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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. Specific treatment depends on the type of MPN, severity, and symptoms. The following are a few examples.

  • For some, watchful waiting may be sufficient for several years. These patients will visit their healthcare practitioners regularly for monitoring and take aspirin to prevent blood clots.
  • Phlebotomy is the procedure for taking blood from a vein. Therapeutic phlebotomy is a procedure similar to making a blood donation and may be use in MPN treatment for removing excess blood cells. For example, with polycythemia vera, frequent therapeutic phlebotomies may be used to decrease the number and volume of red blood cells in the blood. Once the number of RBCs have been lowered as close to normal limits as possible, the person is monitored and occasional therapeutic phlebotomies are used to keep the levels under control.
  • Some medications can also reduce the volume of blood cells. An example is the mild chemotherapy drug hydroxyurea, which can be used to lower RBCs, white blood cells, and platelets.
  • Transfusions are used to add healthy red blood cells or platelets to the bloodstream to replace those destroyed by disease or medicines.
  • If the spleen is enlarged, it may be surgically removed. If surgery is not an option, radiation therapy may be directed at the spleen to kill abnormal blood cells.
  • Chemotherapy, like that used for acute myeloid leukemia (AML), may be used to control production of abnormal blood cells, especially if the MPN reaches a phase called a "blast crisis" when there is an increase in the number of the abnormal blasts (cell precursors) 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.
  • Several targeted therapies are aimed at inhibiting the abnormal proteins related to genetic mutations in MPNs. For example, drugs called tyrosine kinase inhibitors (imatinib, dasatinib, nilotinib) can target the abnormal BCR-ABL protein in chronic myeloid leukemia cells. 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. Other types of targeted therapies are being explored in clinical trials.

For additional details about treatments, see the links in the Related Pages section.

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