What are the treatment options for Polycythemia (excessive red blood cell count)?

From the Guidelines

The treatment for polycythemia should focus on reducing blood thickness and preventing complications, with therapeutic phlebotomy as the cornerstone treatment, aiming to maintain a hematocrit below 45% in men and 42% in women, as recommended by the European LeukemiaNet 1. The goal of treatment is to reduce the risk of thrombotic events and other complications, while also managing symptoms and improving quality of life.

• Key components of treatment include:
  • Therapeutic phlebotomy to reduce blood viscosity and prevent thrombosis
  • Low-dose aspirin to reduce thrombotic risk
  • Cytoreductive therapy with hydroxyurea or interferons for high-risk patients or those with significant symptoms
  • Lifestyle modifications, such as adequate hydration, avoiding extreme heat, and smoking cessation
• The European LeukemiaNet recommends that all patients with polycythemia vera should be managed with phlebotomy to maintain a hematocrit below 45%, together with daily low-dose acetyl salicylic acid 1.
• The NCCN guidelines also recommend therapeutic phlebotomy as the primary treatment for polycythemia vera, with cytoreductive therapy added for high-risk patients or those with significant symptoms 1.
• Regular monitoring of blood counts is essential to adjust therapy and prevent complications like thrombosis, bleeding, or progression to myelofibrosis or leukemia.
• It is worth noting that the CYTO-PV trial demonstrated that maintaining a lower hematocrit target (<45%) was associated with a reduced risk of thrombotic events compared to a higher target (45-50%) 1.

From the Research

Treatment Options for Polycythemia

• Therapeutic phlebotomy is a primary treatment for polycythemia vera, with a goal hematocrit of less than 45% 2, 3, 4, 5
• Low-dose aspirin is recommended for all patients with polycythemia vera, unless there are contraindications 2, 3, 4, 5
• Cytoreductive therapy, such as hydroxyurea or interferon, is recommended for patients at high risk of thrombosis, including those aged 60 years or older or with a prior thrombosis 2, 3, 4, 5
• Ruxolitinib, a Janus kinase inhibitor, can be used as a second-line therapy for patients who are resistant to or intolerant of hydroxyurea 2, 3, 4, 6
• Other treatment options, such as givinostat, are being investigated for the management of polycythemia vera 3

Patient Risk Stratification

• Patients with polycythemia vera can be stratified into low-risk and high-risk categories based on age and prior thrombosis history 3, 5
• High-risk patients are those aged 60 years or older or with a prior thrombosis, and require more aggressive treatment, including cytoreductive therapy 2, 3, 4, 5
• Low-risk patients can be managed with therapeutic phlebotomy and low-dose aspirin alone 3, 5

Special Considerations

• Patients with extreme thrombocytosis (platelet count ≥1000 × 10^9/L) may be at increased risk of bleeding and require special consideration 2
• Women of childbearing age with polycythemia vera may require alternative treatment options, such as interferon, due to the potential risks of hydroxyurea during pregnancy 5