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接受 CD19 CAR-T 细胞治疗的年轻成人患者的真实世界结局

英文原题:Real-world outcomes for young adult patients receiving CD19 CAR T-cell therapy.

查看英文原题

Real-world outcomes for young adult patients receiving CD19 CAR T-cell therapy.

PubMed 2025/06/10(内容时间) Blood Adv Q1 · IF 7.7(JCR 2025)

分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。

中文摘要

Tisagenlecleucel(tisa-cel)和 brexucabtagene autoleucel(brexu-cel)是获批用于复发/难治性 B 急性淋巴细胞白血病青年成人(YA)的 CD19 CAR-T 细胞产品。此前尚无针对接受商业化 CD19 CAR-T 青年成人的独立分析。研究者利用儿科真实世界 CAR-T 联盟和成人 ALL CAR-T 真实世界结局协作项目的回顾性数据,描述 70 例青年成人(18–26 岁;tisa-cel,n = 50;brexu-cel,n = 20)接受 tisa-cel 或 brexu-cel 的疗效和安全性。

brexu-cel 组的细胞因子释放综合征(CRS)和免疫效应细胞相关神经毒性综合征(ICANS)更常见(CRS:85% 对 56%;ICANS:40% 对 18%)。两种产品的完全缓解率相近:brexu-cel 为 80%,tisa-cel 为 88%。12 个月无复发生存率(RFS)在 brexu-cel 组为 46%,在 tisa-cel 组为 36%。缓解持续超过 12 个月的比例分别为 61% 和 41%;brexu-cel 组 12 个月总生存率(OS)为 84%,tisa-cel 组为 68%。多变量分析显示,较低疾病负荷与 OS 改善相关,而 CAR-T 前使用 inotuzumab 与较差结局相关。

本研究显示,不论 CAR 构建体如何,接受 CD19 CAR-T 的青年成人真实世界疗效相近;但 brexu-cel 的毒性发生率似乎更高。

展开英文摘要原文

Tisagenlecleucel (tisa-cel) and brexucabtagene autoleucel (brexu-cel) are approved CD19 chimeric antigen receptor T-cell therapy (CAR T) products for young adults (YA) with relapsed/refractory B-cell acute lymphoblastic leukemia. A distinct analysis of YAs receiving commercial CD19 CAR T has not been reported. Using retrospective data from the Pediatric Real-World CAR T Consortium and the Real-World Outcomes of CAR T in Adult ALL collaboration, we describe the efficacy and safety of tisa-cel and brexu-cel in 70 YAs (18-26 years; tisa-cel, n = 50; brexu-cel, n = 20).

Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were observed more frequently for brexu-cel vs tisa-cel (CRS, 85% vs 56%; ICANS, 40% vs 18%). Complete response rates were similar between products at 80% for brexu-cel and 88% for tisa-cel. Relapse-free survival (RFS) at 12 months was 46% for brexu-cel and 36% for tisa-cel.

Durability of remission over 12 months was 61% for brexu-cel vs 41% for tisa-cel; 12-month overall survival (OS) for brexu-cel was 84% vs 68% for tisa-cel. In multivariate analysis, low disease burden was associated with improved OS, whereas inotuzumab before CAR T was associated with inferior outcomes.

This study demonstrates comparable real-world efficacy among YAs receiving CD19 CAR T irrespective of CAR T construct; however, rates of toxicity seem higher with brexu-cel.

论文信息

作者
Lust H、Schultz LM、Kwon S、Roloff GW、Aldoss I、Baggott C、John S、Rossoff JE
第一作者单位
Division of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.United States
通讯作者单位
Division of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center, Tampa, FL.United States
期刊
Blood advances2025 Jun 10
原文标识
PubMed 40127395 · DOI 10.1182/bloodadvances.2024014846