Interim Results of Betibeglogene Autotemcel Gene Therapy in Pediatric Patients With Transfusion-dependent B-Thalassemia (TDT) Treated in The Phase 3 Northstar-2 (HGB-207) And Northstar-3 (HGB-212) Studies

被引：0

作者：

Thuret, Isabelle ^{[1
]}

Thompson, Alexis ^{[2
,3
]}

Kwiatkowski, Janet ^{[4
,5
]}

Porter, John ^{[6
]}

Hongeng, Suradej ^{[7
]}

Yannaki, Evangelia ^{[8
]}

Kulozik, Andreas ^{[9
]}

Sauer, Martin ^{[10
]}

Thrasher, Adrian ^{[11
,12
]}

Lal, Ashutosh ^{[13
]}

Guo, Ruiting ^{[14
]}

Liu, Weijian ^{[14
]}

Colvin, Richard ^{[14
]}

Walters, Mark ^{[13
]}

Locatelli, Franco ^{[15
]}

机构：

[1] Hop La Timone, Marseille, France

[2] Ann & Robert H Lurie Childrens Hosp, Chicago, IL USA

[3] Northwestern Univ, Feinberg Sch Med, Chicago, IL 60611 USA

[4] Univ Penn, Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

[5] Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA

[6] Univ Coll London Hosp, London, England

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

[8] G Papanikolaou Hosp, Thessaloniki, Greece

[9] Heidelberg Univ, Heidelberg, Germany

[10] Hannover Med Sch, Hannover, Germany

[11] UCL, Great Ormond St Hosp Inst Child Hlth, London, England

[12] Great Ormond St Hosp NHS Trust, London, England

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

[14] Bluebird Bio Inc, Cambridge, MA USA

[15] IRCCS Bambino Gesu Childrens Hosp, Rome, Italy

来源：

BONE MARROW TRANSPLANTATION | 2021年 / 56卷 / SUPPL 1期

关键词：

D O I：

暂无

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

O054

引用

页码：64 / 65

页数：2

共 27 条

[21] Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial
Kwiatkowski, Janet L.
Walters, Mark C.
Hongeng, Suradej
Yannaki, Evangelia
Kulozik, Andreas E.
Kunz, Joachim B.
Sauer, Martin G.
Thrasher, Adrian J.
Thuret, Isabelle
Lal, Ashutosh
Tao, Ge
Ali, Shamshad
Thakar, Himal L.
Elliot, Heidi
Lodaya, Ankit
Lee, Ji
Colvin, Richard A.
Locatelli, Franco
Thompson, Alexis A.
LANCET, 2024, 404 (10468): : 2175 - 2186
[22] Efficacy and safety of LentiGlobin gene therapy in patients with transfusion-dependent β-thalassaemia and non-β0/β0 genotypes: Updated results from the completed phase 1/2 Northstar and ongoing phase 3 Northstar-2 studies
Porter, J. B.
Walters, M. C.
Kwiatkowski, J. L.
Hongeng, S.
Rasko, J. E. J.
Sauer, M. G.
Thrasher, A. J.
Thuret, I.
Schiller, G. J.
Elliot, H.
Deary, B.
Chen, Y.
Tao, G.
Asmal, M.
Locatelli, F.
Thompson, A. A.
BRITISH JOURNAL OF HAEMATOLOGY, 2019, 185 : 23 - 23
[23] 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
[24] 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
Lal, Ashutosh
Locatelli, Franco
Kwiatkowski, Janet L.
Kulozik, Andreas E.
Yannaki, Evangelia
Porter, John B.
Thuret, Isabelle
Sauer, Martin G.
Elliot, Heidi
Chen, Ying
Colvin, Richard A.
Thompson, Alexis A.
BLOOD, 2019, 134
[25] LENTIGLOBIN GENE THERAPY IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THA-LASSEMIA AND NON-β0/β0 GENOTYPES: OUTCOMES FROM THE COMPLETED PHASE 1/2 NORTHSTAR AND ONGOING PHASE 3 NORTHSTAR-2 STUDIES
Locatelli, F.
Walters, M.
Kwiatkowski, J.
Hongeng, S.
Porter, J.
Rasko, J.
Sauer, M.
Thrasher, A.
Thuret, I.
Schiller, G.
Elliot, H.
Deary, B.
Chen, Y.
Tao, G.
Colvin, R.
Thompson, A.
HAEMATOLOGICA, 2019, 104 : 1 - 1
[26] 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
[27] 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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