摘要
儿童慢性髓细胞性白血病(chronic myelogenous leukemia,CML)是一种由BCR/ABL融合基因驱动的造血干细胞恶性血液病。酪氨酸激酶抑制剂(tyrosine kinase inhibitor,TKI)的出现显著提升了CML患儿长期生存率,是CML治疗史上的里程碑。然而,TKI可能对儿童生长发育产生长期影响,需权衡终身治疗的耐受性和生活质量。第二代TKI达沙替尼和尼洛替尼在儿童CML一线治疗中的地位进一步明确;伊马替尼的长期价值在特定人群中获精准评估;新型药物如阿思尼布为克服耐药开辟新路径。现系统梳理近期儿童CML靶向治疗关键进展,深入分析各代TKI的疗效与安全性,探讨个体化选择策略,并展望以精准医疗和功能性治愈为导向的未来方向。
Pediatric chronic myelogenous leukemia(CML),a malignant hematopoietic stem cell disease driven by the BCR/ABL fusion gene.The emergence of tyrosine kinase inhibitor(TKI)has signiffcantly improved long-term survival rates in children with CML,marking a milestone in its therapeutic history.However,TKI may exert long-term effects on pediatric growth and development,necessitating careful consideration of lifelong treatment tolerance and quality of life.The status of the second-generation TKI,dasatinib and nilotinib,in the ffrst-line treatment of pediatric CML has been further clariffed.The long-term value of imatinib has been precisely evaluated in speciffc populations.The novel agents like asciminib,which offer new avenues to overcome resistance.This review systematically summarized key recent progress in targeted therapy for pediatric CML,deeply analyzed the efffcacy and safety proffles of each TKI generation,discussed individualized treatment selection strategies,and envisioned future directions guided by precision medicine and functional cure.
作者
谢志伟
王宁玲
张坤龙
储金华
杨林海
XIE Zhiwei;WANG Ningling;ZHANG Kunlong;CHU Jinhua;YANG Linhai(Department of Pediatrics,the Second Afffliated Hospital of Anhui Medical University,Hefei 230601,Anhui Province,China)
出处
《世界临床药物》
2026年第1期1-6,共6页
World Clinical Drug
