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
供者和患者来源的白细胞介素 6 敲低 CD19 靶向 CAR-T 细胞治疗异基因造血干细胞移植后复发 B 细胞急性淋巴细胞白血病患者的相似疗效与安全性
英文原题：Donor- and patient-derived interleukin 6 knockdown CD19-targeted chimeric antigen receptor T cells exhibit similar efficacy and safety in treating relapsed B-cell acute lymphoblastic leukemia patients after allogeneic hematopoietic stem cell transplantation.
查看英文原题
Donor- and patient-derived interleukin 6 knockdown CD19-targeted chimeric antigen receptor T cells exhibit similar efficacy and safety in treating relapsed B-cell acute lymphoblastic leukemia patients after allogeneic hematopoietic stem cell transplantation.
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研究概要
allo-HSCT 后复发的 B-ALL 患者可能从供者来源或患者来源的 CAR-T 细胞治疗中获益，但供者来源产品的移植物抗宿主病风险可能更高。
研究思路结论见上方概要
背景
嵌合抗原受体（CAR）T细胞疗法显著改善了异基因造血干细胞移植（allo-HSCT）后疾病复发的B细胞急性淋巴细胞白血病（B-ALL）患者的预后。然而，使用供者来源还是患者来源的CAR-T 细胞尚未明确。
方法
这是一项回顾性分析，旨在比较供者来源和患者来源的超级安全型CD19靶向CAR-T 细胞的疗效和安全性。
结果
在疗效方面，供者来源（n = 18）与患者来源（n = 16）CAR-T 细胞亚组之间未发现显著差异。完全缓解/完全缓解伴血细胞计数未完全恢复率（94.4% 对 75.0%，P = 0.1642）、缓解持续时间（中位，633.0 对 373.0 天，P = 0.8562）、总生存期（中位，67.12 个月 对 未定义，P = 0.061）和无事件生存期（中位，17.0 对 12.42 个月，P = 0.773）均相当。在安全性方面，细胞因子释放综合征的发生率（55.6% 对 62.5%，P = 0.7385）和严重程度（P = 0.1802）相似，血清白细胞介素6（中位，30.8 对 40.0 pg/mL，P = 0.8329）和铁蛋白（中位，3788.6 对 1781.3 g/L，P = 0.3552）水平也相似。供者来源超级且安全CAR-T 细胞亚组的急性移植物抗宿主病发生率显著更高（33.33% 对 0.00%，P = 0.0198）。CAR-T 细胞扩增峰值水平（中位，114 169 对 316 000 拷贝，P = 0.2204）及达到峰值的天数（中位，9.5 对 9.5 天，P = 0.8143）均相似。
展开英文摘要原文
METHODS
This was a retrospective analysis to compare the efficacy and safety of donor- and patient-derived super and safe CD19-targeted CAR T cells.
RESULTS
No significant difference was found between the donor-derived (n = 18) and patient-derived (n = 16) CAR T-cell subgroups in terms of efficacy. Complete remission/complete remission with incomplete count recovery rate (94.4% versus 75.0%, P = 0.1642), duration of response (median, 633.0 versus 373.0 days, P = 0.8562), overall survival (median, 67.12 months versus undefined, P = 0.061) and event-free survival (median, 17.0 versus 12.42 months, P = 0.773) were comparable. With regard to safety, the incidence (55.6% versus 62.5%, P = 0.7385) and severity (P = 0.1802) of cytokine release syndrome were similar, as were the serum levels of interleukin 6 (median, 30.8 versus 40.0 pg/mL, P = 0.8329) and ferritin (median, 3788.6 versus 1781.3 g/L, P = 0.3552). The incidence of acute graft-versus-host disease was significantly higher in the donor-derived super and safe CAR T-cell subgroup (33.33% versus 0.00%, P = 0.0198). The peak level of CAR T-cell amplification (median, 114 169 versus 316 000 copies, P = 0.2204) and the day reaching the peak (median, 9.5 versus 9.5 days, P = 0.8143) were both similar.
CONCLUSIONS
B-ALL patients who relapse after allo-HSCT may benefit from either donor- or patient-derived CAR T-cell therapy, but the risk of graft-versus-host disease may be higher for donor-derived products.
论文信息
- 作者
- Sun T、Ma JF、Jia X、Xu SZ、Liu SH、Lyu XY、Huang SM、Wu YJ
- 第一作者单位
- Jiangsu Institute of Hematology, National Clinical Research Center for Hematologic Diseases, First Affiliated Hospital of Soochow University, Soochow University, Suzhou, People's Republic of China; Institute of Blood and Marrow Transplantation, Collaborative Innovation Center of Hematology, Soochow University, Suzhou, People's Republic of China.China
- 通讯作者单位
- Jiangsu Institute of Hematology, National Clinical Research Center for Hematologic Diseases, First Affiliated Hospital of Soochow University, Soochow University, Suzhou, People's Republic of China; Institute of Blood and Marrow Transplantation, Collaborative Innovation Center of Hematology, Soochow University, Suzhou, People's Republic of China. Electronic address: slxue@suda.edu.cn.China
- 期刊
- Cytotherapy2026 Jun
- 原文标识
- PubMed 41930803 · DOI 10.1016/j.jcyt.2026.102118