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.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
干细胞补充输注（SCB）治疗复发/难治性多发性骨髓瘤（RRMM）患者 CAR-T 细胞治疗（CAR-T）相关血液学毒性的疗效——美国多发性骨髓瘤免疫治疗联盟的真实世界经验
英文原题：Efficacy of stem cell boost (SCB) for chimeric antigen receptor-T cell therapy (CAR-T)-related hematologic toxicity in patients with relapsed/refractory multiple myeloma (RRMM)-real world experience from the US multiple myeloma immunotherapy consortium.
查看英文原题
Efficacy of stem cell boost (SCB) for chimeric antigen receptor-T cell therapy (CAR-T)-related hematologic toxicity in patients with relapsed/refractory multiple myeloma (RRMM)-real world experience from the US multiple myeloma immunotherapy consortium.
分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。
中文摘要
持续性血细胞减少是 CAR-T 治疗后公认的并发症。自体 SCB 为促进快速血液学恢复提供了一种潜在策略。我们开展了一项回顾性多机构研究，比较 SCB 与单纯支持治疗在 CAR-T 输注后持续性血细胞减少患者中的结局。若患者在 2021 年 6 月至 2024 年 3 月期间接受商业 CAR-T 后 1 年内接受 SCB，则纳入研究。为确定匹配对照，我们回顾了 590 例 CAR-T 接受者，并选择 ANC 和血小板阈值代表 SCB 队列中血细胞减少严重程度第 75 百分位的患者。SCB 队列中的血液学恢复采用 CIBMTR 植入标准进行评估。对于非 SCB（nSCB）组，在 CAR-T 后第 60 天和第 90 天横断面分析细胞计数，并用 Kruskal-Wallis 检验与 SCB 队列进行比较。PFS 和 OS 采用 Kaplan-Meier 法进行评估。
在 590 例患者中，91 例（15.4%）发生持续性血细胞减少，其中 39 例接受了 SCB。中位 CD34+ 细胞剂量为 2.9 million/kg（范围：1.8-23.6），于 CAR-T 后中位 53 天（范围 24-265）给予。SCB 组除 1 例患者外，所有患者（97.4%）均实现血液学恢复，中位恢复时间为 24 天（范围 9-87）。未观察到归因于 SCB 的新毒性。在 CAR-T 输注后第 90 天，SCB 患者的中位 Hb 更高（10.6 vs. 8.7g/dL，p = 0.002），血小板计数也更高（135 vs. 35K/L，p < 0.001）。在 SCB 队列中位随访 12.6 个月、nSCB 组中位随访 11.6 个月后，mPFS 分别为 11.0 个月和 8.2 个月。SCB 组与 nSCB 组的中位 OS 分别为未达到和 12.3 个月。
总体而言，SCB 使几乎所有患者获得了快速且成功的血液学结局。与匹配对照相比，CAR-T 输注后第 90 天时 Hb 和血小板显著改善。
展开英文摘要原文
Prolonged cytopenias are a well-recognized complication following CAR-T therapy. Autologous SCB offers a potential strategy to promote fast hematologic recovery.
We conducted a retrospective multi-institutional comparing outcomes of SCB versus supportive care alone in patients with prolonged cytopenias after CAR-T infusion. Patients were included if they received SCB within 1 year following commercial CAR-T between 6/2021 and 3/2024. To identify matched controls, we reviewed 590 CAR-T recipients and selected patients with ANC and platelet thresholds representing the 75th percentile of cytopenia severity in the SCB cohort. Hematologic recovery in the SCB cohort was assessed using CIBMTR engraftment criteria. For the non-SCB (nSCB) group, cell counts were analyzed cross-sectionally at day 60 and 90 post-CAR-T and compared to the SCB cohort with Kruskal-Wallis tests. Progression-free survival (PFS) and overall survival (OS) were evaluated using Kaplan-Meier methods.
Of 590 patients, 91 patients (15. 4%) developed prolonged cytopenias, 39 of whom received SCB. Median CD34+ cell dose was 2. 9 million/kg (range: 1. 8-23. 6) administered at a median of 53 days post-CAR-T (range 24-265). All but one patient (97. 4%) in the SCB group achieved hematologic recovery, with median time to recovery of 24 days (range 9-87). No new toxicities attributable to SCB were observed.
On day 90 post CAR-T infusion, SCB patients had higher median Hb (10. 6 vs. 8. 7g/dL, p = 0. 002), and platelet counts (135 vs. 35K/L (p < 0. 001). After a median follow-up period of 12. 6 months in the SCB cohort and 11. 6 months in the nSCB arm, the mPFS was 11. 0 months and 8. 2 months, respectively. Median OS was not reached vs. 12. 3 months in the SCB vs. nSCB groups, respectively.
Overall, SCB lead to rapid and successful hematologic outcomes in nearly all patients. Compared to matched controls, Hb and platelets were significantly improved by day 90 post-CAR-T infusion.
论文信息
- 作者
- Varga C、Robinson M、Davis JA、Hashmi H、Martin TG、Kumar A、Sborov DW、Wagner CB
- 单位
- Levine Cancer Institute/Atrium Health, Charlotte, NC, USA. Cindy.varga@advocatehealth.org.United States
- 文献类型
- 多中心研究
- 期刊
- Blood cancer journal2026 Mar 20
- 原文标识
- PubMed 41862439 · DOI 10.1038/s41408-026-01469-z
在 PubMed 查看 → 出版商原文（DOI） 全文 PDF（PMC）· 可下载 治疗专题与资料阅读指南 资料来源与翻译说明 报告译文或资料问题 →