Department of Hematology, Hospital General Universitario Gregorio Marañón, Madrid, Spain; Instituto de Investigación Sanitaria Gregorio Marañón, Madrid.
Department of Hematology, Vall d'Hebron University Hospital, Experimental Hematology, Vall d'Hebron Institute of Oncology (VHIO), Vall d'Hebron Barcelona Hospital Campus, Passeig Vall d'Hebron 119-129, 08035 Barcelona, Spain; Department of Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra.
Haematologica. 2023 Jan 1;108(1):110-121. doi: 10.3324/haematol.2022.280805.
Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are CD19-targeted chimeric antigen receptor (CAR) T cells approved for relapsed/refractory (R/R) large B-cell lymphoma (LBCL). We performed a retrospective study to evaluate safety and efficacy of axi-cel and tisa-cel outside the setting of a clinical trial. Data from consecutive patients with R/R LBCL who underwent apheresis for axi-cel or tisa-cel were retrospectively collected from 12 Spanish centers. A total of 307 patients underwent apheresis for axi-cel (n=152) and tisa-cel (n=155) from November 2018 to August 2021, of which 261 (85%) received a CAR T infusion (88% and 82%, respectively). Median time from apheresis to infusion was 41 days for axi-cel and 52 days for tisa-cel (P=0.006). None of the baseline characteristics were significantly different between both cohorts. Both cytokine release syndrome and neurologic events (NE) were more frequent in the axi-cel group (88% vs. 73%, P=0.003, and 42% vs. 16%, P<0.001, respectively). Infections in the first 6 months post-infusion were also more common in patients treated with axi-cel (38% vs. 25%, P=0.033). Non-relapse mortality was not significantly different between the axi-cel and tisa-cel groups (7% and 4%, respectively, P=0.298). With a median follow-up of 9.2 months, median PFS and OS were 5.9 and 3 months, and 13.9 and 11.2 months for axi-cel and tisa-cel, respectively. The 12-month PFS and OS for axi-cel and tisa-cel were 41% and 33% (P=0.195), 51% and 47% (P=0.191), respectively. Factors associated with lower OS in the multivariate analysis were increased lactate dehydrogenase, ECOG ≥2 and progressive disease before lymphodepletion. Safety and efficacy results in our real-world experience were comparable with those reported in the pivotal trials. Patients treated with axi-cel experienced more toxicity but similar non-relapse mortality compared with those receiving tisa-cel. Efficacy was not significantly different between both products.
阿基仑赛（axi-cel）和Tisagenlecleucel（tisa-cel）是两种靶向 CD19 的嵌合抗原受体（CAR）T 细胞，已被批准用于治疗复发/难治性（R/R）大 B 细胞淋巴瘤（LBCL）。我们进行了一项回顾性研究，以评估 axi-cel 和 tisa-cel 在临床试验以外环境下的安全性和疗效。
从 2018 年 11 月至 2021 年 8 月，12 个西班牙中心连续收集了接受 axi-cel（n=152）或 tisa-cel（n=155）单采术的 R/R LBCL 患者的数据。其中，261 例（85%）接受了 CAR T 输注（分别为 88%和 82%）。axi-cel 组和 tisa-cel 组从单采术到输注的中位时间分别为 41 天和 52 天（P=0.006）。两组患者的基线特征均无显著差异。axi-cel 组的细胞因子释放综合征（CRS）和神经事件（NE）发生率均高于 tisa-cel 组（88%比 73%，P=0.003；42%比 16%，P<0.001）。axi-cel 组患者在输注后 6 个月内的感染也更为常见（38%比 25%，P=0.033）。axi-cel 组和 tisa-cel 组的非复发死亡率无显著差异（分别为 7%和 4%，P=0.298）。
中位随访 9.2 个月时，axi-cel 和 tisa-cel 的中位 PFS 和 OS 分别为 5.9 个月和 3 个月，13.9 个月和 11.2 个月。axi-cel 和 tisa-cel 的 12 个月 PFS 和 OS 分别为 41%和 33%（P=0.195）、51%和 47%（P=0.191）。多因素分析显示，乳酸脱氢酶升高、ECOG 评分≥2 分和淋巴耗竭前疾病进展与 OS 降低相关。
在我们的真实世界经验中，安全性和疗效结果与关键试验报告的结果相当。与接受 tisa-cel 治疗的患者相比，接受 axi-cel 治疗的患者毒性更大，但非复发死亡率相似。两种产品的疗效无显著差异。
