在单臂、开放标签、多中心、2期PILOT研究中，采用嵌合抗原受体（CAR）T细胞治疗药物lisocabtagene maraleucel（liso-cel）作为二线治疗，治疗复发性或难治性大B细胞淋巴瘤（LBCL）患者，其中不计划进行造血干细胞移植（HSCT），结果显示高响应率、持久性反应，且安全性与先前报告一致。在这里，我们对PILOT中接受liso-cel治疗的患者的健康相关生命质量（HRQOL）变化进行了分析。患者接受liso-cel治疗，是一种自体、针对CD19的、4-1BBCAR T细胞制品，以CD8+和CD4+ CAR+ T细胞等目标剂量总剂量100 × 10⁶ CAR+ T细胞进行治疗。PILOT的预定次要终点为HRQOL，使用3种患者自报结果工具（EORTC QLQ-C30；FACT-LymS；EQ-5D-5L）进行评估。EORTC QLQ-C30疲劳和FACT-LymS在多数治疗后访问中均获得了临床显著改善。总体平均改变从基线到545天显示了EORTC QLQ-C30疲劳、疼痛和食欲减退、FACT-LymS和EQ-5D VAS的显著改善。在患者内分析中，大多数患者在90、180、270和365天的得分上观察到临床显著改善或维持。在PILOT中作为二线治疗接受liso-cel的患者中，HRQOL得到维持或改善。这些发现支持liso-cel作为R/R LBCL患者中未计划进行HSCT的二线治疗的首选方案。

In the single-arm, open-label, multicenter, phase 2 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 × 10⁶ CAR+ T cells. HRQOL, a secondary endpoint of PILOT, was assessed as prespecified using 3 patient-reported outcome instruments (EORTC QLQ-C30; FACT-LymS; EQ-5D-5L). Evaluable datasets for the EORTC QLQ-C30, FACT-LymS, EQ-5D-5L health utility index, and EQ-5D visual analog scale (VAS) included 56 (92%), 49 (80%), 55 (90%), and 54 (89%) patients, respectively. Clinically meaningful improvement was achieved across most posttreatment 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-5D 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.