Ciltacabtagene autoleucel (cilta-cel) CAR-T 疗法于 2022 年获得批准，用于治疗复发/难治性多发性骨髓瘤 (RRMM) 患者。我们报告了标准护理环境中 cilta-cel 的结果。 2022 年 3 月 1 日至 2022 年 12 月 31 日期间，在 16 个美国学术医疗中心接受白细胞分离术以制造 cilta-cel 的 RRMM 患者被纳入研究。结果：255 名患者接受了白细胞分离术，236 名患者 (92.5%) 接受了 cilta-cel。在接受白细胞分离术的患者中，56% 不符合 CARTITUDE-1 试验资格标准。首次尝试和总体制造失败率分别为 6% 和 1%。既往治疗中位数为 6。在接受治疗的患者 (N=236) 中，75% 出现细胞因子释放综合征（>= 3 级：5%），14% 出现免疫效应细胞相关神经毒性综合征（>= 3 级） ：4%），延迟神经毒性为10%。最佳总体 CR 率和≥ CR 率如下：输注患者 (N=236)：89% 和 70%；接受合格 CAR-T 产品的患者 (N=191) 94% 和 74%；符合氟达拉滨/环磷酰胺淋巴清除的 CAR-T 产品 (N=152)：分别为 95% 和 76%。非复发死亡率为 10%，最常见的是感染。 CAR-T 中位随访 13 个月后，中位无进展生存期 (PFS) 尚未达到，12 个月估计为 68%（95% CI：62-74%）。高铁蛋白水平、高风险细胞遗传学和髓外疾病与较差的 PFS 独立相关，并有先前 BCMA-TT 的信号 (p=0.08)。排除非黑色素瘤皮肤癌的第二原发恶性肿瘤 (SPM) 的发生率为 5.5%，骨髓恶性肿瘤/急性白血病的发生率为 1.7%。尽管超过一半的患者不符合 CARTITUDE-1 资格标准，但我们观察到 cilta-cel 标准护理在 RRMM 中的良好疗效。版权所有 © 2024 美国血液学会。

Ciltacabtagene autoleucel (cilta-cel) CAR-T therapy was approved in 2022 for patients with relapsed/refractory multiple myeloma (RRMM). We report outcomes with cilta-cel in the standard-of-care setting. Patients with RRMM who underwent leukapheresis for cilta-cel manufacturing between 3/1/2022-12/31/2022 at 16 US academic medical centers were included. RESULTS: 255 patients underwent leukapheresis and 236 (92.5%) received cilta-cel. Of leukapheresed patients, 56% would not have met CARTITUDE-1 trial eligibility criteria. Manufacturing failure rates at first attempt and overall were 6% and 1%, respectively. Median prior lines of therapy were 6. In treated patients (N=236), cytokine release syndrome was seen in 75% (>= grade 3: 5%), immune effector cell-associated neurotoxicity syndrome in 14% (>= grade 3: 4%), and delayed neurotoxicity in 10%. Best overall and >= CR rates were as follows: infused patients (N=236): 89% and 70%; patients receiving conforming CAR-T product (N=191) 94% and 74%; conforming CAR-T product with fludarabine/cyclophosphamide lymphodepletion (N=152): 95% and 76%, respectively. Non-relapse mortality was 10%, most commonly from infection. After median follow-up of 13 months from CAR-T, median progression-free survival (PFS) was not reached, with 12- month estimate being 68% (95% CI: 62-74%). High ferritin levels, high-risk cytogenetics, and extramedullary disease were independently associated with inferior PFS, with a signal for prior BCMA-TT (p=0.08). Second primary malignancies (SPMs) excluding non-melanoma skin cancers were seen in 5.5% and myeloid malignancies/acute leukemia in 1.7%. We observed a favorable efficacy profile of standard of care cilta-cel in RRMM despite more than half the patients not meeting CARTITUDE-1 eligibility criteria.Copyright © 2024 American Society of Hematology.