Objective: To assess the efficacy of induction chemotherapy followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of FLT3-ITD(+) acute myeloid leukemia (AML) with normal karyotype. Methods: The clinical data of FLT3-ITD(+) AML patients with normal karyotype in the First Affiliated Hospital of Nanjing Medical University from Jan 2018 to March 2021 were retrospectively analyzed. Results: The study included 49 patients with FLT3-ITD(+)AML, 31 males, and 18 females, with a median age of 46 (16-59) years old. All patients received induction chemotherapy, and 24 patients received sequential allo-HSCT (transplantation group) . The median follow-up time was 465 days, the one-year overall survival (OS) from diagnosis was (70.0 ± 7.4) %, and one-year disease-free survival (DFS) was (70.3±7.4) %. The one-year OS was significantly different between the transplantation group and the non-transplantation group [ (85.2 ± 7.9) % vs (52.6 ± 12.3) %, P=0.049]. but one-year DFS [ (84.7 ± 8.1) % vs (55.2 ± 11.9) %, P=0.061] was not. No significance was found in one-year OS between patients with low-frequency and high-frequency FLT3-ITD(+) (P>0.05) . There were 12 patients with high-frequency FLT3-ITD(+) in the transplantation and the non-transplantation groups, respectively. The one-year OS [ (68.8 ± 15.7) % in the transplantation group vs (26.2 ± 15.3) % in the non-transplantation group, P=0.027] and one-year DFS [ (45.5 ± 21.3) % in the transplantation group vs (27.8±15.8) % in the non-transplantation group, P=0.032] were significantly different between the two groups. Conclusion: Induction chemotherapy followed by allo-HSCT can enhance the prognosis of FLT3-ITD(+) patients, particularly those with FLT3-ITD high-frequency mutation.
目的： 评估诱导化疗序贯异基因造血干细胞移植治疗FLT3-ITD突变阳性伴正常染色体核型急性髓系白血病（AML）的疗效。 方法： 对2018年1月至2021年3月南京医科大学第一附属医院收治的FLT3-ITD(+)伴正常染色体核型AML患者进行回顾性分析。 结果： 49例FLT3-ITD(+)AML患者纳入研究，男31例，女18例，中位年龄46（16~59）岁。所有患者均接受诱导化疗，24例患者序贯异基因造血干细胞移植（移植组）。中位随访时间为465 d，确诊后1年总生存（OS）率为（70.0±7.4）%，1年无病生存（DFS）率为（70.3±7.4）%。移植组、非移植组1年OS率分别为（85.2±7.9）%、（52.6±12.3）%（P=0.049），DFS率分别为（84.7±8.1）%、（55.2±11.9）%（P=0.061）。在移植组和非移植组中，FLT3-ITD低频突变与高频突变患者1年OS率差异均无统计学意义（P>0.05）。移植组、非移植组中各有12例FLT3-ITD高频突变患者，1年OS率分别为（68.8±15.7）%、（26.2±15.3）%（P=0.027），1年DFS率分别为（45.5±21.3）%、（27.8±15.8）%（P=0.032）。 结论： 诱导化疗序贯allo-HSCT可改善FLT3-ITD(+)患者（特别是FLT3-ITD高频突变患者）的预后。.
Keywords: Allogeneic hematopoietic stem cell transplantation; FLT3-ITD mutation; Leukemia, myeloid, acute; Prognosis.