肿瘤细胞治疗研究
英文原题：Cesni-cel (ARI0002h) in ultra-high-risk multiple myeloma with plasma cell leukaemia or central nervous system involvement.
共纳入 19 例患者（68% 为女性；中位年龄 61 岁）：12 例为 PCL，5 例为 CNS-MM，2 例为双重受累。
超高危多发性骨髓瘤（uHRMM）定义包括髓外病变（EMD）、中枢神经系统（CNS）受累或浆细胞白血病（PCL），仍是CAR-T 细胞治疗后早期复发的预测因素。我们分析了2020至2025年西班牙3家中心接受cesnicabtagene autoleucel（ARI0002h）治疗的PCL/CNS-MM患者特征、毒性、应答和生存结局。PCL依据国际骨髓瘤工作组（IMWG）2021年标准定义；CNS-MM定义为软脑膜和/或脑实质受累。共纳入19例患者（女性68%；中位年龄61岁）：12例PCL、5例CNS-MM、2例同时受累。50%为原发性PCL。CNS-MM患者均有软脑膜病变，其中1例同时有脑实质病灶。57%接受CNS靶向治疗，74%存在高危细胞遗传学异常。既往治疗中位数为3线；74%接受过造血干细胞移植（HSCT）；2例既往暴露于B细胞成熟抗原（BCMA）。1例患者在单采前死亡，1例在生产期间死亡；17例（89%）接受ARI0002h。76%发生细胞因子释放综合征（CRS），均未达到3级；1例发生3级免疫效应细胞相关神经毒性综合征（ICANS，非CNS-MM患者），18%发生免疫效应细胞相关噬血细胞性淋巴组织细胞增多症样综合征（IEC-HS），其中1例致死。第+100天总体缓解率（ORR）为88%，其中87%达到非常好的部分缓解（VGPR），80%达到CNS完全缓解。中位无进展生存期（PFS）和总生存期（OS）分别为10.5个月和15.9个月。ARI0002h可使复发性uHRMM患者获得深度应答，支持将PCL和CNS-MM纳入临床试验。
Ultra-high-risk multiple myeloma (uHRMM), defined by extra-medullary disease (EMD, including central nervous system [CNS] or plasma cell leukaemia [PCL]), remains as a predictor of early relapse after chimeric antigen receptor T cell (CAR-T) therapy. We analysed the characteristics, toxicities, response and survival of patients with PCL/CNS-multiple myeloma (MM) treated with cesnicabtagene autoleucel (ARI0002h) at three Spanish centres (2020-2025). PCL was defined as per International Myeloma Working Group (IMWG) 2021 criteria; CNS-MM by leptomeningeal and/or intraparenchymal involvement. Nineteen patients (68% female; median age 61) were included: 12 patients had PCL, 5 had CNS-MM, and 2 had dual involvement. Fifty per cent had primary PCL. In CNS-MM, all had leptomeningeal disease and one had also intraparenchymal lesions. Fifty-seven per cent received CNS-directed therapy. Seventy four per cent harboured high-risk cytogenetics. Median prior lines: 3; 74% had haematopoietic stem cell transplantation (HSCT); two had prior B-cell maturation antigen (BCMA) exposure. One patient died pre-apheresis and one during manufacturing; 17 (89%) received ARI0002h. Cytokine release syndrome (CRS) occurred in 76% (none G3), G3 immune effector cell (IEC)-associated neurotoxicity syndrome (ICANS) occurred in one case (not CNS-MM) and IEC-associated haemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) occurred in 18% (one fatal). Overall response rate (ORR) was 88% at day +100, of which 87% achieved very good partial response (VGPR) and 80% complete CNS response. Median progression-free survival (PFS) and overall survival (OS) were 10.5 and 15.9 months respectively. ARI0002h induces deep responses in relapsed uHRMM, supporting inclusion of PCL and CNS-MM in clinical trials.
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