Treatment Options for Polycythemia Vera

A Brief Overview of Treatment of Polycythemia Vera

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Polycythemia vera, a myeloproliferative neoplasm, results from genetic mutation which leads to the production of too many red blood cells (erythrocytosis). The white blood cell and platelet counts may also be elevated. This increase in the number of blood cells (without increase in liquid portion of blood) increases the risk of clot development.  

Learning you have been diagnosed with polycythemia can be distressing and naturally you may wonder, "how can it be treated?" Fortunately, with treatment, survival increases from six to 18 months to 13 years or more.

One of the challenges in treating polycythemia vera is that more than 12 percent of people will transform into myelofibrosis and around 7 percent will develop acute leukemia/myelodysplastic syndrome.  

In contrast to most people with essential thrombocythemia, people with polycythemia vera commonly have symptoms like headache, dizziness, itchiness at diagnosis. These symptoms are generally distressing and require the initiation of treatment.

Phlebotomy for PV

The primary treatment of polycythemia vera is therapeutic phlebotomy. In therapeutic phlebotomy, blood is removed from the body similar to a blood donation to reduce the number of red blood cells in circulation. Therapeutic phlebotomy is continued on a regular basis to keep the hematocrit (concentration of red blood cells) below a certain number, usually 45 percent for men and 42 percent for women. Phlebotomy treats polycythemia vera in two ways: by decreasing the number of blood cells by physically removing them from the body and by causing a deficiency which limits new red blood cell production.

Hydroxyurea for PV

In people with a high risk of developing a blood clot (more than 60 years of age, history of blood clot), additional therapy is needed. Often the first medication added to therapeutic phlebotomy is oral hydroxyurea.

Hydroxyurea is an oral chemotherapeutic agent that reduces the production of red blood cells in the bone marrow.

It also reduces the production of white blood cells and platelets. Similar to other conditions treated with hydroxyurea, it is started at a low dose and increased until the hematocrit is in the goal range.

It is normally well tolerated with few side effects other than the decreased production of blood cells. Other side effects include oral ulcers, hyperpigmentation (dark discoloration of the skin), rash, and nail changes (dark lines under nails). Low dose aspirin is also considered a front line therapy. Aspirin impairs the ability of platelets to stick together thereby reducing the development of blood clots. Aspirin should not be used if you have had a history of a major bleeding episode.  

Interferon Alpha for PV

For people who have unmanageable itching (called pruritus), women who are pregnant or in child bearing years, or refractory to prior therapies, interferon alpha may be used. Interferon alpha is a subcutaneous infection that reduces the red blood cell count. It can also reduce the spleen size and itching.

Side effects include fever, weakness, nausea, and vomiting which limits the useful of this medication.  

Busulfan for PV

For patients that have failed hydroxyurea and/or interferon alpha, busulfan, a chemotherapeutic medication, may be used. The dose is changed to keep the white blood cell count and platelet count in an acceptable range.

Ruxolitinib for PV

Another medication used for patients that failed other therapies is ruxolitinib. This medication inhibits the Janus Associated Kinase (JAK) enzyme that is often affected in polycythemia vera and other myeloproliferaive neoplasms. It is often used for people who develop post-polycythemia vera myelofibrosis. It can also be helpful in people with severe symptoms and splenomegaly (enlarged spleen) that causes significant pain or other issues.  

Remember, although polycythemia vera is a chronic condition, it is a known condition that is manageable and possible to lead a good quality of life with. Talk to your doctor about the various treatment options available. He or she will suggest the best one for you. 

Sources:

Teferri A. Prognosis and Treatment of Polycythemia Vera. In: UpToDate, Post TW (Ed), UpToDate, Waltham, MA. (Accessed on June 22, 2016.)

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