Healthcare resource use and direct costs in severe aplastic anemia (SAA) patients before and after treatment with eltrombopag

<b>Purpose:</b> This study evaluated healthcare resource utilization (HCRU), and direct costs among severe aplastic anemia (SAA) patients treated with eltrombopag (EPAG) using US claims data. <b>Methods:</b> This retrospective, real-world claims database study identified SAA patients aged ≥2 years treated with EPAG who initiated any SAA treatment between 1 July 2014 and 31 December 2017 (identification period) using the Truven MarketScan databases. A subset of 82 patients treated with EPAG during the identification period were evaluated for all-cause and SAA-related HCRU and direct costs as well as blood transfusion 1 month before EPAG initiation (baseline) and at Month 6 after EPAG initiation (follow-up period). <b>Results:</b> The average patient age was 50.8 (<i>SD</i> = 20.6) years old, predominantly female (<i>n</i> = 43, 52.4%), and had a mean CCI at baseline of 1.1 (<i>SD</i> = 1.7). Hospitalizations, and ER, office, and outpatient visits were significantly lower at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (<i>p</i> &lt; .05 for all four all-cause HCRU and SAA-related hospitalizations). An almost two-fold decrease in reliance on biweekly blood transfusions was observed: 1.0 at weeks 1–2 to 0.5 at Month 6 after EPAG initiation. Although prescription costs (mean [<i>SD</i>]) were significantly higher at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (difference of $11,045 USD [<i>SD</i> = $18,801]), these increases were offset by savings in direct costs. Overall, a mean reduction in total all-cause costs of $29,391 USD [<i>SD</i> = $137,770] was reported at Month 6 after EPAG initiation due to substantial reductions in hospitalization ($40,060 USD [<i>SD</i> = $123,198]) and outpatient visits ($2,043 USD [<i>SD</i> = $25,264]). <b>Conclusion:</b> All-cause and SAA-related HCRU were reduced following EPAG treatment. Prescription costs were higher following treatment; however, these costs were generally offset by reductions in direct costs. These results provide real-world evidence around the role of EPAG in SAA treatment.