关键词: 血液系统疾病, 儿童, 免疫治疗, 靶向治疗, 基因治疗

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