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)

被引：3

作者：

Thompson, Alexis A. ^{[1
,2
]}

Olson, Timothy S. ^{[1
,3
]}

Walters, Mark C. ^{[4
]}

Porter, John B. ^{[5
]}

Schneiderman, Jennifer ^{[6
]}

Hongeng, Suradej ^{[7
]}

Kulozik, Andreas ^{[8
,9
,10
]}

Cavazzana, Marina ^{[11
,12
,13
]}

Sauer, Martin G. ^{[14
]}

Thrasher, Adrian J. ^{[15
]}

Thuret, Isabelle ^{[16
]}

Lal, Ashutosh ^{[17
]}

Rasko, John E. J. ^{[18
,19
,20
]}

Yannaki, Evangelia ^{[21
]}

Ali, Shamshad ^{[22
]}

Tao, Gloria ^{[22
]}

Thakar, Himal L. ^{[22
]}

Deora, Ami ^{[22
]}

Gruppioni, Katiana ^{[22
]}

Colvin, Richard A. ^{[22
]}

Locatelli, Franco ^{[23
,24
]}

Kwiatkowski, Janet L. ^{[1
,25
]}

机构：

[1] Univ Penn, Perelman Sch Med, Dept Pediat, Philadelphia, PA USA

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

[3] Childrens Hosp Philadelphia, Div Oncol, Cellular Therapy & Transplant Sect, Philadelphia, PA USA

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

[5] Univ Coll London Hosp, Res Dept Haematol, London, England

[6] Northwestern Univ, Ann & Robert H. Lurie Childrens Hosp Chicago, Div Hematol Oncol Neurooncol & Stem Cell Transpla, Dept Pediat,Feinberg Sch Med, Chicago, IL USA

[7] Mahidol Univ, Ramathibodi Hosp, Dept Pediat, Bangkok, Thailand

[8] Heidelberg Univ, Dept Pediat Oncol Hematol & Immunol, Heidelberg, Germany

[9] Heidelberg Univ, Hopp Childrens Tumor Ctr, Heidelberg, Germany

[10] DKFZ, Heidelberg, Germany

[11] Imagine Inst, Pediat Immunol, Paris, France

[12] Univ Paris 05, Hosp Necker, Dept Biotherapy, Paris, France

[13] Assistance Publ Hop Paris, Biotherapy Clin Invest Ctr, Inserm, Paris, France

[14] Hannover Med Sch, Dept Pediat Hematol & Oncol, Hannover, Germany

[15] UCL, Sect Mol & Cellular Immunol, Great Ormond St Inst Child Hlth, London, England

[16] Hosp Timone, Dept Pediat, Marseille, France

[17] UCSF, Div Hematol, Benioff Childrens Hosp, Oakland, CA USA

[18] Centenary Inst, Gene & Stem Cell Therapy Program, Camperdown, Australia

[19] Univ Sydney, Fac Med & Hlth, Sydney, Australia

[20] Royal Prince Alfred Hosp, Dept Cell & Mol Therapies, Sydney, Australia

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

[22] Bluebird Bio Inc, Somerville, MA USA

[23] IRCCS Osped Pediat Bambino Gesu, Dept Pediat Hematol & Oncol, Rome, Italy

[24] Univ Cattolica Sacro Cuore, Dept Pediat, Rome, Italy

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

来源：

BLOOD | 2023年 / 142卷

关键词：

D O I：

10.1182/blood-2023-174068

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页数：5

共 15 条

[1] 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
[2] 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
[3] Betibeglogene autotemcel (beti-cel) gene therapy for the treatment of Transfusion-Dependent β-Thalassemia (TDT): Updated long-term efficacy and safety results
Kulozik, A. E.
Thuret, I
Thompson, A. A.
Kwiatkowski, J. L.
Porter, J. B.
Hongeng, S.
Yannaki, E.
Sauer, M. G.
Thrasher, A. J.
Lal, A.
Du, L.
Colvin, R. A.
Walters, M. C.
Locatelli, F.
ONCOLOGY RESEARCH AND TREATMENT, 2021, 44 : 8 - 9
[4] 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
[5] 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
[6] 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
[7] 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
[8] 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
[9] 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
[10] 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
Walters, Mark C.
Kwiatkowski, Janet L.
Porter, John B.
Schneiderman, Jennifer
Hongeng, Suradej
Kulozik, Andreas E.
Cavazzana, Marina
Sauer, Martin G.
Thrasher, Adrian J.
Thuret, Isabelle
Lal, Ashutosh
Rasko, John E. J.
Yannaki, Evangelia
Ali, Shamshad
Shestopalov, Ilya
Fincker, Maeva
Colvin, Richard A.
Whitney, Dustin
Locatelli, Franco
Thompson, Alexis A.
BLOOD, 2022, 140 : 5355 - 5357

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