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
在接受 BCMA CAR-T 治疗的复发/难治性患者中验证和优化高危多发性骨髓瘤（HRMM）的新共识基因组分期（CGS）
英文原题：Validation and refinement of the new Consensus Genomic Staging (CGS) of high-risk multiple myeloma (HRMM) in relapsed and refractory patients treated with BCMA CAR-T.
查看英文原题
Validation and refinement of the new Consensus Genomic Staging (CGS) of high-risk multiple myeloma (HRMM) in relapsed and refractory patients treated with BCMA CAR-T.
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中文摘要
高危多发性骨髓瘤(MM)的新共识基因组分期(CGS)最近由国际骨髓瘤学会和国际骨髓瘤工作组更新。我们进行了一项回顾性研究，以在接受BCMA CAR-T 治疗的复发/难治性MM患者中验证修订后的HRMM标准。共纳入158例患者，其中75例(47%)符合HRMM标准。中位随访时间为24.8个月(95% CI 22.5-30.3)。在多变量分析中，接受BCMA CAR-T 后，HRMM患者的PFS(HR 1.89, p = 0.003)和OS(HR 3.06, p < 0.001)较标危MM(SRMM)显著缩短。LDH > 210 U/L和EMD也被确定为独立的不良危险因素。
基于生存分析，我们构建了一个将LDH和EMD纳入CGS-HRMM标准的重新分类分期系统。患者被分为四个离散的风险组：低危(伴LDH正常且无EMD的SRMM；n = 42, 26%)、中低危(伴LDH正常且无EMD的HRMM；n = 31, 20%)、中高危(伴高LDH、EMD或二者兼有的SRMM；n = 41, 26%)和高危(伴高LDH、EMD或二者兼有的HRMM；n = 44, 28%)。低危、中低危、中高危和高危疾病患者的中位PFS分别为35.0、19.6、10.4和6.6个月(p < 0.0001)，估计24个月OS分别为90%、69%、66%和26%(p < 0.0001)。
总之，我们在接受BCMA CAR-T 治疗的复发/难治性MM患者中验证了新的CGS-HRMM标准。我们还提出了一种重新分类分期系统，以进一步改善接受BCMA CAR-T 治疗的MM患者的风险分层。
展开英文摘要原文
The new Consensus Genomic Staging (CGS) of high-risk multiple myeloma (MM) was recently updated by the International Myeloma Society and International Myeloma Working Group.
We performed a retrospective study to validate the revised HRMM criteria in relapsed and refractory MM patients treated with BCMA CAR-T. A total of 158 patients were included, of whom 75 (47%) met criteria for HRMM. The median follow-up was 24. 8 months (95% CI 22. 5-30. 3). On multivariable analysis, patients with HRMM had significantly shorter PFS (HR 1. 89, p = 0. 003) and OS (HR 3. 06, p < 0. 001) compared to standard-risk MM (SRMM) following BCMA CAR-T. LDH > 210 U/L and EMD were also identified as independent adverse risk factors. Based on survival analyses, we constructed a reclassified staging system incorporating LDH and EMD into the CGS-HRMM criteria.
Patients were divided into four discrete risk groups: low risk (SRMM with normal LDH and no EMD; n = 42, 26%), intermediate-low risk (HRMM with normal LDH and no EMD; n = 31, 20%), intermediate-high risk (SRMM with high LDH, EMD, or both; n = 41, 26%), and high risk (HRMM with high LDH, EMD, or both; n = 44, 28%).
Patients with low, intermediate-low, intermediate-high, and high-risk disease had a median PFS of 35. 0, 19. 6, 10. 4, and 6. 6 months, respectively (p < 0. 0001), and an estimated 24-month OS of 90%, 69%, 66%, and 26%, respectively (p < 0. 0001). In summary, we validated the new CGS-HRMM criteria in relapsed and refractory MM patients treated with BCMA CAR-T.
We also propose a reclassified staging system to further improve risk stratification in MM patients treated with BCMA CAR-T.
论文信息
- 作者
- Gustine JN、Attar N、Puliafito BR、Cirstea DD、Branagan AR、Yee AJ、Raje NS
- 第一作者单位
- Massachusetts General Hospital Cancer Center and Harvard Medical School, Boston, MA, USA.United States
- 通讯作者单位
- Massachusetts General Hospital Cancer Center and Harvard Medical School, Boston, MA, USA. nraje@mgh.harvard.edu.United States
- 期刊
- Blood cancer journal2026 Apr 22
- 原文标识
- PubMed 42020379 · DOI 10.1038/s41408-026-01510-1
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