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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

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

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

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

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

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

The Clinical Severity of Alpha-2 Globin Gene Variants: Homozygosity for Hb Agrinio ( HBA2 : c.89T>C) Leads to Severe Antenatal Anemia, about 8 Cases in 3 Families

Claire Berger, MD, Philippe Joly, PhD, Serge Pissard, PhD, Sandrine Baron-Joly, MD, Yoann Huguenin, MD, Aurélie Cantais, MD, Sophie Brun, MD, Cecile GED, MD, Catherine Badens, PhD PharmD, Muriel Giansily-Blaizot, MD, Aguilar-Martinez Patricia, MD PhD, Isabelle Thuret, MD

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-134894

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

Journal Articles

Evaluation of Outcomes and Quality of Care in Children with Sickle Cell Disease Diagnosed By Newborn Screening: A Real-World Nation-Wide Study in France

Valentine Brousse, MD PhD, Cécile Arnaud, MD, Emmanuelle Lesprit, MD, Marie-Helene Odièvre-Montanié, MD PhD, Maryse Etienne-Julan, MD, Gisele Elana, MD, Marie Belloy, MD, Cecile Dumesnil, MD, Beatrice Quinet, MD, Abdourahim Chamouine, MD, Cecile Guillaumat, MD, Nathalie Garnier, MD, Corinne Pondarré, MD PhD, Nathalie Couque, PharmD, Francoise Bernaudin, MD, Mariane de Montalembert, MD PhD, Emmanuelle Boutin, MD, Malika Benkerrou, MD, Isabelle Thuret, MD

Blood (2019) 134 (Supplement_1): 1001.

https://doi.org/10.1182/blood-2019-127221

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

Stenosis Outcome at 1 and 3 Years after Transplantation Vs Standard-Care in Children with Sickle-Cell Anemia and Abnormal Transcranial Doppler with Stroke or No-Stroke History

Blood (2019) 134 (Supplement_1): 2271.

https://doi.org/10.1182/blood-2019-122699

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Probability of TCD-Normalization in the "Drepagreffe" Trial Comparing Transplantation to Chronic Transfusion in Sickle Cell Anemia Children with Abnormal-Transcranial Doppler Is Associated with Lower Ang-2 and BDNF Plasma Levels

Myriam Bernaudin, PhD, Yacine Khelif, Philippe Chadebech, PhD, Gwellaouen Bodivit, Alicia Jouard, Suzanne Verlhac, MD, Catherine Paillard, MDPhD, Isabelle Thuret, MD, Corinne Guitton, MD, Camille Runel, MD, Florence Missud, MD, Cécile Arnaud, MD, Annie Kamdem, MD, Corinne Pondarré, MD PhD, France Pirenne, MD PhD, Francoise Bernaudin, MD

Blood (2018) 132 (Supplement 1): 506.

https://doi.org/10.1182/blood-2018-99-118091

Published: 2018

Journal Articles

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Franco Locatelli, Mark C. Walters, MD, Janet L. Kwiatkowski, MD, John Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Ashutosh Lal, MD, Adrian J. Thrasher, MD PhD FMedSci, Evangelia Yannaki, MD, Heidi Elliot, Ge Tao, PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1025.

https://doi.org/10.1182/blood-2018-99-112667

Published: 2018

Journal Articles

Towards Mechanical Clinical Markers in Sickle Cell Disease: Dynamics of Red Blood Cells in Low Shear Flow

Catherine Badens, Pharm D, PhD, Scott Atwell, PhD, Alexander Hornung, PhD, Imane Agouti, PhD, Anne Charrier, PhD, Isabelle Thuret, MD, Emmanuele Helfer, PhD, Emmanuelle Bernit, MD, Annie Viallat, PhD

Blood (2018) 132 (Supplement 1): 4914.

https://doi.org/10.1182/blood-2018-99-113298

Published: 2018

Journal Articles

Sustainability of Hematological and Clinical Benefits to HU Administration in the Prevention of Sickle-Cell Vaso-Occlusive Crises in Routine Practice

Ersi Voskaridou, MD, Lena Oevermann, MD, Corinne Armari-Alla, MD, Uwe Kordes, MD MRCP, Frédéric Galactéros, MDPhD, Malika Benkerrou, MD, Isabelle Thuret, MD, Dominique Steschenko, MD

Blood (2018) 132 (Supplement 1): 855.

https://doi.org/10.1182/blood-2018-99-116040

Published: 2018

Journal Articles

Health-Related Quality of Life at 1 Year in the Drepagreffe Trial Comparing Chronic Transfusion to Transplantation in Children with Sickle Cell Anemia and a History of Abnormal Cerebral Velocities

Blood (2017) 130 (Supplement 1): 617.

https://doi.org/10.1182/blood.V130.Suppl_1.617.617

Published: 2017

Journal Articles

Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β 0 /β 0 Genotypes

Mark C. Walters, MD, Suradej Hongeng, MD, Janet L. Kwiatkowski, MD, Franco Locatelli, MD PhD, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD, Martin G. Sauer, Adrian J Thrasher, MD PhD FMedSci, Isabelle Thuret, MD, Evangelia Yannaki, MD PhD, Heidi Elliot, Marisa Gayron, Mohammed Asmal, MD PhD, Alexis A Thompson, MD MPH

Blood (2017) 130 (Supplement 1): 526.

https://doi.org/10.1182/blood.V130.Suppl_1.526.526

Published: 2017

NARROW

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

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

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

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

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

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

The Clinical Severity of Alpha-2 Globin Gene Variants: Homozygosity for Hb Agrinio ( HBA2 : c.89T>C) Leads to Severe Antenatal Anemia, about 8 Cases in 3 Families

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

Evaluation of Outcomes and Quality of Care in Children with Sickle Cell Disease Diagnosed By Newborn Screening: A Real-World Nation-Wide Study in France

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Stenosis Outcome at 1 and 3 Years after Transplantation Vs Standard-Care in Children with Sickle-Cell Anemia and Abnormal Transcranial Doppler with Stroke or No-Stroke History

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Probability of TCD-Normalization in the "Drepagreffe" Trial Comparing Transplantation to Chronic Transfusion in Sickle Cell Anemia Children with Abnormal-Transcranial Doppler Is Associated with Lower Ang-2 and BDNF Plasma Levels

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Towards Mechanical Clinical Markers in Sickle Cell Disease: Dynamics of Red Blood Cells in Low Shear Flow

Sustainability of Hematological and Clinical Benefits to HU Administration in the Prevention of Sickle-Cell Vaso-Occlusive Crises in Routine Practice

Health-Related Quality of Life at 1 Year in the Drepagreffe Trial Comparing Chronic Transfusion to Transplantation in Children with Sickle Cell Anemia and a History of Abnormal Cerebral Velocities

Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β 0 /β 0 Genotypes

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American Society of Hematology

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American Society of Hematology

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