Betibeglogene autotemcel (beti-cel) gene therapy for the treatment of Transfusion-Dependent β-Thalassemia (TDT): Updated long-term efficacy and safety results

被引：0

作者：

Kulozik, A. E. ^{[1
]}

Thuret, I ^{[2
]}

Thompson, A. A. ^{[3
]}

Kwiatkowski, J. L. ^{[4
]}

Porter, J. B. ^{[5
]}

Hongeng, S. ^{[6
]}

Yannaki, E. ^{[7
]}

Sauer, M. G. ^{[8
]}

Thrasher, A. J. ^{[9
]}

Lal, A.

Du, L. ^{[10
]}

Colvin, R. A. ^{[11
]}

Walters, M. C. ^{[12
]}

Locatelli, F. ^{[13
]}

机构：

[1] Heidelberg Univ, Univ Med Ctr Children & Adolescents, Heidelberg, Germany

[2] Marseille Univ, Dept Pediat Hematol, Marseille, France

[3] Feinberg Sch Med, Hematol Sect, Chicago, IL USA

[4] Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA USA

[5] UCL, Dept Haematol, London, England

[6] Ramathibodi Hosp, Dept Pediat, Fac Med, Bangkok, Thailand

[7] G Papanicolaou Hosp, Hematol Dept Hematopoiet Cell Transplantat Unit, Gene & Cell Therapy Ctr, Thessaloniki, Greece

[8] Hannover Med Sch, Pediat Hematol & Oncol, Hannover, Germany

[9] Great Ormond St Hosp Sick Children, London, England

[10] UCSF Benioff Childrens Hosp, Oakland, CA USA

[11] Bluebird Bio Inc, Cambridge, England

[12] Univ Calif San Francisco, San Francisco, CA 94143 USA

[13] Osped Pediat Bambino Gesu, Dept Pediat Hematol Oncol, Rome, Italy

来源：

ONCOLOGY RESEARCH AND TREATMENT | 2021年 / 44卷

关键词：

D O I：

暂无

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

V293

引用

页码：8 / 9

页数：2

共 50 条

[1] BETIBEGLOGENE AUTOTEMCEL (BETI-CEL) GENE THERAPY FOR THE TREATMENT OF TRANSFUSION-DEPENDENT β-THALASSEMIA (TDT): UPDATED LONG-TERM EFFICACY AND SAFETY RESULTS
Locatelli, F.
Yannaki, E.
Kwiatkowski, J. L.
Walters, M. C.
Porter, J. B.
Hongeng, S.
Kulozik, A. E.
Sauer, M. G.
Thrasher, A. J.
Thuret, I.
Lal, A.
Cavazzana, M.
Du, Lin
Colvin, R. A.
Rasko, J. E. J.
Algeri, M.
Thompson, A. A.
HAEMATOLOGICA, 2021, 106 (10) : 7 - 8
[2] The First Real-World Experience with Betibeglogene Autotemcel (beti-cel) Gene Therapy Treatment for Transfusion-Dependent β-Thalassemia (TDT)
Kunz, Joachim B.
Roth, Eva
Mirza, Adil
Greil, Johann
Pavel, Petra
Schmitt, Anita
Schmitt, Michael
Kunz, Alexander
Kulozik, Andreas E.
BLOOD, 2021, 138
[3] Betibeglogene Autotemcel (Beti-Cel; Lentiglobin for B-Thalassemia) Gene Therapy Treatment of A Greek Patient with Transfusion-Dependent B-Thalassemia (TDT)
Constantinou, Varnavas
Papayanni, Penelope
Mallouri, Despoina
Batsis, Ioannis
Sotiropoulos, Damianos
Bouinta, Asimina
Papadopoulou, Despoina
Papadimitriou, Vasiliki
Kammenou, Maria
Liu, Weijian
Colvin, Richard
Sakellari, Ioanna
Anagnostopoulos, Achilles
Yannaki, Evangelia
BONE MARROW TRANSPLANTATION, 2021, 56 (SUPPL 1) : 215 - 216
[4] 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
Walters, Mark C.
Chui, David H. K.
Farrell, John J.
Lal, Ashutosh
Locatelli, Franco
Kwiatkowski, Janet L.
Porter, John B.
Sauer, Martin G.
Thuret, Isabelle
Hongeng, Suradej
Kulozik, Andreas E.
Thrasher, Adrian J.
Yannaki, Evangelia
Yang, Julia
Whitney, Dustin
Petrusich, Alexandria
Colvin, Richard A.
Thompson, Alexis A.
BLOOD, 2020, 136
[5] 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 Followup
Kwiatkowski, Janet L.
Olson, Timothy S.
Walters, Mark C.
Porter, John B.
Schneiderman, Jennifer
Hongeng, Suradej
Kulozik, Andreas
Cavazzana, Marina
Sauer, Martin G.
Thrasher, Adrian J.
Thuret, Isabelle
Lal, Ashutosh
Rasko, John E. J.
Yannaki, Evangelia
Ali, Shamshad
Tao, Gloria
Deora, Ami
Thakar, Himal L.
Colvin, Richard A.
Locatelli, Franco
Thompson, Alexis A.
BLOOD, 2023, 142
[6] BETIBEGLOGENE AUTOTEMCEL GENE THERAPY (BETI-CEL) IS COST-EFFECTIVE VERSUS STANDARD OF CARE IN PATIENTS WITH TRANSFUSION-DEPENDENT B-THALASSEMIA (TDT) IN FRANCE
Undreiner, L.
Roze, S.
Caillon, M.
VALUE IN HEALTH, 2020, 23 : S413 - S413
[7] 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)
Thompson, Alexis A.
Olson, Timothy S.
Walters, Mark C.
Porter, John B.
Schneiderman, Jennifer
Hongeng, Suradej
Kulozik, Andreas
Cavazzana, Marina
Sauer, Martin G.
Thrasher, Adrian J.
Thuret, Isabelle
Lal, Ashutosh
Rasko, John E. J.
Yannaki, Evangelia
Ali, Shamshad
Tao, Gloria
Thakar, Himal L.
Deora, Ami
Gruppioni, Katiana
Colvin, Richard A.
Locatelli, Franco
Kwiatkowski, Janet L.
BLOOD, 2023, 142
[8] Durable Clinical Outcomes Following Betibeglogene Autotemcel (BETI-CEL) Gene Therapy With Up to 6 Years of Follow-Up in Patients With Transfusion-Dependent B-Thalassemia (TDT)
Locatelli, F.
Kwiatkowski, J.
Walters, M.
Hongeng, S.
Rasko, J.
Cavazzana, M.
Schmidt, M.
Chen, Y.
Colvin, R.
Thompson, A.
Locatelli, Franco
Kwiatkowski, Janet
Walters, Mark
Hongeng, Suradej
Rasko, John
Cavazzana, Marina
Schmidt, Manfred
Chen, Ying
Colvin, Richard
Thompson, Alexis
BONE MARROW TRANSPLANTATION, 2021, 56 (SUPPL 1) : 69 - 70
[9] Autotemcel betiblogene gene therapy for the treatment of transfusion-dependent β-thalassemia: update of long-term efficacy and safety results
Cavazzana, Marina
Thuret, Isabelle
Yannaki, Evangelia
Locatelli, Franco
Kwiatkowski, Janet L.
Walters, Mark C.
Porter, John B.
Hongeng, Suradej
Sauer, Martin G.
Thrasher, Adrian J.
Lal, Ashutosh
Rasko, John E. J.
Du, Lin
Colvin, Richard A.
Thompson, Alexis A.
HEMATOLOGIE, 2021, 27 (04): : 157 - 158
[10] BETI-CEL GENE THERAPY OUTCOMES IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA
Kwiatkowski, Janet
Walters, Mark
Lal, Ashutosh
Locatelli, Franco
Porter, John
Hongeng, Suradej
Thuret, Isabelle
Yannaki, Evangelia
Kulozik, Andreas
Sauer, Martin
Thrasher, Adrian
Guo, Ruiting
Liu, Weijian
Colvin, Richard
Thompson, Alexis
PEDIATRIC BLOOD & CANCER, 2021, 68 : S25 - S26

← 1 2 3 4 5 →