急性髓细胞白血病的靶向治疗

Targeted Treatment for AML

目前的化疗方案可能已达到其治急性髓细胞白血病（AML）的极限，今后应探索疾病分子学、免疫表型和生物学特征，以及患者的生物学特点，目的是转向更多的靶向治疗。虽然CD33不是白血病特异的抗体，但它在已确定的免疫表型中是一个合适的抗体治疗靶位。单克隆抗体治疗已经在复发AML中产生明显的缓解率，其与化疗联合治疗的几个随机研究接近完成。在AML越来越多的异常分子描述中，FLT-3突变在针对性治疗中似乎是最值得注意的，几个二期研究已显示出一定的疗效，联合治疗的随机研究正在进行。能被特异靶向治疗的其他机理包括法尼化、甲基化状态和组蛋白去已酰化。白血病干细胞群免疫表型和生物学特征的新知识，己为发展针对白血病干细胞特征的治疗提供了方向。

At present ,available chemotherapy has probably reached the limits of its potential in treating acute myeloid leukemia （AML）. Future purpose should be paid to targeted therapy by detating the molecular medicine, immunophenotype and biological features of the disease and the biological feature of patients. Although, CD33 is not the specific antibody of leukemia, but it has been identified to be the feasible therapeutic target for its immunophenotype. Monotberapy can achieve the obviouse remission rate in the recurred AML, and its several random therapy binded with chemotherapy is proceeding to its end. FLT-3 mutation seems to be more notable in its targeted therapy during the accumulated data of abnormal molecules. Its several study （ phase Ⅱ） had achieved somewhat efficacy, the random study of its combinded therapy is on going. The principle of targeted therapy includes farnesylation, methylation status, and histone deacelylation. New knowledge about the immunophenotypic and biological characteristics of the leukemic stem cell population has opened opportunities to develop treatments targeting the feature of its stem cells.