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
复发/难治性急性淋巴细胞白血病患者 CAR-T 治疗后序贯异基因 HSCT：长期随访结果
英文原题：Sequential allogeneic HSCT after CAR-T therapy for relapsed/refractory acute lymphoblastic leukemia patients: A long-term follow-up result.
查看英文原题
Sequential allogeneic HSCT after CAR-T therapy for relapsed/refractory acute lymphoblastic leukemia patients: A long-term follow-up result.
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。
研究概要
我们的研究显示出与临床试验报告一致的有利长期结局，在 4 年随访时观察到持续、持久的缓解。然而，这些获益在老年患者和具有不良风险疾病特征的患者中不太明显。
研究思路结论见上方概要
背景
CAR-T 细胞治疗已彻底改变了 R/R B-ALL 的治疗格局，而桥接 allo-HSCT 具有降低复发率的潜力。然而，现有研究大多仅关注短期结局，导致对整体预后长期可持续性的全面认识不足。
目的
我们的研究旨在为接受序贯治疗的患者提供真实世界的长期随访数据。
方法
纳入2016年1月至2024年5月期间接受CAR-T 治疗后达到MRD - CR并随后接受allo-HSCT的R/R B-ALL患者。主要结局包括总生存期（OS）、无白血病生存期（LFS）、非复发死亡率（NRM）和累积复发率（CIR）。同时还对急性和慢性移植物抗宿主病（GVHD）及无移植物抗宿主病生存期（GRFS）进行了分析。
结果
移植时的中位年龄为32.1岁。在这些患者中，88.2 %接受了单倍体-HSCT，11.8 %接受了无关全合或亲缘全合HSCT。第100天I-IV级和II-IV级aGVHD的累积发生率分别为31.4 %和15.7 %。4年时cGVHD的累积发生率为48.3 %。中位随访时间为43.2个月，4年时OS、LFS和GRFS分别为68.9 %、61.4 %和39.5 %。15例（29.4 %）出现复发，以抗原阳性复发为主（n = 11）。4年时NRM和CIR分别为10.6 %和28.0 %。在多因素分析中，年龄超过45岁且具有高危风险的患者OS（P = 0.018；P = 0.038）和LFS（P = 0.01；P = 0.03）显著较差。
展开英文摘要原文
OBJECTIVES
Our study aimed to provide real-world, long-term follow-up data for patients who underwent sequential therapy.
METHODS
Patients with R/R B-ALL who achieved MRD - CR following CAR-T therapy and subsequently underwent allo-HSCT between January 2016 and May 2024 were enrolled. The primary outcomes included overall survival (OS), leukemia-free survival (LFS), non-relapse mortality (NRM) and cumulative incidence of relapse (CIR). Acute and chronic graft-versus-host disease (GVHD) and graft-versus-host disease-free survival (GRFS) were also investigated.
RESULTS
The median age at transplant of 32.1 years. Of these patients, 88.2 % underwent haploidentical-HSCT, and 11.8 % received either unrelated matched or related matched HSCT. The cumulative incidences of grades I-IV and grade II-IV aGVHD at day 100 were 31.4 % and 15.7 %, respectively. The cumulative incidence of cGVHD at 4 years was 48.3 %. With a median follow-up time of 43.2 months, OS, LFS, and GRFS at 4 years were 68.9 %, 61.4 %, and 39.5 %, respectively. Fifteen cases (29.4 %) experienced relapse, predominantly antigen-positive relapse (n = 11). NRM and CIR at 4 years were 10.6 % and 28.0 %, respectively. In the multivariate analyses, patients over 45 years of age and with poor-risk had significantly dismal OS (P = 0.018; P = 0.038) and LFS (P = 0.01; P = 0.03).
CONCLUSION
Our study exhibits favorable long-term outcomes consistent with those reported in clinical trials, with sustained, durable responses observed at the 4-year follow-up. However, these benefits are less pronounced in older patients and those with poor-risk disease characteristics.
论文信息
- 作者
- Yang T、Dong Y、Shi J、Zhang M、Kong D、Feng J、Fu S、Xiao P
- 第一作者单位
- Bone Marrow Transplantation Center of The First Affiliated Hospital Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China; Institute of Hematology, Zhejiang University, Hangzhou, Zhejiang, China; Zhejiang Province Engineering Laboratory for Stem Cell and Immunity Therapy, Hangzhou, Zhejiang, China.China
- 通讯作者单位
- Bone Marrow Transplantation Center of The First Affiliated Hospital Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China; Institute of Hematology, Zhejiang University, Hangzhou, Zhejiang, China; Zhejiang Province Engineering Laboratory for Stem Cell and Immunity Therapy, Hangzhou, Zhejiang, China. Electronic address: 1313016@zju.edu.cn.China
- 文献类型
- 非美国政府资助研究
- 期刊
- Journal of advanced research2025 Dec
- 原文标识
- PubMed 39947324 · DOI 10.1016/j.jare.2025.02.006
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 →