Ghosh Nilanjan, Sehgal Alison, Liu Fei Fei, Kostic Ana, Crotta Alessandro, De Benedetti Marc, Faccone Jillian, Peng Lily, Gordon Leo I
Atrium Health, Levine Cancer Institute, Charlotte, NC.
University of Pittsburgh Medical Center, Hillman Cancer Center, Pittsburgh, PA.
Haematologica. 2025 Mar 1;110(3):693-705. doi: 10.3324/haematol.2024.285828.
This study assessed the comparative efficacy of lisocabtagene maraleucel (liso-cel) in the open-label, phase II PILOT study (clinicaltrials.gov NCT03483103) versus conventional second-line (2L) chemotherapy regimens in the real world administered to patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) who were not intended for hematopoietic stem cell transplantation (HSCT). The liso-cel-treated cohort (N=61) was based on patients who received liso-cel in the PILOT study. The conventional chemotherapy cohort included patients who met PILOT eligibility criteria and received conventional 2L chemotherapy in the real-world clinical setting (N=273). After using the trimmed stabilized inverse probability of treatment weighting method to balance cohorts according to baseline characteristics, there were statistically significant differences in all tested measures of efficacy. Compared with real-world conventional chemotherapy regimens, liso-cel demonstrated higher overall response rates (79.6% with liso-cel vs. 50.5% with conventional chemotherapy; relative risk [RR]: 1.6; P<0.0001) and complete response rates (53.1% vs. 24.0%; RR: 2.2; P<0.0001), longer median duration of response (12.1 vs. 4.3 months; hazard ratio [HR: 0.40; P=0.0001), longer median event-free survival (7.0 vs. 2.8 months; HR: 0.43; P<0.0001), longer median progression-free survival (7.0 vs. 2.9 months; HR: 0.46; P<0.0001), and longer median overall survival (not reached vs. 12.6 months; HR: 0.58; P=0.0256). Results from analyses applying various additional statistical approaches consistently favored outcomes with liso-cel over real-world conventional chemotherapy regimens. These results reinforce the efficacy of liso-cel as 2L therapy for patients with R/R LBCL who are not intended for HSCT.
本研究在开放标签的II期PILOT研究（clinicaltrials.gov NCT03483103）中评估了利妥昔单抗（liso-cel）与现实世界中给予复发或难治性（R/R）大B细胞淋巴瘤（LBCL）且不打算进行造血干细胞移植（HSCT）患者的传统二线（2L）化疗方案相比的疗效。接受liso-cel治疗的队列（N = 61）基于在PILOT研究中接受liso-cel治疗的患者。传统化疗队列包括符合PILOT纳入标准并在现实世界临床环境中接受传统2L化疗的患者（N = 273）。在使用修剪稳定的治疗权重逆概率方法根据基线特征平衡队列后，所有测试的疗效指标均存在统计学显著差异。与现实世界中的传统化疗方案相比，liso-cel显示出更高的总缓解率（liso-cel为79.6%，传统化疗为50.5%；相对风险[RR]：1.6；P<0.0001）和完全缓解率（53.1%对24.0%；RR：2.2；P<0.0001），更长的中位缓解持续时间（12.1对4.3个月；风险比[HR]：0.40；P = 0.0001），更长的中位无事件生存期（7.0对2.8个月；HR：0.43；P<0.0001），更长的中位无进展生存期（7.0对2.9个月；HR：0.46；P<0.0001），以及更长的中位总生存期（未达到对12.6个月；HR：0.58；P = 0.0256）。应用各种额外统计方法的分析结果一致表明，liso-cel的疗效优于现实世界中的传统化疗方案。这些结果强化了liso-cel作为不打算进行HSCT的R/R LBCL患者的2L治疗的疗效。
