessment by Child Health Questionnaire.

Use of Jak2 Inhibitors to Limit Ineffective Erythropoiesis and Iron Absorption in Mice Affected by β-Thalassemia and Other Disorders of Red Cell Production.

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American Society of Hematology

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American Society of Hematology

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