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
CAR-T 细胞治疗序贯无关脐血移植治疗儿童及年轻成人复发/难治性 B 细胞急性淋巴细胞白血病：生存优越但移植后复发率相对较高
英文原题：Chimeric Antigen Receptor T Cell Therapy followed by Unrelated Cord Blood Transplantation for the Treatment of Relapsed/Refractory B Cell Acute Lymphoblastic Leukemia in Children and Young Adults: Superior Survival but Relatively High Post-Transplantation Relapse.
查看英文原题
Chimeric Antigen Receptor T Cell Therapy followed by Unrelated Cord Blood Transplantation for the Treatment of Relapsed/Refractory B Cell Acute Lymphoblastic Leukemia in Children and Young Adults: Superior Survival but Relatively High Post-Transplantation Relapse.
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中文摘要
多项研究表明，嵌合抗原受体（CAR）T细胞治疗序贯异基因造血干细胞移植有利于治疗复发/难治性（R/R）B细胞急性淋巴细胞白血病（B-ALL）患者。在CAR-T 治疗后的R/R B-ALL中，巩固性非血缘脐血移植（UCBT）是否适用仍不确定。
我们旨在评估CAR-T 治疗序贯UCBT在儿童和年轻R/R B-ALL患者中的疗效和安全性。我们回顾性分析了2012年2月至2020年11月期间在中国科学技术大学附属第一医院接受单份UCBT的43例年龄<18岁的R/R B-ALL患者。其中，21例患者在UCBT前接受CAR-T 治疗后达到完全缓解（CR）（CAR-T 组），其余22例患者在UCBT前未接受CAR-T 治疗且处于未缓解（NR）状态（NR组）。分析了两组的临床结局。从CAR-T 治疗到UCBT的中位时间为62天（范围，42至185天）。两组在II-IV级急性移植物抗宿主病（GVHD）、III-IV级急性GVHD和2年广泛性慢性GVHD发生率方面无显著差异。与NR组相比，CAR-T 组的2年移植相关死亡率累积发生率较低，2年总生存率、无白血病生存率和无GVHD无复发生存率较高（P分别为.037、.005、.028和.017）。
然而，两组的2年累积复发率（CIR）相当高（CAR-T 组为26.7%，NR组为38.3%；P = .41）。在CAR-T 组中，UCBT前MRD阳性的患者相比UCBT前MRD阴性的患者具有更高的CIR（66.7%对19.2%；P = .006）。CAR-T 治疗桥接UCBT在R/R B-ALL中产生了更优的生存，但接受治疗的患者移植后复发率仍然很高。
展开英文摘要原文
Several studies have indicated that chimeric antigen receptor (CAR) T cell therapy followed by allogeneic hematopoietic stem cell transplantation is beneficial for treating patients with relapsed or refractory (R/R) B cell acute lymphoblastic leukemia (B-ALL). Whether consolidative unrelated cord blood transplantation (UCBT) is suitable in R/R B-ALL after CAR-T therapy remain uncertain.
We aimed to assess the efficacy and safety of CAR-T therapy before UCBT in children and young adults with R/R B-ALL.
We retrospectively analyzed 43 patients aged <18 years with R/R B-ALL who underwent single-unit UCBT at the First Affiliated Hospital of the University of Science and Technology of China between February 2012 and November 2020. Among them, 21 patients achieved complete remission (CR) following CAR-T therapy before UCBT (the CAR-T group), and the remaining 22 patients remained in nonremission (NR) without prior CAR-T therapy before UCBT (the NR group). The clinical outcomes in the 2 groups were analyzed.
The median time from CAR-T therapy to UCBT was 62 days (range, 42 to 185 days). There were no significant between-group differences in the incidences of grade II-IV acute graft-versus-host disease (GVHD), grade III-IV acute GVHD, and 2-year extensive chronic GVHD. Compared with the NR group, the CAR-T group had a lower 2-year cumulative incidence of transplantation-related mortality and higher probabilities of 2-year overall survival, leukemia-free survival, and GVHD-free relapse-free survival (P = . 037, . 005, . 028, and . 017, respectively).
However, the 2-year cumulative incidence of relapse (CIR) was comparably high in the 2 groups (26. 7% in the CAR-T group and 38. 3% in the NR group; P = . 41). In the CAR-T group, patients who were minimal residual disease (MRD)-positive before UCBT had a higher CIR compared with those who were MRD-negative before UCBT (66. 7% versus 19. 2%; P = . 006). CAR-T therapy followed by UCBT produces superior survival in R/R B-ALL, but treated patients still have a high post-transplantation relapse rate.
论文信息
- 作者
- Sun G、Tang B、Wan X、Yao W、Song K、Tu M、Geng L、Qiang P
- 第一作者单位
- Department of Hematology, First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China; Anhui Provincial Key Laboratory of Blood Research and Applications, Hefei, China; Blood and Cell Therapy Institute, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China.China
- 通讯作者单位
- Department of Hematology, First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China; Anhui Provincial Key Laboratory of Blood Research and Applications, Hefei, China; Blood and Cell Therapy Institute, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China. Electronic address: xiaoyuz@ustc.edu.cn.China
- 文献类型
- 非美国政府资助研究
- 期刊
- Transplantation and cellular therapy2022 Feb
- 原文标识
- PubMed 34839013 · DOI 10.1016/j.jtct.2021.11.011