Disease Burden, Inadequate Hematocrit Control, and Thromboembolic Events in Patients with Polycythemia Vera Despite Current Standard of Care Treatment: A Retrospective Claims Study.
Key claim: Despite current standard-of-care phlebotomy and cytoreductive therapy, most patients with polycythemia vera do not maintain hematocrit below 45% and continue to face high rates of thromboembolic events, symptoms, treatment burden, and substantial post-TE costs.
Abstract
PURPOSE: To evaluate treatment and disease burden among patients with polycythemia vera (PV) receiving the current standard of care (SoC) in the US. PATIENTS AND METHODS: 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). RESULTS: 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. CONCLUSION: 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.
Why this matters
This large real-world claims study quantifies how often standard-of-care PV management fails on its own terms: most patients do not achieve the guideline HCT <45% threshold, and thromboembolic events plus persistent symptoms continue to accrue at meaningful rates and cost. For the symptom-management area, it anchors why residual fatigue, microvascular symptoms, and thrombotic complications remain live problems even in treated PV — and why non-pharmacologic and adjunctive symptom strategies retain a role alongside cytoreduction.