(221) Transfusion-related Cost and Time Burden Offsets in Patients with Myelofibrosis Treated with Pacritinib Compared to Best Available Therapy Based on PERSIST-2 Trial

Background: : Anemia in patients with myelofibrosis (MF) is associated with a significant disease burden, especially in patients 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) patients 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 ≥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).

Objective: : To estimate the projected differences in transfusion-related cost and time burden with PAC vs BAT

Methods: : An economic evaluation based on transfusion-related data in patients 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. Transfusion-related time burden estimates were based on previously reported RBC transfusion visits in transfusion dependent patients 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 hours (~508 hours vs ~680 hours). Among patients who were non-TI at baseline, projected annual cost and time savings for PAC vs BAT were ~$73K and ~204 hours, respectively. Results remained robust regardless of type of BAT (i.e. RUX or ES therapies).

Conclusions: : 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 patients with MF and anemia.

Sponsorship:: Sobi Inc.