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PDF(638 KB)
PDF(638 KB)
儿童急性白血病治疗格局升级:免疫与靶向的整合与突破
Evolving treatment landscape of pediatric acute leukemia: integration and breakthroughs in immunotherapy and targeted therapy
近年免疫治疗与靶向治疗正推动儿童急性白血病由“方案时代”迈向“策略时代”:其意义不仅在于提高缓解率,更在于实现更深可测量残留病清除、更精准分层、更低累积毒性,并推动相关治疗在真实临床实践中的规范应用与可及性提升。该文结合关键临床研究、真实世界证据与药物审批政策方面的进展,提出“深缓解—低毒性—高可及”三条主线,强调以微小残留病与分子分层为锚点,围绕复发机制优化联合与序贯策略,并通过完善长期随访体系、提升诊疗可及性,推动临床目标从“疗效改善”迈向“高质量治愈”。
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.
急性白血病 / 免疫治疗 / 靶向治疗 / 微小残留病 / 儿童
Acute leukemia / Immunotherapy / Targeted therapy / Measurable residual disease / Child
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所有作者均声明无利益冲突。