中文摘要：

目的探讨Fms样酪氨酸激酶3内部串联重复（FLT3-ITD）突变阳性的急性髓系白血病（AML）患者的临床特征及预后，分析索拉非尼联合化疗一线治疗该类患者的疗效。方法回顾性分析2013年1月至2016年8月河南省肿瘤医院确诊的53例初治伴FLT3-ITD突变的AML患者资料，研究该类患者发病时生物学特征，并对比分析化疗联合与不联合索拉非尼治疗的疗效。结果53例FLT3-ITD突变阳性AML患者中，M5型所占比例最高（41.5%），外周血WBC中位数为61.00（0.98-920.00）×10^9/L，其中高白细胞计数（〉10×10^9/L）患者50例（94.3%）；骨髓原始细胞比例中位数为0.730（0.234-0.966）。53例患者总诱导缓解率为56.6%，索拉非尼联合化疗组与单纯化疗组诱导缓解率分别为86.4%（19/22）和35.5%（11/31），差异有统计学意义（P〈0.001）。索拉非尼联合化疗组与单纯化疗组患者1年总生存率分别为78.3%和50.0%（P=0.041），1年无进展生存率分别为75.9%和42.4%（P=0.044）。结论FLT3-ITD突变阳性AML具有初发时外周血WBC及骨髓原始细胞比例高、与M5亚型高度伴随的临床特征，索拉非尼联合化疗可明显改善该类患者的诱导缓解率及短期生存。

英文摘要：

ObjectiveTo analyze the clinical features of acute myeloid leukemia patients with Fms-like tyrosine kinase 3 internal tandem duplication （FLT3-ITD） mutation and the therapeutic effect of sorafenib in combination with chemotherapy as first-line therapy for these patients. MethodsClinical features and therapeutic effect were retrospectively analyzed in 53 AML patients with FLT3-ITD mutation diagnosed in Henan Cancer Hospital from January 2013 to August 2016. The biological characteristics and clinical efficacy of chemotherapy in combination with or without Sorafeinb were analyzed.ResultsFLT3-ITD mutation was identified in 53 AML patients, 22 cases （41.5%） were M5 subtype. The median of the peripheral WBC was 61.00 （0.98-920.00） ×10^9/L, and there were 50 （94.3%） patients with WBC〉10×10^9/L. The median of blast cell in bone marrow was 0.730 （0.234-0.966） . The total remission rate of all these 53 patients was 56.6% （30/53） . The complete remission （CR） rates in patients treated with chemotherapy in combination with sorafenib and patients with chemotherapy alone were 86.4% （19/22） and 35.5% （11/31） , respectively. The 1-year overall survival rates of the two groups were 78.3%% and 50.0% （P=0.041） , and 1-year progression free survival rates were 75.9% and 42.4% （P=0.044） , respectively.ConclusionAML patients with FLT3-ITD mutation have the characteristics of high peripheral WBC, high blast cells in bone marrow and accompanying with M5 subtype. Sorafeinb combined with chemotherapy can significantly improve CR rate and short term survival.