Chimeric Antigen Receptor T-cell Therapy in the Treatment of Pediatric Acute Lymphoblastic Leukemia: Efficacy, Safety, and Future Directions.

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.