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Journal Articles

Efficacy and Safety of Combination Therapy with Oral Iron Chelators Deferiprone and Deferasirox in Patients with β-Thalassemia Major: A Systematic Literature Review

Janet L. Kwiatkowski, Sujit Sheth, Antonis Kattamis, Francesco Bianchi, Fernando Tricta, Caroline Fradette, Roberta Luzzi, Antonio Piga

Blood (2024) 144 (Supplement 1): 5268.

https://doi.org/10.1182/blood-2024-207562

Published: 2024

Journal Articles

Defining the Impacts of Iron Overload in Children Receiving Hematopoietic Stem Cell Transplantation for Non-Malignant Hematologic Disorders: A Multicenter Analysis

Nora M. Gibson, Sandra Amaral, Jesse Y Hsu, Nhat Nguyen, Meghan Haney, Katelyn E Heimbruch, Anthony Sabulski, Kimberly Ann Davidow, Katherine Lind, Samantha Scanlon, Heather Alva, L. Charles Bailey, Timothy S. Olson, Janet L. Kwiatkowski

Blood (2024) 144 (Supplement 1): 1064.

https://doi.org/10.1182/blood-2024-204339

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2024) 144 (Supplement 1): 512.

https://doi.org/10.1182/blood-2024-205779

Published: 2024

Journal Articles

An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel

Stacey Rifkin-Zenenberg, Julie Kanter, Melissa A. Kinney, Janet L. Kwiatkowski, Robert S. Nickel, Mark C. Walters, Suhag Parikh, Alexis Thompson, Anil P. George, Markus Mapara, Paul L. Martin, Anjulika Chawla, Ankit Lodaya, Lin Pan, Emily Sheldon-Waniga, Francis J. Pierciey, Jr., John Tisdale, Ashish O. Gupta

Blood (2024) 144 (Supplement 1): 511.

https://doi.org/10.1182/blood-2024-199743

Published: 2024

Journal Articles

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Blood (2024) 144 (Supplement 1): 2194.

https://doi.org/10.1182/blood-2024-200152

Published: 2024

Journal Articles

Hydroxyurea Initiation and Occurrence of Cerebrovascular Outcomes in Sickle Cell Disease

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

Blood (2024) 144 (Supplement 1): 2504.

https://doi.org/10.1182/blood-2024-194380

Published: 2024

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

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

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

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

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

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

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

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

NARROW

Efficacy and Safety of Combination Therapy with Oral Iron Chelators Deferiprone and Deferasirox in Patients with β-Thalassemia Major: A Systematic Literature Review

Defining the Impacts of Iron Overload in Children Receiving Hematopoietic Stem Cell Transplantation for Non-Malignant Hematologic Disorders: A Multicenter Analysis

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Hydroxyurea Initiation and Occurrence of Cerebrovascular Outcomes in Sickle Cell Disease

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

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

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

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

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

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

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

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)

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American Society of Hematology

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American Society of Hematology

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