Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent β-Thalassemia

被引：1

作者：

Schneiderman, Jennifer ^{[1
,2
]}

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

Walters, Mark C. ^{[3
]}

Kwiatkowski, Janet L. ^{[4
,5
]}

Kulozik, Andreas E. ^{[6
,7
]}

Sauer, Martin G. ^{[8
]}

Porter, John B. ^{[9
]}

Thuret, Isabelle ^{[10
]}

Hongeng, Suradej ^{[11
]}

Lal, Ashutosh ^{[3
]}

Thrasher, Adrian J. ^{[12
]}

Yannaki, Evangelia ^{[13
]}

Elliot, Heidi ^{[14
]}

Tao, Ge ^{[14
]}

Liu, Weijian ^{[14
]}

Colvin, Richard A. ^{[14
]}

Locatelli, Franco ^{[15
]}

机构：

[1] Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA

[2] Northwestern Univ, Feinberg Sch Med, Pediat Hematol Oncol & Stem Cell Transplantat, Chicago, IL 60611 USA

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

[4] Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

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

[6] Heidelberg Univ, EMBL, MMPU, Heidelberg, Germany

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

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

[9] Univ Coll London Hosp, Haematol Dept, London, England

[10] Hop La Timone, Pediat Hematol, Marseille, France

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

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

[13] George Papanikolaou Hosp, Hematol HCT Unit, Gene & Cell Therapy Ctr, Thessaloniki, Greece

[14] Bluebird Bio Inc, Cambridge, MA USA

[15] IRCCS Bambino Gesu Childrens Hosp, Dept Hematol Oncol Cell & Gene Therapy, Rome, Italy

来源：

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION | 2020年 / 26卷 / 03期

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

118

引用

页码：S87 / S88

页数：2

共 34 条

[1] 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
Thompson, Alexis A.
Kwiatkowski, Janet L.
Porter, John B.
Hongeng, Suradej
Yannaki, Evangelia
Kulozik, Andreas E.
Sauer, Martin G.
Thrasher, Adrian J.
Thuret, Isabelle
Lal, Ashutosh
Guo, Ruiting
Liu, Weijian
Colvin, Richard A.
Walters, Mark C.
Locatelli, Franco
BLOOD, 2020, 136
[2] 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
Thuret, Isabelle
Thompson, Alexis
Kwiatkowski, Janet
Porter, John
Hongeng, Suradej
Yannaki, Evangelia
Kulozik, Andreas
Sauer, Martin
Thrasher, Adrian
Lal, Ashutosh
Guo, Ruiting
Liu, Weijian
Colvin, Richard
Walters, Mark
Locatelli, Franco
BONE MARROW TRANSPLANTATION, 2021, 56 (SUPPL 1) : 64 - 65
[3] Interim results of betibeglogene autotemcel gene-additiontherapy in pediatric patients with transfusion-dependent β-thalassemia (TDT) treated in the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies
Kulozik, A. E.
Kunz, J.
Thuret, I
Thompson, A. A.
Kwiatkowski, J. L.
Porter, J. B.
Hongeng, S.
Yannaki, E.
Sauer, M. G.
Thrasher, A. J.
Lal, A.
Guo, R.
Colvin, R. A.
Walters, M. C.
Locatelli, F.
ONCOLOGY RESEARCH AND TREATMENT, 2021, 44 : 8 - 8
[4] Interim results from the phase 3 Northstar-2 and Northstar-3 studies of LentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassemia
Kunz, J.
Locatelli, F.
Kulozik, A.
Sauer, M.
Kwiatkowski, J.
Porter, J.
Thuret, I.
Hongeng, S.
Lal, A.
Thrasher, A.
Yannaki, E.
Elliot, H.
Tao, G.
Chen, Y.
Colvin, R.
Walters, M.
Thompson, A.
ONCOLOGY RESEARCH AND TREATMENT, 2019, 42 : 301 - 301
[5] Interim results from the phase 3 Northstar-2 and Northstar-3 studies of LentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassaemia
Yannaki, E.
Thompson, A. A.
Kulozik, A.
Sauer, M.
Kwiatkowski, J. L.
Porter, J.
Thuret, I.
Hongeng, S.
Lal, A.
Thrasher, A. J.
Elliot, H.
Tao, G.
Chen, Y.
Colvin, R. A.
Walters, M. C.
Locatelli, F.
HUMAN GENE THERAPY, 2019, 30 (11) : A10 - A11
[6] Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies
Locatelli, Franco
Walters, Mark C.
Kwiatkowski, Janet L.
Porter, John
Sauer, Martin G.
Thuret, Isabelle
Hongeng, Suradej
Kulozik, Andreas E.
Lal, Ashutosh
Thrasher, Adrian J.
Yannaki, Evangelia
Elliot, Heidi
Tao, Ge
Asmal, Mohammed
Thompson, Alexis A.
BLOOD, 2018, 132
[7] Safety and Efficacy of LentiGlobin Gene Therapy for Transfusion-Dependent β-thalassemia (TDT) in Patients with non-β0/β0 Genotypes: Interim results from the HGB-204 (Northstar) and HGB-207 (Northstar-2) Trials
Porter, J. B.
Walters, M. C.
Kwiatkowski, J. L.
Rasko, J. E. J.
Hongeng, S.
Schiller, G. J.
Locatelli, F.
Sauer, M.
Thrasher, A.
Thuret, I.
Yannaki, E.
Elliot, H.
Deary, B.
Gayron, M.
Ramirez-Santiago, A.
Thompson, A. A.
BRITISH JOURNAL OF HAEMATOLOGY, 2018, 181 : 30 - 30
[8] FINAL RESULTS OF THE PHASE 3 STUDIES NORTHSTAR-2 AND NORTHSTAR-3 IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA (TDT): SUBGROUP ANALYSIS OF ITALIAN PATIENTS TREATED WITH BETIBEGLOGENE AUTOTEMCEL (BETI-CEL), LENTIGLOBIN FOR THALASSEMIA
Locatelli, F.
Pagliara, D.
Gaspari, S.
Galaverna, F.
Pira, G. Li
Coletti, V.
Merli, P.
Palumbo, G.
Leone, G.
Guo, R.
Colvin, R. A.
Algeri, M.
HAEMATOLOGICA, 2021, 106 (10) : 32 - 32
[9] Updated results from the ongoing northstar-2 (HGB-207) trial of lentiglobin gene therapy in patients with transfusion-dependent β-thalassemia and non-β0/β0 genotypes
Locatelli, Franco
Thompson, Alexis
Kwiatkowski, Janet
Hongeng, Suradej
Porter, John
Sauer, Martin
Thrasher, Adrian
Thuret, Isabelle
Elliot, Heidi
Tao, Ge
Asmal, Mohammed
Walters, Mark
BONE MARROW TRANSPLANTATION, 2019, 54 : 75 - 76
[10] Clinical outcomes following autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for β-thalassemia) gene therapy in the phase 3 Northstar-2 and Northstar-3 studies for transfusion-dependent β-thalassemia (TDT)
Kulozik, A. E.
Locatelli, F.
Kunz, J.
Kwiatkowski, J. L.
Thompson, A. A.
Yannaki, E.
Sauer, M. G.
Schambach, A.
Schmidt, M.
Porter, J. B.
Thuret, I.
Hongeng, S.
Lal, A.
Thrasher, A. J.
Tao, G.
Liu, W.
Colvin, R. A.
Walters, M. C.
ONCOLOGY RESEARCH AND TREATMENT, 2020, 43 (SUPPL 4) : 33 - 33

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