[接受 CAR-T 细胞疗法治疗急性淋巴细胞白血病的儿童和青少年的管理]。

Chihaya Imai
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摘要

Tisagenlecleucel是一种商业化的CD19靶向CAR-T细胞产品,它极大地改变了复发/难治性B细胞急性淋巴细胞白血病(B-ALL)的治疗方法。Tisagenlecleucel 输注与独特的急性不良反应有关,包括细胞因子释放综合征、神经毒性、嗜血细胞淋巴组织细胞增多症和长期泛发性泛血细胞减少症,而这些不良反应在杀细胞化疗中是罕见的。此外,最近的回顾性研究还揭示了灌注前的预后因素,包括高肿瘤负荷(骨髓白血病细胞分数≥5%)和对另一种 CD19 靶向药物 blinatumomab 无应答。不仅是提供CAR-T细胞疗法的医生,就连转诊患者也应彻底了解这种疗法的适应症和局限性、特征性急性并发症、影响预后的治疗前因素以及晚期并发症。本文概述了目前关于在儿童和青少年 B-ALL 患者中使用替沙格列脲的认识。
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[Management of children and adolescents receiving CAR-T cell therapy for acute lymphoblastic leukemia].

Tisagenlecleucel, a commercially available CD19-targeted CAR-T cell product, has dramatically changed the treatment of relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL). Tisagenlecleucel infusion has been linked to distinct acute adverse events, including cytokine release syndrome, neurotoxicity, hemophagocytic lymphohistiocytosis and prolonged pancytopenia, which are rare with cytocidal chemotherapy. In addition, recent retrospective studies have revealed pre-infusion prognostic factors including high tumor burden (bone marrow leukemia cell fraction ≥5%) and non-response to blinatumomab, another CD19-targeting agent. Not only physicians providing CAR-T cell therapy but also those referring patients for this therapy should thoroughly understand the indications and limitations, characteristic acute complications, pre-treatment factors affecting prognosis, and late complications. This article outlines the current understanding regarding the use of tisagenlecleucel in children and adolescents with B-ALL.

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