Ciltacabtagene autoleucel——治疗复发难治性多发性骨髓瘤的靶向BCMA CAR-T细胞新型疗法

Ciltacabtagene autoleucel——a novel BCMA-directed CAR T-cell therapy in patients with relapsed or refractory multiple myeloma

2022年2月28日,美国食品和药物管理局(FDA)批准Ciltacabtagene autoleucel(Cilta-cel)上市,用于治疗成人复发难治性多发性骨髓瘤(relapsed or refractory multiple myeloma,RRMM)。Cilta-cel是中国首个获FDA批准的细胞治疗产品,亦是全球第二款获批靶向B细胞成熟抗原(B-cell maturation antigen,BCMA)的嵌合抗原受体(chimeric antigen receptor,CAR)-T细胞免疫疗法。最新研究发现,RRMM患者接受Cilta-cel治疗后,总缓解率(overall remission rate,ORR)高达97%,12个月无进展生存率为77%;常见毒性作用包括中性粒细胞减少、血小板减少、贫血、细胞因子释放综合征(cytokine release syndrome,CRS)及神经毒性等。本文将对Cilta-cel的作用机制、适应症、药代动力学、临床研究及不良反应等作一综述。

On February 28,2022,the U.S.Food and Drug Administration(FDA)approved Ciltacabtagene autoleucel(Cilta-cel)for the treatment of relapsed or refractory multiple myeloma(RRMM)in adults.Cilta-cel is the first FDA-approved cell therapy product in China and the second chimeric antigen receptor(CAR)-T cell immunotherapy to target the B cell maturation antigen(BCMA)approved by FDA around the world.Recent studies have found that patients with RRMM treated with Cilta-cel have an overall remission rate(ORR)of 97%and a 12-month progression-free rate of 77%;common adverse effects include neutropenia,thrombocytopenia,anemia,cytokine release syndrome(CRS),neurotoxicity and so on.In this article,we summarize the mechanism of drug action,indications,pharmacokinetics,clinical studies and adverse effects of Cilta-cel briefly.