Autologous Stem Cell Transplantation Shows Improved Relapse-Free Survival Benefit Over Chemotherapy for AML Consolidation Therapy
Key Clinical Summary
- Population and Design: Retrospective real-world cohort study of 1,272 AML patients in first remission receiving ≥ 2 consolidation courses; 657 underwent autoSCT and 615 received chemotherapy (median follow-up, 29.4 mo).
- Efficacy: Median OS 150.6 vs 75.9 mo (HR 0.73; 95% CI, 0.61 to 0.87; P < .001) and RFS 55.1 vs 26.1 mo (HR 0.73; 95% CI, 0.63 to 0.86; P < .001) favored autoSCT; benefits were strongest in <65 y, FLT3–, and normal cytogenetic subgroups.
- Clinical Relevance: autoSCT provides superior relapse-free survival and durable benefit over chemotherapy as consolidation for AML in first remission, supporting its use in select, alloSCT-ineligible patients.
Autologous stem cell transplantation (autoSCT) showed superior relapse-free survival (RFS) compared to chemotherapy (CT) as consolidation therapy for patients with acute myeloid leukemia (AML), according to real-world, retrospective study results published in Leukemia.
Research is limited on the superiority of autoSCT compared with chemotherapy for patients with AML in first remission or ineligible for allogeneic SCT. Researchers docuted a retrospective real-world cohort study to evaluate outcomes of autoSCT versus chemotherapy as consolidation therapy for patients with AML. The primary end points were overall survival (OS) and relapse-free survival (RFS).
The study included 1,272 AML patients in first remission who received at least 2 courses of consolidation therapy. Of which, 657 underwent auto-SCT, and 615 received additional chemotherapy. Patients treated with chemotherapy had a median age of 53.2 years (range, 18.06 to 70.81), while patients who received autoSCT had a median age of 50.37 years (range, 18.22 to 70.87). FLT3-ITD mutations were identified among 16.03% of patients treated with chemotherapy and 18.33% of patients who received autoSCT.
The median follow-up for all patients was 29.37 months (interquartile range [IQR], 14.49 to 68.99). The median OS was higher among patients who received autoSCT (150.62 months) than patients treated with chemotherapy (75.85 months; hazard ratio [HR], 0.73; 95% confidence interval [CI], 0.61 to 0.87; P < .001). Subgroup analyses demonstrated the benefit was more pronounced in patients younger than 65 years, those with normal cytogenetics, and those negative for FLT3-ITD mutations.
The median RFS was also higher among patients who received autoSCT (55.06 months) than patients treated with chemotherapy (26.12 months; HR, 0.73; 95% CI, 0.63 to 0.86; P < .001).
In the propensity score–matched cohort, the RFS advantage for autoSCT was maintained (HR, 0.80; P = 0.092), although OS differences did not reach statistical significance (HR, 0.91; P = 0.563).
A complete response with incomplete hematologic recovery (CRi) was achieved by 2.8% of patients treated with chemotherapy, while 0.9% of patients in the autoSCT group achieved a CRi.
The researchers concluded, “this study provides the most extensive real-world evidence to date on the efficacy of auto-SCT compared to CT as consolidation therapy for AML patients who do not undergo allo-SCT.”
They added, “Our findings confirm a significant RFS benefit associated with auto-SCT, particularly in younger patients, those with favorable risk or normal cytogenetic, and those with FLT3 negativity,” explaining, “The role of autoSCT in the MRD-guided and targeted agent era should not be discarded.”
Source:
Alfonso-Pierola A, Martinez-Cuadrón D, Rodríguez-Veiga R, et al. Long-term benefits of autologous stem cell transplantation versus intensive chemotherapy consolidation for acute myeloid leukemia patients: A propensity score matching analysis from the PETHEMA AML registry. Leukemia. Published online September 1, 2025. doi:10.1038/s41375-025-02744-x
