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
欧洲接受 CAR-T 细胞治疗的血液恶性肿瘤患者的患者报告结局
英文原题：Patient-reported outcomes in patients with hematologic malignancies treated with CAR T-cell therapy in Europe.
查看英文原题
Patient-reported outcomes in patients with hematologic malignancies treated with CAR T-cell therapy in Europe.
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中文摘要
患者报告结局（PRO）可直接反映治疗对患者生活的影响，并补充临床结局信息。然而，自CAR-T 细胞疗法问世以来，PRO报告仍不足。尤其是长期健康相关生活质量（HRQoL）以及心理和社会福祉、工作生活和经济负担等维度，相关信息有限。
因此，我们在欧洲接受CAR-T 治疗的血液系统恶性肿瘤患者中开展横断面研究，评估多维PRO。患者填写经过验证的问卷（EQ-5D-5L、EORTC-QLQ-C30、PCL-5、修订版iPCQ），并回答有关治疗体验、未满足照护需求及HRQoL的自拟问题。问卷于2023年1月至10月以7种语言在线提供。根据具有临床意义差异/问题的既定阈值及回归模型，将结果与欧洲一般人群、匹配的未接受CAR-T 血液系统恶性肿瘤队列以及不同亚组进行比较。来自欧洲10个国家的389名患者参与，其中56%接受CAR-T 治疗已超过1年。EQ-5D视觉模拟评分均值为73.1（标准差18.5）。除角色、社会和认知功能外，患者HRQoL与参照队列相似或更好。最常报告的是躯体功能问题（41%），女性、年长者及发生神经毒性的患者尤为明显；后者也报告更多认知和社会功能问题。患者普遍担心疾病复发（76%）、感染（66%）及长期副作用（59%）。在工作年龄患者中，72%接受CAR-T 后仍能继续有偿工作。年轻患者报告经济困难的比例（32%）高于年长患者（9%）。
本研究显示，与参照队列相比，CAR-T 后总体HRQoL较好，但仍有相当比例患者存在躯体、心理和社会福祉问题。研究识别出需要在随访中重点关注的高风险亚组和照护需求。
展开英文摘要原文
Patient-reported outcomes (PROs) give direct insights into the treatment's impact on patient's life and complement clinical outcomes.
However, since the advent of chimeric antigen receptor T-cell therapy (CAR-T), PROs have been underreported. Particularly, little is known about long-term health-related quality of life (HRQoL) and dimensions such as mental- and social well-being, working life, and financial burden.
Therefore, we evaluated multidimensional PROs in a cross-sectional study among European patients who received CAR-T for hematologic malignancies. Patients completed validated questionnaires (EQ-5D-5L/EORTC-QLQ-C30/PCL-5/modified-iPCQ) and ad hoc items on treatment experiences, unmet care needs, and HRQoL. The survey was available online (January-October 2023) in 7 languages. Outcomes were compared with the European general population, a matched CAR-T-naive cohort with hematologic malignancies and across subgroups, using established thresholds for clinically important differences/problems and regression models. From 10 European countries, 389 patients participated (>1 year post-CAR-T: 56%).
Mean EQ-VAS was 73. 1 (standard deviation, 18. 5). HRQoL was similar or better than reference cohorts, except for role-, social-, and cognitive-functioning. Physical-functioning problems were most frequently reported (41%), particularly by women, older individuals, and those who experienced neurotoxicity.
The latter subgroup also reported more cognitive- and social-functioning problems. Anxiety regarding disease recurrence (76%), infections (66%) and long-term side effects (59%) was common. Among working-age patients, 72% could continue paid work after CAR-T. Younger patients (32%) reported more financial difficulties than older patients (9%).
This study shows favorable general HRQoL after CAR-T compared with reference cohorts.
However, a notable proportion of patients experienced problems in physical-, mental- and social well-being.
We identified high-risk subgroups and care needs that should be addressed during follow-up.
论文信息
- 作者
- Pennings ERA、Spanjaart AM、Thielen FW、Oerlemans S、Fleischer A、Sanges C、Gomes Da Silva M、Cabrerizo Y
- 单位
- Department of Hematology, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.Netherlands
- 期刊
- Blood advances2025 Dec 23
- 原文标识
- PubMed 40991391 · DOI 10.1182/bloodadvances.2025017081
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