CAR-T cells in hematologic disorders: acute myeloid leukemia - A systematic review of efficacy and safety

Introduction. Acute myeloid leukemia (AML) is an aggressive hematologic malignancy with poor survival rates, especially in relapsed/refractory cases. While CAR-T cell therapy has transformed the management of other hematologic cancers, its application in AML remains limited, due to antigen heterogeneity and safety concerns. Methods. This systematic review analyzed 43 studies published between 2020 and 2024 to evaluate the efficacy and safety of CAR-T cell therapies targeting antigens such as CLL-1, CD33, CD123, and CD70 in AML. Data were extracted on survival outcomes, adverse events and antigen-specific responses. Results. CLL-1 demonstrated the most favorable outcomes, with a 60% survival response at 6.2 months in adults and 75% in pediatric patients. CD33 and CD123 showed moderate efficacy but were associated with higher hematologic toxicities. CD70 exhibited promising response rates but significant side effects (85% in preclinical studies). Emerging antigens like B7-H3 and dual-target strategies showed potential to improve outcomes by addressing antigen escape and tumor heterogeneity. Conclusions. CAR-T therapies targeting CLL-1 are highly promising for AML, offering high specificity and manageable toxicity. However, broader clinical adoption requires addressing challenges related to antigen variability and safety. In Colombia, the implementation of CAR-T therapies could revolutionize AML management but requires overcoming barriers such as cost, infrastructure, and local research capacity.