We analyzed 140 patients with a median age of 51 years; 21% had WBC ≥ 100 × 109/L, and 52% had an NPM1 co-mutation. Until 2018, 101 patients received chemotherapy; thereafter, 39 received 3+7+midostaurin. The overall CR rate was 64%, higher in NPM1 mutant patients (73%). Univariate analysis showed that NPM1 mutation (p= 0.032) and WBC < 100 × 109/L (p= 0.013) positively influenced the response, with a trend for FLT3i administration (p= 0.052). Multivariate analysis confirmed WBC count as an independent prognostic factor (p= 0.017). In CR1, 41/90 patients underwent allogeneic and 18 autologous transplantation. The median EFS was 1.1 vs. 1.6 years in autografted and allografted patients, respectively (p= 0.9). The one-year non-relapse mortality was 0.00% for autologous and 28% for allogeneic transplants (p= 0.007); CIR at 1 and 3 years was higher in autologous transplants (39% vs. 15% and 57% vs. 21%,p= 0.004). The median survival was not reached in the FLT3i group. Overall, 69 patients received stem cell transplantation (18 autologous, 51 allogeneic). Post-transplant FLT3i was resumed in eight patients, all alive after a median of 65 months. Allogeneic transplantation is crucial in FLT3-mutated AML, but the next challenge will be to identify which patients can benefit from transplants in CR1 and in which to intensify post-transplant therapy.
本研究共纳入140例患者，中位年龄51岁；其中21%的患者白细胞计数≥100×10⁹/L，52%存在NPM1共突变。截至2018年，101例患者接受化疗；此后39例患者接受"3+7+米哚妥林"方案治疗。总体完全缓解率为64%，NPM1突变患者缓解率更高（73%）。单因素分析显示NPM1突变（p=0.032）和白细胞计数<100×10⁹/L（p=0.013）对治疗反应有积极影响，FLT3抑制剂使用呈现改善趋势（p=0.052）。多因素分析证实白细胞计数是独立预后因素（p=0.017）。在首次完全缓解期，90例患者中41例接受异基因移植，18例接受自体移植。自体移植与异基因移植患者的中位无事件生存期分别为1.1年与1.6年（p=0.9）。一年非复发死亡率自体移植为0.00%，异基因移植为28%（p=0.007）；自体移植患者1年及3年累积复发率更高（39% vs. 15%，57% vs. 21%，p=0.004）。FLT3抑制剂组中位生存期未达到。总体而言，69例患者接受干细胞移植（18例自体移植，51例异基因移植）。8例患者在移植后重启FLT3抑制剂治疗，中位随访65个月后全部存活。异基因移植对FLT3突变急性髓系白血病至关重要，但后续需明确哪些患者能在首次完全缓解期从移植中获益，以及哪些患者需要强化移植后治疗。
Real-Life Management of FLT3-Mutated AML: Single-Centre Experience over 24 Years
肿瘤(癌症)患者之家