Capria Saveria, Trisolini Silvia Maria, Torrieri Lorenzo, Amabile Elena, Marsili Giovanni, Piciocchi Alfonso, Barberi Walter, Iori Anna Paola, Diverio Daniela, Carmini Daniela, Breccia Massimo, Martelli Maurizio, Minotti Clara
Hematology AOU Policlinico Umberto I, 00161 Rome, Italy.
Hematology, Department of Translational and Precision Medicine, "Sapienza" University, 00185 Rome, Italy.
Cancers (Basel). 2024 Aug 17;16(16):2864. doi: 10.3390/cancers16162864.
We analyzed 140 patients with a median age of 51 years; 21% had WBC ≥ 100 × 10/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 ( = 0.032) and WBC < 100 × 10/L ( = 0.013) positively influenced the response, with a trend for FLT3i administration ( = 0.052). Multivariate analysis confirmed WBC count as an independent prognostic factor ( = 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 ( = 0.9). The one-year non-relapse mortality was 0.00% for autologous and 28% for allogeneic transplants ( = 0.007); CIR at 1 and 3 years was higher in autologous transplants (39% vs. 15% and 57% vs. 21%, = 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.
我们分析了140例患者，中位年龄为51岁；21%的患者白细胞计数≥100×10⁹/L，52%的患者存在NPM1共突变。截至2018年，101例患者接受了化疗；此后，39例患者接受了3+7+米哚妥林治疗。总体完全缓解（CR）率为64%，在NPM1突变患者中更高（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）对反应有积极影响，使用FLT3抑制剂有一定趋势（P = 0.052）。多因素分析证实白细胞计数是独立的预后因素（P = 0.017）。在完全缓解1期（CR1），90例患者中有41例接受了异基因移植，18例接受了自体移植。自体移植和异基因移植患者的中位无事件生存期（EFS）分别为1.1年和1.6年（P = 0.9）。自体移植和异基因移植的1年无复发生存率分别为0.00%和28%（P = 0.007）；自体移植1年和3年的累积复发率（CIR）更高（39%对15%，57%对21%，P = 0.004）。FLT3抑制剂组的中位生存期未达到。总体而言，69例患者接受了干细胞移植（18例自体，51例异基因）。8例患者移植后恢复使用FLT3抑制剂，中位65个月后均存活。异基因移植对FLT3突变的急性髓系白血病（AML）至关重要，但下一个挑战将是确定哪些患者能在CR1期从移植中获益，以及哪些患者需要强化移植后治疗。
