Disease Burden, Inadequate Hematocrit Control, and Thromboembolic Events in Patients with Polycythemia Vera Despite...

To evaluate treatment and disease burden among patients with polycythemia vera (PV) receiving the current standard of care (SoC) in the US.

This retrospective study utilized MarketScan® Commercial/Medicare Databases to identify patients with PV diagnosis and treatment claims between 1/1/2011-12/31/2022 (index date=earliest PV treatment date), continuous enrollment (6 months pre-index and ≥12 months post-index), and no pre-index disease progression (myelofibrosis, acute myeloid leukemia, or myelodysplastic syndrome). Patients were categorized by thrombosis risk (high/low-risk) and 12-month post-index treatments: phlebotomy (PHL) only, hydroxyurea (HU) only, PHL+HU, or ruxolitinib/interferons (RUX/IFN). Outcomes included incident TE, iron deficiency anemia, disease progression, and PV-related symptoms. Hematocrit (HCT) control was evaluated in patients with ≥2 HCTs post-index (HCT analysis). All-cause costs during the 12 months post-TE were reported in patients with an incident TE and ≥12 months post-TE follow-up (TE analysis).

Among 11,311 patients (51.8% high-risk; 48.2% low-risk; median follow-up ~2.8 years), 12-month post-index treatments included PHL only (75.0%), HU only (12.2%), PHL+HU (10.9%), and RUX/IFN (1.9%). Frequent PHL (≥3 PHL in 6 months or ≥5 PHL in 12 months post-index; 46.2% of PHL users) and high-dose HU (≥1000mg/day; 41.7% of HU users) were common. Within 12 months post-index, 50.9% of patients experienced burdensome treatment (frequent PHL, high-dose HU) and/or an incident TE. During the full follow-up, 15.3% experienced incident TE, 9.6% experienced incident iron deficiency anemia, 4.4% experienced disease progression, and 83.2% experienced symptoms. For the HCT analysis (N=1,268), 85.3% had uncontrolled HCTs≥45%, with 55.4% having HCTs≥50%. For the TE analysis (N=1,159), mean 12-month all-cause costs post-TE were $71,195, with 29.2% attributable to the index TE.

Patients with PV have high treatment and disease burden with the current SoC. Current PV therapies (PHL, cytoreductive agents) do not consistently maintain HCT<45%, leaving patients at increased risk for life-threatening and costly TEs.