Effective treatment options remain limited for elderly patients with acute myeloid leukemia (AML) , particularly for those with significant frailty. This prospective phase 2 clinical trial evaluated efficacy and safety of azacitidine combined with low-dose cytarabine (LDAC) in newly diagnosed, frail elderly AML patients. As of June 30, 2025, we enrolled 38 patients aged ≥ 60 years who were deemed unsuitable for intensive chemotherapy. The treatment regimen consisted of subcutaneous azacitidine (75 mg/m2 daily on days 1-7) plus subcutaneous LDAC (20 mg twice daily on days 1-10) in 28-day cycles. Primary endpoint was overall response rate (ORR; defined as CR+CRi+MLFS); secondary endpoints included overall survival (OS), duration of response (DOR), safety and tolerability. Median age was 73.1 years (range, 62.2-85.4 years), and 15.8% had secondary AML. According to the 2022 ELN risk classifications, 8 (21.1%), 11 (28.9%) and 19 (50%) patients had favorable-risk, intermediate-risk and adverse-risk AML. Molecular profiling revealed myelodysplasia-related mutations in 36.8% of patients and TP53 mutations in 15.8%. No early death (within 30 days) was observed. ORR was 50% (19/38), median time to first response was 1.7 months, and among them 13 (68.4%) patients achieved MRD negative. Median follow-up duration was 14.6 (95% CI: 8.7-20.5) months and median number of treatment cycles delivered was 2 (range, 1-9). Median OS was 22.5 (95% CI 7.9-37.1) months and median DOR was 9.1 (95% CI: 6.07-12.13) months. Key grade ≥ 3 adverse events were febrile neutropenia (47.3%) and neutropenia (18.4%). Azacitidine plus LDAC exhibits favorable safety profiles and highly effective, and may be a promising option for frail elderly AML patients ineligible for intensive chemotherapy. This trial was registered at chictr.org.cn as ChiCTR2000035292.

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2025
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