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Journal Articles

Rapid and Automated Quantitation of Dense Red Blood Cells: A Robust Biomarker of Therapeutic Response to Early Initiation of Hydroxyurea in Young Children with Sickle Cell Anemia

Alina Sadaf, MD, Charles T. Quinn, MD, Jennifer B Korpik, Amanda Pfeiffer, BS, MA, Mary Reynaud, MA, MT(ASCP), Omar Niss, MD, Punam Malik, MD, Russell E. Ware, MD PhD, Theodosia A. Kalfa, MD PhD, Patrick T. McGann, MD MS

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-137070

Published: 2020

Journal Articles

Increased Hydroxyurea Prescribing Practices over Ten Years with Improved Clinical Outcomes in Children with Sickle Cell Anemia: A Single Center's Experience

Kristine Anne Karkoska, MD, Kevin E Todd, Jr., MD, Kelly Clapp, NP, Lynette Fenchel, NP, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Omar Niss, MD, Charles T. Quinn, MD, Russell E. Ware, MD PhD, Patrick T. McGann, MD MS

Blood (2020) 136 (Supplement 1): 34.

https://doi.org/10.1182/blood-2020-138754

Published: 2020

Journal Articles

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Michael Grimley, MD, Monika Asnani, PhD, Archana Shrestha, Sydney Felker, Carolyn Lutzko, PhD, Paritha I. Arumugam, PhD, Scott Witting, PhD, Jennifer Knight-Madden, MBBS, PhD, Omar Niss, MD, Charles T. Quinn, MD, Chris Lo, BS, Courtney R Little, BSN, Joseph W. McIntosh, MD, Punam Malik, MD

Blood (2020) 136 (Supplement 1): 20–21.

https://doi.org/10.1182/blood-2020-140963

Published: 2020

Journal Articles

Progression of Albuminuria in Sickle Cell Anemia: A Multicenter, Longitudinal Study

Omar Niss, MD, Adam Lane, PhD, Monika Asnani, PhD, Marianne McPherson Yee, MD MSc, Ashok Raj, Susan E Creary, MD, Courtney D. Fitzhugh, MD, Prasad V. Bodas, MD, Santosh L. Saraf, MD, Sharada Sarnaik, MD, Charles T. Quinn, MD, Prasad Devarajan, MD, Punam Malik, MD

Blood (2019) 134 (Supplement_1): 1004.

https://doi.org/10.1182/blood-2019-125860

Published: 2019

Journal Articles

Congenital Dyserythropoietic Anemia Type I Due to Biallelic CDAN1 mutations: Report from the Congenital Dyserythropoietic Anemia Registry (CDAR)

Omar Niss, MD, Robert B. Lorsbach, MD PhD, David K. Buchbinder, MD MSHS, Satheesh Chonat, MD, Morgan L. McLemore, MD, Timothy McCavit, MD, Jeffrey H Schwartz, MD, Jessica Meznarich, MD, Katie Seu, Wenying Zhang, MD PhD, Theodosia A. Kalfa, MD PhD

Blood (2019) 134 (Supplement_1): 3521.

https://doi.org/10.1182/blood-2019-128975

Published: 2019

Journal Articles

VPS4A mutations Cause a Syndrome with Dyserythropoiesis, Hemolytic Anemia, and Neurodevelopmental Delay

Katie Giger Seu, PhD, Lisa Trump, PhD, Sana Emberesh, MBBS, Robert B. Lorsbach, MD PhD, Clarissa E. Johnson, MD, Jessica Meznarich, MD, Hunter Underhill, MD PhD, Haripriya Sakthivel, Omar Niss, MD, Shannon Nortman, Lionel Blanc, PhD, Wenying Zhang, MD PhD, Carolyn Lutzko, PhD, Theodosia A. Kalfa, MD PhD

Blood (2019) 134 (Supplement_1): 339.

https://doi.org/10.1182/blood-2019-128230

Published: 2019

Journal Articles

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Charles T. Quinn, MD, Omar Niss, MD, Amanda Pfeiffer, Jennifer Korpik, Holly Bonar, Mary Reynaud, Russell E. Ware, MD PhD, Patrick T. McGann, MD

Blood (2019) 134 (Supplement_1): 892.

https://doi.org/10.1182/blood-2019-130414

Published: 2019

Journal Articles

Clinical and Laboratory Benefits of Early Initiation of Hydroxyurea with Pharmacokinetic Guided Dosing for Young Children with Sickle Cell Anemia

Patrick T. McGann, MD, Omar Niss, MD, Min Dong, PhD, Anu Marahatta, PhD, Tomoyuki Mizuno, PhD, Karen Kalinyak, MD, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Charles T. Quinn, MD, Russell E. Ware, MD PhD, Alexander A. Vinks, PharmD, PhD FCP

Blood (2018) 132 (Supplement 1): 507.

https://doi.org/10.1182/blood-2018-99-112909

Published: 2018

Journal Articles

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Blood (2018) 132 (Supplement 1): 1021.

https://doi.org/10.1182/blood-2018-99-119591

Published: 2018

Journal Articles

Evaluation of Phenotype-Genotype Correlation in Two Common PIEZO1 Mutations p.R2456H and p.L2495_E2495dup

Mary Risinger, PhD, Vandy Black, MDMSc, Loan Hsieh, MD, Renee C. Prins, MD, Jill Menell, MD, Mohamad Badawi, MD, Cynthia Rutherford, MD, Karen L. Bride, MD PhD, Omar Niss, MD, Charles T. Quinn, MD, Katie Giger Seu, PhD, Wenying Zhang, MDPhD, Theodosia A. Kalfa, MD PhD

Blood (2018) 132 (Supplement 1): 1040.

https://doi.org/10.1182/blood-2018-99-117299

Published: 2018

Journal Articles

Diastolic Dysfunction Is Associated with Impaired Cardiopulmonary Exercise Performance in Sickle Cell Disease

Tarek Alsaied, MD MSc, Omar Niss, MD, Adam Powell, MD, Robert Fleck, MD, Punam Malik, MD, Clifford Chin, MD, Charles T. Quinn, MD MS, Michael D Taylor

Blood (2017) 130 (Supplement 1): 3547.

https://doi.org/10.1182/blood.V130.Suppl_1.3547.3547

Published: 2017

Journal Articles

Temporal Evolution of Diffuse Myocardial Fibrosis in Sickle Cell Anemia

Charles T. Quinn, MD MS, Omar Niss, MD, Robert Fleck, MD, Tarek Alsaied, MD MSc, Punam Malik, MD, Michael D. Taylor, MD

Blood (2017) 130 (Supplement 1): 3539.

https://doi.org/10.1182/blood.V130.Suppl_1.3539.3539

Published: 2017

Journal Articles

Personalized Hydroxyurea Dosing to Reduce Time to MTD and Optimize the HbF Response: Results from the TREAT Study

Patrick T. McGann, MD MS, Omar Niss, MD, Min Dong, PhD, Anu Marahatta, PhD, Kathryn L McElhinney, BS, Thad A. Howard, MS, Tomoyuki Mizuno, PhD, Theodosia A. Kalfa, MD PhD, Karen Kalinyak, MD, Punam Malik, MD, Charles T. Quinn, MD MS, Russell E. Ware, MD PhD, Alexander A. Vinks, PharmD, PhD FCP

Blood (2017) 130 (Supplement 1): 615.

https://doi.org/10.1182/blood.V130.Suppl_1.615.615

Published: 2017

Journal Articles

Prevention of Acute Chest Syndrome By Implementing a Standardized Process to Improve Incentive Spirometry Use in Hospitalized Patients with Sickle Cell Disease

Omar Niss, MD, Cheri Cole-Jenkins, Blair Davis, Tina Brooks, Katherine Woolery, Teri Fetters, Jennifer Rollins, Patrick T. McGann, MD MS, Karen Kalinyak, MD

Blood (2017) 130 (Supplement 1): 132.

https://doi.org/10.1182/blood.V130.Suppl_1.132.132

Published: 2017

Journal Articles

The First Registry for Patients with Congenital Dyserythropoietic Anemia in North America: Design and Preliminary Results

Blood (2017) 130 (Supplement 1): 2210.

https://doi.org/10.1182/blood.V130.Suppl_1.2210.2210

Published: 2017

Journal Articles

RGL2 Deficiency Impairs Human Erythropoiesis By Altering Terminal Erythroid Differentiation and Apoptosis

Harry Lesmana, MD, Georgios E Christakopoulos, MD, Mary Risinger, Haripriya Sakthivel, Omar Niss, MD, Theodosia A. Kalfa, MD PhD

Blood (2017) 130 (Supplement 1): 8.

https://doi.org/10.1182/blood.V130.Suppl_1.8.8

Published: 2017

Journal Articles

VPS4A : A Novel Candidate Gene for Congenital Dyserythropoietic Anemia

Katie Giger, PhD, Georgios E Christakopoulos, MD, Lisa Trump, Clarissa E. Johnson, MD, Haripriya Sakthivel, Mary Risinger, Omar Niss, MD, Kejian Zhang, MD MBA, Lionel Blanc, PhD, Robert B. Lorsbach, MD PhD, Paritha I. Arumugam, PhD, Carolyn Lutzko, PhD, Theodosia A. Kalfa, MD PhD

Blood (2017) 130 (Supplement 1): 923.

https://doi.org/10.1182/blood.V130.Suppl_1.923.923

Published: 2017

Journal Articles

Individualized Dosing of Hydroxyurea for Children with Sickle Cell Anemia Using a Population Pharmacokinetic-Based Model: The TREAT Study

Patrick T. McGann, MD MS, Min Dong, PhD, Anu Marahatta, PhD, Thad A. Howard, MS, Tomoyuki Mizuno, PhD, Karen A. Kalinyak, MD, Theodosia A. Kalfa, MD PhD, Punam Malik, MD, Omar Niss, MD, Charles T Quinn, MD, Russell E. Ware, MD PhD, Alexander A. Vinks, PharmD, PhD

Blood (2016) 128 (22): 3652.

https://doi.org/10.1182/blood.V128.22.3652.3652

Published: 2016

Journal Articles

Diffuse Myocardial Fibrosis Is a Common Feature of Sickle Cell Anemia That Is Associated with Diastolic Dysfunction and Restrictive Cardiac Physiology

Omar Niss, MD, Michael D. Taylor, MD, Robert Fleck, MD, Tarek Alsaied, MD, Jeffrey Towbin, MD, Punam Malik, MD, Charles T. Quinn, MD MS

Blood (2016) 128 (22): 8.

https://doi.org/10.1182/blood.V128.22.8.8

Published: 2016

Journal Articles

Biochemical Surrogate Markers of Hemolysis Do Not Correlate with Directly Measured Erythrocyte Survival in Sickle Cell Anemia

Charles T. Quinn, MD MS, Eric P. Smith, MD, Shahriar Arbabi, Paramjit Khera, PhD, Christopher J. Lindsell, PhD, Omar Niss, MD, Clinton H Joiner, MD PhD, Robert S. Franco, PhD, Robert M. Cohen, MD

Blood (2016) 128 (22): 2484.

https://doi.org/10.1182/blood.V128.22.2484.2484

Published: 2016

NARROW

Rapid and Automated Quantitation of Dense Red Blood Cells: A Robust Biomarker of Therapeutic Response to Early Initiation of Hydroxyurea in Young Children with Sickle Cell Anemia

Increased Hydroxyurea Prescribing Practices over Ten Years with Improved Clinical Outcomes in Children with Sickle Cell Anemia: A Single Center's Experience

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Progression of Albuminuria in Sickle Cell Anemia: A Multicenter, Longitudinal Study

Congenital Dyserythropoietic Anemia Type I Due to Biallelic CDAN1 mutations: Report from the Congenital Dyserythropoietic Anemia Registry (CDAR)

VPS4A mutations Cause a Syndrome with Dyserythropoiesis, Hemolytic Anemia, and Neurodevelopmental Delay

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Clinical and Laboratory Benefits of Early Initiation of Hydroxyurea with Pharmacokinetic Guided Dosing for Young Children with Sickle Cell Anemia

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Evaluation of Phenotype-Genotype Correlation in Two Common PIEZO1 Mutations p.R2456H and p.L2495_E2495dup

Diastolic Dysfunction Is Associated with Impaired Cardiopulmonary Exercise Performance in Sickle Cell Disease

Temporal Evolution of Diffuse Myocardial Fibrosis in Sickle Cell Anemia

Personalized Hydroxyurea Dosing to Reduce Time to MTD and Optimize the HbF Response: Results from the TREAT Study

Prevention of Acute Chest Syndrome By Implementing a Standardized Process to Improve Incentive Spirometry Use in Hospitalized Patients with Sickle Cell Disease

The First Registry for Patients with Congenital Dyserythropoietic Anemia in North America: Design and Preliminary Results

RGL2 Deficiency Impairs Human Erythropoiesis By Altering Terminal Erythroid Differentiation and Apoptosis

VPS4A : A Novel Candidate Gene for Congenital Dyserythropoietic Anemia

Individualized Dosing of Hydroxyurea for Children with Sickle Cell Anemia Using a Population Pharmacokinetic-Based Model: The TREAT Study

Diffuse Myocardial Fibrosis Is a Common Feature of Sickle Cell Anemia That Is Associated with Diastolic Dysfunction and Restrictive Cardiac Physiology

Biochemical Surrogate Markers of Hemolysis Do Not Correlate with Directly Measured Erythrocyte Survival in Sickle Cell Anemia

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American Society of Hematology

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American Society of Hematology

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