OBJECTIVES: Patients with AML in remission following intensive chemotherapy who are not candidates for hematopoietic stem cell transplantation are eligible for oral azacitidine (Oral-AZA) as maintenance treatment. While the QUAZAR AML-001 trial (NCT01757535) demonstrated that Oral-AZA was associated with longer survival outcomes than placebo in this patient population, HCRU and costs associated with Oral-AZA treatment versus no maintenance treatment have not been studied in the real world. Therefore, we assessed HCRU and associated costs of these patient groups in the USA.

METHODS: Using the IQVIA PharMetrics^® Plus claims database, we identified newly diagnosed adult patients with AML who had a maintenance eligibility date for Oral-AZA (MaintDate) on or after September 1, 2020, and were treated with Oral-AZA or did not receive maintenance treatment (NoMaint). Patients were 1:3 propensity score matched on demographic characteristics. We assessed HCRU and costs on a per-patient per-month (PPPM) basis from MaintDate to end of follow-up using generalized linear models.

RESULTS: 43 Oral-AZA and 129 NoMaint patients were identified (mean follow-up was 9.1 and 3.4 months, respectively). The Oral-AZA cohort had less HCRU than the NoMaint cohort in various categories (0.23 vs 0.61 hospitalizations PPPM, 2.47 vs 4.40 non-office outpatient visits PPPM, 2.84 vs 4.53 laboratory tests PPPM; all P<0.01). These differences translated into lower all-cause costs for the Oral-AZA cohort (US dollars [USD] 25,786 PPPM vs USD 38,530, P<0.001), including USD 9222 in maintenance drug costs for Oral-AZA versus none for NoMaint.

CONCLUSIONS: The cost burden (PPPM) of HCRU of patients with AML treated with Oral-AZA is significantly lower than those who did not receive a maintenance treatment. This economic benefit is consistent with the clinical benefits observed in the QUAZAR AML-001 trial among Oral-AZA users compared with patients receiving no maintenance treatment.