Healthcare Utilization and Costs Among Patients with Acute Myeloid Leukemia Receiving Oral Azacitidine Maintenance Therapy Versus No Maintenance: A US Claims Database Study

Abstract

Introduction: The substantial economic burden of acute myeloid leukemia (AML) could be reduced with post-remission maintenance therapies that delay relapse. Real-world healthcare resource utilization (HCRU) data and costs among patients with AML receiving oral azacitidine (Oral-AZA) maintenance therapy or no maintenance are not well understood. We characterize HCRU and costs among these patients in clinical practice in the USA.

Methods: Data from IQVIA PharMetrics® Plus (January 1, 2016-June 30, 2022) were used. Patients ≥ 18 years who were newly diagnosed with AML, received first-line systemic induction therapy, and attained disease remission were eligible. Patients receiving Oral-AZA maintenance and those receiving no maintenance ("watch and wait" [W&W]) were matched 1:3 on baseline characteristics using propensity score matching (PSM) and followed until hematopoietic stem cell transplantation or end of continuous insurance enrollment, whichever occurred first. Outcomes included treatment patterns, inpatient and outpatient visits, and costs.

Results: After PSM, the Oral-AZA cohort included 43 patients and the W&W cohort 129. Of the 43 patients receiving Oral-AZA, 88.4% started at the recommended dose of 300 mg and 11.6% at 200 mg. The Oral-AZA cohort had significantly (p = 0.0025) longer median (95% CI) time to relapse from the index maintenance date (median not reached [NR; 9.0 months-NR] vs 3.3 months [0.8 months-NR]), and fewer per person per month (PPPM) hospitalizations (0.23 vs 0.61; p = 0.0005) and overall outpatient visits (5.77 vs 7.58; p = 0.0391) than the W&W cohort. Despite higher AML drug costs PPPM in the Oral-AZA cohort ($16,401 vs $10,651 for W&W), total healthcare costs PPPM were lower ($25,786 vs $38,530 for W&W; p < 0.0001).

Conclusions: Patients with newly diagnosed AML treated with Oral-AZA maintenance in clinical practice had prolonged remission and lower HCRU and costs than patients receiving no maintenance therapy. These findings underscore the clinical and economic value of Oral-AZA in clinical practice.

Keywords: Acute myeloid leukemia; Costs; Economic burden; Healthcare resource utilization; Maintenance therapy; Oral azacitidine; Real-world data; Remission.

Fig. 1

Study design. Eligible patients were diagnosed with AML, received systemic induction therapy, and attained disease remission. Patients may or may not have received consolidation treatment, defined as taking a regimen involving cytarabine, after remission. The index maintenance date was the date of attaining remission, unless the patient received a consolidation treatment. In such cases, the date following the end of the consolidation treatment was considered the index maintenance date. Treatments received after consolidation and prior to relapse were considered maintenance therapy. Patients were included in the Oral-AZA cohort or in the W&W cohort based on whether they received maintenance therapy with Oral-AZA or received no maintenance therapy, respectively. Patients were followed until HSCT date or the end of continuous insurance enrollment, whichever occurred first. AML acute myeloid leukemia, HSCT hematopoietic stem cell transplantation, Oral-AZA oral azacitidine, W&W watch and wait

Fig. 2

Time to relapse A from index maintenance date and B relapse events. Adapted from Borate U, et al. Value Health. 2023;26(12):S67. ©2023 Elsevier. All rights reserved [32]. CI confidence interval, IQR interquartile range, NR not reached, Oral-AZA oral azacitidine, SE standard error, W&W watch and wait

Fig. 3

HCRU. HCRU was analyzed using a generalized linear model with a negative binomial distribution with a logarithm link function; adjusted for age (60 years), sex (male), insurance status (commercial), and region (South). Individual outpatient components do not add up to the total resource use because each component was modeled separately. ^aLaboratory visits include both visits to a laboratory and lab tests done in an office setting. *Significant difference. ER emergency room, HCRU healthcare resource utilization, Oral-AZA oral azacitidine, PPPM per person per month, W&W watch and wait

Fig. 4

Healthcare costs. Adapted from Borate U, et al. Value Health. 2023;26(12):S67. ©2023 Elsevier. All rights reserved [32]. Costs evaluated over the period from index maintenance date to HSCT date, if applicable, or end of follow-up. Adjusted costs were analyzed using a generalized linear model with a gamma distribution with a logarithm link function; adjusted for age (60 years), sex (male), insurance status (commercial), and region (South). Total costs based on health plan paid amounts were inflated to 2022 US dollars; individual components do not sum up to the total cost because each component was modeled separately. Overall outpatient costs include laboratory costs, office visits, outpatient visits, and other. Laboratory costs could include both visits to a lab and lab tests done in an office setting. ^aAML drugs included azacitidine, cladribine, clofarabine, cytarabine, daunorubicin, decitabine, doxorubicin, enasidenib, etoposide, fludarabine, gemtuzumab, gilteritinib, glasdegib, idarubicin, ivosidenib, midostaurin, mitoxantrone, sorafenib, and venetoclax. *Significant difference. AML acute myeloid leukemia, ER emergency room, HSCT hematopoietic stem cell transplantation, Oral-AZA oral azacitidine, PPPM per person per month, W&W watch and wait