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. 2025 Nov;39(11):2686-2696. doi: 10.1038/s41375-025-02744-x. Epub 2025 Sep 1.

Abstract

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.

Publication types

  • Comparative Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antineoplastic Combined Chemotherapy Protocols* / therapeutic use
  • Consolidation Chemotherapy* / methods
  • Female
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation* / methods
  • Hematopoietic Stem Cell Transplantation* / mortality
  • Humans
  • Leukemia, Myeloid, Acute* / mortality
  • Leukemia, Myeloid, Acute* / pathology
  • Leukemia, Myeloid, Acute* / therapy
  • Male
  • Middle Aged
  • Prognosis
  • Propensity Score
  • Registries
  • Retrospective Studies
  • Survival Rate
  • Transplantation, Autologous
  • Young Adult