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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

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

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

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

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

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

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

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

Potential Use of Jak2 Inhibitors to Limit Ineffective Erythropoiesis and Reverse Splenomegaly in β-Thalassemia.

Luca Melchiori, Ella Guy, Ilaria Libani, Ph.D, Raffaella Schirò, MD, Ph.D, Pedro Ramos, Laura Breda, Ph.D, Amy Chadburn, MD, Yi-Fang Liu, Maria De Sousa, MD, Eliezer Rachmilewitz, M.D, Patricia J Giardina, MD, Robert W Grady, Ph.D, Stefano Rivella, Ph.D

Blood (2008) 112 (11): 1877.

https://doi.org/10.1182/blood.V112.11.1877.1877

Published: 2008

Journal Articles

Leukocyte Apoptosis and Mitochondrial Dysfunction in β-Thalassemia Patients Treated with Deferasirox or Deferoxamine.

Patrick B. Walter, PhD, John Porter, MD, Patricia Evans, PhD, Janet L. Kwiatkowski, MD, Ellis J. Neufeld, MD, Thomas Coates, MD, Patricia J. Giardina, MD, Robert W. Grady, PhD, Elliott Vichinsky, MD, Nancy F. Olivieri, MD, Felicia Trachtenberg, PhD, Daniele Alberti, MD, Jaymes Holland, PhD, Ellen Fung, PhD, Bruce Ames, PhD, Annie Lui, BS, Paul Harmatz, MD

Blood (2007) 110 (11): 2773.

https://doi.org/10.1182/blood.V110.11.2773.2773

Published: 2007

Journal Articles

Efficacy of the Novel Oral Iron Chelator Deferitrin in Metabolic Iron Balance Studies.

Robert W. Grady, PhD, Maria Sitarou, MD, Renzo Galanello, MD, Hannah Tamary, MD, Eliana Lai, MD, Aurelio Maggio, MD, Farrukh Shah, MD, Patricia J. Giardina, MD, Joanne M. Donovan, MD, PhD

Blood (2007) 110 (11): 2775.

https://doi.org/10.1182/blood.V110.11.2775.2775

Published: 2007

Journal Articles

Accelerated Proliferation and Limited Differentiation, Mediated through Jak2, Exacerbate Ineffective Erythropoiesis in β-Thalassemia.

Ella Guy, Ilaria Libani, PhD, Luca Melchiori, Thomas Scholzen, PhD, Raffaella Schiro, MD, Pedro Ramos, Margrit Kernbach, Bettina Baron-Luehr, Laura Breda, PhD, Matteo Porotto, PhD, Maria de Sousa, MD, Maria Domenica Cappellini, MD, Patricia J. Giardina, MD, Robert W. Grady, PhD, Johannes Gerdes, PhD, Stefano Rivella, PhD

Blood (2007) 110 (11): 275.

https://doi.org/10.1182/blood.V110.11.275.275

Published: 2007

Journal Articles

Red Blood Cell Allo and Autoantibody Production in Patients in the Thalassemia Clinical Research Network.

Melody J. Cunningham, MD, Eric A. Macklin, PhD, Sylvia T. Singer, MD, Catherine S. Manno, MD, Patricia J. Giardina, MD, Nancy F. Olivieri, MD, Alexis A. Thompson, MD

Blood (2005) 106 (11): 1890.

https://doi.org/10.1182/blood.V106.11.1890.1890

Published: 2005

Journal Articles

Left Ventricular Cardiac Function during the Course of a One Year Multicenter Trial of the Safety and Efficacy with ICL670 5–40mg/kg/day and Deferoxamine 20–60 mg/kg/day in Beta-Thalassemia Patients with Transfusional Hemosideosis.

Dorothy A. Kleinert, NP, MPH, Erica Jones, MD, Cristina P. Sison, PhD, Peter Marks, MD, PhD, Patricia J. Giardina, MD

Blood (2005) 106 (11): 3853.

https://doi.org/10.1182/blood.V106.11.3853.3853

Published: 2005

NARROW

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

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

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

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.

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.

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.

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

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

Potential Use of Jak2 Inhibitors to Limit Ineffective Erythropoiesis and Reverse Splenomegaly in β-Thalassemia.

Leukocyte Apoptosis and Mitochondrial Dysfunction in β-Thalassemia Patients Treated with Deferasirox or Deferoxamine.

Efficacy of the Novel Oral Iron Chelator Deferitrin in Metabolic Iron Balance Studies.

Accelerated Proliferation and Limited Differentiation, Mediated through Jak2, Exacerbate Ineffective Erythropoiesis in β-Thalassemia.

Red Blood Cell Allo and Autoantibody Production in Patients in the Thalassemia Clinical Research Network.

Left Ventricular Cardiac Function during the Course of a One Year Multicenter Trial of the Safety and Efficacy with ICL670 5–40mg/kg/day and Deferoxamine 20–60 mg/kg/day in Beta-Thalassemia Patients with Transfusional Hemosideosis.

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American Society of Hematology

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American Society of Hematology

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