[Analysis of treatment outcomes of acute myeloid leukemia between different genders].

Objective

To compare the treatment outcomes between male and female patients with acute myeloid leukemia (AML) .

Methods

In this retrospective cohort study, data from adult patients with newly diagnosed AML at Peking University People's Hospital between January 2017 and June 2025 were collected and analyzed. The primary outcomes included complete remission with or without incomplete hematological recovery (CR/CRi) and overall survival (OS) rate.

Results

A total of 2 264 patients were enrolled, including 1 180 males (52%). Males had significantly higher hemoglobin, uric acid, and albumin levels, but a lower estimated glomerular filtration rate than females (P=0.006 for albumin, P<0.001 for other variables). Males harbored a greater number of myeloid gene mutations (P=0.001) and showed significantly higher frequencies of ASXL1, U2AF1, SRSF2, RUNX1, ZRSR2, DDX41, SETBP1, TET2, and FLT3-TKD mutations, as well as -7/7q- and +8 chromosomal abnormalities. Contrarily, males exhibited significantly lower frequencies of NPM1, DNMT3A, and FLT3-ITD mutations than females. Multivariate analysis showed that male sex was significantly associated with a lower rate of CR/CRi after one course of induction therapy (OR=0.80, 95% CI: 0.66-0.97, P=0.023) and inferior relapse-free survival (HR=1.17, 95% CI: 1.00-1.37, P=0.046) and OS (HR=1.27, 95% CI: 1.09-1.47, P=0.002). Among patients classified as having favorable-, intermediate-, or adverse-risk disease according to ELN 2022 risk classification, males had inferior OS compared with females. In patients receiving the azacitidine plus venetoclax regimen, males had a significantly lower CR/CRi rate after one treatment course (62% vs 73%, P=0.004), but the difference was not significant in those receiving intensive chemotherapy (70% vs 71%, P=0.593) or the CAG regimen (54% vs 62%, P=0.222). Among patients who achieved CR/CRi, the 4-year OS rate was significantly lower in males than in females in both the allogeneic hematopoietic stem cell transplantation (allo-HSCT) group with CR1 [74% (95% CI: 69%-78%) vs 81% (95% CI: 77%-85%), P=0.007] and allo-HSCT group with CR2 or no response [54% (95% CI: 43%-66%) vs 69% (95% CI: 58%-80%), P=0.035]. In the non-transplant group, males showed a trend toward inferior 4-year OS rate[55% (95% CI: 49%-60%) vs 59% (95% CI: 54%-64%), P=0.084].

Conclusion

In patients with AML, males are more likely to harbor myelodysplasia-related gene mutations and chromosomal abnormalities, and sex-based disparities in the treatment response and OS rates have also been observed.