Alfonso-Pierola Ana, Martinez-Cuadrón David, Rodríguez-Veiga Rebeca, Gil Cristina, Martínez-Sánchez Pilar, Bernal Teresa, Benavente Celina, Romero-Riquelme Mónica A, Serrano-Lopez Josefina, Bergua Juan M, García-Boyero Raimundo, Tormo Mar, Herrera Pilar, Sossa-Melo Claudia L, Pérez-Simon José A, Rodríguez-Medina Carlos, Bass-Maturana María F, López-Lorenzo José L, Algarra-Algarra Lorenzo, Vidriales-Vicente Belén, Pérez-Encinas Manuel, Barrios-García Manuel, Vives Susana, Sayas-Lloris María J, Capurro Marisa, Hidalgo Sebastián, Olave Mayte, Cuervo-Lozada Diana, Lavilla-Rubira Esperanza, Casado Felipe, Mena-Durán Armando, Valero-Nuñez Marta, Casado-Calderón Soledad, Balerdi Amaia, Torres Vivianne, Fernández Rosa, Noriega Víctor, Stevenazzi Mariana, Labrador Jorge, León-Maldonado Pilar, de Rueda-Ciller Beatriz, Arce-Fernández Olga, Amigo María L, Raposo-Puglia José Ángel, Solé María, Boluda Blanca, Ayala Rosa, Barragán Eva, Montesinos Pau
Hematology and Cell Therapy Department, Cancer Center Clínica Universidad de Navarra (CCUN), IdISNA, CIBERONC, Pamplona, Spain.
Hospital Universitari i Politècnic La Fe, Valencia, Spain.
Leukemia. 2025 Sep 1. doi: 10.1038/s41375-025-02744-x.
While allogeneic stem cell transplantation (allo-SCT) is the preferred consolidation for high and most intermediate-risk acute myeloid leukemia (AML) patients in first remission, the role of autologous SCT (auto-SCT) vs. chemotherapy (CT) when allo-SCT is not feasible or indicated, remains controversial. We conducted a real-world, retrospective cohort study using the PETHEMA AML registry to compare auto-SCT and CT. Multivariate Cox regression and propensity score matching (PS-matching) were used to adjust for confounding factors. A total of 1272 patients in first remission and who received 2 consolidation courses were included (615 receiving additional CT cycles and 657 undergoing auto-SCT). Overall, 78.08% of auto-SCT patients were diagnosed before 2017, compared to 38.11% in the CT cohort (p < 0.001). In the overall cohort, auto-SCT was associated with significantly prolonged overall survival (OS) (HR: 0.73, p < 0.001) and relapse-free survival (RFS) (HR: 0.73, p < 0.001). This benefit was particularly evident in patients ≤65 years, those with normal karyotype, and FLT3-ITD negativity. In the PS-matched cohort, the RFS advantage persisted (HR: 0.80, p = 0.092), but OS differences were not statistically significant (HR: 0.91, p = 0.563). The role of auto-SCT in the genomic and targeted agent era should not be discarded.
虽然异基因干细胞移植（allo-SCT）是初治时高危和大多数中危急性髓系白血病（AML）患者首选的巩固治疗方法，但当allo-SCT不可行或不适用时，自体SCT（auto-SCT）与化疗（CT）相比的作用仍存在争议。我们利用PETHEMA AML登记处进行了一项真实世界的回顾性队列研究，以比较auto-SCT和CT。采用多变量Cox回归和倾向评分匹配（PS匹配）来调整混杂因素。共有1272例初治且接受2个巩固疗程的患者纳入研究（615例接受额外的CT周期治疗，657例接受auto-SCT）。总体而言，78.08%的auto-SCT患者在2017年前被诊断，而CT队列中的这一比例为38.11%（p < 0.001）。在整个队列中，auto-SCT与显著延长的总生存期（OS）（HR：0.73，p < 0.001）和无复发生存期（RFS）（HR：0.73，p < 0.001）相关。这种益处在年龄≤65岁、核型正常以及FLT3-ITD阴性的患者中尤为明显。在PS匹配队列中，RFS优势依然存在（HR：0.80，p = 0.092），但OS差异无统计学意义（HR：0.91，p = 0.563）。在基因组和靶向药物时代，auto-SCT的作用不应被摒弃。
