Qiuxia Yu, Di Wang, Zhe Li, Ning An, Chunhui Li, Yuhan Bao, Xinyu Wen, Xiaolu Long, Jue Wang, Lijun Jiang, Wei Mu, Peiling Zhang, Chang Shu, Huan Ye, Hongyu Gui, Songbai Cai, Guang Hu, Wen Wang, Aihua Du, Chunrui Li
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引用次数: 0
摘要
CT103A 是一种针对 B 细胞成熟抗原 (BCMA) 的全人嵌合抗原受体 T 细胞 (CAR-T) 产品。本研究介绍了CT103A在复发/难治性多发性骨髓瘤(RRMM)患者中长期随访后的最新安全性和疗效。截至2023年7月31日,CAR-T细胞输注后的中位随访时间为45.0个月(范围为0.7-58.3个月)。在长期随访期间,不良事件的发生率随着时间的推移逐渐下降。一名患者的最长反应持续时间接近五年。所有 18 名患者(100%)都获得了部分缓解或更好的缓解;77.8% 的患者(18 人中有 14 人)最终表现出完全应答(CR)或严格完全应答(sCR),应答随时间推移而增加。在数据截止时,9 名患者仍然存活,7 名患者处于 sCR 状态,最小残留病灶(MRD)为阴性。所有18名患者的中位无进展生存期(PFS)为22.6个月,中位总生存期(OS)为50.2个月。中位 CAR 转基因持续时间为 14.0 个月(0.7-57.3 个月)。长期随访表明,基于全人 CAR-T 细胞的持续存在,CT103A 为 RRMM 患者带来了持久的临床获益。
Long-term safety and efficacy of the fully human CAR-T therapy CT103A in relapsed/refractory multiple myeloma.
CT103A is a fully human chimeric antigen receptor T cell (CAR-T) product for targeting B cell maturation antigen. This study presents the updated safety and efficacy profiles of CT103A in patients with relapsed/refractory multiple myeloma (RRMM) after long-term follow-up. As of July 31, 2023, the median follow-up time after CAR-T cell infusion was 45.0 months (range, 0.7-58.3 months). During long-term follow-up, the incidence of adverse events gradually decreased over time. One patient had a maximum duration of response of nearly 5 years. All 18 patients (100%) achieved partial remission or better; 77.8% (14 of 18) of patients eventually exhibited complete response or stringent complete response (sCR), with response increasing over time. At the time of data cutoff, nine patients were still alive and seven patients had an sCR status with negative minimal residual disease. The median progression-free survival was 22.6 months, and the median overall survival was 50.2 months for all 18 patients. The median CAR transgene persistence was 14.0 months (range, 0.7-57.3 months). Long-term follow-up demonstrated that CT103A confers durable clinical benefit for RRMM patients based on the sustained presence of fully human CAR-T cells.
期刊介绍:
Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.