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儿童肿瘤患者的5年生存率已达80%以上,但仍有部分复发难治性肿瘤通过传统治疗手段难以取得理想疗效.嵌合抗原受体(CAR)-T细胞技术的发展为治愈这些肿瘤带来了希望.CAR-T细胞通过非MHC限制性的方式识别肿瘤相关抗原,抗肿瘤能力显著增强,目前已发展到第四代.靶向CD19的CAR-T细胞治疗复发难治性急性淋巴细胞白血病缓解率高达90%,且可以通过桥接造血干细胞移植、供者CAR-T细胞输注等手段辅助白血病的治疗.实体瘤方面,靶向GD2的CAR-T细胞治疗神经母细胞瘤具有良好的反应性,但对其他实体瘤效果欠佳.CAR-T细胞治疗可能出现细胞因子释放综合征、脱靶效应、肿瘤溶解综合征、插入突变等毒副反应.靶向CD19的CAR-T细胞治疗虽有很高的缓解率,但复发率较高,包括CD19+和CD19-复发,其机制尚需进一步研究.“,”Nowadays, the 5-year survival rate of childhood cancer patients can be more than 80%, but some patients with relapse and refractory cancers have shown no good response to traditional strategies. Chimeric antigen receptor engineered T (CAR-T) cell therapy is promising for these patients. CAR-T cells recognize the tumor-associated antigens in a non-major histocompatibility complex-restricted manner, so their anti-tumor ability is enhanced. There are four generations of CAR-T cells now. The complete remission rate of pediatric patients with relapse and refractory acute lymphoblastic leukemia can be as high as 90% when treated with CD19-targeting CAR-T cells. Furthermore, CAR-T cell therapy can also be used to bridge to transplantation and donor CAR-T cell infusion can be a strategy to prevent relapse after hematopoietic stem cell transplantation. As to solid tumors, only patients with neuroblastoma present good response to the GD2-targeting CAR-T cell therapy. The toxic or side effects of CAR-T cell therapy include cytokine release syndrome, off-tumor effect, tumor lysis syndrome, and insertion mutation. Although the CD19-targeting CAR-T cell therapy for childhood cancer can result in a high remission rate, the relapse rate is high, including CD19+ and CD19-relapse. The mechanisms for relapse merit further investigation.