Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent ß-Thalassemia Enrolled in Phase 3 Studies

被引：3

作者：

Kwiatkowski, Janet L. ^{[1
,2
]}

Locatelli, Franco ^{[3
]}

Walters, Mark C. ^{[4
]}

Porter, John B. ^{[5
]}

Hongeng, Suradej ^{[6
]}

Yannaki, Evangelia ^{[7
]}

Kulozik, Andreas E. ^{[8
,9
]}

Sauer, Martin G. ^{[10
]}

Thrasher, Adrian J. ^{[11
]}

Thuret, Isabelle ^{[12
]}

Lal, Ashutosh ^{[4
]}

Guo, Ruiting ^{[13
]}

Colvin, Richard A. ^{[13
]}

Gruppioni, Katiana ^{[13
]}

Thompson, Alexis A. ^{[14
,15
]}

机构：

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

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

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

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

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

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

[7] G Papanikolaou Hosp, Hematol HCT Unit, Gene & Cell Therapy Ctr, Thessaloniki, Greece

[8] Heidelberg Univ, Mol Med Partnership Unit MMPU, European Mol Biol Lab EMBL, Heidelberg, Germany

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

[10] Hannover Med Sch, Dept Hematol & Oncol, Hannover, Germany

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

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

[13] Bluebird Bio Inc, Cambridge, MA USA

[14] Ann Robert H Lurie Childrens Hosp Chicago, Hematol Sect, Chicago, IL USA

[15] Northwestern Univ, Dept Pediat Hematol Oncol & Stem Cell Transplant, Feinberg Sch Med, Chicago, IL USA

来源：

BLOOD | 2021年 / 138卷

关键词：

D O I：

10.1182/blood-2021-150392

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

3085

引用

页数：6

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