达沙替尼治疗核心结合因子相关性急性髓系白血病的理论基础及最新临床研究

Basic theory for the therapy of CBF-AML with dasatinib and the advance in clinical study

伴有c-KIT,FLT3等突变的核心结合因子相关性急性髓系白血病(CBF-AML)在白血病分层上归类于高危AML,预后极差,目前对于这部分患者尚无非常有效的治疗方案。达沙替尼是一种非常有效的双SRC/ABL激酶抑制剂,在众多体外试验中均证实可有效阻断c-KIT,FLT3等突变对下游通路的调控作用,遏制白血病细胞的增长,而且在最新的临床试验中也证实其对KIT阳性的CBF-AML是有效的。

CBF-AML with c-KIT and FLT3 mutations is classified as high-risk disease in association with poor prognosis and lack of effective treatments at present. It is reported that the dual SRC/ABL kinase inhibitor da- satinib can cut off the signal transduction pathway linking mutations including c-KIT and FLT3 to downstream tar- gets, thus prevent the growth of AML cells in exoteric experiment. It is demonstrated that dasatinib is effective to CBF-AML with KIT mutation in the latest clinical trials.