What are the medications used to treat Polycythemia (excessive red blood cell count)?

From the Guidelines

Polycythemia vera should be managed with phlebotomy to maintain the hematocrit below 45%, along with daily low-dose acetyl salicylic acid, and cytoreduction is recommended in high-risk cases, with either hydroxyurea or recombinant interferon alpha (rIFNα) as first-line cytoreductive therapies at any age. The treatment of polycythemia vera involves several medications, including hydroxyurea, interferon alpha, and ruxolitinib, which target different aspects of the disease.

• Hydroxyurea is the most commonly prescribed cytoreductive medication, reducing red blood cell production by inhibiting DNA synthesis, and is typically started at 500-1000 mg daily 1.
• Interferon alpha (pegylated forms like Pegasys) offers an alternative that doesn't increase leukemia risk and is preferred in younger patients or during pregnancy, with a dosage of 45-180 mcg weekly 1.
• Ruxolitinib targets JAK2 mutations and is particularly effective for patients with significant splenomegaly or those intolerant to hydroxyurea, with a dosage of 10 mg twice daily 1.
• Aspirin is typically added to reduce thrombotic complications, with a dosage of 81-100 mg daily 1. The goal of treatment is to maintain hematocrit below 45% in men and 42% in women to prevent thrombotic complications, and regular monitoring of blood counts is essential during treatment to adjust medication dosages and minimize side effects 1. Key considerations in treatment selection include the patient's age, disease severity, and risk factors, with a focus on minimizing morbidity, mortality, and improving quality of life 1. However, the most recent and highest quality study 1 provides the strongest evidence for the recommended treatment approach.

From the Research

Polycythemia Vera Medications

• The primary goal of therapy in polycythemia vera (PV) is to prevent thrombohemorrhagic complications 2, 3.
• First-line cytoreductive therapy in high-risk patients is hydroxyurea; however, its use is associated with toxicities and resistance in a significant proportion of patients 4.
• Other treatment options for hydroxyurea resistant or intolerant PV include interferon, ruxolitinib, and other agents 4.
• Ruxolitinib is a Janus kinase inhibitor that can alleviate pruritus and decrease splenomegaly in patients who are intolerant of or resistant to hydroxyurea 5.
• Cytoreductive therapy is recommended for high-risk PV, but it is not mandatory for low-risk disease 2, 3.
• Aspirin and hematocrit reduction by venesection or cytoreductive therapy are at the cornerstone of management for PV 4.
• The following medications are used for PV:
  • Hydroxyurea: first-line drug of choice for cytoreductive therapy 2, 3
  • Interferon-α: second-line drug of choice for cytoreductive therapy 2, 3
  • Busulfan: second-line drug of choice for cytoreductive therapy 2, 3
  • Ruxolitinib: used for severe and protracted pruritus or marked splenomegaly that is not responding to other drugs 2, 3

Treatment Considerations

• Patients with PV should receive therapeutic phlebotomy to maintain a hematocrit of less than 45% and low-dose aspirin (if no contraindications) 5, 2, 3.
• Patients who are at higher risk of thrombosis include those aged 60 years or older or with a prior thrombosis 5, 2, 3.
• Cytoreductive therapy may be beneficial for patients with persistent PV symptoms or those at high risk of thrombosis 5, 2, 3.