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. Lower hematocrit targets have been proposed for women (42 percent), but researchers are still studying this recommendation. 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, or
- They have a history of thrombosis
Treatment may include:
- Low-dose aspirin
- Medication to reduce the number of blood cells (“cytoreductive” medication)
Low-dose aspirin may reduce the risk of blood clots. 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.
Most PV patients have their blood drawn at regular intervals. Blood is removed from the vein in a way that is similar to donating blood. Phlebotomy reduces the number of blood cells and decreases blood volume. After phlebotomy, the blood is thinner and less likely to cause “sludging,” when red blood cells become massed along walls of blood vessels. The immediate effect of phlebotomy is the decrease of certain symptoms such as headaches, ringing in the ears and dizziness. Eventually, however, phlebotomy will result in iron deficiency.
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®)
- Interferon alfa (Intron® A [alfa-2b] and Roferon®-A [alfa-2a]), and sustained-release preparations of these called PEG-Intron® ([peginterferon alfa-2b] and Pegasys® [peginterferon alfa-2a])
- Ruxolitinib (Jakafi®)
- Busulfan (Myleran®) and Chlorambucil (Leukeran®)
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®) may help itching that does not go away.
- Light therapy (phototherapy) using a medicine called psoralen combined with ultraviolet A (UVA) light.
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.
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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. Sometimes, a clinical trial is the best option for a patient. Some drugs under investigation include:
- Peginterferon alfa-2a (Pegasys®)—In the past, the use of interferon has been restricted because it was not well tolerated by patients. Much interest, however, lies in interferon’s ability to induce a molecular response in some patients. A molecular response occurs in PV patients when there is a reduction in the number of cells with the abnormal JAK2 gene mutation. New formulations of interferon that may be effective in achieving a molecular remission with fewer side effects are now under study.
- Givinostat—Givinostat is a histone deacetylase (HDAC) inhibitor. HDAC inhibitors are substances which cause chemical changes that stop abnormal cells from growing or dividing. Researchers are studying whether givinostat is an effective second-line treatment for patients who are intolerant of or refractory to hydroxyurea. Researchers are also looking at combining givinostat with low-dose hydroxyurea.
- Idasanutlin (RG7388)—This is an oral MDM2 inhibitor that blocks specific protein interactions, and this inhibition results in upregulation of the p53 pathway. This is an important mechanism to induce program cell death (called “apoptosis”) in PV cells.
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.