Rubnitz Jeffrey E
Department of Oncology, St. Jude Children's Research Hospital, 332 N. Lauderdale Street, Memphis, TN 38105-2794, USA.
Curr Treat Options Oncol. 2008 Feb;9(1):95-105. doi: 10.1007/s11864-008-0059-z. Epub 2008 May 28.
Despite the use of intensive chemotherapy and hematopoietic stem cell transplantation, approximately one-third of children with acute myeloid leukemia (AML) still suffer relapse of their disease. It is unlikely that improvements in outcome can be achieved by further intensification of conventional chemotherapy. Instead, advances in the treatment of children with AML will require a greater understanding of the biology of the disease, with particular attention to the genetic abnormalities underlying leukemogenesis and drug resistance. Future clinical trials should include refined risk-directed therapy based on the genetics of the leukemic blasts and the patient's response to therapy. More important, we must develop alternative treatment approaches, such as agents that target specific leukemia-associated abnormalities and agents that selectively eradicate leukemic stem cells.
尽管采用了强化化疗和造血干细胞移植，但约三分之一的急性髓系白血病（AML）儿童仍会出现疾病复发。通过进一步强化传统化疗不太可能改善治疗结果。相反，AML儿童治疗的进展需要更深入地了解该疾病的生物学特性，尤其要关注白血病发生和耐药性背后的基因异常。未来的临床试验应包括基于白血病原始细胞遗传学和患者对治疗反应的精准风险导向治疗。更重要的是，我们必须开发替代治疗方法，例如针对特定白血病相关异常的药物以及选择性根除白血病干细胞的药物。
