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Journal Articles

Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Blood (2025) 146 (Supplement 1): 6089.

https://doi.org/10.1182/blood-2025-6089

Published: 2025

Journal Articles

Real-world comparative effects of curative and disease-modifying therapies on ineffective erythropoiesis in beta-thalassemia

Carlo Castruccio Castracani, Madison Bright, Jaladhikumar Patel, Scott Peslak, Aaron Cheng, Laura Breda, Stefano Rivella, Janet Kwiatkowski

Blood (2025) 146 (Supplement 1): 176.

https://doi.org/10.1182/blood-2025-176

Published: 2025

Journal Articles

Accurate and automated multi-center TCD velocity analysis using natural language processing

Sahal Master, Charles Bailey, David Brousseau, Janet Kwiatkowski, Lauren Beslow, Aleksandra Dain

Blood (2025) 146 (Supplement 1): 175.

https://doi.org/10.1182/blood-2025-175

Published: 2025

Journal Articles

CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial

Blood (2025) 146 (Supplement 1): 4314.

https://doi.org/10.1182/blood-2025-4314

Published: 2025

Journal Articles

Rapid immune reconstitution in patients with transfusion-dependent thalassemia following betibeglogene autotemcel: A real-world experience

Benjamin Philipson, Jonathan Tedesco, Rheanna Congdon, Dominic Duah, Elizabeth Worster, Yi Qi, Sam Barnett Dubensky, Nora Gibson, Janet Kwiatkowski, Laura Vella, Timothy Olson

Blood (2025) 146 (Supplement 1): 4442.

https://doi.org/10.1182/blood-2025-4442

Published: 2025

Journal Articles

Development of a prediction model for maternal pregnancy complications in sickle cell disease

Aaron Cheng, Phyllis Gimotty, David Margolis, Scott Peslak, Hanny Al-Samkari, Andrea Roe, Janet Kwiatkowski

Blood (2025) 146 (Supplement 1): 1091.

https://doi.org/10.1182/blood-2025-1091

Published: 2025

Journal Articles

Efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency who are not regularly transfused: Results from the Phase 3, global, randomized, double-blind, placebo-controlled ACTIVATE-Kids trial

Blood (2025) 146 (Supplement 1): 4654.

https://doi.org/10.1182/blood-2025-4654

Published: 2025

Journal Articles

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Alexis A. Thompson, MD MPH, Janet Kwiatkowski, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD FAAP, MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Alexandria Petrusich, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2016) 128 (22): 1175.

https://doi.org/10.1182/blood.V128.22.1175.1175

Published: 2016

Journal Articles

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew M. Hsieh, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Marcelyne Joseney-Antoine, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, John F. Tisdale, MD

Blood (2016) 128 (22): 1176.

https://doi.org/10.1182/blood.V128.22.1176.1176

Published: 2016

Journal Articles

Iron Unloading By Therapeutic Phlebotomy in Previously Transfused Children with Sickle Cell Anemia: The Twitch Experience

Banu Aygun, MD, Nicole Mortier, PA-C, Zora R. Rogers, MD, William Owen, MD, Beng Fuh, MD, Alex George, MD, PhD, Theodosia A. Kalfa, MD PhD, Janet Kwiatkowski, MD, Margaret Lee, MD, Hamayun Imran, MD, Scott T. Miller, MD, John C Wood, MD PhD, Alan R. Cohen, MD, Sara L Pressel, MS, Russell E. Ware, MD PhD

Blood (2016) 128 (22): 1018.

https://doi.org/10.1182/blood.V128.22.1018.1018

Published: 2016

Journal Articles

Impact of TCD Screening Protocol on the Incidence of Hemorrhagic Stroke in Children and Young Adults with Sickle Cell Disease

Julie Kanter, MD, Janet Kwiatkowski, MD, Heather J. Fullerton, MD, Jenifer Voeks, Ellen Debenham, RN, Lynette J Brown, Robert J. Adams, MD MS

Blood (2015) 126 (23): 3402.

https://doi.org/10.1182/blood.V126.23.3402.3402

Published: 2015

Journal Articles

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew Hsieh, MD, Alexis A. Thompson, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Laura Sandler, MPH, Sandeep Soni, MD, John F. Tisdale, MD

Blood (2015) 126 (23): 3233.

https://doi.org/10.1182/blood.V126.23.3233.3233

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

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Mark C. Walters, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet Kwiatkowski, MD, Gary J Schiller, MD, Morris Kletzel, MD, P. Joy Ho, MBBS, DPhil, Elliott Vichinsky, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Alexis A. Thompson, MD

Blood (2015) 126 (23): 201.

https://doi.org/10.1182/blood.V126.23.201.201

Published: 2015

Journal Articles

TCD with Transfusions Changing to Hydroxyurea (TWiTCH): Hydroxyurea Therapy As an Alternative to Transfusions for Primary Stroke Prevention in Children with Sickle Cell Anemia

Russell E. Ware, MD PhD, Barry R Davis, MD PhD, William H Schultz, MHS PA, Clark Brown, MD PhD, Banu Aygun, MD, Sharada A. Sarnaik, MD, Isaac Odame, MD, Beng Fuh, MD, Alex George, MD, PhD, William Owen, MD, Lori Luchtman-Jones, MD, Zora R. Rogers, MD, Lee Hilliard, MD, Cynthia Gauger, MD, Connie M. Piccone, MD, Margaret T. Lee, MD, Janet Kwiatkowski, MD, Sherron Jackson, MD, Scott T. Miller, MD, Carla W Roberts, MD, Matthew M. Heeney, MD FRCP(C), Theodosia A. Kalfa, MD PhD, Stephen C Nelson, MD, Hamayun Imran, MD, Kerri A Nottage, MD MPH, Ofelia A. Alvarez, MD, Melissa Rhodes, MD, Alexis A. Thompson, MD, Jennifer Rothman, MD, Kathleen J. Helton, MD, Donna Roberts, MD, Jamie Coleman, MD, Melanie J Bonner, PhD, Abdullah Kutlar, MD, Niren Patel, MD, John C Wood, MD PhD, Linda Piller, MD, Peng Wei, PhD, Judy Luden, CCRP, Nicole A. Mortier, MHS, PA-C, Susan Stuber, MA, Naomi L.C. Luban, MD, Alan R. Cohen, MD, Sara L Pressel, MS, Robert J. Adams, MD MS

Blood (2015) 126 (23): 3.

https://doi.org/10.1182/blood.V126.23.3.3

Published: 2015

Journal Articles

Liver Iron Concentration By MRI In Chronically Transfused Children With Sickle Cell Anemia In The Twitch Trial

John C. Wood, MD, PhD, Zora R. Rogers, MD, Isaac Odame, MD, Janet Kwiatkowski, MD, Margaret Lee, MD, William C. Owen, MD, Alan Cohen, MD, Timothy St. Pierre, PhD, Barry R Davis, MD, PhD, Crystal Parker, MS, William Herbert Schultz, PA-C, Russell E. Ware, MD, PhD

Blood (2013) 122 (21): 780.

https://doi.org/10.1182/blood.V122.21.780.780

Published: 2013

Journal Articles

Multicenter Investigation Of Unrelated Donor Hematopoietic Cell Transplantation (HCT) For Thalassemia Major After a Reduced Intensity Conditioning Regimen (URTH Trial)

Blood (2013) 122 (21): 543.

https://doi.org/10.1182/blood.V122.21.543.543

Published: 2013

Journal Articles

A pilot study of subcutaneous decitabine in β-thalassemia intermedia

Nancy F. Olivieri, Yogen Saunthararajah, Vivek Thayalasuthan, Janet Kwiatkowski, Russell E. Ware, Frans A. Kuypers, Hae-Young Kim, Felicia L. Trachtenberg, Elliott P. Vichinsky, for the Thalassemia Clinical Research Network

Blood (2011) 118 (10): 2708–2711.

https://doi.org/10.1182/blood-2011-03-341909

Published: 2011

Includes: Supplemental data

Journal Articles

A Phase 1B Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FBS0701, a Novel Oral Iron Chelator for the Treatment of Chronic Iron Overload

Hugh Y. Rienhoff, Jr, MD, ip Viprakasit, MD, D, Phil, Lay H. Tay, MBBS, FRCPA, Paul Harmatz, MD, Elliott Vichinsky, MD, Deborah Chirnomas, MD, MPH, Janet Kwiatkowski, MD, Amy E Tapper, PhD, John B Porter, MD, Ellis J. Neufeld, MD, PhD

Blood (2010) 116 (21): 2057.

https://doi.org/10.1182/blood.V116.21.2057.2057

Published: 2010

Journal Articles

A Pilot Study of Epigenetic-Differentiation Therapy with Decitabine to Treat β-Thalassemia Intermedia

Nancy F Olivieri, MD, Yogenthiran Saunthararajah, MD, Vivekanandan Thayalasuthan, MD, Janet Kwiatkowski, MD, Russell E. Ware, MD, PhD, Franz Kuypers, PhD, Hae-Young Kim, DrPH, Elliott P Vichinsky, MD

Blood (2010) 116 (21): 2078.

https://doi.org/10.1182/blood.V116.21.2078.2078

Published: 2010

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Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia

Real-world comparative effects of curative and disease-modifying therapies on ineffective erythropoiesis in beta-thalassemia

Accurate and automated multi-center TCD velocity analysis using natural language processing

CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial

Rapid immune reconstitution in patients with transfusion-dependent thalassemia following betibeglogene autotemcel: A real-world experience

Development of a prediction model for maternal pregnancy complications in sickle cell disease

Efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency who are not regularly transfused: Results from the Phase 3, global, randomized, double-blind, placebo-controlled ACTIVATE-Kids trial

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Iron Unloading By Therapeutic Phlebotomy in Previously Transfused Children with Sickle Cell Anemia: The Twitch Experience

Impact of TCD Screening Protocol on the Incidence of Hemorrhagic Stroke in Children and Young Adults with Sickle Cell Disease

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

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

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

TCD with Transfusions Changing to Hydroxyurea (TWiTCH): Hydroxyurea Therapy As an Alternative to Transfusions for Primary Stroke Prevention in Children with Sickle Cell Anemia

Liver Iron Concentration By MRI In Chronically Transfused Children With Sickle Cell Anemia In The Twitch Trial

Multicenter Investigation Of Unrelated Donor Hematopoietic Cell Transplantation (HCT) For Thalassemia Major After a Reduced Intensity Conditioning Regimen (URTH Trial)

A pilot study of subcutaneous decitabine in β-thalassemia intermedia

A Phase 1B Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FBS0701, a Novel Oral Iron Chelator for the Treatment of Chronic Iron Overload

A Pilot Study of Epigenetic-Differentiation Therapy with Decitabine to Treat β-Thalassemia Intermedia

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