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靶向B细胞成熟抗原(BCMA)的嵌合抗原受体T细胞(CAR-T)疗法作为浆母细胞性骨髓瘤患者的挽救疗法

BCMA CAR-T therapy as salvage therapy in patients with plasmablastic myeloma.

作者信息

Jin Chengwei, Deng Jing, Jiang Ying, Zhu Jun, Kang Liqing, Li Su

机构信息

GoBroad Medical Institute of Hematology (Shanghai Center), Liquan Hospital, Shanghai, People's Republic of China.

Department of Pathology, Beijing GoBroad Boren Hospital, Beijing, People's Republic of China.

出版信息

Hematology. 2025 Dec;30(1):2481555. doi: 10.1080/16078454.2025.2481555. Epub 2025 Mar 27.

Abstract

OBJECTIVES

Plasmablastic myeloma (PBM) is a variant of multiple myeloma associated with a poor prognosis. We investigated the efficacy and safety of B-cell maturation antigen (BCMA) chimeric antigen receptor T cell (CAR-T) therapy in patients with PBM.

METHODS

The study comprised six patients diagnosed with PBM between January 1, 2023 and December 31, 2023. The patients received BCMA single-target CAR-T therapy or BMCA/CD19 dual-target CAR-T therapy, with some patients undergoing hematopoietic stem cell transplantation before treatment. The median patient age was 55.5 years (range, 41-63). Four patients exhibited high-risk cytogenetic abnormalities.

RESULTS

The objective response rate (ORR) was 83.3%, with four of six patients achieving a complete response or better and three of six achieving a strigent complete response. Two patients exhibited progression-free survival (PFS) of at least 6 months, one of whom succumbed to a pulmonary infection, whereas four patients died of disease progression. Cytokine release syndrome (CRS) was observed in all patients, three of whom experienced grade 3-4 CRS. Two patients experienced grade 1-2 immune effector cell-associated neurotoxicity syndrome. There were no CRS-related deaths.

CONCLUSION

BCMA CAR-T therapy was safe and effective as a salvage treatment for PBM, and its toxicity was controllable. Future research will examine the use of CAR-T therapy as part of combination regimens.

摘要

目的

浆母细胞性骨髓瘤(PBM)是一种与预后不良相关的多发性骨髓瘤变体。我们研究了B细胞成熟抗原(BCMA)嵌合抗原受体T细胞(CAR-T)疗法在PBM患者中的疗效和安全性。

方法

该研究纳入了2023年1月1日至2023年12月31日期间诊断为PBM的6例患者。患者接受BCMA单靶点CAR-T疗法或BMCA/CD19双靶点CAR-T疗法,部分患者在治疗前接受了造血干细胞移植。患者中位年龄为55.5岁(范围41 - 63岁)。4例患者表现出高危细胞遗传学异常。

结果

客观缓解率(ORR)为83.3%,6例患者中有4例达到完全缓解或更好,6例中有3例达到严格完全缓解。2例患者无进展生存期(PFS)至少为6个月,其中1例死于肺部感染,4例死于疾病进展。所有患者均观察到细胞因子释放综合征(CRS),其中3例经历3 - 4级CRS。2例患者经历1 - 2级免疫效应细胞相关神经毒性综合征。没有与CRS相关的死亡。

结论

BCMA CAR-T疗法作为PBM的挽救治疗是安全有效的,其毒性可控。未来的研究将探讨CAR-T疗法作为联合治疗方案一部分的应用。

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