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Journal Articles

Community Health Worker Engagement with Adolescents and Young Adults with Sickle Cell Disease: Findings from the Comets Study

Jayanne Forrest, Caren Steinway, Eelia Shaw, Sophia Jan, Banu Aygun, Abena Appiah-Kubi, Nataly Apollonsky, Donna Boruchov, Omar Niss, Biree Andemariam, Kim Smith-Whitley, Symme Trachtenberg, Tanisha Belton

Blood (2024) 144 (Supplement 1): 617.

https://doi.org/10.1182/blood-2024-206036

Published: 2024

Journal Articles

Individualized, PK-Guided Dosing of Hydroxyurea for Young Children with Sickle Cell Anemia: Final Results from the Hydroxyurea Optimization through Precision Study (HOPS)

Abena Appiah-Kubi, Seethal A Jacob, Matthew M. Heeney, Connie M. Piccone, Omar Niss, Charles T. Quinn, Stephanie A. Fritch Lilla, Maa-Ohui Quarmyne, Angeli Rampersad, Allison Remiker, Amy Tang, Min Dong, Kathryn McElhinney, Joshua R. Tanzer, Patrick T. McGann

Blood (2024) 144 (Supplement 1): 175.

https://doi.org/10.1182/blood-2024-200426

Published: 2024

Journal Articles

Community Health Worker and Mobile Health Programs to Help Young Adults with Sickle Cell Disease Transition to Using Adult Healthcare Services – the Comets Study; Results from Patient-Reported Outcomes at 6 Months

Sophia Jan, Caren Steinway, Tanisha Belton, Justine Shults, Laura Bennett, Heather Griffis, Banu Aygun, Abena Appiah-Kubi, Nataly Apollonsky, Donna Boruchov, Omar Niss, Lisa A. Schwartz, Lori E. Crosby, Lamia Barakat, Biree Andemariam, David Rubin, Kim Smith-Whitley

Blood (2024) 144 (Supplement 1): 1066.

https://doi.org/10.1182/blood-2024-205273

Published: 2024

Journal Articles

Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub

Omar Niss, Matthew Fenchel, Erin Kingsley, Teresa S. Latham, Punam Malik, Charles T. Quinn

Blood (2024) 144 (Supplement 1): 2312.

https://doi.org/10.1182/blood-2024-206015

Published: 2024

Journal Articles

Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease

Omar Niss, Cara Morin, Sean M Lang, Tarek Alsaied, MacKenzie Tasset, Punam Malik, Charles T. Quinn

Blood (2024) 144 (Supplement 1): 799.

https://doi.org/10.1182/blood-2024-200951

Published: 2024

Journal Articles

Genetic Variants in Canonical Wnt Signaling Pathway Associated with Pediatric ITP

Taylor Olmsted Kim, Jennifer M. Geris, Amanda B. Grimes, Rachael F. Grace, Michele P. Lambert, Melissa J. Rose, Kristin A. Shimano, Omar Niss, Cindy Neunert, Taizo A. Nakano, Derek Macmath, Bogdan Dinu, Susan E. Kirk, Jenny M. Despotovic, Jonathan M. Flanagan, Michael E. Scheurer

Blood (2023) 142 (Supplement 1): 2593.

https://doi.org/10.1182/blood-2023-175013

Published: 2023

Journal Articles

Improving Transition of Emerging Adults with Sickle Cell Disease to Adult Care through a Multidisciplinary Process: The Development of a Transition Clinic to Support Transition Success

Lynette Fenchel, Felisha Jackson, Brittany Walker, Chandra Manuel, Dajia Hooks, Tyesha Allen, Mamie Myo Thant, Kristine Karkoska, Luke R. Smart, Naomi E Joffe, Omar Niss

Blood (2023) 142 (Supplement 1): 5055.

https://doi.org/10.1182/blood-2023-177747

Published: 2023

Journal Articles

Congenital Dyserythropoietic Anemia Type II: An Update from the Congenital Dyseryhtropoietic Anemia Registry of North America (CDAR)

Yasmin Elgammal, Mary Risinger, Ammar Husami, Joseph Walden, Sweta Gupta, Niketa C. Shah, James Boyer, Yasmina L. Abajas, Michael Winstead, David W. Miller, Viviana Vidal-Anaya, Adrianna Vlachos, Ashutosh Lal, Arthur K. Ritchey, Robert Lorsbach, Wenying Zhang, Theodosia A. Kalfa, Omar Niss

Blood (2023) 142 (Supplement 1): 1079.

https://doi.org/10.1182/blood-2023-189389

Published: 2023

Journal Articles

Individualized, PK-Guided Dosing of Hydroxyurea Is Not Associated with Increased Hematologic Toxicity Compared to Weight-Based Initial Dosing: Interim Results from the Hops Trial

Abena Appiah-Kubi, Seethal A Jacob, Matthew M. Heeney, Clark Brown, Susan E. Creary, Christine R Hollenkamp, Emily Riehm Meier, Omar Niss, Connie M. Piccone, Maa-Ohui Quarmyne, Charles T. Quinn, Allison Remiker, Kay L Saving, Min Dong, Patrick T. McGann

Blood (2022) 140 (Supplement 1): 5416–5417.

https://doi.org/10.1182/blood-2022-158623

Published: 2022

Journal Articles

Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia

Omar Niss, Jon Detterich, John C. Wood, Thomas D. Coates, Punam Malik, Michael D. Taylor, Charles T. Quinn

Blood (2022) 140 (11): 1322–1324.

https://doi.org/10.1182/blood.2021015303

Published: 2022

Includes: Supplemental data

Journal Articles

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

Michael Grimley, Monika Asnani, Archana Shrestha, Sydney Felker, Carolyn Lutzko, Paritha I. Arumugam, Scott Witting, Jennifer Knight-Madden, Omar Niss, Charles T. Quinn, Chris Lo, Courtney R. Little, Alisa Dong, Punam Malik

Blood (2021) 138 (Supplement 1): 3970.

https://doi.org/10.1182/blood-2021-147469

Published: 2021

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

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

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

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

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

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

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

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

NARROW

Community Health Worker Engagement with Adolescents and Young Adults with Sickle Cell Disease: Findings from the Comets Study

Individualized, PK-Guided Dosing of Hydroxyurea for Young Children with Sickle Cell Anemia: Final Results from the Hydroxyurea Optimization through Precision Study (HOPS)

Community Health Worker and Mobile Health Programs to Help Young Adults with Sickle Cell Disease Transition to Using Adult Healthcare Services – the Comets Study; Results from Patient-Reported Outcomes at 6 Months

Underutilization of Disease-Modifying Therapies in Sickle Cell Disease: A Real-World Analysis from the ASH Research Collaborative Data Hub

Longitudinal Assessment of Myocardial Fibrosis in Sickle Cell Disease

Genetic Variants in Canonical Wnt Signaling Pathway Associated with Pediatric ITP

Improving Transition of Emerging Adults with Sickle Cell Disease to Adult Care through a Multidisciplinary Process: The Development of a Transition Clinic to Support Transition Success

Congenital Dyserythropoietic Anemia Type II: An Update from the Congenital Dyseryhtropoietic Anemia Registry of North America (CDAR)

Individualized, PK-Guided Dosing of Hydroxyurea Is Not Associated with Increased Hematologic Toxicity Compared to Weight-Based Initial Dosing: Interim Results from the Hops Trial

Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia

Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning

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

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

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

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

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

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

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

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

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American Society of Hematology

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