Inadequate Hematocrit Control in Polycythemia Vera Linked to High Health Care Burden

Key Takeaways

• A US retrospective claims study of 11 311 patients with polycythemia vera (PV) found that 85.3% of patients with available hematocrit measurements did not consistently maintain levels below 45%.
• During follow-up, 15.3% experienced thromboembolic events, with higher rates among high-risk patients.
• Mean all-cause health care costs reached $71 195 during the 12 months following a thromboembolic event, highlighting the economic burden associated with PV complications.

A retrospective US claims study published in the Journal of Blood Medicine found substantial clinical and economic burdens among patients with PV receiving standard-of-care treatment. Researchers reported inadequate hematocrit control, persistent thromboembolic events (TEs), and considerable health care expenditures, highlighting challenges in managing this chronic myeloproliferative neoplasm.

High Rates of Inadequate Hematocrit Control Despite Treatment

Researchers analyzed Merative MarketScan Commercial and Medicare claims data from 11 311 patients with PV who received treatment between January 2011 and December 2022. Patients were followed for a median of 2.8 years and categorized according to thrombosis risk and treatment regimen.

During the first 12 months, 75.0% received phlebotomy alone, 12.2% hydroxyurea alone, 10.9% both treatments, and 1.9% ruxolitinib or interferons.

Among patients receiving phlebotomy, 46.2% required frequent procedures, while 41.7% of hydroxyurea users received high-dose treatment. Overall, 50.9% experienced burdensome treatment, an incident TE, or both during the first year.

Among 1268 patients with at least 2 hematocrit measurements, 85.3% had at least 1 measurement of 45% or higher, and 55.4% had at least 1 measurement of 50% or higher. In patients receiving phlebotomy alone, 90.6% experienced inadequate hematocrit control.

During follow-up, 15.3% experienced an incident TE, 9.6% developed iron deficiency anemia, and 4.4% experienced disease progression. PV-related symptoms were documented in 83.2% of patients.

Thromboembolic Events Drive Substantial Health Care Costs

The findings highlight the challenges of maintaining hematocrit below 45%, an established treatment target intended to reduce thromboembolic risk.

TE incidence was higher among high-risk patients than low-risk patients (21.3% vs 9.0%). However, both groups experienced substantial disease burden, suggesting that inadequate disease control remains a concern across risk categories.

Among 1159 patients with an incident TE and sufficient follow-up, mean all-cause health care costs totaled $71 195 during the subsequent 12 months, measured in 2023 US dollars. Approximately 29.2% of these expenditures were attributable to the initial event.

Mean costs were $77 867 among low-risk patients and $68 332 among high-risk patients. Researchers noted that differences in commercial and Medicare reimbursement may help explain this variation.

For payers, these findings underscore the importance of considering thromboembolic complications, treatment burden, and subsequent health care utilization when evaluating PV management strategies.

Researchers Highlight Gaps in Current PV Management

The researchers emphasized the limitations of existing treatment approaches and concluded that the findings demonstrate a "critical unmet need for more effective therapeutic strategies" capable of maintaining hematocrit control and reducing PV burden.

They also called for more individualized management approaches, including greater consideration of pharmacologic treatment strategies.

Findings Reinforce Need for Improved PV Treatment Strategies

The study's retrospective design, limited laboratory data, and lack of adjustment for baseline differences restrict causal interpretation. Nevertheless, the findings demonstrate substantial clinical and economic burdens associated with PV despite available treatments.

Reference

Gerds AT, Fan Q, Jerry M, Cerretani A, Tran AT, Hernandez L. Disease burden, inadequate hematocrit control, and thromboembolic events in patients with polycythemia vera despite current standard of care treatment: a retrospective claims study. J Blood Med. 2026;17:626464. doi:10.2147/JBM.S626464