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Journal Articles

Defining the Impacts of Iron Overload in Children Receiving Hematopoietic Stem Cell Transplantation for Non-Malignant Hematologic Conditions

Nora M Gibson, Sandra Amaral, Jesse Y Hsu, Timothy S Olson, Janet L. Kwiatkowski

Blood (2023) 142 (Supplement 1): 489.

https://doi.org/10.1182/blood-2023-180171

Published: 2023

Journal Articles

Adults with Transfusion-Dependent Thalassemia Have Variable Clinical Profiles Depending on Where They Receive Care: Thalassemia Treatment Center Vs Community

Farzana A Sayani, Michel Liu, Yvette Frimpong, Scott A. Peslak, Elizabeth A. Traxler, Jennifer Cohen, J. Eric Russell, Janet L. Kwiatkowski

Blood (2023) 142 (Supplement 1): 2382.

https://doi.org/10.1182/blood-2023-191295

Published: 2023

Journal Articles

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Blood (2023) 142 (Supplement 1): 1102.

https://doi.org/10.1182/blood-2023-174068

Published: 2023

Journal Articles

Impact of the 2014 NHLBI Hydroxyurea Guidelines on Cerebrovascular Events in Sickle Cell Disease

Aleksandra S Dain, Yimei Li, Sahal Master, Shri Karthikeyan, Leslie Raffini, Kelly D. Getz, Janet L. Kwiatkowski

Blood (2023) 142 (Supplement 1): 1129.

https://doi.org/10.1182/blood-2023-190822

Published: 2023

Journal Articles

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up

Blood (2023) 142 (Supplement 1): 2480.

https://doi.org/10.1182/blood-2023-173869

Published: 2023

Journal Articles

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Julie Kanter, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Ashish O. Gupta, Lixin Zhang, Emily Sheldon-Waniga, Meghan Gallagher, Katiana Gruppioni, Anjulika Chawla, Heidi Elliot, Francis J. Pierciey, Mark C. Walters, John F. Tisdale

Blood (2023) 142 (Supplement 1): 1051.

https://doi.org/10.1182/blood-2023-174229

Published: 2023

Journal Articles

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2023) 142 (Supplement 1): 1053.

https://doi.org/10.1182/blood-2023-190534

Published: 2023

Journal Articles

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Mark C. Walters, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Y. Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Alex Miller, Lixin Zhang, Anjulika Chawla, Elizabeth R. Macari, Francis J. Pierciey, Jr., John F. Tisdale, Julie Kanter

Blood (2022) 140 (Supplement 1): 26–28.

https://doi.org/10.1182/blood-2022-162288

Published: 2022

Journal Articles

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Blood (2022) 140 (Supplement 1): 5355–5357.

https://doi.org/10.1182/blood-2022-162400

Published: 2022

Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson

Blood (2022) 140 (Supplement 1): 8200–8202.

https://doi.org/10.1182/blood-2022-163178

Published: 2022

Journal Articles

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Blood (2021) 138 (Supplement 1): 561.

https://doi.org/10.1182/blood-2021-147760

Published: 2021

Journal Articles

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Janet L. Kwiatkowski, Franco Locatelli, Mark C. Walters, John B. Porter, Suradej Hongeng, Evangelia Yannaki, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Ruiting Guo, Richard A. Colvin, Katiana Gruppioni, Alexis A. Thompson

Blood (2021) 138 (Supplement 1): 3085.

https://doi.org/10.1182/blood-2021-150392

Published: 2021

Journal Articles

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Blood (2021) 138 (Supplement 1): 573.

https://doi.org/10.1182/blood-2021-148177

Published: 2021

Journal Articles

Ex Vivo Evaluation of Red Blood Cell Adhesion and Whole Blood Thrombosis in Pyruvate Kinase Deficiency

Patrick C. Hines, Xiufeng Gao, Andrew Herppich, Wendy Hollon, Meera B. Chitlur, Janet L. Kwiatkowski, Michael Tarasev

Blood (2021) 138 (Supplement 1): 923.

https://doi.org/10.1182/blood-2021-149744

Published: 2021

Journal Articles

Trial in Progress: A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of the PKR Activator Etavopivat (FT-4202) in Patients with Thalassemia or Sickle Cell Disease

Ashutosh Lal, R. Clark Clark Brown, Thomas D. Coates, Theodosia A. Kalfa, Janet L. Kwiatkowski, Julie Brevard, Von Potter, Ken W. Wood, Sujit Sheth

Blood (2021) 138 (Supplement 1): 4162.

https://doi.org/10.1182/blood-2021-145752

Published: 2021

Journal Articles

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Mark C. Walters, John F. Tisdale, Markus Y. Mapara, Lakshmanan Krishnamurti, Janet L. Kwiatkowski, Banu Aygun, Kimberly A. Kasow, Stacey Rifkin-Zenenberg, Jennifer Jaroscak, Diana Garbinsky, Costel Chirila, Meghan E. Gallagher, Xinyan Zhang, Pei-Ran Ho, Alexis A. Thompson, Julie Kanter

Blood (2021) 138 (Supplement 1): 7.

https://doi.org/10.1182/blood-2021-146905

Published: 2021

Journal Articles

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone: Subgroup Analysis of Pediatric Patients in Iron-Overloaded Patients with Sickle Cell Disease and Other Anemias

Mona Hamdy, Amal El-Beshlawy, Fatma Soliman Elsayed Ebeid, Janet L. Kwiatkowski, Julie Kanter, Baba PD Inusa, Suzan Williams, David Lee, Noemi T Temin, Caroline Fradette, Fernando Tricta, Mohsen Saleh Elalfy

Blood (2021) 138 (Supplement 1): 762.

https://doi.org/10.1182/blood-2021-148064

Published: 2021

Journal Articles

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Julie Kanter, MD, John F. Tisdale, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Ren Chen, Meghan E. Gallagher, MSc, Yuchen Ding, PhD, Sunita Goyal, MD, Clark Paramore, MPH, Alexis A. Thompson, MD, Mark C. Walters, MD

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-136193

Published: 2020

Journal Articles

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Janet L. Kwiatkowski, MD MSCE, Mark C. Walters, MD, Suradej Hongeng, MD, Franco Locatelli, MD PhD, John E.J. Rasko, BSc, MBBS, PhD, Marina Cavazzana, MD PhD, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 51–52.

https://doi.org/10.1182/blood-2020-135850

Published: 2020

Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

https://doi.org/10.1182/blood-2020-135857

Published: 2020

NARROW

Defining the Impacts of Iron Overload in Children Receiving Hematopoietic Stem Cell Transplantation for Non-Malignant Hematologic Conditions

Adults with Transfusion-Dependent Thalassemia Have Variable Clinical Profiles Depending on Where They Receive Care: Thalassemia Treatment Center Vs Community

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Impact of the 2014 NHLBI Hydroxyurea Guidelines on Cerebrovascular Events in Sickle Cell Disease

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Ex Vivo Evaluation of Red Blood Cell Adhesion and Whole Blood Thrombosis in Pyruvate Kinase Deficiency

Trial in Progress: A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of the PKR Activator Etavopivat (FT-4202) in Patients with Thalassemia or Sickle Cell Disease

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone: Subgroup Analysis of Pediatric Patients in Iron-Overloaded Patients with Sickle Cell Disease and Other Anemias

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

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American Society of Hematology

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American Society of Hematology

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