Real-World Treatment Patterns and Outcomes of Patients With Acute Myeloid Leukemia in Japan in the Pre-venetoclax Era.

BACKGROUND

Acute myeloid leukemia (AML) mainly affects elderly patients, and real-world outcomes are often inferior to those observed in clinical trials. More clinically detailed real-world data are needed to better understand AML care.

METHODS

This retrospective study analyzed treatment patterns and outcomes in patients with AML using a population-based dataset linking the Osaka Cancer Registry with hospital administrative data. We included 621 patients diagnosed with AML between 2010 and 2015, before the approval of venetoclax for AML in Japan. Patients were stratified by age group and first-line therapy, and outcomes were compared accordingly.

RESULTS

The 2-year overall survival rate was 33.4% in the entire cohort, with substantial variation by age. Survival decreased sharply with age, ranging from 69.9% in patients aged 18 to 39 to 7.7% in those aged ≥ 80. Intensive therapy (idarubicin or daunorubicin plus cytarabine) was predominantly used in younger patients, while older patients more frequently received low-intensity regimens or supportive care. Among those who underwent allogeneic hematopoietic cell transplantation (allo-HCT) following intensive therapy, 2-year survival reached 78.2% in the 18 to 39 group but declined in older groups. Nonrelapse mortality exceeded relapse mortality in some age groups.

CONCLUSIONS

Our findings underscore age as a key prognostic factor in AML and highlight the importance of optimizing treatment strategies for older patients.