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.
CAR-T疗法已彻底改变复发或难治性B细胞急性淋巴细胞白血病（B-ALL）的治疗格局，在儿童及青少年患者群体中尤为显著。多项研究显示，接受该疗法后患者一年总生存率达60%-80%，一年无事件生存率约为50%-70%，两年后仍有40%-50%的患者维持缓解状态。尽管疗效显著，疾病复发仍是亟待解决的难题。未来CAR-T细胞平台应实现多抗原靶向，其最优构建方案尚需明确。现代临床试验应重点探索CAR-T疗法作为高危ALL患者巩固治疗的价值，包括诱导/巩固治疗结束后持续存在微小残留病灶、携带IKZF1阳性基因表达谱、TP53突变或Ph样基因表达谱的患者群体。通过提升基因编辑技术效率，有望提高CAR-T细胞制备成功率，同时缩短生产周期并降低成本。利用健康供体生产通用型CAR-T细胞可能显著减少制备时间与费用。这些关键问题凸显了B-ALL研究领域持续发展的动态特性。当前多项研究与临床试验正着力突破这些瓶颈，以期改善B-ALL患者预后，并拓展CAR-T细胞疗法的应用范围。
CAR-T Cell Therapies in B-Cell Acute Lymphoblastic Leukemia: Emerging Data and Open Issues
肿瘤(癌症)患者之家