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病例报告:靶向 BCMA 的 CAR-T 细胞治疗在复发髓外浆母细胞性多发性骨髓瘤中诱导完全且持久缓解

英文原题:Case Report: BCMA-targeting CAR T-cell therapy induces complete and durable remission in relapsed extramedullary plasmablastic multiple myeloma.

查看英文原题

Case Report: BCMA-targeting CAR T-cell therapy induces complete and durable remission in relapsed extramedullary plasmablastic multiple myeloma.

PubMed 2025/05/26(内容时间) Front Immunol Q1 · IF 7(JCR 2025)

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中文摘要

浆母细胞型多发性骨髓瘤(PBM)是一种侵袭性强的MM亚型,复发风险高、预后差且有效治疗选择有限。本研究报告一例病例,患者最初诊断为伴双打击遗传学特征的IgG-κ型MM。经硼替佐米、多柔比星和地塞米松(VAD)诱导化疗,以及伊沙佐米、来那度胺和地塞米松巩固治疗后,疾病进展,表现为右侧盆腔浆母细胞瘤。接受两周期卡非佐米、达雷妥尤单抗、环磷酰胺、顺铂、依托泊苷和地塞米松(KD-DECP)后,患者达到部分缓解。患者拒绝自体造血干细胞移植(ASCT),改为接受放疗作为桥接治疗,随后接受靶向B细胞成熟抗原(BCMA)的嵌合抗原受体(CAR)T细胞治疗,并以泊马度胺维持治疗。患者于3个月时达到完全缓解(CR),根据最近随访已无病生存期超过15个月。尽管出现2级细胞因子释放综合征(CRS)及其他不良事件,均可控制。对于侵袭性更强、生存期更短的PBM,BCMA CAR-T 联合桥接放疗和泊马度胺维持治疗提供了一种有前景的治疗方案。仍需进一步研究评估其疗效和长期获益。

展开英文摘要原文

Plasmablastic multiple myeloma (PBM) is an aggressive multiple myeloma (MM) form, identified by a high risk of recurrence and poor prognosis, with limited effective treatment options. Present study reports a case initially diagnosed with IgG-kappa MM with double-hit genetics. Following induction chemotherapy with bortezomib, doxorubicin and dexamethasone (VAD), and subsequent consolidation therapy with ixazomib, lenalidomide, and dexamethasone, the disease progressed, manifesting as a plasmoblastic tumor in the right pelvic cavity. After two cycles of carfezomib, daratumumab, cyclophosphamide, cisplatin, etoposide and dexamethasone (KD-DECP), the patient achieved partial response.

She declined autologous stem cell transplantation (ASCT) and instead received radiotherapy as bridging therapy, followed by B-cell maturation antigen (BCMA)-targeting chimeric antigen receptor (CAR) T-cell therapy with pomalidomide as maintenance therapy. She achieved complete response (CR) at 3 months and has remained disease-free for over 15 months based on the latest follow-up.

Although grade 2 cytokine release syndrome (CRS) and other adverse events were observed, they were manageable. BCMA CAR-T cell accompanied with bridging radiotherapy and pomalidomide as maintenance therapy provided a promising therapy treatment for PBM, which is more aggressive and with shorter survival.

Further studies are demanded to assess the efficiency and long-term benefits for this challenging subtype.

论文信息

作者
Zhou L、Cheng PNM、Mi JQ
第一作者单位
Department of Hematology, Shanghai Sino United Hospital, Shanghai, China.China
通讯作者单位
State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine, Shanghai Institute of Hematology, Ruijin Hospital Affiliated with Shanghai Jiao Tong University School of Medicine, Shanghai, China.China
文献类型
病例报告
期刊
Frontiers in immunology2025
原文标识
PubMed 40491928 · DOI 10.3389/fimmu.2025.1567403