Cheng Yifang, Liu Aiguo
The Second Clinical School, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Front Immunol. 2025 Jul 23;16:1611701. doi: 10.3389/fimmu.2025.1611701. eCollection 2025.
Blinatumomab, a bispecific T-cell engager, has demonstrated substantial clinical benefits in treating pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). Approved by FDA for several indications, blinatumomab is now integral to therapeutic protocols for specific pediatric cohorts, with real-world applications steadily increasing. As one of the representatives of cutting-edge immunotherapy for pediatric ALL, blinatumomab plays a crucial role in precision medicine against the backdrop of current genetic testing. Clinical efficacy is influenced by factors such as tumor burden, endogenous T-cell function, CD19 antigen loss, and lineage switch. Treatment-related complications, such as cytokine release syndrome (CRS), neurotoxicity (ICANS), and infections, necessitate vigilant monitoring. Administration involves continuous intravenous infusion, with consideration for drug interactions. Despite proven short-term efficacy and tolerability, long-term impacts on pediatric patients warrant further investigation. Current studies refine dosing strategies and combinational approaches to enhance therapeutic precision for pediatric patients. This review synthesizes selected literature related to clinical trials of blinatumomab, emphasizing determinants of clinical efficacy and adverse events associated with treatment.
双特异性T细胞衔接器博纳吐单抗在治疗复发或难治性B细胞急性淋巴细胞白血病（R/R B-ALL）的儿科患者中已显示出显著的临床益处。博纳吐单抗已获美国食品药品监督管理局（FDA）批准用于多种适应症，目前已成为特定儿科患者群体治疗方案的一部分，其在现实世界中的应用也在稳步增加。作为儿科急性淋巴细胞白血病前沿免疫疗法的代表之一，在当前基因检测的背景下，博纳吐单抗在精准医学中发挥着关键作用。临床疗效受肿瘤负荷、内源性T细胞功能、CD19抗原丢失和谱系转换等因素影响。治疗相关并发症，如细胞因子释放综合征（CRS）、神经毒性（ICANS）和感染，需要密切监测。给药方式为持续静脉输注，同时需考虑药物相互作用。尽管已证实其具有短期疗效和耐受性，但对儿科患者的长期影响仍需进一步研究。目前的研究正在完善给药策略和联合治疗方法，以提高对儿科患者的治疗精准度。本综述综合了与博纳吐单抗临床试验相关的选定文献，重点强调了临床疗效的决定因素以及与治疗相关的不良事件。
