CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Case Report: BCMA-targeting CAR T-cell therapy induces complete and durable remission in relapsed extramedullary plasmablastic multiple myeloma.
Case Report: BCMA-targeting CAR T-cell therapy induces complete and durable remission in relapsed extramedullary plasmablastic multiple myeloma.
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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。
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.
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