Allogeneic hematopoietic stem cell transplantation is associated with improved survival compared with chemotherapy in patients with acute myeloid leukemia harboring favorable-risk genetic features at initial diagnosis: a single-center retrospective study.
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BACKGROUND: Acute myeloid leukemia (AML) harboring favorable-risk genetic features at initial diagnosis (FR-AML) commonly achieves remission after chemotherapy, but the optimal post-remission strategy remains controversial. This study compared allogeneic hematopoietic stem cell transplantation (allo-HSCT) with chemotherapy and explored prognostic factors. METHODS: We retrospectively analyzed 190 patients with FR-AML treated at Fujian Medical University Union Hospital between 2012 and 2022. Patients were stratified into the chemotherapy (n = 118) and allo-HSCT (n = 72) groups according to their post-induction treatment. Survival outcomes, including overall survival (OS), leukemia-free survival (LFS), cumulative incidence of relapse (CIR), and non-relapse mortality (NRM), were assessed using Kaplan-Meier and competing-risk analyses. Multivariate Cox regression identified independent prognostic factors. RESULTS: At 3 years, the allo-HSCT group had higher OS (86.0% vs. 61.3%, P < 0.0001) and LFS (59.2% vs. 49.3%, P = 0.039) than the chemotherapy group, with lower CIR (3.8% vs. 33.6%, P < 0.0001). Stratified analyses showed that allo-HSCT was associated with improved 3-year OS compared with chemotherapy alone in CR1-AML and R/R AML patients. In CR1-AML, the 3-year OS was 92.8% versus 73.6% (P = 0.0099), whereas in R/R AML, it was 73.5% versus 26.6% (P = 0.00015). By age, outcomes were comparable among patients aged <20 years, whereas allo-HSCT was associated with better survival in those aged 20-60 years. Among 66 transplanted patients with measurable residual disease (MRD) data, 3-year OS did not differ significantly between MRD-positive and MRD-negative groups (86.0% vs. 85.6%, P = 0.35). CONCLUSIONS: Allo-HSCT was associated with improved survival and reduced relapse in FR-AML. This benefit extended beyond patients transplanted in CR1 to those who later develop R/R disease, in whom salvage allo-HSCT confers a marked survival advantage over chemotherapy. Deferred transplantation may remain feasible for selected patients when a suitable donor is available. Prospective studies are warranted to validate these findings and optimize post-remission treatment strategies in this population.