CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
lisocabtagene maraleucel 用于二线复发/难治性大 B 细胞淋巴瘤：PILOT 研究的患者报告结局
英文原题：Lisocabtagene maraleucel for second-line relapsed or refractory large B-cell lymphoma: patient-reported outcomes from the PILOT study.
查看英文原题
Lisocabtagene maraleucel for second-line relapsed or refractory large B-cell lymphoma: patient-reported outcomes from the PILOT study.
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
在单臂、开放标签、多中心 II 期 PILOT 研究中，对于不计划接受造血干细胞移植（HSCT）的复发/难治性（R/R）大 B 细胞淋巴瘤（LBCL）患者，二线采用嵌合抗原受体（CAR）T 细胞疗法 lisocabtagene maraleucel（liso-cel）取得较高缓解率和持久应答，安全性特征也与既往报告一致。
本研究分析 PILOT 中接受 liso-cel 患者健康相关生活质量（HRQOL）的变化。患者接受自体、靶向 CD19 的 4-1BB CAR-T 产品，其中 CD8⁺ 和 CD4⁺ CAR⁺ T 细胞的目标剂量相等，总目标剂量为 100 × 10⁶ CAR⁺ T 细胞。HRQOL 为 PILOT 的次要终点，按预设方案使用三种患者报告结局工具评估：EORTC QLQ-C30、FACT-LymS 和 EQ-5D-5L。EORTC QLQ-C30、FACT-LymS、EQ-5D-5L 健康效用指数和视觉模拟量表（EQ-VAS）的可评估数据集分别包括 56 例（92%）、49 例（80%）、55 例（90%）和 54 例（89%）患者。
在大多数治疗后访视中，EORTC QLQ-C30 疲劳评分及 FACT-LymS 均达到有临床意义的改善。从基线至第 545 天的总体平均变化显示，EORTC QLQ-C30 疲劳、疼痛和食欲下降评分、FACT-LymS 及 EQ-VAS 均显著改善。患者内分析显示，在第 90、180、270 和 365 天，多数患者的评分达到有临床意义的改善或维持。PILOT 研究中接受二线 liso-cel 的患者 HRQOL 得以维持或改善。这些发现支持将 liso-cel 作为不计划接受 HSCT 的 R/R LBCL 患者优选的二线治疗（ClinicalTrials.gov 注册号：NCT03483103）。
展开英文摘要原文
In the single-arm, open-label, multicenter, phase II PILOT study, second-line treatment with the chimeric antigen receptor (CAR) T-cell therapy lisocabtagene maraleucel (liso-cel) in patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) for whom hematopoietic stem cell transplantation (HSCT) was not intended resulted in high response rates, durable responses, and a safety profile consistent with previous reports.
Here, we analyzed changes in health-related quality of life (HRQOL) in patients who received liso-cel in PILOT. Patients received liso-cel, an autologous, CD19-directed, 4-1BB CAR T-cell product administered at equal target doses of CD8+ and CD4+ CAR+ T cells, for a total target dose of 100 10 CAR+ T cells. HRQOL, a secondary endpoint of PILOT, was assessed as prespecified using three patient-reported outcome instruments (EORTC QLQ-C30; FACT-LymS; EQ-5D-5L).
Evaluable datasets for the EORTC QLQ-C30, FACT-LymS, and EQ-5D-5L health utility index, and visual analog scale (EQ-VAS) included 56 (92%), 49 (80%), 55 (90%), and 54 (89%) patients, respectively. Clinically meaningful improvement was achieved across most post-treatment visits for EORTC QLQ-C30 fatigue and FACT-LymS.
Overall mean changes from baseline through day 545 showed significant improvements in EORTC QLQ-C30 fatigue, pain, and appetite loss, FACT-LymS, and EQ VAS. In within-patient analyses, clinically meaningful improvements or maintenance in scores were observed in most patients at days 90, 180, 270, and 365. HRQOL was maintained or improved in patients who received liso-cel as second-line therapy in PILOT.
These findings support liso-cel as a preferred second-line treatment in patients with R/R LBCL not intended for HSCT (clinicaltrials gov. Identifier: NCT03483103).
论文信息
- 作者
- Gordon LI、Liu FF、Braverman J、Hoda D、Ghosh N、Hamadani M、Hildebrandt GC、Peng L
- 单位
- Northwestern University, Feinberg School of Medicine, Robert H. Lurie Comprehensive Cancer Center, Chicago, IL. l-gordon@northwestern.edu.United States
- 文献类型
- 多中心研究 · 非美国政府资助研究
- 期刊
- Haematologica2024 Mar 1
- 原文标识
- PubMed 37646670 · DOI 10.3324/haematol.2023.283162
在 PubMed 查看 → 出版商原文（DOI） 全文 PDF（PMC）· 可下载 治疗专题与资料阅读指南 资料来源与翻译说明 报告译文或资料问题 →