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

共 50 条

[1] BETIBEGLOGENE AUTOTEMCEL IN PEDIATRIC PTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA IN PHASE 3 TRIALS
Lal, Ashutosh
Kwiatkowski, Janet
Thompson, Alexis
Thuret, Isabelle
Thrasher, Adrian
Kulozik, Andreas
Hongeng, Suradej
Yannaki, Evangelia
Sauer, Martin
Guo, Ruiting
Colvin, Richard
Locatelli, Franco
Walters, Mark
PEDIATRIC BLOOD & CANCER, 2022, 69
[2] Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia
Locatelli, Franco
Lang, Peter
Corbacioglu, Selim
Wall, Donna
Li, Amanda M.
de La Fuente, Josu
Kohli, Puja
Li, Nanxin
Rubin, Jaime
Zhang, Siyu
Zhang, Lanju
Hobbs, William
Frangoul, Haydar
BLOOD, 2023, 142
[3] Adherence and Health-related Quality of Life of Transfusion-Dependent Thalassemia Patients
Jin, Lee Wan
Tahir, Nurul Ain Mohd
Islahudin, Farida
Chuen, Li Shu
ARCHIVES OF PHARMACY PRACTICE, 2023, 14 (04) : 89 - 95
[4] Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent ß-Thalassemia
Locatelli, Franco
Walters, Mark C.
Kwiatkowski, Janet L.
Cavazzana, Marina
Hongeng, Suradej
Porter, John B.
Thrasher, Adrian J.
Kulozik, Andreas E.
Thuret, Isabelle
Rasko, John E. J.
Yannaki, Evangelia
Sauer, Martin G.
Ali, Shamshad
Thakar, Himal
Gruppioni, Katiana
Thompson, Alexis A.
BLOOD, 2022, 140 : 8200 - 8202
[5] Health-related Quality of Life and its Predictors Among Transfusion-dependent Thalassemia Patients
Tuysuz, Gulen
Tayfun, Funda
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY, 2017, 39 (05) : 332 - 336
[6] 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)
Thompson, Alexis A.
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
Thakar, Himal L.
Deora, Ami
Gruppioni, Katiana
Colvin, Richard A.
Locatelli, Franco
Kwiatkowski, Janet L.
BLOOD, 2023, 142
[7] Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial
Cappellini, Maria Domenica
Taher, Ali T.
Piga, Antonio
Shah, Farrukh
Voskaridou, Ersi
Viprakasit, Vip
Porter, John B.
Hermine, Olivier
Neufeld, Ellis J.
Thompson, Alexis A.
Tang, Derek
Yu, Peiwen
Guo, Shien
Shetty, Jeevan K.
Miteva, Dimana
Zinger, Tatiana
Backstrom, Jay T.
Oliva, Esther Natalie
BLOOD, 2020, 136
[8] Health-related quality of life in pediatric and adolescent patients with transfusion-dependent ß-thalassemia in upper Egypt (single center study)
Gehan L Abdel Hakeem
Suzan O Mousa
Asmaa N Moustafa
Mohamed H Mahgoob
Ebtesam E Hassan
Health and Quality of Life Outcomes, 16
[9] Health-related quality of life in pediatric and adolescent patients with transfusion-dependent β-thalassemia in upper Egypt (single center study)
Hakeem, Gehan L. Abdel
Mousa, Suzan O.
Moustafa, Asmaa N.
Mahgoob, Mohamed H.
Hassan, Ebtesam E.
HEALTH AND QUALITY OF LIFE OUTCOMES, 2018, 16
[10] Factors Related to the Quality of Life in Children with Transfusion-Dependent Thalassemia
Permana, Dea Aprilianti
Susanah, Susi
Adrizain, Riyadi
Rahayuningsih, Sri Endah
Dhamayanti, Meita
Rakhmilla, Lulu Eva
PEDIATRIC HEMATOLOGY AND ONCOLOGY, 2024, 41 (03) : 179 - 197

← 1 2 3 4 5 →