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
骨外髓外病变对接受标准治疗 CAR-T 细胞治疗的复发/难治性多发性骨髓瘤患者结局的影响
英文原题：Impact of Extraosseous Extramedullary Disease on Outcomes of Patients with Relapsed-Refractory Multiple Myeloma receiving Standard-of-Care Chimeric Antigen Receptor T-Cell Therapy.
查看英文原题
Impact of Extraosseous Extramedullary Disease on Outcomes of Patients with Relapsed-Refractory Multiple Myeloma receiving Standard-of-Care Chimeric Antigen Receptor T-Cell Therapy.
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中文摘要
髓外病变（EMD）与复发/难治性多发性骨髓瘤（RRMM）患者不良结局相关。本文报告接受标准治疗（SOC）嵌合抗原受体（CAR）T 细胞疗法、且输注前存在活动性骨外 EMD 的 RRMM 患者结局。回顾性收集美国 3 家机构计划接受 SOC CAR-T 治疗患者的数据。根据国际骨髓瘤工作组标准评估缓解。共 152 例患者接受输注，其中 47 例（31%）有 EMD（EMD 组），105 例（69%）无 EMD（非 EMD 组）。两组基线患者特征相近。与非 EMD 组相比，EMD 组高级别 CRS、类固醇和阿那白滞素使用，以及输注后第 +30 天血小板减少的发生率较高。
此外，EMD 组总缓解率较低（58% 对 96%，p<0.00001），中位 PFS 较短（5.1 对 12.4 个月；p<0.0001），OS 较短（12.2 对 27.5 个月；p=0.00058）。
我们进一步将非 EMD 患者分为仅旁髓病变（PMD）组（n=26[17%]）和既无 EMD 也无 PMD 的骨髓局限组（BM-only，n=79[52%]）。仅 PMD 患者与 BM-only 患者的中位 PFS（11.2 对 13.6 个月，p=0.3798）和 OS（未达到[NR]对 27.5 个月，p=0.6446）相近。
然而，与 BM-only 组相比，EMD 患者的中位 PFS（5.1 对 13.6 个月，p<0.0001）及 OS（12.2 对 27.5 个月，p=0.0008）较差。真实世界 RRMM 患者接受 SOC CAR-T 治疗后获得了有意义的临床结局，但与无 EMD 患者相比，其缓解和生存结局欠佳。只有 EMD、而非 PMD，与 CAR-T 输注后显著较差的生存结局相关。
展开英文摘要原文
The presence of extramedullary disease (EMD) has been associated with poor outcomes in patients with relapsed-refractory multiple myeloma (RRMM).
Herein, we report the outcomes of RRMM patients who were treated with standard-of-care (SOC) chimeric antigen receptor (CAR) T-cell therapy and had active extraosseous EMD before the infusion. Data were retrospectively collected from patients at three US institutions with the intent to receive SOC CAR T. Responses were assessed per the International Myeloma Working Group criteria.
A total of 152 patients proceeded with infusion, of whom 47 (31%) had EMD (EMD group) and 105 (69%) did not (non-EMD group). Baseline patient characteristics were comparable between the two groups. The EMD group had a higher incidence of high-grade CRS, steroid and anakinra use, and thrombocytopenia on day +30 compared to the non-EMD group.
In addition, the EMD group had an inferior overall response rate (58% vs 96%, p < 0. 00001), median progression-free survival (PFS) (5. 1 vs 12. 4 months; p < 0. 0001), and overall survival (OS) (12. 2 vs 27. 5 months; p = 0. 00058) compared to the non-EMD group.
We further subdivided the non-EMD patients into those with paramedullary disease (PMD-only group, n = 26 [17%]) and those with neither EMD nor PMD (bone marrow-contained group or BM-only group, n = 79 [52%]). Patients with PMD-only had similar median PFS (11. 2 vs 13. 6 months, p = 0. 3798) and OS (not reached [NR] vs 27. 5 months, p = 0. 6446) compared to patients with BM-only disease.
However, patients with EMD exhibited inferior median PFS (5. 1 vs 13. 6 months, p < 0. 0001) and OS (12. 2 vs 27. 5, p = 0. 0008) compared to patients in the BM-only group. Treatment with SOC CAR T yielded meaningful clinical outcomes in real-world RRMM patients with extraosseous EMD, though responses and survival outcomes were suboptimal compared to patients without EMD. The presence of only EMD but not PMD was associated with significantly worse survival outcomes following the CAR T infusion.
论文信息
- 作者
- Dima D、Abdallah AO、Davis JA、Awada H、Goel U、Rashid A、DeJarnette S、Anwer F
- 第一作者单位
- Department of Hematology/Oncology, Cleveland Clinic, Taussig Cancer Institute, Cleveland, OH, USA. dimad@ccf.org.United States
- 通讯作者单位
- US Myeloma Innovations Research Collaborative (USMIRC), Westwood, KS, USA. hamzahashmi87@hotmail.com.United States
- 期刊
- Blood cancer journal2024 May 31
- 原文标识
- PubMed 38821914 · DOI 10.1038/s41408-024-01068-w
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