Treatment patterns and blood count control in 10,112 patients with polycythemia vera

Elevated blood counts in polycythemia vera (PV) are associated with increased thrombotic risk, which contributes to morbidity and mortality. This retrospective study describes treatment patterns and blood count control in patients with PV managed at community oncology practices (January 2014–February 2023; Integra Precision Q database). Of a total 10,112 patients, most received phlebotomy (68.1%) or hydroxyurea (HU; 28.2%) as initial treatment, with median follow-up of 32.1 (IQR, 13.5–58.5) months and 31.5 (IQR, 16.8–54.9) months, respectively. Changing treatment was less common than remaining on initial treatment despite 67.8% of patients on phlebotomy and 30.4% on HU having elevated hematocrit (≥45%) after 1 year of treatment. In contrast, 85.4% of patients who switched to ruxolitinib from HU achieved hematocrit &lt; 45% after 1 year, and fewer required phlebotomy during ruxolitinib treatment than with HU treatment (RUX, 29.3%; HU, 53.5%). Additionally, 54.2% of patients who switched to ruxolitinib achieved white blood cell counts &lt; 11 × 10⁹/L, and 57.5% achieved platelet counts ≤ 400 × 10⁹/L after 1 year of ruxolitinib treatment. This real-world evidence highlights the importance of considering alternative therapies for patients whose initial treatment regimen does not provide adequate clinical benefit. <b>Why was the study done?</b> Polycythemia vera (PV) is a rare blood cancer that can cause life-threatening blood clots. Clots usually occur in patients with too many red or white blood cells. To better understand how effective PV treatments are in real-world conditions, the researchers asked which treatments patients were given by their doctors and whether their blood counts remained high. <b>What did the researchers do and find?</b> Researchers analyzed data from 10,112 patients who received PV treatment between 2014 and 2023. Most patients started with standard therapies, with about two-thirds receiving therapeutic blood draws and about one-quarter receiving the chemotherapy hydroxyurea. Patients often remained on their initial treatment, even when that treatment did not meet therapy goals. Even after 1 year of initial treatment, two-thirds of patients treated with blood draws and nearly one-third with hydroxyurea still had high red blood cell counts. In contrast, 85% of patients who switched from hydroxyurea to ruxolitinib had well-controlled red blood cell counts after 1 year and required fewer blood draws. In addition, over half of patients who switched to ruxolitinib for 1 year had well-controlled white blood cell and platelet counts. <b>What do the results mean?</b> Patients with PV may be able to overcome persistent high blood counts by switching to ruxolitinib. Lowering blood counts is known to reduce chances of developing life-threatening clots. <b>What is the objective influence on the wider field?</b> This study highlights the importance of treatment changes when patients’ initial therapy does not adequately control their disease.