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Myeloproliferative Neoplasms

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Treatment

MPNs are usually not preventable or curable. The goals of MPN treatment are to slow the progression of the disease and to alleviate the symptoms and complications brought on by excessive, insufficient, and dysfunctional blood cell production.

For some, watchful waiting may be sufficient for several years. These patients will visit their doctors regularly for monitoring and take aspirin to prevent blood clots.

Treatment may include removal of the volume of problematic blood cells. For example, with polycythemia vera, frequent phlebotomies, the removal of pints of blood, are used to decrease the number and volume of red blood cells in the blood. Once RBCs have been lowered as close to normal limits as possible, the person is monitored and occasional phlebotomies are used to keep the levels under control.

Some medications can also reduce the volume of problematic blood cells. These include the mild chemotherapy drug hydroxyurea, which can be used to lower RBCs, white blood cells, and platelets. Interferon-alpha, a human protein, can be used to lower all three types of blood cells, while anagrelide, lowers the platelet count

In advanced disease and when the bone marrow doesn't produce enough healthy blood cells, steps may be recommended to reduce symptoms caused by low levels of blood cells, known as supportive care. These steps may include transfusions and medicines, including the growth factor erythropoietin (EPO) to increase red blood cells, the growth factor granulocyte colony-stimulating factor (G-CSF) or granulocyte macrophage colony-stimulating factor (GM-CSF) to increase white blood cells, and antibiotics and antiviral drugs that fight infections when white blood cells cannot fight on their own.

Transfusions are used to add red blood cells or platelets to the bloodstream if medicines do not increase a person's levels enough and if the patient develops anemia.

Chemotherapy, like that used for acute myelogenous leukemia (AML), may be used to control production of abnormal blood cells, especially if MPN reaches a phase called a "blast crisis" when there is an increase in the number of the abnormal stem cells in the bone marrow or blood.

People with severe or advanced MPN may be treated with bone marrow transplantation known as hematopoietic cell transplantation. This is currently the only type of treatment that has the potential to cure MPN. MPN patients receive allogeneic transplants, or stem cells from a donor, preceded by chemotherapy to kill diseased stem cells in their marrow. A transplant is not appropriate for everyone. For instance, a transplant may be too difficult older people, especially if they have other health problems besides MPN.

Several novel treatments aimed at inhibiting the abnormal proteins related to genetic mutations in MPNs have been developed. For example, a few drugs called tyrosine kinase inhibitors (imatinib, dasatinib, nilotinib) can target the abnormal BCR-ABL protein in chronic myeloid leukemia cells and have produced a high rate of remission in most patients, meaning no detectable disease on continuous drug treatment. These drugs are now the standard treatment for patients with chronic myeloid leukemia. Ruxolitinib inhibits the JAK2 protein and is used to treat intermediate to high risk primary myelofibrosis and myelofibrosis derived from preexisting polycythemia vera or essential thrombocythemia.

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