Wang Ni-Na, Ye Qi-Dong
Department of Hematology, Shanghai Children's Hospital, Shanghai Jiao Tong University, Shanghai 200062, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2017 Jul;19(7):832-836. doi: 10.7499/j.issn.1008-8830.2017.07.020.
At present, acute myeloid leukemia (AML) accounts for about 15%-20% of childhood acute leukemia. Although overall survival rate is increasing with the help of risk stratification, stratification of chemotherapy, and supportive treatment, conventional pharmacotherapy still has a limited clinical effect and certain limitations in improving remission rate in previously untreated patients and reducing recurrence after remission. With the development of precision medicine, the mechanisms of targeted therapy, including abnormal activation of AML-related signaling pathways and epigenetic modification, have been found in recent years. Molecular-targeted drugs can therefore act on specific receptors and target genes to improve clinical effect and the prognosis of AML patients.
目前，急性髓系白血病（AML）约占儿童急性白血病的15%-20%。尽管在风险分层、化疗分层和支持治疗的帮助下总体生存率有所提高，但传统药物治疗在提高初治患者的缓解率以及降低缓解后的复发率方面，临床效果仍然有限且存在一定局限性。随着精准医学的发展，近年来已发现靶向治疗的机制，包括AML相关信号通路的异常激活和表观遗传修饰。因此，分子靶向药物可作用于特定受体和靶基因，以提高AML患者的临床疗效和预后。
