AML-068- Survival Differences in Patients With Acute Myeloid Leukemia (AML) Treated With Oral Azacitidine (Oral-AZA) as Maintenance and Those Eligible but Not Treated in a US Electronic Health Record (EHR) Database (vol 23S1, S1-S593, 2023)

Context: Oral-AZA maintenance treatment has improved outcomes for patients with AML in remission following intensive induction/consolidation chemotherapy (IC) and ineligible for hematopoietic stem cell transplant (HSCT). We compared outcomes between patients in real -world settings who received Oral-AZA and those who were eligible but did not receive maintenance treatment. Design : Using Flatiron Health's EHR database, we identified patients with newly diagnosed AML who received IC, attained remission, and were >= 18 years. Exclusion criteria included acute promyelocytic leukemia diagnosis, treatment with arsenic trioxide/tretinoin, or clinical study enrollment during follow-up (diagnosis to last active date). Patients were eligible for maintenance after front -line IC on/after September 1, 2020, and either treated with Oral-AZA or did not receive any maintenance treatment (NoMaint, "watch-and-wait"). Cohorts were matched 1:3 on baseline demographic and clinical characteristics using propensity scores to minimize differences. Overall survival (OS) and relapse-free survival (RFS) were estimated using Kaplan-Meier analysis from maintenance eligibility date (later of remission/consolidation), censoring patients at the time of HSCT/last active date. Results: In total, 431 patients met the inclusion criteria; 12% received Oral-AZA maintenance. After matching, 52 patients were in OralAZA and 156 in the NoMaint cohort. Median age at diagnosis was 58 years (interquartile range, 47-67), 46% were female, 32% had poor cytogenetic risk (where known), 20% had Eastern Cooperative Oncology Group performance status (ECOG PS) 2 + (where known), and 13% had prior history of myeloproliferative disorder/myelodysplastic syndromes. Four patients in the Oral-AZA cohort (7.7%) and 39 in the NoMaint cohort (25.0%) received HSCT. Median follow-up from maintenance eligibility date was 11.6 and 3.7 months, respectively. Median (95% CI) RFS was significantly longer for Oral-AZA (20.0 months [12.0-not reached [NR]) vs NoMaint (8.2 months [5.0-10.9]; P < 0.001) as was median (95% CI) OS (Oral-AZA, [14.6-NR] vs NoMaint, 13.6 months [9.1-NR]; P = 0.001). Conclusions : Patients treated with Oral-AZA maintenance had significantly better survival outcomes than eligible watch -and -wait patients. These data confirm the pivotal QUAZAR AML -001 trial results showing OS benefit of Oral-AZA versus no active maintenance therapy, further supporting consistent drug use in all eligible patients in clinical practice.