酪氨酸激酶抑制剂作为儿童急性白血病造血干细胞移植后延续治疗的作用：一项系统评价。
完整摘要 · 中文译文
目的：造血干细胞移植（HSCT）后复发是儿童急性白血病患者移植失败的主要原因，凸显了需要有效的移植后延续治疗。本系统综述评估酪氨酸激酶抑制剂（TKIs）作为儿童急性白血病HSCT后延续治疗的作用。 方法：根据PRISMA指南对PubMed、Cochrane、Web of Science和VHL进行了文献检索。如果原始研究与研究问题相关且符合纳入标准，则予以纳入。 结果：初步检索共识别出765篇文章，经过筛选后纳入16项研究。TKIs适用于存在导致激酶信号组成性激活的染色体改变的患者。常用药物包括伊马替尼、达沙替尼、帕纳替尼、索拉非尼和吉瑞替尼，选择基于疾病状态和分子谱分析。延续治疗持续时间从10个月至3年不等，剂量取决于药物和耐受性。TKIs通常耐受良好，已报告的副作用包括血细胞减少、肝功能障碍和皮肤状况。在纳入的研究中，接受HSCT后TKI延续治疗的特定患者报告了微小残留病（MRD）阴性和持续分子学缓解，但治疗中止后或耐药性疾病中也有复发报告。然而，在儿童人群中最佳持续时间和长期安全性仍不明确。 结论：TKIs可作为HSCT后靶向延续 在选定的儿童急性白血病患者中，尤其是那些具有高危、突变驱动疾病的患者，治疗。分子谱分析和微小残留病（MRD）监测对于指导药物选择、时机和突变导向的治疗调整至关重要。这些发现支持个体化酪氨酸激酶抑制剂（TKI）治疗以降低造血干细胞移植（HSCT）后的复发风险，同时强调需要前瞻性研究来定义标准化治疗策略。
完整摘要 · 英文原文
PURPOSE: Relapse following hematopoietic stem cell transplantation (HSCT) is a leading cause of transplant failure for pediatric acute leukemia patients, emphasizing the need for an effective post-transplant continuation therapy. This systematic review evaluates the role of tyrosine kinase inhibitors (TKIs) as a post-HSCT continuation therapy in pediatric acute leukemia. METHODS: A literature search of PubMed, Cochrane, Web of Science, and VHL was conducted according to PRISMA guidelines. Original studies were included if they were relevant to the research question and fit the eligibility criteria. RESULTS: The initial search identified 765 articles, with 16 studies included after filtration. TKIs were indicated for patients with chromosomal alterations causing constitutively active kinase signaling. Common agents included imatinib, dasatinib, ponatinib, sorafenib, and gilteritinib, and selection was based on disease status and molecular profiling. Duration of continuation ranged from 10 months to 3 years, with dosage dependent on the agent and tolerability. TKIs were generally well-tolerated, with reported side effects including cytopenia, liver dysfunction, and skin conditions. Across the included studies, MRD negativity and sustained molecular remission were reported in selected patients receiving post-HSCT TKI continuation therapy, while relapse was also reported after treatment discontinuation or in resistant disease. However, optimal duration and long-term safety remain unclear in the pediatric population. CONCLUSION: TKIs can serve as a targeted post-HSCT continuation therapy in select pediatric acute leukemia patients, particularly those with high-risk, mutation-driven disease. Molecular profiling and MRD monitoring are central to guiding agent selection, timing, and mutation-directed therapy adjustments. These findings support individualized TKI treatment to reduce relapse risk following HSCT, while highlighting the need for prospective studies to define standardized treatment strategies.