Long-term follow-up of BCMA CAR-T cell therapy in patients with relapsed/refractory multiple myeloma

作者：Chun Mei Jin, Rongrong Chen, Shan Fu, Mingming Zhang, Yuanyin Teng, Tingting Yang, Fengmei Song, Jingjing Feng, Ruimin Hong, Jiazhen Cui, Simao Huang, Huijun Xu, Yanlei Zhang, Guoqing Wei, Zhen Cai, Yok-Lam Kwong, Thomas Sau Yan Chan, Alex H. Chang, He Huang, Yongxian Hu · 发表于：Journal for ImmunoTherapy of Cancer · 年份：2025 · DOI：10.1136/jitc-2024-010687 · 被引用次数：14 · 研究领域：CAR-T cell therapy research、Multiple Myeloma Research and Treatments、Immunotherapy and Immune Responses

BACKGROUND: B-cell maturation antigen (BCMA)-targeting chimeric antigen receptor (CAR) T-cell immunotherapy has shown promising results in the treatment of relapsed or refractory multiple myeloma (R/RMM). This study presents the updated long-term outcomes from our center. METHODS: cells/kg. The study evaluated overall response rates, long-term efficacy, safety profiles, and their associations with clinical and disease characteristics. RESULTS: At a median follow-up of 20.2 months, the safety profile of the therapy was manageable. Grade 3/4 cytokine release syndrome occurred in 36.2% of patients, with no cases of severe neurotoxicity reported. 1-month post-infusion, grade ≥3 anemia persisted in 39.6% of patients, while neutropenia (43.3%) and thrombocytopenia (52.2%) were observed. The objective response rate (ORR) among evaluable patients was 94.8%, with 50.7% achieving a complete response (CR). The 4-year progression-free survival and overall survival rates were 37.4% (95% CI, 29.1% to 48.1%) and 63.2% (95% CI, 54.8% to 72.8%), respectively, with survival curves showing gradual flattening over time. Patients with a history of autologous stem cell transplantation (ASCT) and those with extramedullary disease demonstrated significantly inferior efficacy and survival outcomes. Peak CAR-T cell expansion was positively correlated with ORR (p<0.001) and CR (p<0.001). Notably, patients with prior ASCT exhibited significantly lower CAR-T cell expansion compared with those without pri...