Abstract: This article reviews the integrated application of immunotherapy and targeted therapy in pediatric leukemia，guided by molecular subtyping and measurable residual disease（MRD），and highlights their clinical value in achieving deeper remissions，optimizing treatment de-intensification，and enabling practical，scalable care pathways.It also proposes that immune reconstitution，infection prevention and control，and long-term functional outcomes should be included in routine follow-up frameworks based on the strategic bridging between CAR-T therapy and hematopoietic stem cell transplantation（HSCT）and the platformbased management. In the field of inherited hematologic disorders，gene therapy/gene editing is advancing“functional cure”from concept toward a viable clinical option；however，this progress requires a closed-loop governance framework encompassing screening and risk stratification，centralized implementation，and lifelong registry-based follow-up. For non-malignant conditions including aplastic anemia，hemophagocytic lymphohistiocytosis（HLH），and immune thrombocytopenia（ITP），the article emphasizes evidence-based risk stratification and standardized supportive treatment. Overall，future gains are most likely to arise from multi-modality synergy and health-system capacity building：positioning MRD and disease biology as the primary decision axis，and underpinning care with standardized monitoring and longitudinal，end-to-end management to achieve populationlevel，reproducible long-term benefit.

Key words: hematologic disorders, child, immunotherapy, targeted therapy, gene therapy

摘要： 概述儿童白血病免疫治疗与靶向治疗在分子分型与微小残留病指导下的整合应用，强调“更深缓解、减毒优化与可落地路径”的临床价值；同时结合嵌合抗原受体T 细胞免疫疗法与造血干细胞移植的策略衔接与平台化管理，提出免疫重建、感染防控及远期功能结局应纳入常规随访体系。在遗传性血液病领域，基因治疗 / 基因编辑正推动“功能性治愈”从概念走向可选路径，但需建立筛查分层、中心化实施与终身注册随访的闭环治理。针对再生障碍性贫血、噬血细胞综合征及免疫性血小板减少症等非肿瘤性疾病，该文强调循证基础上的风险分层与规范化支持治疗。总体而言，未来进展更可能来自多策略协同与系统能力建设：以微小残留病与生物学为决策轴，以标准化监测与全程管理为基础，推动人群层面、可复制的长期获益。

关键词: 血液系统疾病, 儿童, 免疫治疗, 靶向治疗, 基因治疗