肿瘤细胞治疗研究
英文原题：BCMA CAR-T therapy as salvage therapy in patients with plasmablastic myeloma.
BCMA CAR-T 疗法作为 PBM 的挽救治疗安全有效，且其毒性可控。
目的：浆母细胞性骨髓瘤（PBM）是预后不良的多发性骨髓瘤变异类型。本研究评估 B 细胞成熟抗原（BCMA）CAR-T 细胞疗法治疗 PBM 患者的疗效和安全性。方法：研究纳入 2023 年 1 月 1 日至 2023 年 12 月 31 日诊断的 6 例 PBM 患者。患者接受 BCMA 单靶点 CAR-T 或 BCMA/CD19 双靶点 CAR-T 治疗；部分患者在治疗前接受了造血干细胞移植。患者年龄中位数为 55.5 岁（范围 41–63 岁），4 例存在高危细胞遗传学异常。结果：客观缓解率（ORR）为 83.3%；6 例中 4 例达到完全缓解或更佳，3 例达到严格完全缓解。2 例患者 PFS 至少为 6 个月，其中 1 例死于肺部感染；另有 4 例死于疾病进展。所有患者均发生 CRS，其中 3 例为 3–4 级。2 例患者发生 1–2 级免疫效应细胞相关神经毒性综合征。未发生 CRS 相关死亡。结论：BCMA CAR-T 疗法用于 PBM 挽救治疗安全且有效，毒性可控。未来研究将考察 CAR-T 疗法纳入联合治疗方案的应用。PBM 的背景与挑战：PBM 是一种侵袭性多发性骨髓瘤亚型，预后差。免疫调节剂、蛋白酶体抑制剂和自体干细胞移植等标准疗法疗效有限，中位生存期仅 4.5 个月，迫切需要有效治疗策略改善结局。研究新进展：本研究发现 BCMA CAR-T 对 PBM 患者疗效显著。具体结果包括：缓解率高，多数接受 BCMA CAR-T 患者病情显著改善甚至完全缓解；生存获益，该疗法显著延长患者生存；安全性可管理，虽然可发生 CRS 和神经毒性等副作用，但多数情况下可通过医疗干预有效控制。影响：BCMA CAR-T 为 PBM 患者提供一种有效且相对安全的治疗选择，有望改善预后和生活质量。未来研究将探索将 CAR-T 纳入联合治疗方案。
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. What's the background and challenges for PBM? Plasmablastic myeloma (PBM) is an aggressive subtype of multiple myeloma with a poor prognosis.Standard therapies including immunomodulators, proteasome inhibitors and autologous stem cell transplantation have limited efficacy with a median survival of only 4.5 months.The urgent need for effective therapeutic strategies to improve outcomes for patients with PBM. What is new? For PBM patients, the study found that BCMA CAR-T therapy showed significant efficacy. Specific results include:High remission rate: Among patients who received BCMA CAR-T therapy, the majority of patients experienced significant improvement or even complete remission.Survival benefit: The therapy significantly prolonged patients survival.Manageable safety: Although some side effects, such as cytokine release syndrome (CRS) and neurotoxicity, can occur, they can be effectively managed with medical intervention in most cases. What's the impact? BCMA chimeric antigen receptor T-cell (CAR-T) therapy offers an effective and relatively safe treatment option for patients with PBM and is expected to improve the prognosis and quality of life of these patients. Future studies will explore CAR-T therapy as part of a combination therapy regimen.
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