Shamim Hina, Jhakri Kiran, Al-Shudifat Moath, Sumra Bushra, Kocherry Cyril, Malasevskaia Iana
Pediatrics, Baqai Medical University, Karachi, PAK.
Internal Medicine, Shahjalal University of Science and Technology, Sylhet, BGD.
Cureus. 2025 Jul 31;17(7):e89172. doi: 10.7759/cureus.89172. eCollection 2025 Jul.
Pediatric acute lymphoblastic leukemia (ALL) poses significant treatment challenges, particularly in relapsed or refractory cases. This review synthesizes recent studies evaluating the safety and efficacy of chimeric antigen receptor T (CAR-T) cell therapy, specifically tisagenlecleucel, in achieving complete remission (CR) and improving overall survival rates among pediatric patients with ALL. A comprehensive literature search identified 12 studies published between January 2014 and July 2024, encompassing cohort studies and clinical trials. Findings indicate that CAR-T cell therapy demonstrates superior CR rates (up to 100% in some studies) and manageable safety profiles, with common adverse effects, including cytokine release syndrome (CRS) and neurotoxicity. This review points out some of the important aspects such as the identification of biomarkers for response prediction, understanding of the mechanisms of resistance, and the crucial requirement for long-term outcome data. Challenges remain in the management of adverse effects, particularly CRS and neurotoxicity. This review underscores the transformative potential of CAR-T cell therapy in pediatric oncology while emphasizing critical areas for further investigation to optimize patient outcomes and enhance the therapeutic landscape for pediatric ALL.
小儿急性淋巴细胞白血病（ALL）带来了重大的治疗挑战，尤其是在复发或难治性病例中。本综述综合了近期评估嵌合抗原受体T（CAR-T）细胞疗法，特别是tisagenlecleucel，在小儿ALL患者中实现完全缓解（CR）和提高总生存率方面的安全性和有效性的研究。全面的文献检索确定了2014年1月至2024年7月期间发表的12项研究，包括队列研究和临床试验。研究结果表明，CAR-T细胞疗法显示出较高的CR率（在某些研究中高达100%）和可控的安全性，常见的不良反应包括细胞因子释放综合征（CRS）和神经毒性。本综述指出了一些重要方面，如用于反应预测的生物标志物的识别、耐药机制的理解以及长期结局数据的关键需求。在不良反应的管理方面，尤其是CRS和神经毒性方面，仍存在挑战。本综述强调了CAR-T细胞疗法在小儿肿瘤学中的变革潜力，同时强调了需要进一步研究的关键领域，以优化患者结局并改善小儿ALL的治疗前景。
