Are CAR-T therapies living up to their hype? A study using real-world data in two cohorts to determine how well they are actually working in practice compared with bone marrow transplants.
Are CAR-T therapies living up to their hype? A study using real-world data in two cohorts to determine how well they are actually working in practice compared with bone marrow transplants.
复制标题
DOI：
10.1136/bmjebm-2019-111226
复制
发表时间：
2021-06
影响因子：
5.8
通讯作者：
Jagasia M
中科院分区：
文献类型：
--
作者：
Schulthess D;Gassull D;Makady A;Ludlow A;Rothman B;Have PT;Wu Y;Ekstrom L;Minnema M;Jagasia M
随着新型监管工具的越来越多的使用，例如食品药品监督管理局的突破性设计，欧洲卫生技术评估者（HTAS）面临越来越多的挑战，以准确评估嵌合抗原受体T细胞的长期价值和性能（CAR-T）疗法，特别是针对孤儿的疗法，例如急性淋巴细胞白血病。从医学中心的电子健康记录中提取的纵向现实世界数据作为临床试验地点，以对CAR-T的性能进行准确分析与次要治疗选项相比，即同种异体造血细胞移植（ HCT）在CAR-T队列中，生存率为46％（95％CI：08％至79％），HCT队列中有68％（95％CI：46％至83％）。 -T队列与HCT队列相比，作者假设CAR-T队列的疾病水平要高得多。网站。更大的研究。
With the increasing use of new regulatory tools, like the Food and Drug Administration’s breakthrough designation, there are increasing challenges for European health technology assessors (HTAs) to make an accurate assessment of the long-term value and performance of chimeric antigen receptor T-cell (CAR-T) therapies, particularly for orphan conditions, such as acute lymphoblastic leukaemia. The aim of this study was to demonstrate a novel methodology harnessing longitudinal real-world data, extracted from the electronic health records of a medical centre functioning as a clinical trial site, to develop an accurate analysis of the performance of CAR-T compared with the next-best treatment option, namely allogeneic haematopoietic cell transplant (HCT). The study population comprised 43 subjects in two cohorts: 29 who had undergone HCT treatment and 14 who had undergone CAR-T therapy. The 3-year relapse-free survival probability was 46% (95% CI: 08% to 79%) in the CAR-T cohort and 68% (95% CI: 46% to 83%) in the HCT cohort. To explain the lower RFS probability in the CAR-T cohort compared with the HCT cohort, the authors hypothesised that the CAR-T cohort had a far higher level of disease burden. This was validated by log-rank test analysis (p=0.0001) and confirmed in conversations with practitioners at the study site. The authors are aware that the small populations in this study will be seen as limiting the generalisability of the findings to some readers. However, in consultation with many European HTAs and regulators, there is broad agreement that this methodology warrants further investigation with a larger study.
登录
查看更多内容
DOI：
10.1056/nejmoa1707447
发表时间：
2017-12-28
期刊：
The New England journal of medicine
影响因子：
--
作者：
Neelapu SS;Locke FL;Bartlett NL;Lekakis LJ;Miklos DB;Jacobson CA;Braunschweig I;Oluwole OO;Siddiqi T;Lin Y;Timmerman JM;Stiff PJ;Friedberg JW;Flinn IW;Goy A;Hill BT;Smith MR;Deol A;Farooq U;McSweeney P;Munoz J;Avivi I;Castro JE;Westin JR;Chavez JC;Ghobadi A;Komanduri KV;Levy R;Jacobsen ED;Witzig TE;Reagan P;Bot A;Rossi J;Navale L;Jiang Y;Aycock J;Elias M;Chang D;Wiezorek J;Go WY
通讯作者：
Go WY
DOI：
10.1056/nejmoa1709919
发表时间：
2018-02-01
期刊：
The New England journal of medicine
影响因子：
--
作者：
Park JH;Rivière I;Gonen M;Wang X;Sénéchal B;Curran KJ;Sauter C;Wang Y;Santomasso B;Mead E;Roshal M;Maslak P;Davila M;Brentjens RJ;Sadelain M
通讯作者：
Sadelain M
影响因子：
3.7
作者：
KAPLAN, EL;MEIER, P
通讯作者：
MEIER, P
DOI：
10.1111/j.1752-8062.2010.00175.x
发表时间：
2010-02
期刊：
Clinical and translational science
影响因子：
--
作者：
Pulley J;Clayton E;Bernard GR;Roden DM;Masys DR
通讯作者：
Masys DR
影响因子：
6.7
作者：
Roden, D. M.;Pulley, J. M.;Masys, D. R.
通讯作者：
Masys, D. R.