EFFICACY AND SAFETY OF A SINGLE DOSE OF EXAGAMGLOGENE AUTOTEMCEL FOR TRANSFUSION-DEPENDENT.-THALASSEMIA AND SEVERE SICKLE CELL DISEASE

被引：0

作者：

Locatelli, Franco ^{[1
]}

Frangoul, Haydar ^{[2
]}

Corbacioglu, Selim ^{[3
]}

de la Fuente, Josu ^{[4
]}

Wall, Donna ^{[5
]}

Cappellini, Maria Domenica ^{[6
]}

de Montalembert, Mariane ^{[7
]}

Kattamis, Antonis ^{[8
]}

Lobitz, Stephan ^{[9
]}

Rondelli, Damiano ^{[10
]}

Sheth, Sujit ^{[11
]}

Steinberg, Martin ^{[12
]}

Walters, Mark C. ^{[13
]}

Bobruff, Yael ^{[14
]}

Simard, Chris ^{[14
]}

Song, Yang ^{[14
]}

Zhang, Lanju ^{[14
]}

Sharma, Anjali ^{[15
]}

Imren, Suzan ^{[14
]}

Hobbs, Bill ^{[14
]}

Grupp, Stephan ^{[16
]}

机构：

[1] Osped Pediat Bambino Gesu, Rome, Italy

[2] Childrens Hosp, Sarah Cannon Ctr Blood Canc, Tri Star Centennial, Nashville, TN USA

[3] Univ Regensburg, Regensburg, Germany

[4] Imperial Coll Healthcare NHS Trust, St Marys Hosp, London, England

[5] Univ Toronto, Hosp Sick Children, Toronto, ON, Canada

[6] Univ Milan, Milan, Italy

[7] Univ Paris, Necker Enfants Malades Hosp, Assistance Publ Hop Paris, AP HP, Paris, France

[8] Univ Athens, Athens, Greece

[9] Gemeinschaftsklin Mittelrhein, Koblenz, Germany

[10] Univ Illinois, Chicago, IL USA

[11] Cornell Univ, Joan & Sanford Weill Med Coll, New York, NY USA

[12] Boston Univ, Sch Med, Boston, MA USA

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

[14] Vertex Pharmaceut Inc, Boston, MA USA

[15] CRISPR Therapeut, Cambridge, England

[16] Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA USA

来源：

BONE MARROW TRANSPLANTATION | 2023年 / 58卷 / SUPP1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：

O005

引用

页码：16 / 17

页数：2

共 50 条

[21] Umbilical cord blood transplantation for transfusion-dependent thalassemia and sickle cell disease
Mark, Walters
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2006, 12 (11) : 1222 - 1222
[22] MODEL-PROJECTED CLINICAL OUTCOMES OF EXAGAMGLOGENE AUTOTEMCEL (EXA-CEL) IN PATIENTS WITH TRANSFUSION-DEPENDENT BETA-THALASSEMIA IN CANADA
Udeze, C.
Gargano, M.
Kaindl, L.
Kane, S.
Zhou, A.
Jain, S.
Lopez, A.
VALUE IN HEALTH, 2024, 27 (12) : S130 - S130
[23] Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia
Chandra, Jagdish
Parakh, Nupur
Sidharth
Singh, Neha
Sharma, Sunita
Goel, Manish
Pemde, Harish
INDIAN PEDIATRICS, 2021, 58 (07) : 611 - 616
[24] Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia
Jagdish Chandra
Nupur Parakh
Neha Sidharth
Sunita Singh
Manish Sharma
Harish Goel
Indian Pediatrics, 2021, 58 : 611 - 616
[25] Safety and Efficacy of Blood Exchange Transfusion for Priapism Complicating Sickle Cell Disease
Ballas, Samir K.
Lyon, David
JOURNAL OF CLINICAL APHERESIS, 2016, 31 (01) : 5 - 10
[26] Safety and Efficacy Results with a Single Dose of Autologous CRISPR-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells (HSPCs) in Transfusion-Dependent β-Thalassemia (TDT) and Sickle Cell Disease (SCD)
Ho, T. W.
Frangoul, H.
Bobruff, Y.
Cappellini, M. D.
Corbacioglu, S.
Fernandez, C. M.
de la Fuente, J.
Grupp, S.
Handgretinger, R.
Imren, S.
Kattamis, A.
Lekstrom-Himes, J.
Locatelli, F.
Lu, Y.
Mapara, M.
Mulcahey, S.
de Montalembert, M.
Rondelli, D.
Shanbhag, N.
Sheth, S.
Soni, S.
Steinberg, M. H.
Weinstein, M.
Wu, J.
Wall, D.
MOLECULAR THERAPY, 2021, 29 (04) : 64 - 65
[27] Evaluation of home desferal care in transfusion-dependent children with thalassemia and sickle cell disease
Weissman, L
Treadwell, M
Vichinsky, E
INTERNATIONAL JOURNAL OF PEDIATRIC HEMATOLOGY/ONCOLOGY, 1997, 4 (01): : 61 - 68
[28] Safety and efficacy of elective automated red cell exchange transfusion programmes for patients with sickle cell disease at a single centre
Miller, P. D. E.
Ortizo, J.
Baconguis, A.
Perez, M.
Stylianou, J.
Feane, K.
Rhodes, E. S.
BRITISH JOURNAL OF HAEMATOLOGY, 2015, 169 : 104 - 104
[29] Transfusion and sickle cell disease: Axes of transfusion safety optimization
Noizat-Pirenne, F.
TRANSFUSION CLINIQUE ET BIOLOGIQUE, 2014, 21 (02) : 77 - 84
[30] Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
Yannaki, Evangelia
Locatelli, Franco
Thrasher, Adrian J.
Tisdale, John F.
Orchard, Paul J.
Duncan, Christine N.
Kuehl, Joern-Sven
De Oliveira, Satiro
Sauer, Martin G.
Kulozik, Andreas E.
Hongeng, Suradej
Mapara, Markus Y.
Krishnamurti, Lakshmanan
Hermine, Olivier
Blanche, Stephane
Aubourg, Patrick
Smith, Nicholas
Shi, Weiliang
Colvin, Richard A.
McNeil, Elizabeth
Ribeil, Jean-Antoine
Cavazzana, Marina
Walters, Mark C.
Williams, David A.
BONE MARROW TRANSPLANTATION, 2020, 55 (SUPPL 1) : 75 - 76

← 1 2 3 4 5 →