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.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
大 B 细胞淋巴瘤中 CAR-T 细胞制备失败和患者结局的风险因素：来自英国国家 CAR-T 专家组的报告
英文原题：Risk factors for CAR T-cell manufacturing failure and patient outcomes in large B-cell lymphoma: a report from the UK National CAR T Panel.
查看英文原题
Risk factors for CAR T-cell manufacturing failure and patient outcomes in large B-cell lymphoma: a report from the UK National CAR T Panel.
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中文摘要
CAR-T 细胞制备失败（MF）是指制备过程未能产出产品，或产出的产品不符合质量标准（OOS）的情况。我们开展了一项多中心回顾性研究，分析导致 MF 的因素及患者结局。在 981 例获批接受 CAR-T 细胞治疗的大 B 细胞淋巴瘤（LBCL）患者中，38 例（3.87%）发生 MF。在 21 次重新制备尝试后，11 例患者接受了符合质量标准产品的延迟输注（延迟输注组）。13 例输注了 OOS 产品（OOS 输注组），14 例未输注。为进行比较，我们纳入 38 例无 MF 的 LBCL 对照；29 例接受了输注（对照输注组）。既往苯达莫司汀治疗是唯一与 MF 风险相关的基线变量，主要归因于 6 个月内接受治疗；MF 组为 23.7%，对照组为 0%（P = 0.0029）。
输注患者的总生存期（OS）和无进展生存期（PFS）无显著差异，OOS 输注组、延迟输注组和对照输注组的 1 年 OS（PFS）分别为 52.8%（46.2%）、46.8%（24.2%）和 68.4%（41.4%）（PFS HR：OOS 输注组 vs 对照输注组 1.41，P = 0.40；延迟输注组 vs 对照输注组 1.64，P = 0.25；OOS 输注组 vs 延迟输注组 0.86，P = 0.76）。各队列之间的 CRS、ICANS 和血细胞减少无显著差异。MF 后输注 OOS 产品的 LBCL 患者结局令人鼓舞。重新制备使约 50% 的患者得以输注符合质量标准的产品，对于无法获得合适 OOS 产品的患者，这可能是一种选择。
展开英文摘要原文
CAR T-cell manufacturing failure (MF) is a situation where the manufacturing process fails to yield a product or results in one which is out-of-specification (OOS).
We conducted a multicentre retrospective review of factors contributing to MF and patient outcomes. Of 981 large B-cell lymphoma (LBCL) patients approved for CAR T-cell therapy, 38 (3. 87%) had MF. Eleven patients received delayed infusion with a product in-specification (delayed-infused) following 21 remanufacturing attempts.
OOS product was infused in 13 (OOS-infused), and 14 were not infused. For comparison, we included 38 LBCL controls without MF; 29 received infusion (controls-infused). Prior bendamustine was the only baseline variable associated with MF risk, largely due to therapy within 6 months; 23. 7% MF vs 0% controls (P = 0. 0029).
Overall survival (OS) and progression-free survival (PFS) were not significantly different for infused patients, with 1-year OS (PFS) of 52. 8% (46. 2%), 46. 8% (24. 2%) and 68. 4% (41. 4%) for OOS-infused, delayed-infused and controls-infused respectively (PFS HR OOS-infused vs controls-infused 1. 41, P = 0. 40; delayed-infused vs controls-infused 1. 64, P = 0.
25; and OOS-infused vs delayed-infused 0. 86, P = 0. 76). CRS, ICANS and cytopenias were not significantly different between cohorts. Outcomes for OOS-infused LBCL patients following MF are encouraging. Remanufacturing led to infusion of a product in-specification in around 50% and may be an option for patients where a suitable OOS product is not available.
论文信息
- 作者
- Dulobdas V、Kirkwood AA、Serpenti F、Gautama B、Panopoulou A、Mathew A、Gabriel S、Malladi R
- 单位
- Centre for Clinical Haematology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK. vaishali.dulobdas@uhb.nhs.uk.United Kingdom
- 文献类型
- 多中心研究
- 期刊
- Blood cancer journal2025 Mar 4
- 原文标识
- PubMed 40032870 · DOI 10.1038/s41408-025-01225-9
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