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B 细胞急性淋巴细胞白血病中的 CAR-T 细胞治疗:新数据与未决问题

英文原题:CAR-T Cell Therapies in B-Cell Acute Lymphoblastic Leukemia: Emerging Data and Open Issues.

PubMed 2025/09/16(内容时间) Cancers (Basel) Q2 · IF 4.8(JCR 2025)

研究概要

多项研究报告,治疗后 1 年总生存率在 60% 至 80% 之间。

中文摘要

CAR-T疗法改变了复发或难治性B细胞急性淋巴细胞白血病(B-ALL)的治疗格局,尤其是在儿童和年轻成人患者中。多项研究报告治疗后1年总生存率为60%–80%;1年无事件生存率约为50%–70%,治疗后2年仍处于缓解的患者约占40%–50%。尽管结果令人鼓舞,疾病复发仍是问题。未来CAR-T平台应靶向多个抗原,并需确定此类构建体的最佳设计。现代临床试验应探索CAR-T作为高危ALL巩固治疗的作用,包括诱导/巩固治疗结束时仍有微小残留病、IKZF1阳性基因表达谱、TP53突变或Ph样基因表达谱的患者。提高基因编辑效率有望提升CAR-T细胞制备成功率,并减少制造时间和成本。使用健康供者制备通用型CAR-T细胞也可显著降低生产时间和费用。这些问题凸显了B-ALL研究的动态发展特征。目前正在开展的研究和临床试验正努力应对这些挑战,以改善B-ALL患者结局并拓展CAR-T疗法的应用范围。

展开英文摘要原文

CAR-T therapy has transformed the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL), particularly in pediatric and young adult patients. Many studies report one-year overall survival rates of between 60% and 80% following therapy. Event-free survival rates at one year are around 50-70%, with 40-50% of patients in remission after two years. Despite these impressive results, disease relapse remains a problem. Future CAR-T cell platforms should target multiple antigens, and the optimal design of such constructs must be determined. Modern trials should explore the role of CAR-T cell therapy as a consolidation treatment for patients with high-risk ALL, including those with persistent minimal residual disease at the end of induction/consolidation therapy, an IKZF1-positive gene expression profile, or a TP53 mutation or Ph-like gene expression profile. Improving the efficiency of gene-editing methods could lead to higher success rates in creating CAR-T cells, as well as reducing manufacturing time and costs. Producing universal CAR-T cells from healthy donors could significantly reduce production time and costs. These issues underscore the dynamic and evolving nature of B-ALL research. Ongoing studies and clinical trials are addressing many of these challenges in order to improve outcomes for B-ALL patients and expand the applications of CAR-T cell therapy.

论文信息

作者
Alati C、Pitea M、Molica M、Scalise L、Porto G、Bilardi E、Lazzaro G、Micò MC
单位
Hematology and Stem Cell Transplantation and Cellular Therapies Unit (CTMO), Department of Hemato-Oncology and Radiotherapy, Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli", 89133 Reggio Calabria, Italy.Italy
文献类型
综述
期刊
Cancers2025 Sep 16
原文标识
PubMed 41008869 · DOI 10.3390/cancers17183027