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

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

Ana Alfonso-Pierola  1 , David Martinez-Cuadrón  2 , Rebeca Rodríguez-Veiga  2 , Cristina Gil  3 , Pilar Martínez-Sánchez  4 , Teresa Bernal  5 , Celina Benavente  6 , Mónica A Romero-Riquelme  7 , Josefina Serrano-Lopez  8 , Juan M Bergua  9 , Raimundo García-Boyero  10 , Mar Tormo  11 , Pilar Herrera  12 , Claudia L Sossa-Melo  13 , José A Pérez-Simon  14 , Carlos Rodríguez-Medina  15 , María F Bass-Maturana  16 , José L López-Lorenzo  17 , Lorenzo Algarra-Algarra  18 , Belén Vidriales-Vicente  19 , Manuel Pérez-Encinas  20 , Manuel Barrios-García  21 , Susana Vives  22 , María J Sayas-Lloris  23 , Marisa Capurro  24 , Sebastián Hidalgo  25 , Mayte Olave  26 , Diana Cuervo-Lozada  27 , Esperanza Lavilla-Rubira  28 , Felipe Casado  29 , Armando Mena-Durán  30 , Marta Valero-Nuñez  31 , Soledad Casado-Calderón  32 , Amaia Balerdi  33 , Vivianne Torres  34 , Rosa Fernández  35 , Víctor Noriega  36 , Mariana Stevenazzi  37 , Jorge Labrador  38 , Pilar León-Maldonado  39 , Beatriz de Rueda-Ciller  40 , Olga Arce-Fernández  41 , María L Amigo  42 , José Ángel Raposo-Puglia  43 , María Solé  44 , Blanca Boluda  2 , Rosa Ayala  4 , Eva Barragán  2 , Pau Montesinos  45 ; PETHEMA Group
Collaborators, Affiliations
Comparative Study

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

Ana Alfonso-Pierola et al. Leukemia. 2025 Nov.

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.

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Conflict of interest statement

Competing interests: The authors declare no competing interests.

Figures

Fig. 1
Fig. 1. Kaplan–Meier curves for RFS and OS in the overall population.
A RFS: relapse-free survival. B OS: overall survival.
Fig. 2
Fig. 2. Kaplan–Meier curves for RFS and OS in the propensity score-matched population.
A RFS: relapse-free survival. B OS: overall survival.
Fig. 3
Fig. 3. Kaplan–Meier curves for RFS and OS in AML patients diagnosed since 2017: overall and PS-matched cohorts.
A RFS in the overall cohort. B OS in the overall cohort. C RFS in the PS-matched cohort. D OS in the PS-matched cohort. RFS: relapse-free survival. OS overall survival, PS propensity score.
Fig. 4
Fig. 4. Forest plots of univariate analysis for OS and RFS in the overall population.
A OS: overall survival. B RFS: relapse-free survival.
Fig. 5
Fig. 5. Forest plots of univariate analysis for OS and RFS in the propensity score-matched population.
A OS: overall survival. B RFS: relapse-free survival.
See this image and copyright information in PMC

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