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Journal Articles

Improvement of Iron Overload Parameters in Patients with Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Data from the Phase 3b Long-Term Rollover Study Following the BELIEVE Trial

John B. Porter, Maria Domenica Cappellini, Kevin H.M. Kuo, Maria Dimopoulou, Marta Reverte, Loyse Felber Medlin, Shweta Sharma, Kefeng Wang, Jennie Zhang, Ali T. Taher

Blood (2024) 144 (Supplement 1): 2475.

https://doi.org/10.1182/blood-2024-200157

Published: 2024

Journal Articles

Observational Data on Parvovirus B19 Cases in Patients with Red Cell Disorders in the United Kingdom from 2023-2024

Chloe Merrion, Ethan Troy-Barnes, Ryan Mullally, Ana Ortuno Cabrero, John B. Porter, Emma Drasar

Blood (2024) 144 (Supplement 1): 3895.

https://doi.org/10.1182/blood-2024-208518

Published: 2024

Journal Articles

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

Blood (2024) 144 (Supplement 1): 409.

https://doi.org/10.1182/blood-2024-200867

Published: 2024

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

Iron Chelation Efficiency in Human Hepatocytes Is Enhanced By Exogenous Hepcidin

Pimpisid Koonyosying, Maciej Garbowski, Ute Schaeper, Alberto Martinez, John B. Porter

Blood (2023) 142 (Supplement 1): 2468.

https://doi.org/10.1182/blood-2023-182214

Published: 2023

Journal Articles

Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent β-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial

Ali T Taher, Vip Viprakasit, Antonis Kattamis, Silverio Perrotta, Paolo Ricchi, John B. Porter, Thomas D Coates, Gian Luca Forni, Khaled M. Musallam, Oriana Esposito, Ana Carolina Giuseppi, Wen-Ling Kuo, Marta Reverte, Richard Wei, Luciana Moro Bueno, Maria Domenica Cappellini

Blood (2023) 142 (Supplement 1): 3847.

https://doi.org/10.1182/blood-2023-174097

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

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

Effect of Luspatercept on Red Blood Cell (RBC) Transfusion Burden, Iron Chelation Therapy (ICT), and Iron Overload in Adults with Transfusion-Dependent β-Thalassemia (TDT) from the BELIEVE Trial: A Long-Term Analysis

Olivier Hermine, Maria Domenica Domenica Cappellini, Ali T. Taher, Thomas D. Coates, Vip Viprakasit, Antonis Kattamis, Jeevan K. Shetty, Marija Bosilkovska Weisskopf, Natalia Holot, Sadanand Vodala, Wen-Ling Kuo, John B. Porter

Blood (2022) 140 (Supplement 1): 8215–8217.

https://doi.org/10.1182/blood-2022-156985

Published: 2022

Journal Articles

Erythroid Response in Patients with Non-Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Long-Term Data from the BEYOND Trial

Ali T. Taher, Vip Viprakasit, Maria Domenica Domenica Cappellini, Antonio Piga, John B. Porter, Thomas D. Coates, Khaled M. Musallam, Gian Luca Forni, Jeevan K. Shetty, Marija Bosilkovska Weisskopf, Richard Wei, Wen-Ling Kuo, Antonis Kattamis

Blood (2022) 140 (Supplement 1): 8210–8212.

https://doi.org/10.1182/blood-2022-157512

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

Investigations into the Mechanisms and Clinical Implications of Modulation of Hepcidin Levels By Luspatercept in TD MDS and TD b-Thalassemia

Aarif Ahsan, Wei Fang, Manuel Ugidos, Danny V Jeyaraju, Pierre Fenaux, Uwe Platzbecker, Olivier Hermine, John B. Porter, Rajasekhar N.V.S. Suragani, Sadanand Vodala, Anita K. Gandhi

Blood (2022) 140 (Supplement 1): 8188–8189.

https://doi.org/10.1182/blood-2022-166559

Published: 2022

Journal Articles

Effect of Luspatercept in β-Thalassemia Patients with β0/β0 Genotype: A Subgroup Analysis of the BELIEVE Study

Sujit Sheth, Olivier Hermine, Ali T. Taher, Kevin H.M. Kuo, John B. Porter, Antonio Piga, Thomas D. Coates, Antonis Kattamis, Loyse Felber Medlin, Wen-Ling Kuo, Natalia Holot, Maria Domenica Domenica Cappellini

Blood (2022) 140 (Supplement 1): 1946–1948.

https://doi.org/10.1182/blood-2022-157511

Published: 2022

Journal Articles

A Closer Look at Changes in Hemoglobin Levels in Patients with Non-Transfusion Dependent β-Thalassemia Treated with Luspatercept: Post Hoc Analysis of the Phase 2 BEYOND Trial

Khaled M. Musallam, Ali T. Taher, John B. Porter, Antonis Kattamis, Mrudula B. Glassberg, Luciana Bueno, Jeevan K. Shetty, Frederik Lersch, Barbara Rosettani, Maria Domenica Domenica Cappellini

Blood (2022) 140 (Supplement 1): 5358–5359.

https://doi.org/10.1182/blood-2022-157113

Published: 2022

Journal Articles

Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency

Eduard J. van Beers, Hanny Al-Samkari, Rachael F. Grace, Wilma Barcellini, Andreas Glenthøj, Melissa Dibacco, Penelope A. Kosinski, Emily Xu, Vanessa Beynon, Parija Patel, John B. Porter, Kevin H.M. Kuo

Blood (2022) 140 (Supplement 1): 2460–2462.

https://doi.org/10.1182/blood-2022-169098

Published: 2022

Journal Articles

Luspatercept Improves Quality of Life and Reduces Red Blood Cell Transfusion Burden in Patients with Non-Transfusion-Dependent β-Thalassemia in the BEYOND Trial

Antonis Kattamis, Vip Viprakasit, Maria Domenica Cappellini, Ersi Voskaridou, Antonio Piga, John B. Porter, Thomas D. Coates, Gian Luca Forni, Jeevan K. Shetty, Dimana Miteva, Oriana Esposito, Wen-Ling Kuo, Shien Guo, Christopher Pelligra, Jennifer Lord-Bessen, Aylin Yucel, Ali T. Taher

Blood (2021) 138 (Supplement 1): 3081.

https://doi.org/10.1182/blood-2021-148535

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

Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Blood (2021) 138 (Supplement 1): 848.

https://doi.org/10.1182/blood-2021-147711

Published: 2021

Journal Articles

Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-Term Treatment with Mitapivat

Hanny Al-Samkari, Rachael F. Grace, Andreas Glenthoej, Oliver Andres, Wilma Barcellini, Frédéric Galactéros, Kevin H.M. Kuo, D. Mark Layton, Marta Morado, Vip Viprakasit, Yan Dong, Feng Tai, Liz Grekas, Karishma Khoja, Sarah Gheuens, Bryan McGee, John B. Porter, Eduard J. Van Beers

Blood (2021) 138 (Supplement 1): 924.

https://doi.org/10.1182/blood-2021-147156

Published: 2021

NARROW

Improvement of Iron Overload Parameters in Patients with Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Data from the Phase 3b Long-Term Rollover Study Following the BELIEVE Trial

Observational Data on Parvovirus B19 Cases in Patients with Red Cell Disorders in the United Kingdom from 2023-2024

ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia

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

Iron Chelation Efficiency in Human Hepatocytes Is Enhanced By Exogenous Hepcidin

Luspatercept for the Treatment of Anemia in Non-Transfusion-Dependent β-Thalassemia: Final Safety and Efficacy Data from the BEYOND Trial

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)

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

Effect of Luspatercept on Red Blood Cell (RBC) Transfusion Burden, Iron Chelation Therapy (ICT), and Iron Overload in Adults with Transfusion-Dependent β-Thalassemia (TDT) from the BELIEVE Trial: A Long-Term Analysis

Erythroid Response in Patients with Non-Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Long-Term Data from the BEYOND Trial

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

Investigations into the Mechanisms and Clinical Implications of Modulation of Hepcidin Levels By Luspatercept in TD MDS and TD b-Thalassemia

Effect of Luspatercept in β-Thalassemia Patients with β0/β0 Genotype: A Subgroup Analysis of the BELIEVE Study

A Closer Look at Changes in Hemoglobin Levels in Patients with Non-Transfusion Dependent β-Thalassemia Treated with Luspatercept: Post Hoc Analysis of the Phase 2 BEYOND Trial

Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency

Luspatercept Improves Quality of Life and Reduces Red Blood Cell Transfusion Burden in Patients with Non-Transfusion-Dependent β-Thalassemia in the BEYOND Trial

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

Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained over Time in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat in a Long-Term Extension Study

Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-Term Treatment with Mitapivat

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American Society of Hematology

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American Society of Hematology

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