Abstract
In recent years, immunotherapy and targeted therapy are reshaping the therapeutic paradigm of pediatric acute leukemia, shifting it from a protocol-driven era toward a strategy-oriented era. The significance is not only in improving remission rates, but also in achieving deeper measurable residual disease (MRD) clearance, enabling more precise risk stratification, reducing cumulative toxicity, and advancing standardized implementation with enhanced accessibility in real-world clinical practice. Integrating pivotal clinical studies, real-world evidence, and advances in drug approval and policy, this article proposes three core themes-deep remission, low toxicity, and high accessibility. It highlights MRD and molecular stratification as anchors for therapeutic decision-making, emphasizes optimizing combination and sequential strategies based on relapse mechanisms, and advocates strengthening long-term follow-up systems and improving access to diagnosis and treatment, aiming to shift clinical practice from efficacy improvement toward high-quality cure.