Department of Haematology, King's College Hospital, London, UK.
Department of Haematology, University College London Hospitals, London, UK.
Br J Haematol. 2022 Aug;198(3):492-502. doi: 10.1111/bjh.18209. Epub 2022 Apr 29.
CD19 CAR-T have emerged as a new standard treatment for relapsed/refractory (r/r) large B-cell lymphoma (LBCL). CAR-T real-world (RW) outcomes published to date suggest significant variability across countries. We provide results of a large national cohort of patients intended to be treated with CAR-T in the UK. Consecutive patients with r/r LBCL approved for CAR-T by the National CAR-T Clinical Panel between December 2018 and November 2020 across all UK CAR-T centres were included. 404/432 patients were approved [292 axicabtagene ciloleucel (axi-cel), 112 tisagenlecleucel (tisa-cel)], 300 (74%) received the cells. 110/300 (38.3%) patients achieved complete remission (CR) at 6 months (m). The overall response rate was 77% (52% CR) for axi-cel, 57% (44% CR) for tisa-cel. The 12-month progression-free survival was 41.8% (axi-cel) and 27.4% (tisa-cel). Median overall survival for the intention-to-treat population was 10.5 m, 16.2 m for infused patients. The incidence of grade ≥3 cytokine release syndrome and neurotoxicity were 7.6%/19.6% for axi-cel and 7.9%/3.9% for tisa-cel. This prospective RW population of CAR-T eligible patients offers important insights into the clinical benefit of CD19 CAR-T in LBCL in daily practice. Our results confirm long-term efficacy in patients receiving treatment similar to the pivotal trials, but highlight the significance of early CAR-T failure.
CD19 CAR-T 已成为复发/难治性（r/r）大 B 细胞淋巴瘤（LBCL）的新的标准治疗方法。迄今为止，已公布的 CAR-T 真实世界（RW）结果表明，各国之间存在显著差异。我们提供了一个大型全国性患者队列的结果，这些患者计划在英国接受 CAR-T 治疗。纳入了 2018 年 12 月至 2020 年 11 月期间，经国家 CAR-T 临床专家组批准用于 CAR-T 的所有英国 CAR-T 中心的 r/r LBCL 连续患者。404/432 例患者获得批准[292 例 axicabtagene ciloleucel（axi-cel），112 例 tisagenlecleucel（tisa-cel）]，其中 300 例（74%）接受了细胞治疗。在 6 个月时，110/300 例（38.3%）患者达到完全缓解（CR）。axi-cel 的总缓解率为 77%（52%CR），tisa-cel 为 57%（44%CR）。12 个月无进展生存率为 axi-cel 为 41.8%，tisa-cel 为 27.4%。意向治疗人群的中位总生存期为 10.5 个月，输注患者为 16.2 个月。axi-cel 和 tisa-cel 的 3 级及以上细胞因子释放综合征和神经毒性发生率分别为 7.6%/19.6%和 7.9%/3.9%。这项前瞻性 RW 可接受 CAR-T 的患者人群为 CD19 CAR-T 在 LBCL 中的临床获益提供了重要的见解。我们的结果证实了接受与关键试验相似治疗的患者的长期疗效，但强调了早期 CAR-T 失败的重要性。
