Fang Yi, Lin Weiwei, Shen Lijing, Ni Beiwen, Zhang Minyue, Cai Yongmei, Zhou Yi, Hou Jian
Department of Hematology, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Department of Clinical Laboratory, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Front Immunol. 2025 Apr 16;16:1564774. doi: 10.3389/fimmu.2025.1564774. eCollection 2025.
Chimeric antigen receptor T (CAR-T) cells targeting B-cell maturation antigen (BCMA) have been used as an effective therapy against relapsed/refractory multiple myeloma (MM). However, the relapse rates in these patients are still high, which may be related to the poor quality of T cells after multiple chemotherapies. The case reported here demonstrated the effectiveness and safety of first-line anti-BCMA CAR-T cell therapy for high-risk MM patients, even in frailty with multiple comorbidities.
A 75-year-old woman was diagnosed with biclonal gammopathy and high-risk MM with extramedullary mass in the right caput femoris. The patient was fragile with multiple comorbidities, including pneumonia, left lower limb deep venous thrombosis, and epilepsy secondary to cerebral hemorrhage. Considering the patient's fragility and comorbidities, commercial equecabtagene autoleucel, a fully human anti-BCMA CAR-T cells, as first-line CAR-T cell therapy, was proposed and accepted by the patient and her family. After one cycle of bortezomib, cyclophosphamide, and dexamethasone (VCD) regimen), she reached very good partial response (VGPR). Then her leukapheresis was performed, and the harvested cells were sent to the manufacturer for preparation. After lymphodepletion was performed using fludarabine and cyclophosphamide (FC) chemotherapy, her equecabtagene autoleucel was transfused. On day 21 after infusion, she achieved stringent complete remission (sCR) with minimal residual disease (MRD) negativity without severe toxicity. The CAR-T cells/CD3 T cell ratio gradually increased, reaching a maximum of 54.97% on day 14, and gradually decreased, remaining at 0.03% on the 153rd day. The patient received right hip replacement plus pelvic lesion curettage 7 months after CAR-T transfusion due to pain in her right hip, but no MM cells were found in postoperative pathology. Hitherto, her deep remission persisted for 12 months without any maintenance therapy.
First-line anti-BCMA CAR-T cell therapy is effective and safe for high-risk MM patients, even in fragile patients with multiple comorbidities.
靶向B细胞成熟抗原（BCMA）的嵌合抗原受体T（CAR-T）细胞已被用作治疗复发/难治性多发性骨髓瘤（MM）的有效疗法。然而，这些患者的复发率仍然很高，这可能与多次化疗后T细胞质量差有关。本文报道的病例证明了一线抗BCMA CAR-T细胞疗法对高危MM患者的有效性和安全性，即使是在伴有多种合并症的虚弱患者中。
一名75岁女性被诊断为双克隆丙种球蛋白病和高危MM，右股骨近端有髓外肿块。该患者身体虚弱，伴有多种合并症，包括肺炎、左下肢深静脉血栓形成以及脑出血继发的癫痫。考虑到患者的虚弱状态和合并症，建议并经患者及其家属同意，使用商业化的全人源抗BCMA CAR-T细胞equecabtagene autoleucel作为一线CAR-T细胞疗法。在接受一周期硼替佐米、环磷酰胺和地塞米松（VCD）方案治疗后，她达到了非常好的部分缓解（VGPR）。然后进行了白细胞分离术，采集的细胞被送去制造商处进行制备。在使用氟达拉滨和环磷酰胺（FC）化疗进行淋巴细胞清除后，输注了她的equecabtagene autoleucel。输注后第21天，她达到了严格完全缓解（sCR），微小残留病（MRD）阴性，且无严重毒性。CAR-T细胞/CD3 T细胞比值逐渐升高，在第14天达到最高值54.97%，然后逐渐下降，在第153天降至0.03%。由于右髋疼痛，患者在CAR-T输注7个月后接受了右髋关节置换术加骨盆病变刮除术，但术后病理未发现MM细胞。迄今为止，她的深度缓解持续了12个月，未进行任何维持治疗。
一线抗BCMA CAR-T细胞疗法对高危MM患者有效且安全，即使是在伴有多种合并症的虚弱患者中。
