Treatment Options

Myeloproliferative Neoplasms (MPN) may cause only mild symptoms, with little effect on health, but in some case progress to very severe.  Because most people with MPNs have survivals of greater than 5 years and the MPN are not cured by chemotherapy (except for CML), treatment is geared toward symptom relief and prevention of complications.  The development of clots is the most common and sometimes life threatening complication of MPNs.  Prevention of clots may include aspirin, a platelet activity inhibitor, with or without drugs or phlebotomy  to lower the blood counts.  The choice depends on tthe disease type, age, and history of previous clot.  Treatment for CML, mastocytosis, and eosinophilia are discussed elsewhere.
 
Treatment can vary greatly from person to person. Your plan will be customized to you.

Watchful Waiting 

If your disease is mild and interferes only a little or not at all with your usual activities, your doctor may recommend “watchful waiting.” This means you receive no treatment or aspirin alone, but you visit your doctor on a regular basis (or if you notice changes) to check your health. Some people with MPN go for several years with no major health changes.

Phlebotomy

Phlebotomy is the removal of blood from the body.  In MPNs large volume phlebotomy (usually 1 unit of blood equals 500 ml) is removed to lower the red blood cell counts.  This is an effective treatment to relieve symptoms and with aspirin to reduce clot complications to people with polycythemia vera and low clot risk.

Cell-Reducing Medications

In patient with high risks for or previous clot or with very high non-RBC blood counts, may be treated with medications that decrease the productions or release of blood cells from the bone marrow.  The mild chemotherapy agent Hydroxyurea (Hydrea) can be used to lower RBCs, platelets and white cells.  It is generally well tolerated but may slightly increase the low risk for transformation to leukemia in polycythemia vera.  You should discuss this with your physician.  Interferon-alpha, a human protein, can be used to lower all three blood cell types but is not well tolerated by all people due to the common side effect of flu-like symptoms.   Anagrelide is a drug that lowers the platelet count with little other effects on the bone marrow cells.  It is well tolerated except for some light headedness and headaches.  In head to head comparison, hydrea was superior to anagrelide in preventing arterial clots and is currently the preferred therapy to lower blood counts in high risk MPNs.

Supportive Care

In advance disease and burn out phases, MPN treatment may include supportive care—measures to help reduce symptoms. For MPN, these measures may include medicines and transfusions.
 
Medicines used in supportive care for MPN include the following:

• The growth factor erythropoietin (EPO) to increase your red blood cell count
• The growth factor granulocyte colony-stimulating factor (G-CSF) or granulocyte macrophage colony-stimulating factor (GM-CSF) to increase your white blood cell count 
• Antibiotics and antiviral drugs to fight infections that your white blood cells cannot fight on their own

Induction Chemotherapy

If the MPN progresses to a blast crisis where there is increase in the number of the abnormal stem cells in the bone marrow or blood, the treatment is similar to acute leukemia.   Chemotherapy is given intravenously, by mouth or directly into the spinal fluid (if you need treatment in your central nervous system, which is rare in patients with MPN). These medicines are distributed throughout the body through the bloodstream. They can help control your body’s production of abnormal blood cells.
 
Side effects are an important concern for many people receiving chemotherapy. Besides killing cancer cells, chemotherapy can damage healthy tissue and cause side effects like fatigue, hair loss, digestive problems, mouth sores, skin irritation, and low levels of healthy blood cells. Your doctor will talk with you about the specific side effects you might develop and what to watch for. We will help you take the best possible care of yourself to prevent, minimize, or relieve side effects so you can focus your energy on your recovery.

Transplantation

Patients with severe or advanced MPN often are treated with a hematopoietic cell transplant (a bone marrow transplant or a transplant using peripheral blood stem cells, those circulating in the blood). This is currently the only type of treatment that has the potential of curing MPN.
 
Patients having an allogeneic transplant  undergo conditioning with high dose of chemotherapy . Some have myeloablative conditioning; others have lower-intensity conditioning, called nonmyeloablative. (You may also have heard this called mini-transplant.) This type of regimen destroys some of the bone marrow cells. The intent is to suppress the immune system enough so that the donor cells are not rejected and can help to kill remaining abnormal cells in the marrow by an immune attack on those cells.  Following the donor cell infusion, immunosuppressive agents are use to prevent graft-versus-host disease, a complication of transplantation that can affect the skin, liver or GI tract and potentially be fatal.
 
A transplant is not appropriate for all patients. For instance, a transplant may be too challenging for older individuals, especially if they have other health problems besides MPN, as many older people do. Though some patients have had successful transplants at age 70 years, it is not the standard treatment in this age group. For some patients who cannot undergo a myeloablative transplant, a nonmyeloablative transplant may be an option. Read more about our Transplant Program.

New Treatments

With the discovery of specific gene mutations in MPNs, medications were designed to specific inhibit these abnormal proteins.  The drug imatinib was developed because of its ability to inhibit the abnormal BCR-ABL protein in CML cells and has been successful at producing a very high rate of remissions.  (see CML treatment)  Other drugs are in various stages of development and testing for the mutated JAK2 gene product.   These drugs are not commercially available, but may be available at the Seattle Cancer Care Alliance as part of clinical trials.