Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial.

Abstract

11061 Background: Anemia in patients (pts) with myelofibrosis (MF) is associated with a significant disease burden, especially in pts who require red blood cell (RBC) transfusions, as it negatively impacts quality of life and disease prognosis. In the PERSIST-2 trial, treatment with pacritinib (PAC), a JAK1 sparing inhibitor of JAK2/IRAK1/ACVR1, was associated with anemia benefit. A significant proportion of non-transfusion independent (non-TI) pts at baseline on PAC compared to best available treatment (BAT) achieved transfusion independence (TI) (37% vs 7%) in any 12 weeks over a 24-week interval and significantly more had a ≥50% reduction in transfusion burden (49% vs 9%) (Oh ST, et al. 2023) with lower RBC transfusion rates (Mean [2.45 vs 3.54/30-day]) (Data on file). This study aimed at estimating the projected differences in transfusion-related cost and time burden with PAC vs BAT. Methods: An economic evaluation based on transfusion-related data in pts treated with PAC or BAT (including ruxolitinib [RUX] and erythroid support [ES]) from the PERSIST-2 trial (NCT02055781). Transfusion status (TI and non-TI) at baseline and over any 12-week interval within the 24-week study period was defined based on the Gale criteria (i.e. presence or absence of RBC transfusions). RBC transfusion rates over 30-day periods, including all reported transfusions within the initial 24-week study period, were annualized and used as proxy for transfusion-related visits. Annual transfusion-related cost estimates by transfusion status were based on a previous MF burden of illness study which utilized IBM MarketScan data (Gerds AT, et al. 2022) and was adjusted to 2024 US dollars using the medical component of the Consumer Price Index. Transfusion-related time burden estimates were based on previously reported RBC transfusion visits in transfusion dependent pts with β-thalassemia (Knoth RL, et al. 2022). Results: Annual transfusion-related cost with PAC was projected to be 19.5% lower, with a cost saving of ~$61K compared to BAT (~$252K vs ~$313K). Annual transfusion-related time burden with PAC vs BAT was lower by 25.3% with a time saving of ~172 hrs (~508 vs ~680 hrs). Among pts who were non-TI at baseline, projected annual cost and time savings for PAC vs BAT were ~$73K and ~204 hrs, respectively. Results remained robust regardless of type of BAT (i.e. RUX or ES therapies). Conclusions: The reduction in transfusion rates associated with PAC treatment relative to BAT is projected to result in decreased transfusion-related medical cost and time burden for pts with MF and anemia.