PETHEMA AML: Auto-HSCT vs chemotherapy consolidation for AML in first remission

A real-world retrospective cohort study compared long-term outcomes following consolidation with autologous hematopoietic stem cell transplantation (auto-HSCT) vs intensive chemotherapy (IC) in 1,272 patients with acute myeloid leukemia (AML) following first complete remission (CR1), utilizing data from the Spanish Programa Español para Tratamientos en Hematología (PETHEMA) AML registry (NCT02607059). Eligible patients received either three cycles of IC (n = 615) or two cycles of IC followed by auto-HSCT (n = 657), without subsequent allogeneic HSCT (allo-HSCT). Propensity score-matching was applied to auto-HSCT (n = 240) and IC (n = 237) cohorts. The primary endpoints were overall survival (OS) and relapse-free survival (RFS), and Alfonso-Pierola et al. published the results in Leukemia.

Key data: Overall, auto-HSCT was associated with prolonged OS (hazard ratio [HR], 0.73; 95% confidence interval [CI], 0.61–0.87; p < 0.001) and RFS (HR, 0.73; 95% CI, 0.63–0.86; p < 0.001). The survival outcomes were particularly beneficial in patients aged ≤65 years, those with a normal karyotype, and those with FLT3-ITD negativity. In the propensity score-matched analysis, the RFS benefit was sustained (HR, 0.80; 95% CI, 0.62–1.04; p = 0.092), but the OS differences were not significant (HR, 0.91; 95% CI, 0.66–1.25; p = 0.563) in the auto-HSCT vs IC cohorts. 

Key learning: Consolidation therapy with auto-HSCT vs IC provides significant RFS benefit for patients with AML in CR1, particularly in those who are younger or with favorable molecular profiles. Auto-HSCT is an effective option for low- and intermediate-risk AML and is a feasible alternative for high-risk AML, supporting its continued role in the measurable residual disease (MRD)-guided and targeted therapies setting.