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Polycythemia Vera (PV) is a chronic disease: It's not curable, but it can usually be managed effectively for very long periods. The goal of therapy is to reduce the risk of thrombosis and to ease symptoms by lowering the number of extra blood cells. 

Many treatment options are designed to manage PV by lowering hematocrit levels below 45 percent for men and 42 percent for women. Careful medical supervision and therapy is important to keep the hematocrit concentration at normal levels. 

Treatment for Low-Risk Patients

Patients are generally considered low risk if: 

  • They are younger than 60 years, and 
  • They have no history of thrombosis 

Treatment may include:

Treatment for High-Risk Patients 

Patients are generally considered high risk if: 

  • They are 60 years or older, and/or 
  • They have a history of thrombosis 

Treatment may include:

Low-Dose Aspirin

Low-dose aspirin may reduce the risk of blood clots, heart attacks and strokes. Low-dose aspirin helps prevent platelets from sticking together, making it less likely for blood clots to form. The most common side effects of aspirin are upset stomach and heartburn.

Therapeutic Phlebotomy

Most PV patients have their blood drawn regularly to reduce the number of blood cells and decrease blood volume. Phlebotomy is a procedure in which blood is taken from a vein similarly to what is done when donating blood. After phlebotomy, the blood is thinner and less likely to cause “sludging” (which occurs when red blood cells build up along walls of blood vessels). The immediate effect of phlebotomy is to decrease certain symptoms, such as headaches, itchiness, vision problems, ringing in the ears and dizziness.

Medications to Reduce the Number of Blood Cells

High-risk PV patients may be prescribed cytoreductive drugs to reduce the number of blood cells. These drugs may include any or a combination of the drugs listed below

  • Hydroxyurea (Hydrea®)
  • Ruxolitinib (Jakafi®)
  • Interferon alfa (Intron® A, BESREMi® [alfa-2b], and Roferon®-A [alfa-2a]), and sustained-release preparations of these called PEG-Intron® ([peginterferon alfa-2b] and Pegasys® [peginterferon alfa-2a])
  • Busulfan (Myleran®)

Treatments to Reduce Itching

A troublesome symptom that occurs in many PV patients is itchy skin (“pruritus”). Treatment options include:

  • Bathe less frequently.
  • Bathe or shower in cool water and use a gentle soap.
  • Avoid hot tubs, heated whirlpools and hot showers or baths. 
  • Keep skin well moisturized with lotion and try not to scratch it because that can damage the skin.
  • Antihistamines such as diphenhydramine (Benadryl®) or doxepin may help itching that does not go away.
  • Light therapy (phototherapy) using a medicine called "psoralen" combined with ultraviolet A (UVA) light.
  • Medications such as gabapentin or pregabalin that block neurotransmitters in the central nervous system from sending signals that trigger itching.

For information about the drugs listed on this page, visit Drug Listings.

Clinical Trials

Taking part in a clinical trial may be the best treatment choice for some  PV patients. Patient participation in clinical trials is important in the development of new and more effective treatments for PV and may provide patients with additional treatment options. 

Click here to read more about clinical trials.

Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

There are clinical trials for newly diagnosed patients, for patients with advanced disease, and for patients who are intolerant of or resistant to their current medications. Researchers are currently studying:

  • Gene mutations and cellular pathways involved in the development of PV to help develop new drugs to target PV
  • New treatments to reduce the risk of thrombosis and cardiovascular complications and to improve quality of life for PV patients
  • New therapies to prevent PV from progressing to more serious blood diseases
  • New treatments to help high-risk PV patients live a normal life span

Some drugs under investigation include:

  • Ropeginterferon alfa-2b. In the past, the use of interferon was restricted because it was not well tolerated by patients. However, there is a good deal of interest in interferon’s ability to induce a molecular response (remission) in some patients. This type of treatment response occurs in patients with PV when there is a decrease in the number of cells with the abnormal JAK2 gene mutation. New formulations of interferon may be effective in achieving a molecular remission with fewer side effects and less frequent doses. Researchers are evaluating “pegylated” formulations of interferon that can be given less often, since they remain in the body longer. High molecular response rates have been demonstrated in studies. In 2019, ropeginterferon alfa-2b was approved by the European Medicines Agency for the treatment of polycythemia vera in patients without symptoms of an enlarged spleen. It is now being studied in clinical trials in the United States.
  • Histone deacetylase inhibitors (HDAC). HDAC inhibitors are substances that cause chemical changes that stop abnormal cells from growing or dividing. They show promise as “salvage therapies,” which are given when first-line treatment options fail. Researchers are studying whether givinostat is an effective second-line treatment for patients who are intolerant or refractory to hydroxyurea. Researchers are also evaluating the use of givinostat in combination with low-dose hydroxyurea.
  • JAK Inhibitors. The FDA-approved drug ruxolitinib (Jakafi®) is being studied for use in patients with PV who have had an inadequate response to or are intolerant of hydroxyurea. It is also being evaluated in combination with other drugs, including pegylated interferon, for PV treatment.

Treatment Planning

Treatment decisions are based on the patient's risk for clotting complications (thrombosis). The two main risk factors for thrombosis are:

  •  A previous clot or clots
  • Age 60 years or older

Every patient’s medical situation is different and should be evaluated individually by a hematologist-oncologist. As you develop a treatment plan with your doctor,  it is important to discuss:

  • The results you can expect from treatment
  • Potential side effects
  • All treatment options, including treatments being studied in clinical trials

You may find it helpful to bring a loved one with you to your doctor's visits for support and to take notes and ask follow-up questions. It's a good idea to prepare questions you'd like to ask when you visit your doctor. You can also record your conversations with your doctor and listen more closely when you get home.

To download lists of suggested questions to ask your healthcare providers, click here.

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